Nano Medicine

FDA files injunction again Regenerative Sciences citing Regenexx violates regulations

§ August 12th, 2010 § Filed under Regenerative Medicine Comments Off

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Money for regenerative medicine

§ July 7th, 2010 § Filed under Regenerative Medicine Comments Off

This is simple. You can give real money to regenerative medicine without it costing you a dime. JPMorgan Chase & Co’s Chase Community Giving program is giving away $5 million to the charities with the most votes. Your vote could mean money for regenerative medicine.

Don’t be silly. Just click here and vote.

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The changing face of PR and why it matters to regenmed

§ June 12th, 2010 § Filed under Regenerative Medicine Comments Off

This may seem a little off-topic for those who don’t know me but for those of you who do, you’ll know this is a little mix of many of my primary loves – cell therapy/regenerative medicine, communications/public relations, social media, and all things internet/technology.

This is a reprint (with permission) of an article I was invited to write for the 2009 World Stem Cell Report.

______________________________________________________________________

You may be one of them. The scientist who can’t get the job or promotion you want. The company not securing customers fast enough. The investigator not finding collaborators. The company not finding quality employees. The company failing to secure much needed investment. The big company not effectively penetrating new, niche markets like cell therapy-regenerative medicine. The executive or investigator not getting the speaking invites to create the buzz needed to help move things forward.

Whatever the challenge, consider a healthy dose of marcom. [2]

Ok, I’m biased and it certainly isn’t a single panacea for all these problems, but seriously. A good profile (personal or corporate) – built from a solid reputation and strong relationship network – can help address almost any challenge you or your company is facing.

INTRODUCTION

I know. You don’t believe me. Humor me and answer this one question. If you could double the number of people that know about your company or technology, the number of people that learn, hear, or read something about your research, product or service in the average 30–day period, and/or the number of people that interact with one of your employees or colleagues every week, would that – on the whole – likely help or hinder you in addressing at least some of the primary challenges you are currently facing?

See where I’m going with this? Even if your challenges are deeply scientific or technical, is it possible to imagine that if only you knew the right person …?

Your existence depends on human interaction. Your success depends on how well you interact and the impressions you make. The impressions are not solely or even primarily driven by the brilliance of your technology or the compelling weight of your data. This is likely more true than you may think. For those of you who have read Malcolm Gladwell’s book, Blink, you will appreciate just how much people’s perceptions are influenced not by logic, data, or comprehensive consideration but by the judgments of the subconscious in a matter of seconds.

The PR (public relations) I discuss in the following pages is not spin and it’s not advertising. It’s about establishing and maintaining relationships that result in profile among your various kinds of constituents be they your collaborators, peers, investors, employers, employees, customers, or potential strategic partners.

Successful people and companies use effective PR to create relationships, turn them into interactive networks, and then leverage them to help solve the challenges they face.

This has always been true. What are so radically changing today are the tools which people and companies can now employ to conduct effective PR, create relationships, and build networks all at a fraction of the cost, with greater returns, and performed by individuals or small companies not previously capable of affecting global impact. These tools are, of course, online.

A successful strategy for PR is now increasingly determined by how well online tools are employed to engage in conversation, create relationships, and build profile.

As it turns out, there are, in my opinion, too few people in this industry who, as yet, understand the power of PR and fewer still who understand the potential of web2.0 to change their fate.

What I will share with you in the next couple pages is:

1. why if you’re breathing, you’re in PR – if you can’t avoid it (and I don’t recommend you try), you should learn to embrace it;

2. the cost of dodging the embrace; and

3. how you can embrace it.

POINT #1: YOU’RE IN PR. REALLY.

There are a zillion reasons why you underestimate the importance of PR in your career or to your company. There are a zillion other reasons why you think online social media is irrelevant to what you do. Let’s look at five.

1. My company is a research-based, early-stage company conserving burn-rate, with few employees, and nothing to sell. Ergo we don’t have a need for PR stuff like marketing, communications, or profile building.

There is nothing new but certainly nothing outdated in the adage: Everyone is selling something.

Don’t pretend you have no one to impress, you have nothing to sell, or that content is the only thing that impresses in the science business. If you thing you have nothing to market, you just haven’t figured out what it is yet.

Are you looking to recruit quality people? Do you anticipate needing to find and impress new investors sometime this year or next? Would you be interested in engaging in discussions from parties potentially interested in licensing or acquiring some of your IP? Are you looking to impress potential academic or corporate collaborators? Do or will you need to recruit patients for a clinical trial? Do you need to start educating doctors about the potential of your therapeutic or engage them in giving advice about your trial or product? Would you benefit from increased government funding for cell therapy and regenerative medicine? Do you believe there are policy changes that could be made that would benefit this industry?

A better profile and a wider network of contacts can’t help but help you address these needs. If you have anyone to impress (and everyone does), then you need to be talking with them. Will a bigger and better profile hurt your efforts to find good employees, seduce investors, recruit patients, identify collaborators, secure partners, etc? It’s hard to imagine how.

At the very minimum, as a member of the life sciences industry you have an obligation to get out of your lab and communicate to the world around you about the science you are so passionate about. As President Obama encouraged in his address to the April 27, 2009 National Academy of Sciences [3], we all have a duty to do a better job of engaging the people around us in a discussion about the importance of science and the science we’re doing.

2. My company is a big, public, multi-national company with departments that handle marketing, communications, PR, and advertising. Ergo I don’t need to pay attention to these things because they do that.

Firstly, unless you have tenure, chances are between now and retirement you may want/need/be recruited for another job. A strong, personal “brand” (profile) is certainly a huge asset in terms of ensuring you are successful in finding that job and negotiating the right remuneration.

Secondly, if you are ever let out of the lab/office or given internet access, you have the capacity to (and likely already) influence your company’s profile and brand. You are either complementing or distracting from your company’s primary messages and brand.

Thirdly, if you care about your company’s success, don’t assume your company’s department or external marcom firm is doing what needs to be done. Investor relations, marketing and communications firms or departments may be good at investor relations, marketing, and communications but that doesn’t necessarily mean they are good at the kind of profile building that is going to attract and support meaningful business development opportunities or potential investigators, customers, licensees, collaborators, employees, or clinical trial patients.

Finally, too many big companies haven’t yet figured out how or how to successfully use Social Media. If you’re using Social Media (e.g., Facebook, Twitter, FriendFeed, Digg, etc), you might be able to play a leadership role in being the face of your company on these platforms – just don’t do it without permission!

3. My company is a science-based company with a technical sale to scientists who are not receptive to glitzy marketing, advertising, PR, and-the-like. Ergo there is little ROI on spending money on these kinds of things. We don’t “do” marketing.

You may be in a company that believes putting out press releases is an unnecessary distraction from the business of science; or the company that believes that communications is something done only big companies, public companies, stock hustlers, or companies with something to sell.

Wrong. First you’re working on the assumption that all marketing looks like a drug ad.

Second your target audience is human. Humans are susceptible to having their perceptions influenced via all the usual means. Even if you don’t want to look at it so crassly, you would have to agree they are susceptible to forming more positive impressions of your company or product if information about it is available, packaged nicely, and easily accessible. You would also have to agree that any impression or sale is easier to make when there is a personal relationship/contact.

Your network is one of your most valuable assets to you and to your company. Building a global network and industry profile is marketing under what ever name you want to call it.

4. All this web 2.0 and social media stuff is for tech geeks and kids. They are not my target market and don’t have a critical mass of the kinds of people I need to building profile among.

Let’s assume for a moment, this is true. If you’re looking to be an attractive employer to potential, young employees you may want to reconsider.

Secondly, young people grow up and become industry leaders and executives. Is it too early to start shaping their perceptions?

Thirdly, there is the occasional executive that is actually occasionally influenced by input from their younger employees. I know – it’s crazy!

Furthermore, it’s not true. For example, I have a very high percentage of c-level executives on my LinkedIn Cell Therapy Industry Group, Jim Till who proved the existence of the stem cell in his ground-breaking nature paper in 1963, is an active poster on Twitter, and the average age of social media users is much older than you believe – check your data.

5. Twitter-schmitter. Today’s social media sites are tomorrow’s relics. They’re all hype when they launch and then they fall out of favor and are no longer useful. I’m a serious business professional with no time or need for these gimmicks that have no real business applications or executives like me on them.

If you think LinkedIn, Twitter, or Facebook (my three favorite and most used social media sites) are not going to be around and successful for long enough to have a real impact on your business, you’re definitely one to bet against the odds.

If you think serious business is not being done and useful business connections being made on these sites (see the last paragraph in the point above), you’re dead wrong. I’ve found customers, employees, and collaborators for my customers using social media in addition to getting them speaking engagements and highlighted in publications.

The point? Everyone has some kind of PR (marketing, communications, profile building, networking, advertising, community participation, publishing, speaking, etc) to do. Your potential audience, network, and impact are all much bigger than likely believe to be true.

My guess? You have more than likely significantly underestimated the value of engaging in PR. And, you have likely significantly overestimated the time and financial cost of engaging in PR.

That brings me to Point #2.

POINT #2: THERE IS A REAL COST TO YOU AND YOUR COMPANY IN PRETENDING YOU DON’T DO OR NEED TO DO PR.

All I should really have to do here is refer you back to point #1. The benefits of engaging in PR are the costs for not. Nevertheless, I will briefly expound.

Your next job, employee, collaboration, investor, customer, patient, etc will be influenced by the profile of you and/or your company. While your CV – comprised of its peer-reviewed publications, presentations, and collegial references – is still an important driver in people’s perceptions in our industry what often makes someone or some company stand out among the others (perhaps even imperceptibly) is the overall profile one has created. One’s profile is increasingly judged online.

That brings me to revise Point #2 to the following:

There is a real cost to you and your company in pretending you can/should ignore the PR power of web2.0’s social media.

I’m going to steal now from my October 2008 blog posting on the subject. [4]

Only a few minutes ago (relatively speaking in the passage of time) most companies didn’t think they needed to have a website or a domain name strategy. This seems as foolish now as ignoring social medial will seem in a few minutes from now.

In 1995 – a mere 14 years ago – I was a young associate in a downtown law firm making a pitch to a skeptical management committee at a weekend retreat that the firm needed a website and an email “system”. It was not an easy sell. They “knew” lawyers would never send their own emails and certainly not use it to communicate with clients. Similarly they were convinced a website would likely not bring in any new clients and existing clients would not likely find a website useful.

How quickly they were proven wrong.

We are at a similar technological threshold. Most companies are ignoring the importance of “social media” to their corporate strategy. Most consider using social media an unnecessary “luxury” in exactly the same way websites, domain names, and email were considered by people in their positions only a few years ago. They do not understand how social media is already changing the way they do business, the importance of staking position/profile early, nor the pain they will feel in the very near future for having failed to be proactive in building and leveraging online communities.

This is very understandable. In times of fiscal constraint one tends to focus on that which hurt you yesterday or causes you the most pain today. Nonetheless, the companies that will succeed tomorrow are looking at tomorrow now and preparing for it. Tomorrow’s mainstream business activities involve social media as much as today’s involves website and email.

For those thinking that this will be true only for businesses that deal direct with the retail consumer, they’re dead wrong. If you have a constituency that you need to keep informed about and actively engaged in your company, then this applies to you. Whether its business (B2B) customers, investors, media, patients, recruits, employees, or collaborators, they are online and engaging in social media. If you’re not there, they’re listening to and engaging with someone else.

LinkedIn now has 42 million members with 1,945,047 members who identify themselves in biotech, pharma, or healthcare. Some 300,000+ of them are in biotechnology. There are 293 biotechnology groups in LinkedIn and 375 biotechnology groups on Facebook. The LinkedIn cell therapy industry group has 400+ members engaging in active discussions and exchanges of information in the public forum and using the platform to make and strengthen connections offline.

Bottom line? There are so many different kinds of social media out there that can be used to reach so many different kinds of audiences at a fraction of the cost of traditional media that it’s just good business to figure out how to use it to your advantage. In fact, you may be feeling the pain of ignoring it already and just don’t know it…

Which brings me to point #3.

POINT #3: WEB 2.0 HAS PUT THE ABILITY TO DO EFFECTIVE, LOW-COST PR IN YOUR HANDS. THERE ARE NO MORE GATEKEEPERS.

The internet has blown the sense of community up and redefined it. Gone are the days when it was feared the web would destroy “community”. Instead web 2.0 is all about creating more and larger communities than we ever thought possible eliminating all kinds of barriers and cutting across all kinds of definitions.

It used to be (before web 2.0) that corporate profile, marketing, communications, PR, advertising was all quite carefully controlled by managing the one-way push of information to the open receptacles we called “readers” or “audience”. If you wanted a global profile, you needed a global PR firm.

Just like the internet has taken the magic out of the business of travel agents, web 2.0 has forever changed the grip that marketing, communications and PR firms had on the business of managing global corporate profiles. Similarly, a global profile is no longer only the purview of companies sufficiently large to retain the services of such firms or have big, internal PR departments.

Just as the information on how to build long-range missiles is no longer the exclusive domain of rocket scientists, with the empowering tools of web 2.0, successful marcom can now be effectively done by subject-matter experts who are not big-budget marcom professionals.

It’s why newspaper are dying and bloggers are thriving. It’s why small companies are building niche profiles that beat out their Fortune 500 competitors. It’s why startups can have a global profile in weeks on next-to-no budget.

All of that is a rather lengthy introduction to primary point of this article:

In this new world of interconnectedness and easy-to-access information, it is easier than ever for everyone and anyone to influence the profile of a company and what it is offering to the world. That’s the good news. The bad news is that you are expected to. All of which leads to one not-so-original observation of the impact of web 2.0 on companies.

If you’re breathing with an internet connection, you can and should have significant and immediate impact on the awareness and perceptions of your company among people around the globe.

If you’re not creating global awareness and positive perceptions, it may be assumed you’re terminally short of breath.

Like it or not. You can’t help it and you certainly can’t avoid it. And the people forming these perceptions are not just the potential customers of companies with something to sell – they’re current or potential investors, partners, collaborators, patients, purchasers, journalists, regulators, policy-makers, employees, employers, etc.

LIGHTS, CAMERA, ACTION

So what can you do to begin leveraging the power of the PR tools at your fingertips and turn a bungalow profile into a chateau; a respectable list of contacts into a vibrant, enviable network of relationships?

10 Ways to a Better Profile for RegenMed and Cell Therapy Organizations

I’ll start with some rules that apply regardless of the PR platform you’re employing (e.g., traditional ones like conference presentations or journal publications or web2.0 ones like blogging or social networking).

1. You need to have a communications strategy and then mandate and empower your people to communicate your message/story to your audiences.

This doesn’t have to be complicated. It’s about defining your primary/secondary audiences, messages, keywords, and tactics to accomplish your primary/secondary goals.

2. Talk about what you’ve done not about what you’re going to do.

It is very rare that news about what you are going to do is really newsworthy. Don’t be tempted to put out a press release that’s not news just because you haven’t put one out in awhile. For example, a press release that your executive is going to speak at a conference is not news unless they are presenting new data or they are the keynote at the biggest conference of the year. That doesn’t mean you can’t post it on your website or let your network know in other ways but the press release should be used judiciously.

3. Make sure you tell the market story not just the science story. Similarly, be clear about what problem your technology solves not just how brilliant it is.

4. Be upfront about the limitations of your product/service. Don’t try to pretend you have the perfect product/service. Don’t try to be everything to everyone – defining who you are (your company and its product/service profile) is just as much what about what it is than what it is not.

5. Avoid the temptation to promise when you will do something by (e.g., begin or end your trial, submit your IND, launch your product, etc) unless it’s the subject of a contractual obligation. There are too many unknowns that may prevent you from meeting the expectation you created. The ensuing disappointment will hurt your profile and perceptions of your company and its product/service and management.

6. Don’t over communicate your regulatory interactions with the FDA – this is one area where the less said is often better.

