Currently, I am in the middle of moving my family to Frederick, Maryland. I am starting a new position at Lonza, and it may take a couple weeks to figure out how the blog fits with the new position/company.
Please stay tuned, and I should be able to start back up in one form or another in the next 1-2 weeks.
I will be experimenting with Twitter in the meantime…..
Q Therapeutics, a cell therapy company focusing on neural applications, recently announced a $15M series B financing that included funding from multiple venture capital (VC) groups as well as biotech tools provider Invitrogen. Q’s lead product, Q-Cells™, is an allogeneic product intended to restore function to damaged neurons by providing trophic (e.g. cytokines, growth factors) support and re-insulating damaged neurons in a variety of therapeutic indications. According to Q’s website, the Q-Cell platform technology is potentially applicable in a range of demyelinating diseases, including transverse myelitis, multiple sclerosis, cerebral palsy, spinal cord injury, and other neurodegenerative diseases such as ALS (Lou Gehrig’s Disease) and Parkinson’s Disease. The companies technology is centered around patented research from Mahendra Rao’s lab. Currently, Dr. Rao is the VP of Stem Cell Research at Invitrogen, where some of Q’s series B funding came from.
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More than 12 million minimally invasive vascular surgeries are done every year. These procedures require catheterization of major blood vessels to gain access to the vasculature. While catheter technology has dramatically improved, closure of the vessel puncture wound post surgery remaines a challenge. Manual compression of the puncture site is the traditional method used to stop bleeding, but this often requires hours and renders the patient immobile. Rapid closure of the puncture site is critical to stop bleeding and to get the patient out of the hospital and back to normal activity.
About ten years ago, biomaterial-based vascular closure devices were introduced, such as St. Jude Medical’s Angio-Seal or Datascope Inc.’s VasoSeal. These first generation devices were essentially collagen based products that were used to “plug” the vessel puncture site Continue reading
According to an article on CNN.com, Geron’s CEO, Dr. Tom Okarma, confirmed timelines for Geron to initiate human clinical trials with their embryonic stem (ES) cell-based treatment for spinal cord injury (SCI). He stated that the trial should start by the end of the second quarter (~June). This is really big news, as this will be the first-in-man studies using ES cells in a regulated clinical trial. Dr. Okarma made sure to mention the ‘high bar’ related to safety that the FDA has placed on this unprecedented therapeutic cell product. However, Geron has assured investors that they have worked very closely with the FDA, and Okarma’s public statements this week tells me that Geron believes that they have generated sufficient safety data to satisfy the FDA for initiating clinical testing.
You can get an idea of what this ‘high bar’ set by the FDA relates to by reading the recent press release from Advanced Cell Technologies, Inc. Continue reading
Geron yesterday announced the issuance of US patent #7,326,572, helping to extend their patent protection around their embryonic stem (ES) cell platform technology in the area of treating type 1 diabetes. Currently, Geron owns an exclusive license from the Wisconsin Alumni Research Foundation (WARF) for James Tomson’s original ES patents. This license allows Geron the right to develop and commercialize pancreatic islet cells, cardiomyocytes, and neural cells that are derived from ES cells for therapeutic applications. Patent #7,326,572 covers important and frequently used culture conditions for creating endoderm tissue from ES cells, a critical intermediate cell type on the differentiation pathway towards insulin-secreting islet cells. Continue reading
I always found it extremely useful to read and monitor job posts, even well before I had any reason to send out my resume. Reading about various jobs give you an understanding of 1) what jobs are out there that you might be interested in, and more importantly 2) what gaps you have in your background that would help you get those jobs. At least some time should be spent on planning your career during school. By the time I was done with grad school I had 2-3 pages of skills and experiences that I wanted to obtain from jobs over the next 5-10 years that would help get me from Scientist to CEO (not there yet, nor do I have all the skills). I generated that list based on postings of various positions at different levels that I found on Biospace.
