ISCT 2009 Corporate Tutorials and Symposia

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

			Sunday May 3, 2009 Time: 3:00pm – 5:00pm / Grand Ballroom B Miltenyi Biotec
			Emerging treatments enabled by cellular therapy ……………………………………………………………………………………………………………… Chairperson: Shelly Heimfeld, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA, USA Topics and Speakers: Notch-mediated expansion of human cord blood stem/progenitor cells: cell processing and translation to the bedside ……………………………………………………………………………………………………………… Shelly Heimfeld, PhD / Colleen Delaney, MD, MSc, Fred Hutchinson Cancer Research Center, Seattle, WA, USA NK cell therapy in the pediatric leukemia ……………………………………………………………………………………………………………… Wing H. Leung, MD, PhD, St. Jude Children's Research Hospital Memphis, Memphis, TN, USA Antiviral T cell immunotherapy—present and future perspectives ……………………………………………………………………………………………………………… Mark W. Lowdell, PhD, FRCPath, MICR, Royal Free & University College London, London, UK The role of cell processing in the emerging regenerative medicine applications ……………………………………………………………………………………………………………… Denis-Claude Roy, MD, FRCP(C), Hopital Maisonneuve-Rosemont, University of Montreal, Canada

Monday May 4, 2009 Time: 7:30am – 8:30am / Nautilus 4 WuXi AppTec

Topics and Speakers: Validation of a rapid PCR method for the detection of mycoplasma contaminants according to European Pharmacopoiea 5.8, 2.6.7, 2007. Use in release testing of cell therapy products. ……………………………………………………………………………………………………………… Garry B. Takle, Ph.D. Vice President, Operations. WuXi AppTec Philadelphia. Recent publication of a revised section for mycoplasma testing in the European Pharmacopoeia (5.8, section 2.6.7) has led to interest in validating a nucleic acid amplification technique for use in detection of mycoplasma contaminants in cellular therapies. The replacement or supplementation of the existing culture based methods with a PCR-based method has several advantages for the biopharmaceutical industry, mainly with respect to turnaround time for results, and cost. Replacement or substitution of existing methods by a PCR method requires the demonstration of equivalent assay LOD and specificity. The experimental requirements for this comparability validation have been spelled out in detail in the EP section referenced above. In this presentation, we describe the validation and comparability analysis of a PCR method exactly according to the EP guidance. Completion of this validation activity has resulted in the availability of an assay that meets or exceeds EP compliance requirements for a mycoplasma detection method. Comprehensive testing panels for cellular therapeutics. ……………………………………………………………………………………………………………… Chris Larson, MS., Lab Manager, Cell Biology and Flow Cytometry. WuXi AppTec, Philadelphia. In this presentation WuXi AppTec will describe the current regulatory landscape for appropriate testing programs for cellular therapies, focusing on safety and identity assays. A multiuse contract manufacturing facility for the production and release of cellular therapies. ……………………………………………………………………………………………………………… John Bermel, Sr Manager, Cell banking/Cell therapy. WuXi AppTec, Philadelphia. WuXi AppTec will present a summary of the design and operation of the contract GMP cell therapy and cell banking facility located within the Philadelphia site. Design, start up and validation activities will be discussed with reference to specific projects and case studies.

Monday May 4, 2009 Time: 12:15pm - 1:30pm/ Nautilus 2 CellGenix Technologie Transfer GmbH and American Fluoroseal Corporation

Topics and Speakers Application of DC Maturation and T Cell Expansion in a Clinical Setting ……………………………………………………………………………………………………………… Dr. Gunnar Kvalheim, Radium Hospital - Oslo, Norway Purpose: Description of methods and materials for DC maturation and autologous T cell expansion in cancer therapy and a report of clinical outcomes. Expansion of Young Tumor Infiltrating Lymphocytes (Y-TIL) as a Therapeutic Agent in Cancer Treatment ……………………………………………………………………………………………………………… Dr Nina Garlie, Aurora-St. Lukes Medical Center - Milwaukee, WI. Purpose: Description of materials and methods used for a therapeutic Y-TIL expansion protocol Description of Materials and Methods for Cord Blood Expansion with Clinical Outcomes ……………………………………………………………………………………………………………… Dr. Colleen Delaney, Fred Hutchinson Cancer Center - Seattle, WA Purpose: The application of double cord transplants with one expanded cord using cytokines and notch ligand, and one cord left unmanipulated in cancer therapy.