7. Wait on that hiring announcements just a little while. It’s critically damaging to put out a press release that now has a very, long online life outside of your control announcing the hire of a key executive only to have him/her not last beyond the first few weeks.

8. Understand that communications, marketing and profile building are very different from advertising. Act accordingly.

9. Value the untapped potential of your existing customers and prospects to spread your gospel.

One of the real discoveries from social media is the alarming willingness of people to zealously proselytize on behalf of companies and products win which they believe. Find ways to actively engage them in doing so. [5]

10. Engage online.

The 8-step Plan to Enhance your Online Profile and Network

1. SEO.

If you’ve decided your profile matters, then your online profile really matters. The fastest way to an impressive online profile is Search Engine Optimization. There are a lot of tricks up the SEO tricksters sleeve and I’m not about to give them all away but suffice it to say that SEO is a multi-pronged attack and there are many arrows in the quiver each with a different use to the same end: higher rankings on the major search engines = increased traffic to the website = greater profile = whatever your end goal is (sales, investors, collaborations, changing the world, etc).

2. Listen & Monitor.

Whether you employ Google Alerts, Twitter search tools, or simple subscriptions to topical news services or blogs, there is a lot of content and discussions being added to the internet every day. You will likely be amazed how much (or how shockingly little) your company and product/service is being discussed and who is behind the discussions. Listening to and reading what’s going on the internet will get you intel about your competitors, new ideas for potential products or studies, new leads for potential collaborators or customers, etc.

3. Follow/Subscribe.

Find your favorite content providers, writers, resources, publications, etc and follow or subscribe to the content their pushing out and discussions they are leading.

4. Join

There’s no harm in joining. People join for all different reasons. There’s no problem in being an observer. In fact it’s likely best you just observe for a while after you join to assess the flavor of the group and discussion so when you do participate you don’t offend.

5. Participate

Once you’re comfortable, start to participate. Engage in discussion, exchange information, and above all make sure you are providing value to the other participants not just talking about yourself. This will be punished.

6. Connect (yourself and others)

There’s a reason why these are called online networks or communities. Use them to connect with people not just for the sake of building a bigger ‘friends’ list but because you have something in common and want to begin building a relationship.

7. Create

Be original. Create something of value you can share. That might just be facilitating a channel in which 3rd party information is exchanged. It might be you writing original content. It might be facilitating introductions.

8. Enjoy and don’t offend.

Most importantly find the online networks that you enjoy, that give something of value to you, then contribute value in return and ensure you don’t offend the rules. Nothing slaps harder than an online, viral slap from a community you’ve offended online. [6]

CONCLUSION

Don’t pretend you have no one to impress. Everyone needs to engage in PR.

Even in the business of science, man cannot live on data alone. You need more than just solid content to affect the kind of impressions that will be critical to your success. Don’t let your data or career speak for itself – it can’t talk.

Web 2.0 has made it exponentially easier – if not expected – to engage in PR and profile building.

What’s missing from this article is the web 2.0 tools I’d recommend you use to put the 8-step plan in place. For that, watch here for further blog postings on the subject. See what I did there?

[1] In this article, I use “PR” not to mean “public relations” as you may have come to understand it used but rather simply to mean any kind of relating to the public via marketing, communications, writing, profile building, networking, advertising, community participation, publishing, speaking, proselytizing, lobbying, etc. It’s a not-so-dirty little secret that we all do it in one form or another or several all at once.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Biotech tax credit appears perfectly designed for cell therapy companies to recoup research dollars spent in 2009-10

§ May 22nd, 2010 § Filed under Regenerative Medicine Comments Off

Stewart Lyman of Lyman BioPharma Consulting posted a great article in today’s Xconomy summarizing some key points and links to more information about the rules governing the Therapeutic Discovery Project Credit which have now been released by the US Treasury Department. Today, a detailed fact sheet was released about the tax credit program and it seems almost perfectly designed for most cell therapy companies.

Lyman points out a few important details about the application schedule including:

1. The Formal IRS applications (Form 8942) will not be available until June 21st or thereabouts.

2. The application period opens on June 21 and ends on July 21. The postmark on the application is deemed to be the date of delivery. Preliminary review of the applications is to be completed by Sept. 30; this is to ensure that applicants are eligible taxpayers and that their applications are complete. Applicants will receive determinations as to whether or not they qualify for credits and/or grants, and how much they will receive, by Oct. 29.

By way of a little more background, the following is excerpted from a March Forbes.com article by Dean Zerbe:

What does the credit cover?

The credit/grant covers research in tax years beginning in 2009 and 2010. The taxpayer is provided a 50% credit/grant for qualified investments in “qualifying therapeutic discovery projects.” What expenses count as qualified investments? The aggregate amount of costs paid or incurred in the taxable year for expenses necessary for and directly related to the conduct of a qualifying discovery project. What doesn’t count? The pay of employees covered by 162(m)(3) of the tax code–think CEOs–doesn’t count. Other excluded items: interest expenses; facility maintenance expenses (e.g. mortgage or rent payments, insurance, utility and maintenance and costs of employment of maintenance personnel); and certain indirect costs (basically general and administrative costs) as defined in the Treasury Regulations at 1.263A-1(e)(4).

What is a qualifying therapeutic discovery project?

According to the legislation, it’s a project designed to do one of three things:

–Treat or prevent diseases or conditions by conducting pre-clinical activities, clinical trials and clinical studies, or carrying out research protocols for the purpose of securing federal government approval by the FDA.

–Diagnose diseases or conditions or to determine molecular factors related to diseases or conditions by developing molecular diagnostics to guide therapeutic decisions.

–Develop a product, process or technology to further the delivery or administration of therapeutics.

Finally, to qualify, a venture may not have more than 250 employees in all businesses of the taxpayer–meaning a small biotech project at a big company wouldn’t qualify.

Which biotech companies might benefit?

Those that are investing significant resources in pre-clinical or clinical studies, which may take years to come to fruition to ultimately satisfy FDA requirements, could now recoup a significant portion of their expenses. Additionally, biotech start-ups focusing on the development of diagnostic assays or applications to advance therapeutics and treatments can also benefit. Finally, companies currently engaged in basic or applied research which may ultimately contribute to curing caner within the next 30 years may also be excellent candidates. Along these lines, companies studying signal transduction pathways, gene therapy and stem cell research seem like prime candidates.

The Cell Therapy Group will be collecting more information about the tax credit and service providers who might be recommended to assist in the application if needed. Contact CTG for more details or watch here for more information.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Google to Invest in Regenerative Medicine

§ May 5th, 2010 § Filed under Regenerative Medicine Comments Off

While the US government prints money to shore up failing and broken business models which no one likes but are considered simply too big (not too important or significant or even useful) to fail, Google is making money and investing it in start ups who expect nothing less than to create “disruptive, even world-changing technologies”.

No suggestion here that GOOG is being altruistic, just that this is the way the new entrepreneur and investor class thinks. Opportunity and money are to be found in technologies that improve the way we live, work, play, eat, and think… and perhaps even improve the world.

To Google Ventures this has already meant wind farms, carbon emission reduction systems, green vehicles, and medical cures. To former Microsoft chief scientist Nathan Myhrvold and his high-level think tank, Intellectual Ventures, this means creating TerraPower – a company intending to revolutionize the nuclear power by developing reactors run on waste uranium – and also actively looking at regenerative medicine technologies.

Having formed the fund a little over a year ago, Google is only now starting to make a splash with the fund. Officially the fund has no specific industry focus saying on the Google Ventures website FAQ:

We are interested in a wide range of industries, including (but not limited to) consumer Internet, software, hardware, clean-tech, biotech, health care and others. First and foremost, we’re looking for entrepreneurs who are tackling problems in creative and innovative ways, with the potential for significant financial return.

Unofficially and yet not so quietly, Google has named a few broad areas of interest. An article in Monday’s New York Times quoted Google Ventures’ managing partner, Bill Maris as saying that while they were not going to name particular investment themes, a few broad ares of interest include:

regenerative medicine, bioinformatics, cloud storage, companies that use large data sets, online monetization and mobile.

There it is. Regenerative medicine right there front and center.

In typical Google tradition, Maris, who looks all of 30 years old on the website, has a successfull and multidisciplinary track record. He was involved in founding Web hosting pioneer Burlee.com (now part of Web.com), where he built much of the key computing, network and technological infrastructure.Prior to that, Bill was a biotechnology and healthcare portfolio manager for Stockholm, Sweden-based Investor AB. Bill’s background also includes research at the Duke University Medical Center, Department of Neurobiology.

Google Ventures is said to be aiming at investing about $100 million a year. Any portion of that for regenerative medicine is more than welcome.

While traditional VC money remains reticent to back RM in any signifant way, Google’s move confirms a trend we’ve been seeing and talking about at the Cell Therapy Group for the past 12 months or so. The multinational lifescience, biopharmaceutical, and healthcare companies along with strategic investors all now have regenerative medicine on their radar. They are all quietly and not-so quietly developing internal and external regenerative medicine strategies.

Please join us in welcoming regenerative medicine to the radar screen. It’s bound to be an exciting ride ahead.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Latest Cell Therapy Approval by FDA. Dendreon’s Provenge.

§ April 30th, 2010 § Filed under Regenerative Medicine Comments Off

It has been a long-time coming. It has been hyped and scoffed, bet against and hoped for, but now none of that matters. It’s here. Dendreon has brought Provenge to market. Here, in the word’s of the FDA…

FDA NEWS RELEASE

For Immediate Release: April 29, 2010

FDA Approves a Cellular Immunotherapy for Men with Advanced Prostate Cancer

The U.S. Food and Drug Administration today approved Provenge (sipuleucel-T), a new therapy for certain men with advanced prostate cancer that uses their own immune system to fight the disease.

Provenge is indicated for the treatment of asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment.

Prostate cancer is the second most common type of cancer among men in the United States, behind skin cancer, and usually occurs in older men. In 2009, an estimated 192,000 new cases of prostate cancer were diagnosed and about 27,000 men died from the disease, according to the National Cancer Institute.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Karen Midthun, M.D., acting director of the FDA’s Center for Biologics Evaluation and Research.

Provenge is an autologous cellular immunotherapy, designed to stimulate a patient’s own immune system to respond against the cancer. Each dose of Provenge is manufactured by obtaining a patient’s immune cells from the blood, using a machine in a process known as leukapheresis. To enhance their response against the cancer, the immune cells are then exposed to a protein that is found in most prostate cancers, linked to an immune stimulating substance. After this process, the patient’s own cells are returned to the patient to treat the prostate cancer. Provenge is administered intravenously in a three-dose schedule given at about two-week intervals.

The effectiveness of Provenge was studied in 512 patients with metastatic hormone treatment refractory prostate cancer in a randomized, double-blind, placebo-controlled, multicenter trial, which showed an increase in overall survival of 4.1 months. The median survival for patients receiving Provenge treatments was 25.8 months, as compared to 21.7 months for those who did not receive the treatment.

Almost all of the patients who received Provenge had some type of adverse reaction. Common adverse reactions reported included chills, fatigue, fever, back pain, nausea, joint ache and headache. The majority of adverse reactions were mild or moderate in severity. Serious adverse reactions, reported in approximately one quarter of the patients receiving Provenge, included some acute infusion reactions and stroke. Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5 percent of patients in the Provenge group compared with 2.6 percent of patients in the control group.

Provenge is manufactured by Seattle-based Dendreon Corp.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Stem cells for a Webby!

§ April 28th, 2010 § Filed under Regenerative Medicine Comments Off

I know I haven’t been a very good blogger for quite some time but I wanted to pass on a letter I just received from my friend James Price as a Charter member of the Canadian Stem Cell Foundation. They’re going for a Webby award. You don’t have to be Canadian to support their cause – you just have to:

• believe in the power of interactive, online network-based activism,
  
• support the potential of stem cells to change people’s lives, and
• wanna have a little fun raising awareness for our otherwise-sometimes-stodgy-science!

Go vote – it’s good for you and the world! (WARNING: you might find out some things about some pretty cool stuff nominated in the other categories while you’re there).

www.celltherapyblog.com hosted by www.celltherapygroup.com

Errata Statement re: Regenexx

§ March 23rd, 2010 § Filed under Regenerative Medicine Comments Off

It has been pointed out to me by legal counsel that the 25 July 2008 letter from the FDA to Regenerative Sciences, Inc regarding what FDA observed as apparent “violations” of FDA regulation by Regenexx, was not a “Warning Letter” as that term is officially defined but rather an “Untitled Letter” which has less or no formal effect.

My previous reference to the letter as a “warning letter” was a colloquial rather than legal use of the term. In any event, to avoid possible confusion, I hereby formally point out that the letter was not a “Warning Letter”.

Despite the FDA’s observations of what it then said appeared to them as Regenexx “violations” of FDA regulation in that Untitled Letter, since the date of the letter the FDA has not followed up with any Warning Letter, Cease and Desist Letter, or sought any other relief in court either in an action of its own or in the action brought against the FDA by Regenerative Sciences, Inc.

This fact has emboldened many to believe that the FDA will not take any action to remove Regenexx from the market despite its observations in 2008 that Regenexx appeared to violate the existing FDA regulatory framework.

Some take this one step further and believe that by its lack of action the FDA has demonstrated it now believes the product does not, in fact, violate the regulations. Others are convinced the FDA’s lack of action is merely a lack of action and not a change of conviction. In the face of a lawsuit by RSI, after all, the FDA has to pick its battles carefully.

Only time will tell…

www.celltherapyblog.com hosted by www.celltherapygroup.com

Cell Therapy Industry HiLites 2009-07-17

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

As Late, Late Show host, Craig Ferguson, says…”It’s a great day for America. ‘Why is it a great day for America,’ you ask? Well, let me tell you”.

The US House of Representatives has passed what promises to be (if approved by the Senate) the largest single budget increase for the FDA in the agency’s history. Now many an economist or financial wizard may argue why this is fiscally irresponsible given America’s economic woes but it certainly is promising for the biotech industry which has suffered due to a lack of resources in the Agency overseeing the sector. This lack of resources permeates into almost every interaction with the agency whether it be awaiting new Guidance, submission reviews, staff attrition, etc.

As it happens it is also a great day for the mother country from which America declared its independence because of their dastardly tea-taxing ways. “Oh, why ever it that so?”, you might ask with a fake English accent. Well I’lltell you. It’s because the UK Office for Life Sciences (OLS) has announced a “Life Sciences Blueprint” that will attempt to capitalise (well, that’s how the English spell it) fully on its “leading global position in regenerative medicine”. The OLS has announced £18 million funding from the Technology Strategy Board for a Regenerative Medicine programme (again with the English spelling) of investment to support key areas of commercial R&D and the development of R&D partnerships, supported by additional funding of £3.5 million from the Medical Research Council, the Engineering and Physical Sciences Research Council and the Biotechnology and Biological Sciences Research Council.
__________________________________________________________________

I fell in love with this conference, the venue, and the charming city of Leipzig when it was held in 2007. For a unique and international take on the multi-disciplinary world of regenerative medicine, you should consider meeting at the WCRM 29-31 October 2009.

www.wcrm-leipzig.com

___________________________________________________________________

Well I seem to be on a roll with anything related to Wake Forest recently so ‘why stop now’ I ask myself, without any compelling reply to the negative. This is just a little FYI. The Babcock Demon Incubator (BDI), a new venture incubator at WFU, promises to be a unique new venture incubator opportunity for regenerative medicine startups. The BDI has has fully equipped wet lab space with available equipment including chemical and tissue culture hoods, PCR, Gel systems, HPLC, microscopes, centrifuges, etc. In addition, BDI clients have access to collaborative opportunities at the Wake Forest Institute of Regenerative Medicine and to a wide range of services and specialized equipment. The incubator provides both residential and non-residential client models depending on client needs. All clients have access to office or lab space, can take advantage of business consulting, and access to an extensive services network designed to provide difficult-to-obtain resources to help young companies succeed. For more information, visit the BDI web site.