Cytori Therapeutics announced that they have enrolled their first two patients in an acute myocardial infarction clinical trial using adipose-derived cells prepared using their Celution™ device. The Celution™ device is an automated, tissue processing unit that isolates cells from adipose tissue derived via liposuction. Continue reading
You’ve been submitting resumes like nobody’s business. And contacted everyone you know asking if they know of any job opportunities. You’ve attended all the local networking events and career fairs. After what seems like forever, you get called in for an interview. Congrats! Continue reading
You have to hand it to Tengion, they are trying to tackle one of the largest challenges I can think of: producing tissue engineered organs and tissues from a patients own cells in unrelated indications (bladder, blood vessel, and kidney). While moving through clinical trials Tengion will surely be transforming patients lives with these technologies. They have already had early-stage clinical success with their tissue engineered bladder (highlighted at CNN), but there are several challenges to overcome in creating a business from autologous tissue engineered products. Continue reading
When looking for a job, a good fit is very important. You want to be able to get as much out of a job as possible, as well as feel like you are making an impact on the direction of the company. What your own wants and needs are will be dictated by your personality. However, what you can can expect from a company is mostly (not always) dictated by whether it is a large, well funded company, or a small company where profits aren’t expected for years. This post will generally outline the biggest differences in these two very different work environments, and will be followed by individual posts outlining what it is like to work in each type of environment. Continue reading
Pervasis Therapeutics announced that they have raised $9.75M to further develop their allogenic tissue engineered endothelial cell (EC) product for vascular health indications. Their Vascugel product is comprised of ECs grown in 3D collagen sponges that are implanted externally on blood vessels to prevent them from occluding. Pervasis has proven safety in a Phase I study concluded in 2006, and they have recently finished enrollment of two end stage renal disease (ESRD) Phase II studies in 2007. Continue reading
When we have a cold or a headache, we pop a pill. We get sick, we go to the doctor and get a shot. Taking medication orally via a pill or getting drugs injected via a syringe are some of the oldest forms of delivering therapeutic molecules. In the past, “drug delivery” simply meant getting the therapeutic inside the patient,whereas now it has evolved to include “controlled release,” allowing control over the timing, dosage and also location of drug release.
A transformation in the field of controlled release is being pioneered by Mimetics Solutions, LLC (Austin, TX), with the development of their proprietary Affinimer™ technology. This technology shifts the current controlled release paradigm from one of Continue reading
Osiris Therapeutics announced that they won a DoD contract worth up to $224.7 million for developing their adult mesenchymal stem cell (MSC)-based therapeutic for treating acute radiation exposure. The contract allows for reimbursement of up to $24.7 million for continued development of their Prochymal product, and then, once the product is FDA approved for acute radiation syndrome (ARS), 20,000 doses will be purchased in 5,000 dose increments at $10,000 per dose . This contract provides Osiris with much needed cash to cover clinical development while they complete their various other clinical trials.
Importantly, Osiris has partnered with Genzyme, a niche-disease development expert Continue reading
Just a quick note on a topic that keeps catching my attention: China. Three interesting things have come up in the last month:
With the US dollar so low and the Chinese markets so hot, it is highly possible we see even more investment from Chinese firms in US regenerative medicine companies as we are seeing in other industries. But I wonder how much more US-based capital will make it into China?
The WuXi acquisition of Apptec (posted last week) is continuing the trend of consolidation in the cell manufacturing sector. It is similar to Lonza’s (a Swiss company) purchase of Cambrex’s biologics division in 2006, and GE’s purchase of Wave Biotech in mid-2007. Lonza is currently the largest manufacturer of cells for therapy, and has process development and manufacturing relationships with many of today’s clinical-stage regenerative medicine companies. GE is making a big splash with Continue reading
Biomimetics Therapeutics, Inc. (Franklin, TN), a company which develops drug-device combination products for orthopedic applications, recently announced positive results from its Canadian clinical study evaluating its GEM OS®1 Bone Graft for the open surgical treatment of foot and ankle fusions. The GEM OS®1 is a combination product comprised of a β-tricalcium phosphate matrix and recombinant-human platelet-derived growth factor (rhPDGF-BB) and is based on the same platform technology as their first product, GEM 21S®, only the second FDA recombinant protein approved product for orthopedic applications.