As regular readers of my Industry HiLites will know, from time-to-time I occasionally profile a company here in the front-end of the post providing some background, analysis, and recent developments. I’ll formalize this a little by calling it a COMPANY PROFILE but won’t promise to include one every time.

On another house-keeping matter you will note that I’ve changed the section title “FINANCIAL” to “FINANCIAL and DEALS”.

COMPANY PROFILE

Bioheart, Inc., (OTC Bulletin Board: BHRT) has announced that they will proceed to the final phase of their clinical trial with muscle stem cells for treatment of cardiovascular disease.

The company convened a panel of experts to review pre-clinical data dating back to 1988 and clinical data from controlled studies which began in early 2000 (involving nearly 400 patients). Bioheart’s latest Phase II/III MARVEL study has undergone interim analysis. While the data has not been disclosed pending publication confidentiality, this panel of experts was reportedly asked to also review this data.

The panel is reported to have “unanimously and enthusiastically endorsed moving forward” to complete the final phase of clinical trials (MARVEL) to advance muscle stem cell (myoblast) therapy for treating heart failure with the goal of bringing the product to market with FDA approval and CMS reimbursement.

It’s not entirely clear to me how or why this panel of experts was called together but a listen of the audio of the panel discussion frankly makes it sound a little bit like a love-in for Bioheart. In the accompanying slide deck, you will find an overview by Chairman and CEO, Howard J. Leonhardt listing an extremely aggressive list of deliverables for 2009 for a dizzying array of clinical products & programs.

On the financial front, Leonhardt states the company has to-date invested $125 million to support heart failure-related research – $85 million of which was in paid capital.

From its May 20, 2009 Form 10-Q Quarterly Report filed with the SEC, “The Company has incurred significant operating losses over the past several years and has a deficit accumulated during the development stage of $98.6 million as of March 31, 2009. In addition, as of March 31, 2009, the Company’s current liabilities exceed current assets by $12.7 million. Current liabilities include notes payable of $5.4 million. …the Company also has an obligation of $3 million to the Company’s Chairman and spouse [they repaid the principal on a company loan and a pro rata portion of accrued interest ]. The Company does not have sufficient cash to support its operations through December 2009. The Company will need to secure significant additional sources of capital in June 2009 to develop its business and product candidates as planned.”

On July 9, the company issued a press release saying that between October 1, 2008, and July 7, 2009, Bioheart received proceeds in the amount of $2,855,830 from the placement of restricted common stock and warrants under its current offering which it has now extended through October 2009. There’s a little positive spinning here because this would appear to include the approximately $1.8 million raised in the sale of stocks and warrants which it announced back in October 2008. The new ~$1M appears to be coming in dribs and drabs from the sale of small chunks of stock or warrants including, if I’m reading it right, the granting of stock in repayment of an outstanding loan (see the May 2009 Quarterly Report for clarification and details).

FINANCIAL and DEALS

Bioheart, Inc., (OTCBB: BHRT) has raised approximately $1 million over the past few month in an ongoing offering (see details above).

Aastrom Biosciences, Inc. (Nasdaq:ASTM) has gained a modest extension from NASDAQ to comply with the $1.00 minimum closing bid price rule in order to remain listed on the Nasdaq Capital Market. This means they now have until October 1, 2009 to regain compliance by achieving a $1.00 closing bid price for a minimum of 10 consecutive trading days anytime before that date. If not, they get kicked off the NASDAQ and forced to find another market for their listing. Absent a stock split it seems an unlikely goal give that the stock is currently trading in the region of .35 cents and the 52 range for the stock is 0.15 – 0.78.

Quantum Immunologics, Inc, a Tampa, Florida company focusing on the research and development of cancer immunotherapies, announced that the first patient in its breast cancer trial has begun to receive its dendritic cell therapy. QI is currently sponsoring and conducting an FDA-authorized Phase I/II clinical trial testing the safety and efficacy of its immunotherapy on 27 Stage IV breast cancer patients who have failed conventional therapy. Each patient will receive three monthly injections of the patient’s own dendritic cells that have been sensitized to OFA (oncofetal antigen) – a cancer antigen (a protein found on cancer cells that can be targeted by the body’s own immune system) found in many tumor cell lines or fetal tissue, but absent on normal, healthy tissue. When the sensitized cells are injected back into the patient, QI expects the patient’s T-cells will locate the OFA found on the patient’s cancer cells, thereby generating an immune response with the goal of killing the cancer cells and preventing further spread of the disease.

Harvest Technologies Corp. announced that the company-sponsored 60-patient clinical trial conducted in Chennai, India using the company’s BMAC point-of-care System to treat patients with non-reconstructable Critical Limb Ischemia (CLI) has completed enrollment. Over 300 CLI patients have been treated with the Harvest BMAC treatment protocol in an ongoing multi-center FDA study in the U.S. and other studies in India, Germany and the Czech Republic.

COMMERCIAL

Dendreon Inc (NASDAQ:DNDN) is currently eying Atlanta, GA as the location for a new $80 million, 300-person manufacturing facility. “Why Atlanta”, you ask? “Ah, good question”, I say. It all comes down to transportation logistics. An autologous cell therapy like Provenge requires the acquisition of a biological sample from the patient which is then used to manufacture a unit of the therapeutic that is then shipped back to the patient’s clinical stie for administration. This makes logistics a primary consideration for the business model. Atlanta, has one of the world’s busiest and most connected airports.

Cord Blood America, Inc. (OTCBB:CBAI) has signed a lease for a 17,000 square foot building in Las Vegas, Nevada, for a state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Aggressively the company intents to start processing and storing its customer’s umbilical cord blood stem cells in the facility in the fourth quarter of 2009. According the the company CEO, in 2010, CBAI intends to expand its cryogenic storage services to other forms of stem cells, including peripheral blood stem cell and adipose tissues. Presumably this move to self-manufacture will mean that its Corcell banking brand will no longer be able to claim the advantage of cGMP-quality processing and storage which it has made a part of its pitch since contracting with Progenitor Cell Therapy in 2007.

Meanwhile CBAI has picked the pockets of Bio-Matrix Scientific Group Inc (OTCBB:BMSN) by hiring its former Vice President and Chief Operating Officer Geoffrey John O’Neill as its new Laboratory Director and Brian Pockett as Vice President of Laboratory Operations.

Meanwhile in a weird twist of almost Seinfeld-like interconnectedness, in researching SEC filings for this post I discovered that on August 11, 2008, MSN entered into a Letter of Intent with CBAI regarding the processing and storage by BMSN of cord blood specimens newly acquired by CBAI for consideration acceptable to BMSN. It would appear, however, that deal never materialized given CBAI’s recent moves.

Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) has announced the formation of a subsidiary, LCT Biomedical Limited in Russia, to facilitate the commercial development of DIABECELL®, its lead product for the treatment of type 1 diabetes. Reportedly the regulatory process has already been initiated and it is envisaged that the pivotal study and product would be registrable by late 2011. They recently implanted their 8th patients with DIABECELL, their porcine xenogeneic cell therapy product in clinical trial for type 1 diabetes. The plan is for the product to be manufactured and supplied from New Zealand, where the company is located, for the foreseeable future.

Progenitor Cell Therapy, LLC has appointed a Vice President of Manufacturing Operations with extensive pharmaceutical production experience to head its two U.S. contract manufacturing facilities. PCT recent announced the recent appointment of Daryl LeSueur as Vice President of Manufacturing Operations. As head of Manufacturing Operations, Daryl is responsible for managing and supervising the day-to-day conduct of the manufacturing, packaging, and operational functions of PCT’s two North American contract manufacturing facilities.

MISCELLANIA

The ERA Consulting Group is proud to announce the next intERActions seminars entitled:

The Regulation of “Advanced Therapy Medicinal Products*” in Europe, from the Clinic to Approval (*Cell Therapy, Gene Therapy and Tissue Engineered Products)

Topics to be covered:
* The EU regulatory framework for Advanced Therapy Medicinal Products (gene, cell therapy and tissue engineered products)
* Opportunities in the EU regulatory landscape, especially for rare diseases: How can they benefit your company?
* Current and future regulatory guidelines: Latest developments and implications for your company and product portfolio
* What are the EU CMC/quality issues/requirements at various stages of development?
* Preclinical development program: EU regulatory expectations – rational approach
* Clinical trials with Advanced Therapy Medicinal Products in the EU

Including Europe in your Strategy to Add Value to Early-Stage Biotech Product Development

Topics to be covered:
* The value of including an EU component into your overall regulatory strategy
* Optimizing interactions with European regulators and how this can aid development and add value
* The benefits of orphan medical product designation in the EU and an explanation of the procedure
* Clinical Development in the EU: The European Clinical Trials Directive
* Opportunities in the EU regulatory framework to add value and reduce time to approval, such as accelerated review, conditional approval or exceptional circumstances
* Compassionate use programs

The seminars will be held at The GreenV Sustainable Center of South San Francisco on 14th – 15th September 2009. For a complete program and registration information, please visit their website at: www.eraconsulting.com.

After 15 years in the biotech conference industry, the Williamsburg BioProcessing Foundation has shuttered its operation in financial distress. Hasta la vista, Wilbio.

Sign-off

Ok, this is a stretch for inclusion in this blog which is why I leave it to this humble location in the post but two fellow Canadians have been named among the six researchers to receive prestigious awards from the American Society of Hematology later this year. Congratulations to stem cell researchers Connie Eaves and John Dick.

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www.celltherapyblog.com hosted by www.celltherapygroup.com

The true status of the cell therapy industry…

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

I apologize for the quality of this image but hopefully its just clear enough for you to make out this week’s Genetic Engineering News poll. If you’re reading this in realtime you will see on GEN’s homepage.

In case you can’t read it, the poll is this:

“Which type of stem cells will be the first to move from the laboratory into clinical testing?

• embryonic stem cells
• induced pluripotent stem cells
• adult stem cells
• none of the above
• undecided”
  

The introduction to the poll is as follows:

“The 2009 Lasker Awards honored two scientists who developed nuclear reprogramming, a process that instructs specialized adult cells to form pluripotent stem cells. This has been widely hailed in the life science research community and the biotech/biopharma industry as one of the most significant findings of the past decade. While embryonic stem cell research has been plagued by ethical concerns, some believe that the newfound ability to induce pluripotency in adult stem cells will lead to breakthroughs in the therapeutic applications of stem cells. So we want to know which type of stem cells you think will win the race.”

C’mon GEN, are you serious? If you honestly missed the point that adult stem cells are already in clinical testing and use, then shame on you. If you’re trying to catch people in their own ignorance by this trick question then I suppose that’s a little more tolerable but a poll like this still perpetuates the misinformation – at least until you do everything you can to expose it.

This kind of misunderstanding about the true status of the development of cell therapies is pervasive. Late last year I commented on an article by Tom Feilden on BBC’s website in which he said things like,

• “…a sizeable number of exciting stem cell projects are now reaching the stage where they should be moving on from the research lab and into clinical trials” and
  
• “Regenerative medicine may finally be moving out of the lab and into the clinic.”

As we all know, stem cell research moved from the lab into clinical use decades ago (with stem cell transplantation) and there are now literally hundreds of clinical trials currently underway testing the use of different kinds of stem cell products for almost every imaginable condition not the least of which is Osiris’ Prochymal which has been tested now in two phase III clinical trials.

Perhaps the educated readers of this blog will think that it is not possible that readers of GEN could believe that adult stem cells are not already in clinical testing. Let me assure you there is a lot of misinformation about the maturity of the cell therapy sector even among those in biotechnology. As proof, a click on “View Results” shows that as of today the poll stands almost neck-and-neck. A mere 48.6% of respondents believe adult stem cells will beat ESC or iPS cells to clinical testing. 45.7% believe either ESC or iPS cells will beat adult stem cells into clinical trial.

What more can I say? We – you and I – must continue to do everything we can to raise awareness about the true status of the cell therapy industry and the products we are developing – even among our biotech colleagues.

–Lee

p.s. I know my Cell Therapy Industry HiLites has disappeared this summer. I’m doing everything I can do resurrect it from among the rubble left by summer holidays, home renos, new baby, and more work from clients than I could have ever expected.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Cell Therapy Industry HiLites 2009-06-26

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

Let’s get the public services announcement out of the way right up front.

• CellTherapy 2010. Save the date. Submit your session ideas. www.celltherapy2010.com

• While you’re planning your calendar, plan on the 2009 World Stem Cell Summit September 21-23, 2009. Baltimore Convention Center Baltiomore, Maryland. (the 2008 World Stem Cell Summit webcast and downloadable World Stem Cell Report are at www.worldstemcellsummit.com).

• Curiously, the Regenerative Medicine Foundation (I know. I hadn’t heard of it either until now.) has announced that its “Translational Issues in Regenerative Medicine” conference is the “premiere event for leaders in regenerative medicine translation, representing academic research, clinical, healthcare policy, regulatory, reimbursement, investment and biotechnology industry interests.” Curious because it has yet to ever occur. That being said, if they do what they promise, it may just well be. I’m trying to learn more and when I do I’ll let you know. Meantime, this is about all I know:
  

Spring 2010
Twin City Quarter
Winston-Salem, NC

“…to facilitate business development opportunities, the conference will feature a venture forum including 15 regenerative medicine companies, as well as partnering sessions.“

____________________________________________________
Do yourself a favor. Stop monkeying around with home-made cryopreservation brew or, for than matter, any media not built to minimize cellular damage from freezing and thawing.

Do your cells a favor. Try CryoStore and HypoThermosol.

www.biolifesolutions.com

_____________________________________________________

I’m dedicating this issue of Cell Therapy Industry HiLites to TiGenix NV (NYSE EURONEXT: TIG). TiGenix wins the Cell Therapy Group “kick-ass cell therapy company of the week” award which is, of course, something I just made up but they had have a great week raising several million dollars, announcing plans for a new European facility, and receiving a positive opinion from the EMEA CHMP which all but assures them of an MAA within 90 days applicable in 30 European countries. See details below.

FINANCE

TiGenix (NYSE EURONEXT: TIG) has secured the necessary financing to fund the set-up of a GMP manufacturing facility in Europe through a private placement for a total amount of EUR 5,4 million. The investment agreement was subscribed by NV Industriebank LIOF, Particon BV, Limburg Ventures BV and LRM NV. The funds will be used to set up a new cell expansion facility in Europe for the commercial production of its cell based products in the Netherlands. The financing for the facility has been secured through a private placement. After carefully evaluating a number of options throughout Europe, taking into consideration technical, logistical, regulatory and financial criteria, TiGenix has selected a building on the Chemelot Campus, near to Maastricht, the Netherlands, to locate its new cell expansion facility. The site is centrally located in TiGenix’ key European markets, in a region that is strong in distribution and (bio)logistics and that is highly committed to develop as a transnational knowledge centre in life sciences and regenerative medicine.

This financing has now been completed following the execution of a long term lease agreement for the facility in question.

**

After raising $40+ million last year and over $14 million so far this year, Cytori Therapeutics Inc (NASDAQ: CYTX) has announced it has entered into an agreement to raise up to another $20 million over the next year. The company announced recently that it had raised $850,000 in what is described as an agreement by Orlando, FL-based fund, Seaside 88, to buy 7.15 million shares over the next year by buying a specified allotment every two weeks at an agreed discount of the then current stock price. The agreement could being in roughly $20 million for the company depending on share price. Cytori intends to use the proceeds to “accelerate marketing efforts and continue our investment in clinical trials to broaden the number of potential applications for which the Celution® System may be applied”.

**

A bankruptcy court hearing is scheduled for July 6 in which Isolagen Inc. (AMEX:ILE) will request final approval of its debtor-in-possession financing plan under Chapter 11 bankruptcy reorganization protection.

**

Always a master at the art of spinning good profile, NeoStem, Inc. (NYSE: NBS) (AMEX: NBS), in the business of collection, processing and long-term storage of adult stem cells for future medical need, garnered a feature in Gene Marcial’s prominent Business Week column, “Inside Wall Street,” on June 18, 2009. The same week saw the company profiled in a piece on the investment potential for adult stem cell businesses in a Fortune Magazine article distributed by CNNMoney.com. The company has been on a deal-making, press release and publications tear the past few months. “Big plans by tiny NeoStem (NBS),” Mr. Marcial observed, “have helped catapult its stock to $2.27 a share from 50 cents on March 9.” It must be the plans because the company which has been doing what it does since 2006, generated a mere $45,100 in total revenuefor the three months ended March 31, 2009. The good news is that is up from the $700 generated in the three months ended March 31, 2008.

The company also announced another deal saying it has signed an exclusive 10-year agreement with Enhance BioMedical Holdings Limited, a Shanghai corporation, to develop a Stem Cell Collection and Treatment Network using NeoStem’s “technologies” Taiwan, Shanghai and the Chinese provinces of Jiangsu, Zhejiang, Fujian, Anhui and Jiangxi. Enhance BioMedical Holdings is a subsidiary of Enhance Holding Corporation, a multinational conglomerate whose CEO, Jackson Ling, recently invested $5 million in an $11-million private placement financing for NeoStem.

CLINICAL

Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced six-month interim data from a Phase II clinical trial evaluating Prochymal, the Company’s formulation of adult mesenchymal stem cells, for the treatment of chronic obstructive pulmonary disease (COPD). The trial failed to demonstrate an improvement in pulmonary function in COPD patients. The company, quickly becoming practised at spinning what might be considered bad news, said they were pleased the data did provide additional evidence of safety for the product in addition to demonstrating significantly decreased inflammation in the COPD patients. Sixty-two patients were enrolled and are being followed for two years in the placebo-controlled study.

**

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX: PJT) received approval from the Paul Ehrlich Institute to begin clinical trials evaluating the company’s placental-derived adherent stromal cell product, PLX-PAD, for the treatment of critical limb ischemia.

**

Aastrom Biosciences’ (NASDAQ:ASTM) trial of its cell therapy for severe chronic heart failure was released from a clinical hold by the FDA. The the agency determined the therapy did not cause the death of a participant which resulted in the hold in May.

**

The University of Pittsburgh Cancer Institute began enrolling patients in a Phase III study evaluating the safety and efficacy of Gamida Cell Ltd’s StemEx cord blood stem cell therapy for the treatment of blood cancers.

**

Having received regulatory approval late last year to proceed with its clinical trial in New Zealand, Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) CEO Paul Tan said enrolment of patients should be complete in about two months to begin testing their pig islet cells implanted in humans to treat diabetes.

**

Neuralstem, Inc. (NYSE Amex: CUR) has entered into a sponsored research agreement with the China Medical University & Hospital of Taiwan, in Taichung, Taiwan, to prepare for a human clinical trial using Neuralstem’s human spinal cord neural stem cells to treat stroke patients. The therapy will focus on patients whose post-stroke symptoms, including complete or partial paralysis, have stopped improving more than six months after an ischemic stroke.

COMMERCIAL

Not to be outdone by finance and operational teams, the regulatory team at TiGenix also announced reason to celebrate this week. TiGenix (NYSE EURONEXT: TIG) announced that the company received a positive opinion from the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) on the European Marketing Authorisation application (MAA) for
its lead product ChondroCelect.

CHMP opinions are forwarded to the European Commission for adoption of the final community Marketing Authorisation, which is typically issued between 60 and 90 days from adoption of the CHMP opinion. I’m told that European Commission generally follows the advice of the CHMP but, as Dendreon, there’s little reason to celebrate until the formal approval is formalized. For the EMEA take and related info on the decision, click here.

Upon approval, ChondroCelect will be, I believe, the first product approved under the new ATMP regulatory framework. It will certainly be the first cell based product to be centrally approved in the 27 member states of the European Union as well as in Iceland, Lichtenstein and Norway under the new Advanced-Therapy Medicinal Products (ATMP) legislation.

**

Further to my March 31 speculation about who might be the buyer of York Pharma plc (and its two cell therapy products on the market) and my April 13 post that ULURU Inc. (NYSE Amex: ULU) had signed a non-binding offer letter to acquire the company, ULURU Inc. (NYSE Amex: ULU) has announced that at this time discussions to acquire York Pharma, plc have been terminated. ULURU has notified York that the $1 million USD line of credit it had extended York are due plus accrud interest. The revolving credit facility is secured by substantially all of the assets of York and its subsidiaries. York is now actively pursuing alternative funding facilities in order to repay the line of credit within 30 days and to meet its ongoing working capital requirements.

Meanwhile, rumor has it the company is already in talks with another potential suitor.

**

Here are a couple hints for your next press release. It’s rarely newsworthy to have as the primary point of your release an announcement about what you are going to do, rather than what you’ve done; secondly, don’t announce seemingly contradictory information. I’m just saying….

Stem Cell Therapy International, Inc. (OTCBB: SCII), through its wholly owned subsidiary AmStem International, Inc., announced that it has “started the process” to enable it distribute Histostem’s products in anticipation of the finalization of the merger between Stem Cell Therapy International, Inc. and Histostem. This is intented, according to the SCTI President and CEO, to “enable AmStem International, Inc. to begin US distribution and immediately bolster the company’s cash flow”. However, the product has yet to be the subject of a clinical study in the U.S. for which they are seeking “investigators…of the highest caliber, to compare its Stem Cell Facial Cream with others on the market.”

The companies reportedly continue “to work on the necessary steps to finalize the merger, and both sides are working diligently to manage its many details.”

**

Community Blood Services has entered into a licensing agreement with AuxoCell Laboratories, Inc. to use AuxoCell’s exclusive patent rights and propriety process to extract fetal mesenchymal stem cells from the Wharton’s Jelly of donated umbilical cords, allowing Community Blood Services to operate the first Wharton’s Jelly derived stem cell bank in the United States.

**

After netting $221 million last month by nearly 12 million shares at $19.20 apiece, Dendreon Therapeutics Inc (NASDAQ:DNDN) expects to spend up to $50 million in a two-phase expansion of its manufacturing facility in Morris Plains, New Jersey. Monday in a regulatory filing. Additional quality control laboratories, data center, training areas, infrastructure and offices are scheduled to by done by mid-December. The final phase, with additional manufacturing clean-room work stations, production support areas, warehouse, infrastructure and offices, is to be substantially complete by April 23 of next year. Dendreon finished the initial build-out of its 158,000-square-foot facility in July 2006, before its FDA application was tabled to await additional clinical results.

**

Apparently yet another way has been invented to derive stem cells from placenta – reportedly a source much richer in stem cells than umbilical cord blood. The inventors are looking for commercial partners.

**

Cell Genesys, Inc. (NASDAQ:CEGE) – the company that just won’t go away – is still pursuing “strategic alternatives”. Meanwhile, it expects its cash balance will be approximately $36 million upon imminent completetion of its stock exchange offer.

MISCELLANIA

This week’s issue of Science has a special focus on stem cells including a piece on “FDA Regulation of Stem Cell–Based Products” by Donald W. Fink, Jr. of FDA, CBER, an article on stem cell tourism and 3 reviews. 26 June 2009. Vol 324, Issue 5935, Pages 1603-1754

**

The Production Assistance for Cellular Therapies Group (PACT) Educational Web Seminar: Topic: “Deviation Management of Type 351 & 361 Cell Products” Date: Thursday, July 16, 2009 Time: 12:00pm-1:00pm (Eastern US Time) Register is now open for this Web Seminar at www.pactgroup.net.

Sign-off

Thanks again to BioLife Solutons. Congratulations to Tigenix. May the force be with all of you and for those going to ISSCR later this week….safe travels and please try not to enjoy Barcelona too much without me!

www.celltherapyblog.com hosted by www.celltherapygroup.com

Cell Therapy Industry HiLites 2009-07-03

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

Last week I mentioned the Regenerative Medicine Foundation and the conference they have announced for Spring 2010. I promised to find out more and bring you what I learned.

I’ve discovered that the driver behind the RMF is Anthony Atala and Case Western. It is a 501(c)(3) charity, non-profit organization. It was formed in 2005 by Dr. Atala to host a meeting with the U.S. Food and Drug Administration (FDA) around the topic of regenerative medicine. In 2006, the Foundation was instrumental in the formation of STRAC, the Soldier Treatment and Rengeneration Consortium, a national partnership of leading military and academic research centers and industry. STRAC formed the basis for the Armed Forces Institute of Regenerative Medicine (AFIRM).

Now the Foundation announced the first annual “Translational Issues in Regenerative Medicine” Conference, to be held in Winston-Salem, NC, December 8-10. The conference promises to focus on best practices in clinical trials and GMP facilities, as well providing a forum to discuss regulatory and reimbursement challenges. In addition, the conference will feature a venture forum to introduce early-stage and later-stage companies to accredited and institutional investors.

As its name suggests, the organization functions like a “foundation” rather than a member-based organization. This is reinforced by the fact the “Participate” page on the website has no content.

All of this is interesting but nothing more than you would learn from the RMF’s website. What I DID discover this week that isn’t obvious from anything yet online is that Atala has also been a primary driver behind the creation of a newly announced organization, this one a 501(c)(4) organization intended to be much more about lobbying.

This week the Alliance for Regenerative Medicine was formerly launched. (no website yet). Headquartered in Washington, DC, the Alliance is “dedicated to promoting regulatory, research, and reimbursement policies that will foster innovation in regenerative medicine” in addition to serving as a “source of information about regenerative medicine for policy makers, the media, and the general public”.

With Atala’s ‘encouragement’, Michael Wener of DC law firm Holland & Knight and Morrie Ruffin of Adjuvant Global Advisors signed up to be the driving force in the creation and evelopment of the Alliance intented to bring together academic institutions, non-profit organziations, and companies that commit to supporting and participating in a lobbying organization to promote much-needed policies in support of the regenerative medicine sector.

The organization intends to announce its final list of charter members later this month. Thus far its a respectable list including Wake Forest Institute for Regenerative Medicine, Stanford University, the University of Washington, Georgia Tech University, the Genetics Policy Institute, Geron, Johnson & Johnson, Aldagen, iZumi, Fate Therapeutics, Maxcyte, Kleiner, Perkins, Caufield and Byers, and Proteus Ventures.

Werner and Ruffin both have a great deal of experience with Capitol Hill and the biotechnology sector. I had a great conversation with Werner this week and am very encouraged by the plans for the Alliance and their intended inclusiveness. In my opinion, the Alliance promises to fill a much-needed gap unmet by any existing organizations. I will dedicate a post to more information about the Alliance in the days to come.

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More stakeholders united in one place at one time than any other.

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FINANCIAL

Here’s the cool part: Check out their product animation on YouTube.

CLINICAL

Ok, this isn’t exactly a clinical story but it’s the closest thing I’ve got this week and I hate leaving sections bare.

Geron Corporation (Nasdaq: GERN) has published in advance of print in the journal Regenerative Medicine a paper that reports that dendritic cells (DCs) scalably manufactured from human embryonic stem cells (hESCs) exhibit normal functions of naturally occurring human DCs found in the bloodstream. This, they say, supports their planned use of hESC-derived DCs in their therapeutic vaccine currently in Phased II for AML but employing autologous dendritic cells. (see GEN’s summary here)

COMMERCIAL

The big deal of the week, of course, was between GE Healthcare, a unit of General Electric Company (NYSE: GE), and Geron Corporation (Nasdaq: GERN).

The companies announced an agreement to co-develop human embryonic stem cell (hESC)-derived assay products for in vitro drug screening with the aim of having products on the market in by “early 2010″.

Under the worldwide, exclusive license and collaboration agreement, GE Healthcare obtained an exclusive license to Geron’s hESC IP and a sublicense under Geron’s rights to foundation hESC patients held by the Wisconsin Alumni Research Foundation. IP arising from the alliance will be shared between the companies, with GE Healthcare retaining rights to the resulting technologies for drug discovery applications and Geron for cell therapy applications.

When Geron announced that its IND had been accepted by the FDA earlier this year, Okarma made quite a bit about how they had figured out how to scale the production of their product ” unlike adult stem cell therapies” and that their product was “scalable in the same way as a monoclonal antibody”.

This theme continues in this announcement with Geron’s David Earp saying ““Geron is intensely focused on developing hESC-based cell therapies, and the expertise that we have developed in scalable manufacturing and differentiation of hESCs to specific cell types is directly applicable to the production of these dells for drug discovery.”

Interestingly, GE claims it is bringing “expertise in cell manufacturing” to the deal. GE Healthcare will fund the R&D program and will be responsible for manufacturing, sales and distribution of products developed under the agreement.

Click here for more background information

As has been discussed here several times, ever since Cell Genesys Inc (NASDAQ: CEGE) pulled the plug on its late-stage trials of its GVAX immunotherapy triggering Takeda Pharmaceuticals to pull out of its co-development deal for the product, Cell Genesys has been busy dealing with downsizing, lawsuits, and shopping itself around to potential suitors looking primarily to take it on for its cash.

This week BioSante Pharmaceuticals, Inc. (NASDAQ: BPAX) anted up to announce it is merging with Cell Genesys in an all-stock transaction valued at about $38 million – pretty much the exact amount of cash CEGE has in its bank account. the surviving entity will be BioSante.

Cash value is actually a generous purchase price for biotech companies which usually come with a burn rate that makes then worth less than their cash. CEGE, however has no clinical program, had sold most of its assets, and had downsized to 9 staff in a rented office.

There are, of course, persistent rumors of back alley shenanigans that took place in CEGE’s demise but we’ll leave these on the street. What is interesting to me is that BioSante has suggested they will examine options for the future of Cell Genesys’ GVAX Immunotherapies, including potential combination with BioVant, BioSante’s vaccine adjuvant, as well as possible external collaborations. The combined entity is also on record saying they will try to outlicense other Cell Genesys technologies.

GEN this week carries an interesting article on a company applying its existing technology to cell therapy bioprocessing scale-up. The article sites “…large-scale culturing of embryonic and induced pluripotent stem (ES, iPS) cell lines for use in regenerative medicine and drug discovery applications” as still a “major challenge” for the industry. Cyntellect, Inc. reports that its Stem Cell Manager “is a series of stem cell management processes including automated physical passage of stem cells, automated embryoid body generation, and purification of specialized cell types derived from stem cells” which can help alleviate some of the major challenges in large-scale automation of the processes required to manipulate sensitive therapeutic cells.

MISCELLANIA

ProChon Biotech, Ltd., a company working with tissue regenerative technologies announced that it recently received the Genzyme Award for Excellence in Cartilage Research for its poster presentation entitled, “Comparative Analysis of Cell Sources for Cartilage Cell Therapy.” The company reports that the research recognized by the award is incorporated into their development pipeline and will compliment its BioCart™ Cartilage Regeneration System.

Prochon’s BioCart^™ Cartilage Regeneration System is an autologous chondrocyte implant system that – and here’s why I love this story because this is just another example of a commercially-available cell therapy that people in the US never talk about – is commercially available in Italy, Greece and Israel and is undergoing an FDA Phase II multicenter clinical study in the United States. The company reports that to-date over 70 patients have received the BioCart^TM implant with some patients over five years post-implantation.

I came across a nice piece this week discussing Arteriocyte Medical Systems‘ work on its DARPA-funded project to create human blood from stem cells at a rate that could allow the military and hospitals to produce units of blood on site. The company claims that with a technology developed by a company called Nanex, they are able to grow stem cells at a rate 10 to 100 times faster than with more traditional methods. The Nanex technology mimics a bone marrow environment in which stem cells can grow quickly. The trick now is to automate and scale-up the system to meet the specs DARPA required in its project paramaters.

Sign-off

The latest issue of the journal Regenerative Medicine has been released and it looks well worth the read. Here the table of contents.