This product is similar to the market leading INFUSE® Bone Graft by Medtronic Sofamor Danek (the first FDA recombinant protein approved product (2002)) in that they are biomaterials releasing molecular therapeutics to drive local bone regeneration. The differences between the two products are the molecules (growth factor in this case) used to drive bone regeneration and the material used to guide where the bone regenerates: Continue reading
WuXi (pronounced Woo-See) Pharmatech, a contract research organization (CRO) based in China, announced the acquisition of MN-based Apptec Laboratory Services. WuXi acquired Apptec to gain an immediate US footprint, better access to the US market, and important biologics expertise to expand outside the world of small molecules. This biologics expertise includes Apptec’s cell therapy bioproduction business which they were aggressively moving in to.
These two fast growing companies may be a match made in heaven Continue reading
I have recently signed up for the StemCellPatents.com weekly newsletter, and it is great to keep updated on all the patents that get issued in regenerative medicine. In the most recent newletter, they reported on two patents that issued last week that strengthen Celgene’s patent estate in adult stem cells. Celgene got into the stem cell business in late 2002 when they aquired Anthrogenesis (also see Celgene’s History graffic from their website below). Anthrogenesis had technology for harvesting placenta-derived stem cells from post-birth placentas, as well as Continue reading
With the New Year upon us, I thought it would be good to make a set of New Year’s Resolutions for the regenerative medicine industry. This set of resolutions is not company specific, but things that will be required to build long term value for this nascent industry.
The FDA has scheduled an April 10, 2008 meeting to “discuss scientific considerations for safety testing for cellular therapy products derived from human embryonic stem cells.” This meeting will be followed by another meeting on April 11 that will “discuss updates on 1) research management related to the September 29, 2005 review of research programs of the Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research; 2) FDA’s Somatic Cell Therapy Letter; and 3) recently released FDA Guidance documents.” Both meetings will be open to the public, and they are allowing for 30 minutes each day for limited public presentations for those that would like to contribute and provide public input to the discussion.
This meeting has been called “unusual” in the press since Continue reading
Top Ten Events in the Regenerative Medicine Industry in 2007 (according to Jon Rowley, and in no particular order):
Athersys and their cardiac parter, Angiotech, announced today that they got the ok from the FDA to begin a Phase I clinical study using Athersys’ Multistem adult stem cell product in acute myocardial infarction (MI). This is the second IND approval for Athersys in just over a month (and a welcome Christmas present for the Cleveland-based company). In my last post on Athersys, I suggested that they were accepting some market risk by their choice of indications in order to minimize clinical risk. However, cardiac regeneration is the Holy Grail of cell therapy today, as this is one of the most substantial markets available for cell technologies. The first (and maybe second or third) cell therapy company on the market with a successful cardiac product that can be manufactured at the necessary scale will begin building a Genzyme-like market capitalization. This is a very important milestone for this company.
The two press releases (1, 2) actually provide some interesting details:
Continue reading
A guest post by Susan Hsiong, PhD
You worked hard as a lab rat for the past several years and you’re eager to move on to the next step. However, the transition from academia to industry is a tricky one. Companies are often wary of hiring someone fresh out of grad school without any industrial experience. Industrial R&D is very different from academic research (product and timeline driven) and they’re not sure how well you’ll make that transition. You may be able to do quality research but how well will you contribute to their objectives and bottom-line? I’ve been told again and again that the first industrial job is often the most difficult to obtain. However, once you’ve gotten some industrial experience under your belt, it’s much easier to find the company and position that you want.
Below, I have provided some links that were useful during my own job search, as well as some of the best advice I got. Continue reading
There have been some interesting news releases this week, including Osiris’ fast track designation, Cytori obtaining a 510k for their fat cleaner (not their stem cell isolation device, just a clinical fat cleaner), and the angiogenesis and liver regeneration capabilities of Aldagen’s aldehyde dehydrogenase positive sorted cells. However, the press release that really caught my attention is BioTime’s spinnout of Embryome Sciences.