Hope this was useful.

www.celltherapyblog.com hosted by www.celltherapygroup.com

SBIR Funding – Should it be open to VCs?

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

I don’t have much time to give you an extensive blog about the issue but I did want to bring this to your attention. Here’s the nutshell.

The NIH’s SBIR (Small Business Innovation Research Program) funding mechanism has been an important one for small businesses who are not big/mature/saavy/desperate-enough to land venture capital funding, have very limited access to other NIH funding, and have tapped out the available angel/seed/friends_n_family resources at their disposal.

Since 2003, one of the hallmarks of the rather small SBIR funding pot ($2.2 billion a year) has been that it was not available to companies that are majority-backed by venture capital. Before 2003, venture-backed firms were allowed to participate in the SBIR program.

The program is scheduled to expire next month and so is up for renewal. The House committee responsible for the program has proposed that the VC-restriction be removed thus re-opening the program to a whole slew of companies previously ineligible. The House just passed this bill. A senate version of the bill tries to reach a compromise by limiting the amount available to venture-backed firms.

Whatever your opinion on the judiciousness of this move, there is no doubt that opening up eligibility to the program will dilute the available funding.

Here is a link to yesterday’s article on the subject in the Washington Post.

Fellow blogger Steven S. Clark (BioScience Biz) has a just posted his most recent update and opinion on the matter that is well worth the read.

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Cell Therapy Industry HiLites 2009-06-12

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

“Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan. “Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive“. GEN News Highlights. 12 June 2009.

Amen. Glad to hear someone else saying it.

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FINANCIAL

Neuralstem, Inc (NYSE AMEX: CUR) received notice from the NYSE Amex Exchange that its shareholder’s equity has dropped below the $2,000,000 threshold required by the NYSE Amex for continued listing. The Company intends to submit a plan to the Exchange. CEO and President Richard Garr said, “We are confident that the company will be in compliance.”

National Institutes of Health has awarded a $5 million grant to Q Therapeutics (co-founded by former NIH researcher Mahendra Rao), the University of Utah’s Cell Therapy Facility and Johns Hopkins University School of Medicine to start human clinical trials of Q-Cells for myotrophic lateral sclerosis, or ALS — better known as Lou Gehrig’s disease.

Opexa Therapeutics, Inc. (NASDAQ: OPXA) received a favorable ruling from NASDAQ allowing an extension to regain compliance and continue the listing of the Company’s securities on The NASDAQ Capital Market.

CLINICAL

StemCells, Inc. (NASDAQ: STEM) announced positive data from its Phase I clinical trial of its proprietary HuCNS-SC® product candidate (fetal-derived purified human neural stem cells). The six-patient Phase I trial was designed primarily to assess the safety associated with transplanting HuCNS-SC cells as a treatment for infantile and late infantile NCL, often referred to as Batten disease

Celsense, Inc. is one of the recipients in a 4-year $1.95M grant from the National Institutes of Health to test its in vivo cell trafficking MRI reagent in human clinical studies. The funded studies will be the first effort to evaluate Cell Sense, the Company’s fluorocarbon-based magnetic resonance imaging (MRI) cell tracking reagent, for clinical use. Additionally, the studies will garner preliminary data critically important for improving immunotherapeutic delivery strategies in metastatic colorectal (CRC) and other forms of cancer.

The Cell Sense technology makes transplanted cells visible by MRI. Therapeutic cells are labeled ex vivo with the Cell Sense reagent prior to administration to the patient. Cell migration and biodistribution are subsequently monitored using fluorine-19 (19F) MRI or magnetic resonance spectroscopy (MRS).

The key advantage of Cell Sense is purported to be that the 19F images are extremely selective for the labeled cells, with no background signal from the host’s tissues. Furthermore, accurate cell quantification in regions of interest is possible. Existing data from pre-clinical studies using human cells is said to show that the Cell Sense reagent is highly biocompatible inside cells and does not affect their innate function.

A clinical-grade version of the Cell Sense reagent has been formulated by Celsense, Inc. and is currently being subjected to mandatory in vitro and animal safety studies in preparation for human trials.

Never a company to shy away from making bold and early claims, Advanced Cell Technology, Inc. (OTC:ACTC.PK) Chairman and CEO William M. Caldwell IV said earlier this week that they are completing the “finishing touches” on a submission to seek FDA approval to conduct human clinical trials with a cell therapy that may be able to address many of the 200 plus known retinal diseases.

COMMERCIAL

It’s hard to put this story under any one category but since the financial terms weren’t released and it’s not yet clinical, I’ll call it “commercial”. J&J’s Ortho Biotech Oncology Research & Development, a unit of Centocor Research & Development, Inc., has announced that it has entered into a five-year Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), with Steven A. Rosenberg, M.D., Ph.D., chief, Surgery Branch, serving as the NCI principal investigator, to research and develop novel cell therapy technologies as potential treatments for a variety of cancers.

The deal bring together Ortho’s and Rosenberg’s adoptive immunotherapy technologies designed to work by helping the immune system fight cancer.

Rosenberg has been a pioneer in the field of adoptive immunotherapy of cancer developing Tumor Infiltrating Lymphocytes (TILs) – T cells obtained from a patient’s tumor, expanded and then re-administered to actively seek and destroy cancer cells. In recent years, Dr. Rosenberg’s team developed a new technology in which T cells obtained from a patient’s blood are genetically engineered to express receptors that give them specific immunity against cancer cells and then re-administered.

Meanwhile, researchers at Ortho Biotech Oncology Research & Development independently developed a different and proprietary adoptive immunotherapeutic approach that uses tumor antigens and other materials to stimulate T cells from a patient’s blood to become Cytotoxic T Lymphocytes (CTLs), which recognize and attack tumor cells.

Both technologies show postive early clinical results in melanoma, a type of skin cancer that ranks sixth among U.S. men and seventh in U.S. women for the most commonly diagnosed cancer, according to the NCI.

Under the CRADA, Dr. Rosenberg’s lab will conduct a clinical trial in melanoma patients using Ortho Biotech Oncology Research & Development’s proprietary technology. It is hoped that the technology will be effective in other types of cancer, as well.

They expect to begin clinical testing of the immunotherapy technology in melanoma patients by the end of 2009, with the possibility of additional studies for other types of cancer and other technologies in years to come.

Cellular Dynamics International, Inc. has secured an exclusive license on a patent portfolio surrounding cardiovascular progenitor cells. The license covers differentiation of stem cells into all cell types of the human heart. The patent portfolio was generated by and obtained from Gordon Keller, Ph.D. and Mount Sinai School of Medicine.

Cardiovascular progenitor cells are capable of further differentiation into the multiple cell types of the heart, including cardiomyocytes, endothelial cells, and vascular smooth muscle cells, both in vitro and in vivo. CDI intends to commercialize the technology to the pharma and biotech industry for predictive toxicology and screening purposes. Iit’s not clear whether the license also includes clinical applications but if so they may be looking for licensing partners given their current lack of interest in developing these cells into therapeutics.

In a not-so-surprising move, StemCells Inc (NASDAQ: STEM) has decided to close StemCell Sciences in Melbourne, Australia after its acquisition of the company in March. British staff will now be holding their breath to see if the same will happen to them. STEM says it has offered relocation packages to employees should they wish to move to sunny California. This leaves one “stem cell” firm in Australia where previously there were four (SCS, Bresagen, Embryonic Stemcells Inc and Mesoblast). If there is good news it is that, if I recall correctly, the three others were acquired and not just shuttered.

After many months of actively looking to offload its contract manufacturing subsidiary, Fresenius Biotech, has finally sold EUFETS AG to BioNTech AG. BioNTech is looking ot leverage the acquisition to further its quest to develop innovative molecular immunotherapies and biomarker based diagnostic approaches for individualized treatment of cancer and other severe human diseases.

StemCyte Therapeutics is collaborating with Chennai-based Apollo Hospitals in joint venture with Ahmedabad-based Cadila Pharmaceuticals to set up what reports to be a first-of-its-kind umbilical cord blood (UCB) public bank for India.

“No more pads or tampons, period.” Lifecell International is set to introduce in India next month menstrual cups as a cost-effective option to control menstrual flow in women and as a preparatory move for the company’s menstrual-blood banking project called Femme, which is scheduled to begin in July

MISCELLANIA

Xconomy’s Luke Timmerman does a nicewriting up an informative piece on Denderon’s recent AGM and the thinking around the company of what will be involved in taking the company “all the way”.

Ok its another pre-clinical item (which I rarely cover) but again this one’s just too cool to ignore. The New Scientists did a nice job of looking at Doris Taylor’s work at University of Minnesota. Her team is looking to expand on their success creating rat hearts using stem cells. The ultimate goal is to some day create a limitless supply of transplant organs for humans. The team took stripped-down scaffolding of one rat heart and coated it with tissue produced from another rat’s stem cells. They now want to repeat the procedure using hearts and other organs from human cadavers or larger animals.

The NIH received 49,015 comments in response to its draft guidelines for human stem cell research. One of those was submitted by CIRM.

President Barack Obama should appoint a commission on stem cell research to help the therapy fulfill its promise to save lives and reduce medical costs, says Geron Corp. Chief Executive Officer Tom Okarma.

The California Institute for Regenerative Medicine (CIRM) received 73 preapplications for the CIRM Disease Team Awards RFA 09-01. The principal investigator for 15 of those applications is based with a for-profit entity. All of the teams proposed a wide variety of collaborators with several of the not-for-profit principal investigators citing collaborators in the for-profit sector. After review by a panel of outside experts and the CIRM in-house science team, a subset of these teams have been asked to prepare a full application, which will be due to CIRM July 16. Word on the street is that to-date just over 20 of the 73 have been given the green light to the next step.

Sign-off

Organogenesis, Inc has established an annual scholarship program with Canton High School to honor a graduating senior pursuing a career in life sciences research or biomedical engineering. Kudos for them. We need more of that! Me? I sponsored a child this week. No big deal but it reminds me to remind you…

…find a way to make a difference in your world today.

One more thing. You want to make sure you hear cell therapy-related news faster? Get on Twitter and follow me. I’m @celltherapy.

Ok. Hope you found that useful. I’m trying to get back to my every Friday schedule here. I’ll do my best. Happy weekend.

Lee Buckler, BEd, LLB
Cell Therapy Group
Principal

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A Therapeutic Long Tail. Does Cell Therapy Fit the Model? (Part I)

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

I’m always skeptical of companies or products or technologies that depend on a ‘paradigm-shift’ or ’sea-change’ in the long-entrenched habits of people before they will be widely adopted and/or successful.

It’s why, for example alternative fuel autos might succeed in the U.S. where public transit has not – because they don’t require as big a change. People can stay in their car and change their habits less.

Disruptive Technologies Lead to Paradigm Shifts

Nevertheless there are from time-to-time disruptive technologies that do arise which are paradigm-shifting. These are readily (though perhaps not quickly) adopted because the benefits are seen to outweigh the trade-offs and/or effort required to make the change. In many instances there are entire industries built around the pre-existing technologies that foolishly resist such change rather than learn to adopt and adapt. It’s the classic mistake of railway companies believing they were in the business of trains rather than transporting people and goods and thus missing the opportunity to dominate transport by road and plane when these industries started to compete for the transport customer.

Will Cell Therapies Disrupt Sufficiently to Shift the Therapeutics Paradigm?

There are a lot of people talking about how regenerative medicine, personalized medicine, and cell therapies are going to revolutionize health care. I’m one of them. There are, however, many good reasons to be skeptical – not the least of which is the sheer size of the industries built around the pharmaceutical model and the power they can exert in resisting change. This might be comparable to Detroit’s resistance to alternative transport systems or Houston’s resistance to the use of fossil-fuels. It’s massive.

But just as Detroit is now embracing the need for change and Houston is touting alternative energies, could it be that New Jersey is having its own ‘come-to-Jesus’ moment and realizing there may be – perhaps must be – a future for pharma beyond the blockbuster pill model? The other big ‘industry’ to convince is the healthcare inudstry. How readily will hospitals and doctors adapt to a therapeutics paradigm shift?

Last week, I had an interesting conversation with someone from a struggling cell therapy company that has completed a semi-successful phase II trial and is very quickly running out of money despite having been on a road show for over a year. They are engaged in a “number” of “partnering” discussions with pharma companies who understand the science, the manufacturing, and the cost-of-goods data but say they are still uncomfortable with the “business model for cell therapy.”

The know the market, have good data on the COGs and can assume a certain product price and reimbursement…so what’s left? I’m told that “business model” is code – at least in this instance – for two concerns:

(1) what will clinical adoption rates be like, i.e., will doctors adopt these new therapies and, if so, at what rate? and
(2) the model they are being presented does not offer them the type of margins they’ve come to expect.

Let’s look at each of these in turn.

Health 2.0

Doctors have traditionally been driven by clinical results and reimbursement. But there’s a new driver in town – it’s the informed patient and their participation in Health 2.0. Patients no longer rely solely on their physician for healthcare information.

Deputy Editor of Wired magazine, Thomas Goetz, author of “The Decision Tree” (blog and upcoming book), describes this new era of healthcare as follows:

“We are entering a Third Phase of medicine – one that spots disease and illness based on risks rather than one that responds to symptoms (phase 1, from pre-history to the mid 19th c), or one that seeks out the causes (phase 2, from circa-1850 to just about now).

These three phases closely parallel our technological capacity to spot disease: Phase 1 was primed to spot symptoms because humans relied on our senses – and nothing more – to recognize illness. If we couldn’t see it (or smell it), it wasn’t yet evident. Phase 2 emerged with the perfection of the microscope and the discovery of the germ theory of disease: finally we could see disease in its pathogenic form – bacteria, mostly – and thus began a race to find the little buggers that were causing disease. The late 19th century saw a flurry of diagnosis, as the cause of one disease after another was identified, and the Pasteurs and Kochs went about devising antibodies or vaccines to vanquish them… So now this third phase dovetails with the bevy of new diagnostics emerging – devices and techniques that look for certain molecules and DNA strands to not only detect the presence of disease, but predispositions towards certain conditions as well…

This third phase contains elements of what many are calling “personalized medicine,” but I hate that term – it sounds too servicey and implies that this is simply regular medicine/clinical practice that’s been tailored for you, as if we have a computer watching our health (it’s “personalized!”).

To my mind, what’s happening now is much more than simply a move to personalization; it’s an entirely new way of relating to disease and illness, one that’s as impactful and as new as the realization that germs – not bad air or bad luck – caused disease. What’s happening now makes disease/illness an optional state, one that can be opted out of (ideally) or engineered against (more likely).”

This is, he says, “…a new phase of health and medical care, where more decisions are being made by individuals on their own behalf, rather than by physicians, and that, furthermore, these decisions are being informed by new tools based on statistics, data, and predictions. This is a good thing – it will let us, the general public, live better, happier, and even longer lives. But it will require us to be stewards of our health in ways we may not be prepared for. We will act on the basis of risk factors and predictive scores, rather than on conventional wisdom and doctors recommendations.

We will act in collaboration with others, drawing on collective experience with health and disease, rather than in the isolation and ignorance that can come with “privacy” concerns. And we will act early, well before symptoms appear, opting to tap the science of genomics and proteomics in order to mitigate our risks down the road.

Together, these tools will create a new opportunity and a new responsibility for people to act – to make health decisions well before they become patients. This can be characterized as a decision tree, a series of informed choices we will make to minimize uncertainty and optimize our outcomes. Indeed, we will use decision trees to navigate most of our health decisions, sometimes in overt ways – new decision support tools will both inform us and guide us, and they’ll be steeped in statistics, prediction, and the power of collective experience.”

For better or for worse, patients have an increasing multitude of sources and options. Not only are patients now much more informed of their diagnoses and conditions but also of their treatment alternatives. More each year are demonstrating a willingness to access cell-based treatments out-of-country. Others are lobbying for the loosening of the regulatory sphincter around cell-based therapies such as stem cell transplantation, Dendreon’s PROVENGE, etc.