BioTime, a blood preservation company, hit my radar recently when they hired Michael West, PhD, as CEO (he was already a board member). Dr. West has a long history building companies around embryonic stem (ES) cell technology. He was a founder of Geron, the first ES cell company, and more recently the President and CSO of Advanced Cell Technologies. Embryome will be focusing their business on the service and bioprocess supply sector of the cell therapy industry. Their initial product pipeline is planned to be:
Organogenesis, one of the pioneering tissue engineering companies, is partnering with China’s National Tissue Engineering Center (NTEC) to bring Organogenesis’ regenerative therapies to the Chinese marketplace. NTEC is a Shanghai-based private company that is funded by the Chinese government, and it was launched by a Harvard/MIT-trained plastic surgeon, Dr. Yiling Cao. The two companies look to have a three-phase agreement in place that starts with Organogenesis exporting it’s tissue products into China for distribution by NTEC. If certain milestones are met (likely based on sales and successful deployment of the living tissue product to patients), phase II would see the transfer of manufacturing to NTEC for distribution throughout Asia. Phase III would include joint development of new products, which Organogenesis would retain certain regional rights to outside of Asia.
One of the aspects of my science career that I really enjoy is the career coaching that I provide, and receive. I actually spend a lot of time telling students that are graduating, whether it is at the Bachelors, Masters, or PhD level, about industry, and helping them get a feel for what the job search will be like. Since I repeat a lot of what I say to different people, I thought maybe I could save myself some time and organize a series of posts about job searching and finding different types of positions in regenerative medicine.
It is interesting that the majority of jobs that I have noticed over the last year at regenerative medicine companies have been in cell production or bioprocessing, more so than in R&D. Continue reading
The regenerative medicine industry is banking on cellular therapy to help regenerate tissues and restore function in clinical indications with high unmet needs. The mechanism of action, or how stem cells actually drive tissue regeneration, is still hotly debated – but it is likely a mixture of several actions (see item #6 in this post) that result from the cells producing multiple factors and releasing them into the local environment.
A more controlled method of driving tissue regeneration would be to have locally transplanted biomaterial-based devices present the soluble or insoluble factors at the desired site, without the use of cells. It is possible to present adhesion/migration signals from materials, as well as release a variety of soluble drugs or growth factors at different rates and times. If sufficient knowledge is acquired on how and why therapeutic cells work, and the mechanism is not dependent on long term cell engraftment, then it could be possible to engineer sophisticated biomaterial systems to provide necessary signals for driving tissue regeneration.
Not only is this possible, but proof of concept has been demonstrated with simple systems, and there are a variety of companies that are pursuing these strategies. These biomaterial companies include (there are several more):
The author of CELLWISE, one of the newer bio-Blogs out there, asked a question related to the Patent Reform Act of 2007 in a comment on the Pfizer post. He asked:
The article that is cited is right; our patent system is not optimized for today’s innovations or our different industries. The patent office is overwhelmed by too many applications. The applications are too broad and poorly thought out, making them difficult to review and it can take 2-3 years before your patent is reviewed for the first time. The patent office has a huge backlog, lawyers fight tooth and nail for their clients on every claim, and patent reviewers are evaluated by how many patents they get off their desk and not issuing high quality patents (it is easier to allow claims than to reject claims, as no one argues). As a result, a huge number of low quality patents are being issued. The Patent Reform Act of 2007 is trying to address several problems at once, and two of the primary stakeholders in the outcome of the reform, the info tech (IT) and pharma/biotech industries, have very different interests. Continue reading
I have been playing with the idea of a post related to the area of therapeutic cell bioprocessing, and today one of the leaders in the field became an acquisition target. Progenitor Cell Therapy (PCT) provides process development and manufacturing support for institutions and cell therapy companies, and has over 30,000 cell procedures under it’s belt and a combined 125 years of cell therapy management experience. Stem Cells Inc., the proposed purchaser, outsources it’s cell manufacturing to PCT for their cell product that is currently being tested in a Phase I Batten disease trial. Stem Cells must like what they see.
Stem Cells Inc. is several years (5-8) away from generating any revenue from their neural stem cell therapy. They just finished the 1 year safety follow-up to their first patient in a 6 patient clinical trial – pretty far from BLA and marketing. What PCT would give them, if the purchase goes through, is a revenue generator (yes, PCT actually generates revenue) to potentially bring in cash while Stem Cells continues through clinical trials. This could be the best way for Stem Cells to weather the tight capital markets, and gives them a way to remain in business if follow-on funding dries up. It also brings a cell manufacturing expertise to Stem Cells that they, and most other cell therapy companies, do not have. Most cell therapy companies don’t have the money to invest in manufacturing equipment or expertise, so they outsource cell production to one of several companies including Cognate Bioservices, PCT, Apptec, or Lonza.