Increasingly patients’ demands are influencing doctors as much or more than anything else. Pricewaterhouse Coopers recently listed “the internet and social network” as the Health Industry’s “top nine issues for 2009” citing web2.0 as “changing how healthcare is navigated and experienced by consumers”.

It remains to be seen how doctors will adopt cell therapies and this will depend in large part on the adequacy of reimbursement but certainly patients are driving their doctor’s decisions like never before and patients seem enthused – perhaps even overly so – about cell-based therapies.

Therapeutic Profit Margins

In terms of margins, even pharma executives are now realizing – in the face of diminishing pipelines and massive R&D costs per drug – that the models that have worked so well to-date financed by the kinds of margins they have received to-date may not be possible going forward. M&A, the creation of decentralized divisions, increased academic collaborations, etc…. they are all measures to try and address the same problem of diminishing margins.

Decreasing product margins are being tackled like it’s the disease when at least some would suggest it’s the symptom. The disease may not be lower margins at all but the model which demands such massive margins to succeed. But more on that in just a minute…

Personalized Medicine

I believe there is a third challenge to pharma’s “business model” concerns. From one direction we have pharma’s increasing desperation for new therapeutics; from another direction comes the public’s increasing demand for access to cell-based therapies, and – to complete the trifecta – we have the fast-growing sector(s) of personal genomics, theragnostics, and/or personalized medicine.

The ability to analyze the effects of therapeutics on patient sub-populations and use that to target such groups and/or tailor therapeutics accordingly opens up so many promises therapeutically but also puts incredible additional pressures on the blockbuster model with its low-rate of success per overall prescription rate. Some insurers are now catching on to the notion that reimbursement can be – perhaps should be – tied to therapeutic success and are willing to pay a higher price tag for success but nothing at all for failure.

Others before me have described the increasingly hard-to-ignore crack in the blockbuster model much better than I. One of the better analyses I’ve read recently can be read here.

To predict that cell-based therapies will contribute to and succeed because of a shift in the healthcare paradigm may not be that much of a stretch at all. The stars do appear to be aligning for a rather wholesale change to be readily adopted by all involved – or at least the major players.

…

(click here to continue to Part II)

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A Therapeutic Long Tail. Does Cell Therapy Fit the Model? (Part II)

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

…this is a continuation of Part I…

So are Personalized Medicines the Long Tail of the Therapeutics Industry?

In 2006, Chris Anderson, Editor-in-Chief of Wired magazine, released “The Long Tail” – a book describing the Web 2.0-induced business-to-consumer economics (r)evolution enabling businesses to now deliver product at outrageously affordable prices to global micro-markets.[i] The book quickly became one of the most influential business books of the ‘new economy’.

Indeed this flat new world – as Thomas L. Friedman describes it[ii] – is all about micro-trends and micro-markets, empowering small companies and niche products to participate successfully in the marketplace along with Fortune 100 companies and blockbuster products. More than that, this paradigm shift is forcing product and service companies to revolutionize their business and pricing models, the latest and rabidly successful of which is the pricing model of “Free!”. The blockbuster-based business model that depends on the economics of a ‘hit’ (whether it’s a platinum-selling CD or a ‘blockbuster’ drug) is no longer seen to be the only way to make money and, indeed, may not have any future at all.

To date, this internet-driven Long Tail (r)evolution has had its largest impact on – if not been largely restricted to – the retail sector. Business-to-consumer (B2C) companies are now enabled like never before to profitably supply unique global products to meet customer demands. In this respect, the (r)evolution has had a visible impact on the retail side of the pharmaceutical and biotech industries. However the ongoing realization of “personalized products” as the future of medicine for consumers seems a perfect fit for the Long Tail economic model.

But does this apply to therapeutics?

To the extent that the Long Tail economy is less about the mass market and more about businesses succeeding in serving niche markets with unique and/or customized products, the analogy fits.

Certainly, it would appear the sun is setting on the “blockbuster hit” days of the therapeutics industry. In June 2008 Genentech’s Susan Desmond-Hellmann, president of product development, when outlining the biotech giant’s development strategy to a group at the Goldman Sachs healthcare conference, told the group that the blockbuster drugs of the past may never be duplicated. “Future drugs,” she stated, “will cater to a smaller, better defined group of patients” and “the days of big cholesterol-lowering drugs for millions of people may be behind us.” (link).

In a June 8, 2009 post, authors of the IN VIVO Blog stated that pharma “managers these days are learning to leverage only very modest top-line growth into faster bottom-line growth through cost-savings, bolt-on acquisitions, and rapid introductions of incrementally-improved new products. The traditional, high-risk, high-growth R&D-based model is gone, in case you hadn’t noticed.”

The second indication that personalized medicine fits with the Long Tail supply-demand model is that therapeutics can now be customized taking advantage of personalizing elements such as HLA-typing and advances in genetic, diagnostic, and discovery testing that will permit us to more effectively both pre-select which medicines are best suited for which patient sub-groups and develop therapies that are better suited to particular genetic groups. On the clinical end of the spectrum, advances in theragnostic tools and surrogate biomarkers (e.g., imaging) are allowing clinicians to much more rapidly assess personal responses to treatment and adjust their therapeutic regimines accordingly before any symptomtic response can be observed.

The ability to match therapeutics to genetic make-up, disease stage/presentation, or predisposition will allow the provision of treatments with more confidence in their expected efficacy, fewer patient-specific adverse reactions, and result in cost savings to the health care industry and a better return to fund the premium for the development of such products.

In a recent interview with Xconomy’s Luke Timmerman, Alan Frazier, founder and managing partner of Seattle-based Frazier Healthcare Ventures, one of the world’s biggest life sciences venture capital funds, said,

The trend is toward smart pharmaceuticals that address smaller populations, narrower indications, with better results. It’s based on a better understanding of disease from genomics and proteomics. You’ll see more targeted drug delivery. You’ll have incredibly focused pharmaceuticals, which will take care of some of the safety issues you read about.

Does the model apply to cell therapies?

At the intersection of the personalized and regenerative medicine sectors, lies the therapeutic industry’s response to (or reflection of) both the demand for and the ability to deliver a more personalized therapeutic to the consumer (the patient).

If the tail of the therapeutics market is being stretched longer to include products better targeted to smaller markets, this therapeutics Long Tail will almost certainly include regenerative medicine (regen 2.0) products and many of these will be cell-based therapies.

The cell therapy sector now encompasses the convergence of three distinct technologies, i.e. cell transplantation, tissue engineering, and biomaterials. Often also drawn in are elements of gene and molecular (e.g., proteins and antibodies) therapies.

Autologous cell therapies are the ultimate personalized medicine and while such medicines are often not included in definitions of personalized medicine, any approach to regenerative medicine – whether using tissue engineered products, cell-based therapies, small-molecule drugs, scaffolds, nano or biobots, etc – intended to trigger in vivo regeneration or repair… is personal!

Again, to the extent the Long Tail economy is about “selling less of more” then medicines such as cell therapies will certainly be part of the long tail of the therapeutics industry giving access to highly-personalized, tailored products to meet the unique needs of the consumer.

But does the Long Tail economic model apply?

While there may well be blockbuster cell therapies in the future, concern around the scarcity of potential blockbusters in cell therapy are seated in the notion that the blockbuster economy is the only successful model.

Who thought you could make money by giving free access to internet searches and email? There are a lot of ways the analogy breaks down but what the Long Tail economy has shown us is modern corporate ingenuity in finding profitable business models in niche markets despite the long-standing dominance of blockbuster products.

Where the paradigm falls short, in my opinion, is when one realizes that a fundamental pillar to the Long Tail economy is the commoditization of products and production processes as well as the standardization of delivery systems. The Long Tail economic model is enabled by the fact that the cost of goods sold has been brought sufficiently low enough – by new technologies which allow for small batches to be produced and shipped – that the products in question are affordable to most. Highly unique and/or customized goods were always available to those with sufficient ability to pay. The Long Tail brings these kinds of products to micro-market at the same price or lower than their mass-market counterparts.

We are a long way from this kind commoditization of personalized medicines and, in particular, of cell-based therapies. There is little-to-no standardization of production systems at any scale. The next generation of these therapies will still be highly priced goods which many people, insurers, and/or healthcare systems will be pressed to afford.

In once sense, this is not surprising. Even in the world of electronic or digial products, Moore’s principle only applies because the cost of goods for new technologies when they first enter the market are prohibiitively expensive. If the model applies in the therapeutics industry, technological advances in production will bring down the cost of goods and therfore the price.

On the other hand, what drives the price of therapeutics has not been so much the cost of goods (production) but the need to recoup massive upfront R&D costs which must be recovered (with a reasonable profit) during the life of a patent.

Will this change with personlized medicines and cell therapies? One could argue it might if personlized medicines and cell therapies can deliver on the promise to have a much higher effective rate among those given the therapies. Pharmaceuticals have a notoriously low effective rate among those prescribed any given drug. New research methodologies employing surrogate biomarkers, imaging, cell-based assays, etc promise the potential to significantly lower R&D costs by (a) getting to a much quicker “go/no-go” decision point in therapeutic development, and (b) identifiying patient sub-groups for which the therapeutic will have a high rate of effectiveness. One the one hand, this has the potential to lower the cost of goods (driven by R&D costs) and simultaneously justify a higher price point because of a high rate of therapeutic success.

However, even if we were able to materially lower upfront R&D costs, this still does not change the fact that cell therapies are costly to produce on a per-unit basis and the opportunity to “scale-up” is limited particlarly with autologous cell therapies which require lot sizes to be an individual batch. While this is a seperate discussion, I am confident technological and/or commercial innovation will find a way to economically bring therapeutics to market that are proven effective. I am not alone. Recenly Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan as quoted as saying that “Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” (“Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive”. GEN News Highlights. 12 June 2009)

There are other reasons the Long Tail business model as it is currently defined does not fit cookie-cutter with the therapeutics industry not the least of which is that the consumer demand does not solely regulate supply. In highly regulated industries, the economies are different. Some would argue this should not be the case particularly if personalized medicines, genomics,theragnostics, etc advance to point where the consumer has an abundance of therapeutic information and options but that is the subject of another discussion.

In the end, it is certainly clear to me that the emerging technologies are enabling new therapeutic paradigms that will introduce elements of the Long Tail business model into the therapeutics industry.

How much Impact will Cell Therapies Have on New Therapeutic Paradigms and Systems?

Despite the exponential escalation of cell therapy innovation, is cell therapy ready to be a viable business that will make any material impact on the therapeutic industry? For reasons which I will continue to explore in this blog in the months to come, I believe the answer is ‘yes’.

The cell therapy industry has more products in late-stage development than many believe. While the existing commercial products are far from blockbusters and questions remain regarding the business models and potential margins for cell therapy products, ground-breaking research and market enthusiasm is driving a renewed interest among an early second-round of investors including participation by mature industry players making cautious plays in the sector. Regulatory issues in the primary markets of the US and EU are rapidly becoming less problematic though harmonization remains a significant challenge.

In blogs to come I will outline the commercial developments I see transpiring that make me believe the primary issues around the ‘business models’ question are being addressed. Regenerative and personalized medicines are enough of a force that creative business people will (a) invent ways to lower the cost of goods and (b) figure out how to price and reimburse appropriately to ensure effective therapies get to people that need them and incentivize doctors to prescribe them.

Most indicia point to this sector being poised for a phase that will start demonstrating real answers to those challenges which have to-date prevented many from participating in a sector that they believe presents too many unanswered risks.

Conclusion

As with so many of the paradigm-shifting economic revolutions in the past, once the underlying technologies exist, business and economic models radically adjust to reflect and accommodate that change. The latest of these is the Long Tail economy (r)evolution with it ability to deliver niche (if not personalized) products to niche markets. Pesonalized medicines – regenerative medicine and cell therapies in particular – are the therapeutic industry’s answer for the demand for personalized products. For a host of reasons, however, the economic model is not yet one which makes for profitable delivery of such products to niche markets.

Nonetheless, while questions about the viability of the business and economic models for these paradigm-shifting therapies are reasonable, the next 5 years will show that today’s questions about the business models for cell therapy were rooted in limited imagination about the potential for technological and commercial innovation as well as outdated concepts of the therapeutics industry and its economies.

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[i] Anderson C: The Long Tail: Why the Future of Business is Selling Less of More. Hyperion (2006).

[ii] Friedman TL: The World is Flat. A Brief History of the Twenty-First Century. Farrar, Straus and Giroux (2005).

www.celltherapyblog.com hosted by www.celltherapygroup.com

Cell Therapy Industry HiLites 2009-05-26

§ October 16th, 2009 § Filed under Regenerative Medicine Comments Off

This week I’m ROTFLMAO loving this cartoon so I thought I’d share the chuckles (thanks to www.onstartups.com). I can’t tell you how many times in the past years I’ve seen the inevitable ‘hockey stick’ market projections graph from companies, analysts, and investors when discussing the coming 5 years in the cell therapy sector. It’s become a standing joke about how we just keep pushing the hockey stick out to the right but the curve is inevitably the same. This pokes a little fun at this much maligned tradition.

Maybe it’s just because I’m Canadian that I love this so much but c’mon the beloved hockey stick has become almost more a marketing tool than the venerable piece of sports equipment every Canadian boy must own! Maybe we can adapt this new hockey stick model to give the over-used Gartner cycle a run for its money!

Down to business…

I was told last week that Senators have been inviting people to Capital Hill to speak to them about FDA regulation of adult stem cells perhaps at the instigation of Chris Centeno’s lobbying through ASCTA (www.stemcelldocs.com) that the medical use of moderately expanded autologous adult stem cells should not be regulated by the FDA but rather self-regulated by physicians. This could, of course, have serious implications on business models for autologous cell therapy companies. I’ve heard nothing of the Senators asking for ISSCR or ISCT counsel to-date. It would, of course, likely be Centeno’s position that anyone involved in those orgs are “pharma hacks” as he has accused me of being.

ASCTA which is comprised of physicians none of whom are acknowledged leaders in stem cell research recently proudly announced the release of their stem cell guidelines which they suggest should replace FDA regulation. In what appears to be simple ignorance of the years of work being done by AABB & FACT, ASCTA described their guidelines as “the world’s first clinical guidelines to allow U.S. doctors to safely begin using the patient’s own stem cells to treat disease.” Their lab practice guidelines contain such gems as:

• Any facility used in the processing A-ASC’s must be of suitable size, construction, and location to prevent contamination.
• The facility should be in a good state of repair.
  
• Equipment used in the facility should be adequately maintained
• In humid areas, dehumidifiers should be used to control the spread of infectious agents in the lab.
• Any reagents used must be approved for human use (where practical).

In an online posting entitled “The FDA is Killing Adult Stem Cell Therapy Thus Killing Patients“, ASCTA supporter, Don Margolis, states:

In a startling new interview on stem cell research and adult stem cells in the near future, Dr. Christopher Centeno warns that Big Pharma and the FDA are teaming to control the use of a person’s own stem cells thus slowing innovation and the use of Adult Stem Cells to help patients now.

FINANCIAL

ReNeuron Group plc (LSE: RENE.L) has raised the £3 million it announced it intended to raise. ReNeuron recently received regulatory approval to commence a Phase I clinical trial in the UK with its lead ReN001 stem cell therapy for disabled stroke patients. The Company is developing stem cell therapies for a number of other conditions, including peripheral arterial disease and diseases of the retina. ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.

Pfizer expects to pump $100 million into its international stem cell development program which aims to focus both on small molecules that work by modifying cells in the body and cell therapy where the cells are manipulated outside the body. The first major use of the Pfizer money is for a collaboration with University College London (UCL) for the treatment of certain forms of blindness through the London Project to Cure Blindness. The collaboration will attempt to develop stem cell-based therapies primarily for wet and dry macular degeneration (AMD), which Pfizer has the rights to progress through clinical trials and then commercialize. Pfizer’s work on this could potentially provide a boost for the cash-strapped and searching-for-a-buyer, Intercytex Group plc (AIM: ICX), whose involvement on the London Project could yield cash royalties. But that’s not where the Pfizer-Intercytex connection may end, apparently. Reports indicate that Pfizer is in fact eyeing the troubled Intercytex as a potential acquisition or investment. The company is on the auction block and actively seeking suitors.

Cytori Therapeutics, Inc. (NASDAQ: CYTX) expected to close a private placement for $4.2 million by May 11. No word yet…

Aastrom Biosciences, Inc. (Nasdaq:ASTM) temporarily suspended enrollment and patient treatment in its U.S. Phase II IMPACT-DCM clinical trial following a report that a patient died at home after being released from the hospital following treatment in the trial. IMPACT-DCM is a clinical trial to evaluate the surgical delivery of autologous cells directly into the human heart muscle for the treatment of congestive heart failure associated with dilated cardiomyopathy (DCM) in both ischemic and non-ischemic patients. The patient’s cause of death has not yet been determined and is the subject of a pending investigation at the clinical site. An independent Data Safety Monitoring Board (DSMB) will also assess the circumstances of the event. The Company has voluntarily suspended patient enrollment and treatment in the trial and the FDA placed the trial on temporary clinical hold pending an investigation.

ImmunoCellular Therapeutics, Ltd. (OTC: IMUC.OB ) (IMUC) announced that clinical data from the company’s Phase 1 trial of its antigen-pulsed dendritic cell immunotherapeutic cancer vaccine ICT-107 for patients with brain stem glioma and glioblastoma will be presented at this year’s ASCO. Having said that, they also said that while “encouraging data” was observed from the phase I trial of ICT-107, IMUC’s primary focus going forward will be on its lead product candidate, ICT-121, which is an peptide-based “off-the-shelf” cancer vaccine that targets cancer stem cells and may have applicability to multiple types of cancer. IMUC anticipates filing an Investigational New Drug (IND) application in the third quarter of 2009 for a Phase I trial of ICT 121 in the treatment of glioblastoma (brain cancer).

COMMERCIAL

Cytori Therapeutics (NASDAQ:CYTX) and GE Healthcare, a unit of General Electric Company (NYSE:GE) announced an agreement by which GE Healthcare will commercialize Cytori’s StemSource(R) technology in the North American stem cell banking and research markets. The StemSource technology includes automated equipment to process stem and regenerative cells found in adipose tissue, cryopreserve them or use them directly for research purposes. In January 2009, Cytori and GE Healthcare formed a separate agreement to commercialize Cytori’s products in ten European countries. This includes selling the Celution(R) 800/CRS System in the European cosmetic and reconstructive surgery market as well as selling StemSource products in the European cell banking and research markets. The recent partnership is similar in nature to the European agreement, but is limited to the sale of StemSource banking and research products in the U.S., Canada and Mexico for 18 months starting in the second quarter of 2009. The agreement does not include U.S. commercialization of Cytori’s Celution System, which is currently under review by the FDA.

After securing a $12M equity financing facility and closing a $1.5M private placement, formerly on-the-rocks Melbourne-based Prima BioMed (ASX: PRR), which had less than $500,000 in the bank at the end of December, is now preparing IND filing in the US for a phase IIb / III pivotal trial of CVac for ovarian cancer. As previously reported here, the company has also commenced CVac™ ovarian cancer treatments on selected patients in Australia made possible through the Australian Government’s Australian Regulatory Control Mechanism’s Special Access Scheme under the Therapeutic Goods Administration. Meanwhile the company is looking at taking the product to other countries as a means of generating cash flows in the short-term through the sale of CVac™ treatments. Now the company’s executive director, Mr Martin Rogers, is touring North America in discussions with a number of hedge funds and other biotech focused investment funds about new investment and partnering opportunities for the commercialization of CVac.

Progenitor Cell Therapy, LLC has done a deal with NeoStem, Inc. (NYSE Amex: NBS) as their exclusive provider of commercial adult stem cells processing and storage. Progenitor founder, Andrew L. Pecora, has also joined NeoStem’s Advisory Board.

NeoStem, meanwhile, – the new favorite company of alternative “medical” products promoter, Suzanne Sommers – has been busy the last few weeks creating a curious empire of sundry types of bedfellows. Recently the company announced it had signed an agreement to license the exclusive worldwide rights to a technology, with pending patent applications, developed by Vincent Falanga, M.D., Chairman of the Department of Dermatology and Skin Surgery at Roger Williams Medical Center, Providence, R.I. Dr. Falanga’s stem cell product under development, Primcel, is a mesenchymal stem cell (MSC) product. Early clinical studies are said to indicate that Primcel can be used to accelerate or jump start healing of chronic wounds. Dr. Falanga’s work seeks to develop a prepackaged product created from a patient’s own cells, ready for physician use in a clinical setting, consisting of three applications over a 12-week period.

Almost in the same breath, the company filed a patent application claiming the proprietary stem cell technology of Vincent C. Giampapa, M.D., F.A.C.S. relating to cosmetic facial rejuvenation, which NeoStem first licensed in February 2009. Dr. Giampapa is director of the Giampapa Institute for Anti-Aging Medical Therapy, a board-certified plastic reconstructive surgeon and Assistant Clinical Professor of Plastic and Reconstructive Surgery at the University of Medicine and Dentistry of New Jersey. His cosmetic stem cell face lift technology is said to comprise a “non-surgical procedure for complete facial rejuvenation that involves injecting pluripotent cells, including stem cells, into the skin of individuals whose skin has lost its firmness and texture due to age“.

Days later NeoStem announced it had signed an exclusive royalty-bearing license agreement for the Asia territory for a procedure developed by Regenerative Sciences, LLC and marketed for the treatment of chronic orthopedic conditions, under the name, Regenexx. Additionally, Regenerative Sciences, through its founder and CEO, Christopher Centeno, M.D., will serve as a consultant to NeoStem, Inc. in the area of stem cell therapy in orthopedics.

Undoubtedly Centeno will also embroil and leverage NeoStem in his fight with the FDA about the marketing of Regenexx in direct flagrance of FDA regulation. In any event this is clearly a solid “plan B” for Centeno should the FDA ever get around to shutting him down in the U.S. In the interim, Centeno continues to strengthen his network of supporters and is reportedly actively working Capital Hill for support for his fight.

To mix it up a little, NeoStem then announced they had signed a deal to promote and glean royalties from the sale of a liquid, nutritional supplement – AIO Premium Cellular Health. Not kidding. They have signed an agreement for Ceres Living, Inc., a developer and direct marketer of health and wellness products. AIO Premium Cellular Health was apparently developed in conjunction with NeoStem’s scientists and Advisory Board members, based on certain nutraceuticals that have been shown to “optimize stem cell functions”. The promotion of AIO is said to be “part of NeoStem’s corporate commitment to advancing anti-aging and regenerative medicine therapies and technologies.”

Novocell, Inc. announced that it has received U.S. Patent # 7,534,608 with with wide-ranging method claims covering the Company’s innovative stem cell therapy for the production of functional pancreatic, insulin-producing cells from human embryonic stem cells (hES). Novocell’s therapy is being developed as a method for the use of hES cells to replace insulin-producing pancreatic cells that are destroyed in people with diabetes.

Neuralstem, Inc. (NYSEAmex:CUR) recently published a reaction to a press release by StemCells, Inc. (STEM) refuting many of their statements in what has become a very public dispute. Included in the reaction is the claim that “the PTO upheld the patentability of Neuralstem’s core technology in May, 2006, in response to a challenge from STEM” and that the Neuralstem “patents are not being challenged in the PTO or in any of the suits with STEM”.

This is not a cell therapy story but it could be. Takeda Pharmaceutical Company Limited (TSE: 4502) and IDM Pharma, Inc. (Nasdaq: IDMI) today announced that Takeda America Holdings, Inc., a wholly-owned subsidiary of Takeda (Takeda America), and IDM Pharma have entered into an agreement for Takeda America to acquire IDM Pharma. Takeda America has established Jade Subsidiary Corporation as a wholly-owned subsidiary to effect that transaction. Under the agreement, Takeda America will purchase all of IDM Pharma’s outstanding shares for US$2.64 per share in an all cash tender offer followed by a merger.

You may recall that on April 1 last year (omen perhaps?), Cell Genesys, Inc. (“Cell Genesys”, Nasdaq: CEGE) and Takeda announced that the companies had formed a global alliance for the development and commercialization of GVAX immunotherapy for prostate cancer, Cell Genesys’ lead product candidate then in Phase 3 clinical development. Under the agreement, in exchange for exclusive worldwide commercial rights to GVAX immunotherapy for prostate cancer, Takeda was to pay Cell Genesys an upfront payment of $50 million and additional milestone payments totaling up to $270 million relating to regulatory approval and commercialization of GVAX immunotherapy for prostate cancer in the United States, European Union and Japan.

This deal quickly unraveled when they decided to mothball both phase 3 studies of GVAX on statistical likelihood of failure. This was Takeda’s big venture into cell therapy and it is an ugly, recent memory. Now they have bought IDM Pharma. They ‘ve bought it for the company’s primary asset, MEPACT, (mifamurtide), a macrophage activator drug therapy indicated for the treatment of non-metastatic osteosarcoma (malignant bone cancer) following surgical removal of the tumor (resection) in children, adolescents and young adults.

Here’s the cell therapy part: IDM Pharma has 3 autologous cell therapy products currently on developmental hold as a means of perserving resources to get them to the home run on MEPACT. BEXIDEM is activated macrophages (Monocyte-derived Activated Killer cells or MAK® cells) for bladder cancer. UVIDEM, for melanoma, and COLLIDEM, for colorectal cancer, are both therapies using dendritophages (specialized immune cells derived from the patient’s own white blood cells). It will be very interesting to see what Takeda does with these products, if anything.

Veterinary stem cell company, Vet-Stem, Inc, has now expanded from its US base into Canada, providing commercially available autologous, adipose-derived stem cell tranplants for horses and dogs.

MISCELLANEA

International Stem Cell Corporation (OTCBB:ISCO), the company behind human stem cells from unfertilized eggs (called “parthenogenetic stem cells”), submitted comments to NIH on their draft stem cell research guidelines issued by the National Institutes of Health (NIH) on April 17, 2009. ISCO then published its comments in a press release and then their CEO published a blog to address questions regarding their comments.

Genetics Policy Institute also submitted a letter to the National Institutes of Health (NIH) commenting on the Draft Guidelines for Human Stem Cell Research. The complete letter is available at www.genpol.org for public viewing.

The California Institute for Regenerative Medicine is working its way through Congress seeking $500 million in federal funds it hopes to apply toward guaranteeing the long-planned small-business loan program.

Forbes published what is actually quite an informative article on cell therapy medical tourism for a change.

Sign-off

If you’ve read this far you’re obviously interested in the cell therapy industry. If you’re on LinkedIn make sure you have joined the LinkedIn Cell Therapy Industry Group.

Thank you all for all your good wishes about the new addition to my family – my own little sample of regenerative medicine! Now I gotta go and spend some time with her before I’m enrolling her in college…

Remember. Cell Therapy Means Business.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Cell Therapy Industry HiLites 2009-05-15

§ September 21st, 2009 § Filed under Regenerative Medicine Comments Off

I’m back.

That on the left my friends is the new squawking reason I’ve been quite on the blogosphere and Twitter scene for the past few weeks!

I know this issue of my Industry HiLites has some rather stale news but it also has some recent news. What I can’t promise is that I’ve captured all the cell therapy related news from the past few weeks. I’ll attempt to back fill as time permits.

“Cell Therapy is expected to be one of the most important innovation drivers of modern medicine”. See below who is the source of such zeal!

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If you missed the Invitrogen-sponsored breakfast session at the 2009 Phacilitate Cell & Gene Therapy Forum in Washington, DC, the session videos are now posted for your convenience.

Realizing the potential of cell therapy: new enabling platforms
Brian Newsom, Cell Therapy Business Development Leader, Invitrogen/Life Technologies

Exciting lentiviral platform technology applicable to clinical indications from leukemia to HIV
Dr. Boro Dropulic, President and Chief Scientific Officer, Lentigen, Inc.

20-year history of Organogenesis
Katie Faria, Director, Process Development, Organogenesis, Inc.

Contact them at celltherapysystems@invitrogen.com if you want to engage in a discussion about how Invitrogen™ Cell Therapy Systems can deliver proven products and expertise to help with your translational research and clinical development plans. Find more videos and information about their products and services (including immunotherapy, stem cells, and tissue engineering) go to www.invitrogen.com/celltherapy.
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The race is on between Dendreon and Osiris to be the first cell-based therapeutic approved in over 10 years. Osiris Therapeutics, Inc. (NASDAQ:OSIR) has confirmed with the U.S. Food and Drug Administration, receipt of the first portions of its BLA (Biological License Application) for Prochymal as part of a rolling submission. Dendreon expects to file revisions to its already-filed BLA by Q4 of this year. If things go as Mitch Gold (Dendreon CEO) predicts, it looks like Dendreon will win the race to market but they are not competitive products and everyone wins if they are both approved regardless of the order!

Meanwhile, troubled cell therapy company Isolagen, Inc. (AMEX: ILE) received good news from FDA recently that its BLA has been accepted for full review – decision due by Jan 4.

FINANCIAL

Cell therapy company Isolagen, Inc. (AMEX: ILE) received bridge financing to allow it to seek debtor-in-possession financing in conjunction with a possible chapter 11 filing. Meanwhile, they’ve filed their latest 10Q with the SEC for those interested in the gory details.

The California Institute for Regenerative Medicine (CIRM) has handed out 15 grants worth $67.7 million to 13 not-for-profit and two for-profit organizations, BioTime and Novocell. An additional 12 grants which were recommended are on hold and money will made available only if funds allow. They will consider those grants in June. Novocell has been awarded $5,405,397 for its embryonic stem cell-derived diabetes therapeutic program and BioTime has been granted $4,721,706 to facilitate the use of its ACTCellerate™ technology for manufacturing patient-specific therapeutic products through the expansion of over 140 highly purified primitive human embryonic progenitor cells (hEPCs) from hES or iPS cells.

Novocell will receive two funding awards totaling $6.2 million from CIRM. The grants cover development of methods for delivery and monitoring safety of human embryonic stem cell (hESC)-derived pancreatic cells for diabetes cell therapy.

Virxsys Corp. of Germantown, Md., and Rockville’s GlobalStem Inc. are the only two private companies among the recipients of nearly $19 million of funding for stem cell projects granted recently by the Maryland Stem Cell Research Commission to 59 projects out of 147 applications that it received. GlobalStem’s project is entitled “Scale-Up Manufacturing, Authentication, and Banking of Human Pluripotent Stem Cells” and curiously Virxsys’ is “Developing safer methods for induced Pluripotent Stem (iPS) cell generation using Spliceosome-mediated RNA trans-splicing (SMaRTTM) technology”.

While functionally the company is in a deep coma for the moment, according to a recent SEC filing, Cell Genesys, Inc. (NASDAQ:CEGE) is continuing to explore financing alternatives and consider strategic alternatives, including merger with or acquisition by another company, further restructuring, other sales of non-core assets, and other potential technology outlicensing or liquidation of the company.

Embryome Sciences, Inc. parent co BioTime, Inc. (OTCBB:BTIM) has raised $4M with a possible $16M in total with the potential sale of additional shares and warrants.

CLINICAL

Pervasis Therapeutics received Orphan Drug Designation for Vascugel cell therapy as they
prepare for a phase III trial of the product.

Osiris Therapeutics, Inc. (NASDAQ:OSIR) completed patient enrollment in the second of its Prochymal Phase III trials for Graft vs. Host Disease (GvHD).