Stem Cells Inc. must see cell production as a growing market that they can capitalize on with a strategic acquisition. I must say that I am more surprised to see Stem Cells Inc. as the purchaser and not one of the bigger players like Lonza or Apptec. I guess we have to see if this acquisition actually goes through, and what the repercussions will be through out the field.
Pfizer Global Research and Development (PGRD) has been going through some major restructuring, and in an effort to spur innovation has opened an independent research center in the Bay Area. This “biotherapeutics and bioinnovation center” will be headed up by experienced bioentrepreneur Corey Goodman. Is it possible that this could lead to some cell therapeutic-based products to enter the Pfizer pipeline? Continue reading
Osiris Therapeutics last week announced positive data that came out of their Phase I/II double blinded Chondrogen trial. They demonstrated statistically significant improvements in joint pain in patients with osteoarthritis, however this seems to be an about face compared to when they announced their “disappointing” (the CEO’s words) interim 6 month data from this trial. Osiris is getting beat up for this in the media, notably at my favorite stock website TheStreet.com. The articles author, Adam Feuerstein, didn’t like the fact that Osiris cherry picked the data and only announced an analysis from just over half of the patients (and apparently they wouldn’t comment on the other half). Osiris got a similar left hook from an analyst when they announced their cardiac trial results in March (I am still trying to find this reference), for similar reasons. Continue reading
Senators Sam Brownback (R-Kan) and Mary Landrieu (D-La) introduced the Human-Animal Hybrid Prohibition Act to Congress last week that would make outlaws of scientists that create part human, part animal beings. According to a white paper published by Friends of the Earth, “A human-animal hybrid is an organism with both human and animal genetic material.” These hybrids are apparently dangerous to both human health and the environment, and could change ecosystems in unprecedented ways (somewhat alarmist, wouldn’t you say?).
If passed, this bill would have major impacts on basic science research (covered nicely by Brandon Keim at Wired). Important for industry; the definitions in the bill (see below) have been thought through sufficiently to not cover animal safety and efficacy studies that are required by the FDA for IND data packages. If all human material (eg. stem cells) being placed into animals were outlawed, it would bring the regenerative medicine industry to a halt. This is not the case at this time though.
While this is likely not another Galileo Affair, it is not unprecedented for similar legislation to make it through in other countries. Maybe it is time to follow the lead of our colleagues in England and do more public outreach to educate the masses on stem cells research and technology. Continue reading
CellCyte Genetics Corporation recently announced that US Patent No 7,282,222 was issued, a patent which they have exclusive rights to. The press release states that the patent covers unique methods and compositions “to deliver and direct stem cells to target organs in the body”, including cardiac tissue.
The focus of the patent addresses cell retention at a site after delivery – a very important area of cell therapy, especially as it relates to cardiac applications. While no one really knows how cells work therapeutically, Continue reading
I do not want to undermine the importance of the great work that was done in generating pluripotent stem cells from adult skin cells, but the only thing I think about when hearing this news is TIME and MONEY. I know I will be fielding questions from my friends and relatives over Thanksgiving weekend on whether or not Aastrom (my employer) is doing this type of work or if we have to change our business model. I will have to explain that this type of research is at such an early stage, that it will not be impacting what is going on in Biotech for 20 years, if not more. It is challenging enough to manufacture and distribute an autologous cell product based on the expansion of a patient’s bone marrow. I cannot imagine trying to develop and implement a process where every patient needs their cells to be genetically modified with multiple genes prior to expansion, and then there is the potential need to differentiate the cells or engineer them into vascularized tissues – it is enough to give a process engineer a migraine. Not only is something like this difficult to scale, but the in-process quality control testing would be so expensive and time consuming that the only companies making any money would be the supply companies, such as Invitrogen.