I have to confess that until I read this recent press release from ReNeuron I was suffering under the misconception that the regulatory framework for cell therapies in Europe was becoming clearer but listen to this nightmarish-sounding bureaucratic cluster-schmuck. ReNeuron Group plc (LSE: RENE.L) provided a regulatory update regarding its ReN001 stem cell therapy for stroke. In January of this year, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) granted regulatory approval for a Phase I clinical trial with ReN001 in disabled stroke patients, to be undertaken in Scotland at Glasgow Southern General Hospital. The decision was ratified by the Commission on Human Medicines, with the support of its Clinical Trials Expert Advisory Group which was also involved in reviewing the application.The Company has since submitted the clinical trial application to the Gene Therapy Advisory Committee (GTAC) who, in addition to their existing gene therapy remit, were recently given responsibility to act as the national research ethics committee for stem cell therapy clinical trials in the UK. The Company has been notified by GTAC that a Provisional Opinion has been granted in respect of the applicatio , subject to the resolution of a small number of points raised pertaining to non-safety-related pre-clinical data and the clinical trial protocol. The Company is reportedly currently working to respond to the points raised, the majority of which involve straightforward amendments to the clinical trial protocol. Further clarification is being sought by the Company, particularly within the context of the above-mentioned regulatory approvals already received, regarding one request for further non-safety-related data by GTAC, the provision of which would potentially delay the start of the clinical trial by a few months. Meanwhile their IND in the U.S. remains on hold after well more than a year has passed since its submission.

Cellerant Therapeutics, Inc. announced the dosing of the first patient in a phase 1 trial of CLT-008 for hematological malignancies. CLT-008 is an allogeneic cell therapy designed to rapidly produce mature neutrophils and platelets in vivo and facilitate long-term engraftment in patients undergoing bone marrow or cord blood transplantation, or being treated for chemotherapy- or radiation-induced neutropenia among other applications. CLT-008 is said to have the potential to make cord blood transplants safer and more available for everyone regardless of age, size, or ethnic and racial background. In preclinical testing CLT-008 has reportedly shown to provide hematopoetic support to enable long-term engraftment of blood-forming stem cells, which could lead to broader use of cord blood transplants as an effective therapy for multiple life-threatening blood diseases. The company intends to study CLT-008 to addresses several significant markets and including neutropenia in the near future.

Led by Dr. John Wagner, Professor of Pediatrics and Director of the Division of Hematology-Oncology and Blood and Marrow Transplantation at the University of Minnesota, the Phase 1 study is an open-label, dose-escalation trial and will investigate the safety and tolerability of CLT-008 in 20 to 25 patients undergoing cord blood transplants.

Ever-innovative Medistem Inc. (PINKSHEETS: MEDS) is bringing pre-clinical research, commercial cell therapy clinical practice outside the U.S., and veterinary stem cell protocols together to report positive multiple sclerosis data using fat stem cells. A recent paper describes the scientific rationale and preliminary results of three patients with multiple sclerosis treated with their own fat derived stem cells. The paper provides some evidence that a stem cell-rich component of adipose tissue, called the Stromal Vascular Fraction, can concurrently immune modulate, while inducing regenerative activities in multiple sclerosis patients.

Publication collaborator, Dr. Robert Harman, CEO of Vet-Stem, has treated over 3,500 horses and 1,500 dogs with fat derived stem cells for inflammatory conditions such as osteoarthritis immune-mediated polyarthritis. The article, titled “Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis,” appeared today in the Journal of Translational Medicine (http://www.translational-medicine.com), and was a collaboration between scientists and clinicians from Medistem; the University of Western Ontario; Hospital CIMA, San Jose, Costa Rica; Indiana University; Cell Medicine Institutes; University of California, San Diego; and University of Utah

COMMERCIAL

Finally, we hearing some more about David Mooney’s company InCytu, Inc. which makes “smart biopolymer devices that enable in vivo cell programming”. I hear a lot of great talks but I have to confess hearing Mooney speak last year topped the list for 2008. This stuff might be too intelligent (read “complicated”) to be commercializable but it certainly doesn’t lack for brilliance.

The first of their in situ bioreactive devices (iBDs), coined Cellarium(TM) by the company, will be an intelligent biopolymer scaffold that once implated releases a factor that selectively attracts dendritic cells (DCs) into the scaffold, presents antigenic (melanoma specific) proteins to them, and then, upon maturation, releases the activated DCs to home to the lymph nodes where they stimulate T cells to specifically attack the melanoma.

This takes the whole notion of cell therapy (traditionally involving the ex vivo manipulation of harvested cells for implantation) and turns it on its head by doing in vivo what we have believed can/must be done in cGMP facilities.

Now this is all a long way off and the Lord Almighty knows we have had more than our share of cell-based cancer vaccine failures but other applications in the pipeline include chronic ischemia, peripheral vascularization, muscle regeneration and even potentially stroke and CNS disease.

InCytu plans to develop partnerships for product sales and marketing but retain control of the cGMP production of its iBD devices. The company’s product development facility is in Lincoln, RI likely for reasons associated with its CEO – serial CEO and cell therapy entrepenuer, Alfred Vasconcellos, who has been involved with CytoTherapeutics, ETEX, LCT, Sertoli Technologies. LCT Biopharma, Multicell, and cut his early teeth in Pfizer’s Strategic Market Development group. Vasconcellos was most recently the CEO of LCT Biopharma Inc. which is the wholly owned subsidiary of diabetes cell therapy company Living Cell Technologies Ltd (listed on the Australian Securities Exchange (ASX:LCT) and the US-based OTCQX (OTCQX:LVCLY) from New Zealand and now conducting a trial in Russia.

Dendreon (NASDAQ:DNDN) CEO Mitchell Gold’s ship came in when he recently sold 600,000 shares of the company at $24.92 apiece on April 29, generating gross proceeds of $15 million.

Meanwhile Dendreon is going on a hiring binge in prep for FDA approval of Provenge.

It is interesting to see the different approaches companies take to competitive IP. Whereas Osiris appears content for the moment to let companies develop all kinds of MSC products without interference, Stem Cells, Inc couldn’t be more aggressive in litigating any IP they believe infringes on their patents, like that of Neuralstem. Cytori and Vesta had a similar battle.

StemCells, Inc. (NASDAQ:STEM) recently announced that the U.S. Patent and Trademark Office (PTO) has upheld the validity of the remaining two neural stem cell patents which were subjected to reexamination proceedings commenced by Neuralstem, Inc. The upheld patents are the subject of two related lawsuits initiated by StemCells against Neuralstem, which allege infringement of a total of six patents. These six patents collectively claim the manufacture and use of human neural stem and progenitor cells as tools for drug discovery and as therapeutic agents. The PTO’s decision to uphold the two patents is final and cannot be appealed. Consequently, StemCells is asking the federal district court in Maryland to resume the infringement lawsuits against Neuralstem.

“The PTO’s latest action is a reaffirmation of the validity of the patents asserted against Neuralstem. Each of our reexamined patents has now been twice tested and twice granted by the Patent Office,” said Martin McGlynn, President and CEO of StemCells, Inc. “We now look forward to our day in court. Litigation can be a slow and meticulous process, but we plan to ensure that those who wish to commercialize neural stem cells for drug discovery or therapeutic uses, recognize the validity and value of our patents.”

Pfizer is clearly seeing ocular therapies as an important franchise for its regenerative medicine enterprise. After making a seed-like investment of $3M last year in the startup EyeCyte Inc, Pfizer has now announced a collaboration with University College London to advance development of stem cell-based therapies for age-related macular degeneration (AMD). The London Project to Cure Blindness, led by Professor Pete Coffey (UCL Institute of Ophthalmology), is involved in producing a cell replacement therapy from human embryonic cells; a therapy which it aims to introduce into clinics by 2011. The goal is to replace cells essential for “seeing” lost through disease at the back of the eye. The UCL–Pfizer collaboration will accelerate the research process by bringing together the pioneering work of UCL’s researchers in the field of cell-based therapies and Pfizer’s expertise in design and delivery of therapeutics.

Under the terms of the agreement, Pfizer will provide funding to UCL to enable research into the development of stem cell-based therapies for AMD as well as other retinal diseases. Pfizer will also contribute expertise in the design and execution of clinical studies, interaction with global regulators, and in product manufacturing techniques. After the completion of preclinical safety studies, Pfizer will have the option to conduct clinical trials to determine efficacy of treatment and commercialize any resulting product.

Pfizer also recently signed deal with WARF for rights to human embryonic stem (hES) cells for dev of new drug therapies

One of the topics of discussion among those of us trying to lead the charge in commercializing cell therapies is the lack of experienced executive leadership at the helm of many cell therapy companies. Novocell, Inc, as it has done before on several fronts, is leading by example by hiring a seasoned biotech executive as its new President & CEO to replace Alan Lewis who went to lead the JDRF.

Novocell, Inc. recently announced the appointment of John West as President, Chief Executive Officer

“Novocell addresses one of the largest commercial and medical opportunities in stem-cell-derived therapeutics, and its team is increasingly recognized for its scientific and IP leadership,” said Mr. West. “I look forward to working with Novocell through clinical development and market launch and to growing a major new business.”

West, former CEO of Solexa, Inc. (NASDAQ:SLXA – News), brings more than 27 years of experience building profitable businesses from transformational technologies in the life sciences industry. Mr. West served as Chief Executive Officer of Solexa from 2004 until the company’s acquisition in 2007. When Mr. West joined Solexa, it was a private venture backed company with a radically different biochemistry and molecular biology for DNA sequencing. During Mr. West’s tenure, the company became public through a reverse merger, raised almost $100M and launched its first commercial product. This product lowered DNA sequencing costs over 100-fold, achieved almost $100M revenue in its first full year and became the dominant system for genome-scale genetic research. Mr. West led sale of the company in 2007 to Illumina, Inc (ILMN) for over $600M, and the business market cap subsequently exceeded $1B. Prior to Solexa, Mr. West served as VP of DNA Platforms, approximately a $1B business, at Applied Biosystems (ABI) (now Life Technologies). Mr. West’s responsibilities included the company’s businesses in DNA sequencing, preparative and real-time PCR, DNA synthesis and arrays. Prior to joining ABI, Mr. West was President of Princeton Instruments, a private company with transformational technology for low light imaging and spectroscopy. During Mr. West’s tenure, the company grew over 10 times in revenue, with much of the growth leveraging advances in cell biology. The company was sold to Roper Industries (NYSE:ROP – News), where Mr. West continued as subsidiary President. Mr. West holds B.S. and M.S. degrees in engineering from MIT, and an M.B.A. in finance from the University of Pennsylvania’s Wharton School.

Advanced Cell Technology, Inc. (Advanced Cell, ACT) (OTC: ACTC) and Korea-based CHA Bio & Diostech Co., Ltd. (CHA Bio), formerly known as CHA Biotech, Co., Ltd. and whose shares are publicly traded on KOSDAQ, announced that the parties have entered into a licensing agreement under which Advanced Cell will license its proprietary “single blastomere technology,” which has the potential to generate stable cell lines, including retinal pigment epithelium (RPE) cells for the treatment of diseases of the eye, to CHA Bio for development and commercialization exclusively in Korea. ACT received an undisclosed up-front license fee.

At this year’s ISCT conference, Invitrogen, a division of Life Technologies (NASDAQ:LIFE), announced a new technology to separate undifferentiated stem cells from those that are differentiated by utilizing magnetic beads that latch onto a common marker on embryonic and induced pluripotent stem cells, which are not considered as suitable for transplantation as those which are differentiated because of the potential of unregulated cell growth, removing them from a culture in less than 45 minutes. This leaves behind highly pure and differentiated cells that are unaffected by the process.

Histogen Aesthetics, a division of Histogen focused on providing unique, clinically proven, human skin cell-derived products for aesthetics medicine, has announced the launch of the ReGenica™ Rejuvenation System for anti-aging and post-resurfacing.

Histogen Aesthetics utilizes a proprietary manufacturing process in which newborn dermal fibroblast cells are grown on beads in controlled bioreactors that maintain an embryonic-like environment, including low oxygen and low gravity conditions. During this process, the cells secrete growth factors such as Keratinocyte Growth Factor and Vascular Endothelial Growth Factor, and soluble extracellular matrix proteins such as collagens, laminin and decorin, which are associated with skin renewal and scarless healing. The resulting product is ReGenica, a complex made up of the naturally produced cell signaling materials and embryonic-like proteins which support the epidermal stem cells that renew skin.

Stem cell therapy faces more scrutiny in China but regulations remain unclear for companies that supply treatments.

Hemogenix, Inc. is stepping up the heat a little in its competitive battle with Stem Cell Technologies, Inc. over the preferred assay method for stem cell quality control and potency. Hemogenix recently sent out an email and posted a statement on the homepage of its website saying “It has taken Stem Cell Technologies 6 years to acknowledge one of the basic concepts of the HALO Platform: By counting undifferentiated colonies at 7 days, they are actually counting clusters of “proliferation units” which can, in turn, be measured using ATP without any manual enumeration of colonies at 7 days. “

MISCELLANEA

FDA CBER’s Cellular, Tissue and Gene Therapies Advisory Committee held a meeting over the last couple day. On May 14 in the morning, in open session, the Committee was scheduled to discuss the potential for Chlamydia trachomatis and Neisseria gonorrhea transmission by human cells, tissues, and cellular and tissue-based products (HCT/Ps) that are recovered from the reproductive system or gestational tissues (e.g., amnionic membrane and placenta, cells recovered from menstrual blood, foreskin, placental/umbilical cord blood derived cell products), or other sources. In the afternoon, in open session, the Committee will discuss animal models for porcine xenotransplantation products intended to treat Type 1 diabetes or acute liver failure. On May 15, in open session, the Committee intended to: (1) Receive an update on Guidance documents from the Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research and the Center for Veterinary Medicine and (2) discuss clinical issues related to the FDA draft guidance “Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee
Cartilage.”

FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its Web site prior to the
meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA’s Web site after the meeting. Meeting material is available online here.

BrightTalk recently hosted a stem cell webcast summit including presentation by ISCO – check it out here.

Sign-off

So with that I’m easing back in. As mentioned, I’ll try to back fill any news I missed and get back on schedule. Until then… ciao.

www.celltherapyblog.com hosted by www.celltherapygroup.com

ISCT 2009 Corporate Tutorials and Symposia

§ September 21st, 2009 § Filed under Regenerative Medicine Comments Off

Here is a listing of the corporate tutorial and symposia sessions being held at the International Society for Cellular Therapy conference Sunday-Wednesday in San Diego. For more information see www.celltherapy.org.

	ISCT 2009 Corporate Tutorial and Symposium Listing

Tuesday May 5, 2009
Time: 12:30pm – 1:30pm / Nautilus 3


BioLife Solutions Inc.

www.celltherapyblog.com hosted by www.celltherapygroup.com

Final Results of Dendreon’s phase III IMPACT Trial of Provenge

§ September 21st, 2009 § Filed under Regenerative Medicine Comments Off

Dendreon Corporation (Nasdaq: DNDN) will host a conference call tomorrow, Tuesday, April 14, 2009, at 9:00 AM ET (6:00 AM PT) to review the outcome of the FINAL analysis of its IMPACT (IMmunotherapy for Prostate AdenoCarcinoma Treatment, also known as D9902B) clinical trial of PROVENGE(R) (sipuleucel-T), the Company’s investigational active cellular immunotherapy for the treatment of advanced prostate cancer.

Those interested may access the call with the following information:

  Time:     9:00 AM ET/6:00 AM PT  Date:     April 14, 2009  Dial-in:  1-877-419-6594 (domestic) or +1-719-325-4855 (international)  Webcast:  www.dendreon.com (homepage and investor relations section

A recorded rebroadcast will be available for interested parties unable to participate in the live conference call by dialing 888-203-1112 or 719-457-0820 for international callers; the conference ID number is 8182435. The replay will be available from 12:00 pm ET on April 14, 2009 until midnight ET on April 16, 2009.

In addition the webcast will be archived for on-demand listening for 30 days at www.dendreon.com.

www.celltherapyblog.com hosted by www.celltherapygroup.com