On another note: I wonder if our healthcare system will ever evolve to reimburse a therapeutic strategy that this work would enable. One would need to generate and bank a patient’s pluripotent cells well before they would be needed for most diseases. Does the average cardiac patient have 6 months to wait for their drug to be manufactured? What insurance company would pay for this type of expensive processing, and banking, just in case you get a disease? This may be a therapy only for the rich.
Overall, this is really exciting science and I am enthusiastic to see how this will expand our knowledge base and alter the political landscape. However, the reality is that there will be several cell-based products on the market before this technology is ready for prime time (and therefore fewer unmet medical needs) that there will be little need, or smart venture money available, to develop it.
On November 15, 2007, Athersys announced in a press release that they had received a letter from the FDA giving the green light to start a Phase I safety study of MultiStem, their adult, allogeneic stem cell product. Athersys will start an open label, dose escalation trial to test their cells after bone marrow transplantation.
This is an interesting first clinical trial choise for Athersys to begin testing their cells in. This MultiStem cell product is very similar to Continue reading
An interesting study was published this week in the Journal of Neuroimmunology spurring a press release from Geron that stated “GERON’S HUMAN EMBRYONIC STEM (ES) CELL-BASED THERAPEUTIC FOR SPINAL CORD INJURY EVADES DIRECT ATTACK BY THE HUMAN IMMUNE SYSTEM”. The title is a little misleading since the cells really only evade immune cells in an in vitro model. However, the work was published in a peer-reviewed journal so it has some scientific credibility.
This study’s major limitation is that Continue reading
I am not sure if you noticed, but the stem cell stocks haven’t been performing all that great in 2007, especially compared to technology companies like Apple or Google. So now seems as good a time as any to start a blog that will help to demystify the area of regenerative medicine and cell-based therapies for the masses. Since I work at one of the companies on the above graph (Aastrom, not Apple), you might think that I am kicking myself for PhDing in the biological science and not getting a computer science degree. However, I could not be more enthusiastic to be in this field today, especially at a clinical stage company such as Aastrom. I believe that regenerative medicine is at an exciting crossroads, and I am thrilled to be in the trenches addressing the challenges of shepherding cell-based therapies through the FDA and towards commercialization.
When I finished my PhD in 2001, I felt extremely fortunate to get a relevant job in the field of regenerative medicine. I spent four years at BD Technologies (the corporate research center of BD) in a budding cell therapy group. I realized as the pioneering companies were filing for bankruptcy in 2002 (Advanced Tissue Sciences & Organogenesis), many large pharma or medical device companies were evaluating the cell therapy technology space. Companies such as BD and JNJ were starting up internal research programs and spreading their bets out between various start-ups in the form of equity investments. BD’s venture arm has investments in companies like Novocell, Cellerant, Progenitor Cell Therapies, and now defunct RenaMed, to name a few. JNJ has also been active investing in regenerative medicine start-ups, and provided much of the initial funding for Tengion (http://www.tengion.com/news/press/20050803.cfm ), and is currently a major shareholder of Novocell as well (http://geneticsandsociety.org/article.php?id=3445). This is very standard for how technology-based fields evolve. Early adopter companies form and often fail, but they do succeed in removing some risk from the technology. As risk decreases, large companies try to figure out how to play in the new technology sandbox that is littered with small companies. Today, even Baxter is running a cell therapy clinical trial for cardiac regeneration, PerkinElmer is buying up cord blood banks, Celgene is dabbling in placenta-derived cells, and Teva Pharmaceuticals has an autologous MSC long bone trail underway.
There is still a lot of risk left in the field, but I believe that Chris Mason was pretty accurate when he coined the term Regenerative Medicine 2.0 (this is a great read), drawing parallels between some of today’s regenerative medicine companies and Web 2.0 companies such as YouTube and Facebook. There are currently some companies with reasonably strong balance sheets and novel cell-based therapies targeting significant markets. Some senior managers bring 15+ years of experience to the field, and they have learned from the mistakes that have been made along the way. I am most definitely looking forward to a bright future for Regenerative Medicine 2.0, and you can follow the field right here at The Regeneration Station.
Full disclosure: I own stock in Apple, Google, Celgene, JNJ, and Aastrom.
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