Tuesday May 5, 2009 Time: 7:30am – 8:30am / Nautilus 5 Invitrogen

Come join the leading innovators in cell therapy for a breakfast session on safer cell therapies. One of the key concerns of regulatory agencies around the world is ensuring that candidate cell therapies for clinical trials and ultimate approval have adequate safety profiles. During this session, four leading industry experts will share the technologies and strategies they have identified and employed at different stages of cell therapy development to ensure phase-appropriate safety profiles and streamlined regulatory review of their clinical products. Chair Eric Roos, Business Development Leader, Stem Cell Therapy, Life Technologies Corporation Panelists Leslie Wolfe, PhD, VP, Technology Development, Cellular Therapies, Genzyme Biosurgery Patricia Whelton, Senior Director, Regulatory and Process Development, Osiris Therapeutics, Inc. John T. Kemshead, PhD, Director, Clinical Affairs, Baxter Cellular Therapies For more information on the session: www.invitrogen.com/isctmeeting Register for the breakfast session: www.invitrogen.com/isct-register

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

Culture and expansion of cord blood MNCs enriched by the Pall Purecell Select System, a new alternative to density gradient Description Enriched MNCs are used for variety of research and clinical applications. The new Pall Purecell™ Select System is a rapid, easy to use, single use disposable for the isolation of Mononuclear cells (MNC) from whole blood and other samples. Purecell Select System was evaluated for enrichment of MNCs from cord blood samples in two different research studies with direct comparison to cells isolated by density gradient method. In one study, enriched cells were subsequently used for CD34+ cell selection followed by hematopoietic progenitor cell expansion. In the second study, MNC enriched cells were used for production of Endothelial Colony Forming Cells (ECFCs®). Results of these studies will be presented and participants will walk away from the tutorial with an awareness of how the Purecell Select System can be suitable for certain research applications and it’s potential future use in clinical cell manufacturing processes. Speaker Christine Smith-Steinhart, PhD EndGenitor Technologies, Inc.

Tuesday May 5, 2009 Time: 12:30pm – 1:30pm / Nautilus 2 Biosafe SA

From Stem Cell Banking to Bedside Biosafe is honoured to have 2 renowned experts presenting some of the leading advances in the field of cellular therapy. Topics and Speakers Dr. Joanne Kurtzberg, Chief of Pediatrics and Blood & Marrow Transplantation at Duke University, NC, USA will provide an overview of the Carolinas Cord Blood Bank, one of the world’s largest and most respected public cord blood banks, before presenting recent developments in the use of cord blood to treat non-haematopoietic diseases. Dr. Riccardo Saccardi from the Haematology Department, Careggi University Hospital, Florence, Italy, will present the different work performed by his laboratory in the study and therapeutic use of cellular products. Careggi is Europe’s leading transplant centre for autoimmune diseases and Dr. Saccardi will go on to discuss the use of stem cells in the treatment of such diseases.

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


BioLife Solutions Inc.

Biopreservation Yield in Cell Therapy: Process Optimization From Source Material to Finished Product Description Participants in the Biopreservation Yield in Cell Therapy: Process Optimization workshop will experience presentation and discussion of a new class of preservation media products which result in the optimization of supply chain logistics and biopreservation processes in hypothermic storage and cryopreservation of various biologic source materials and manufactured cell therapy products. In addition, this workshop will provide a new and detailed understanding of preservation induced cellular injuries and the resultant impact on functional yield of source material and clinical therapeutic yield of manipulated cell therapy products. Critical analysis of traditional post-preservation assay type and timing and how the status quo impacts therapeutic effectiveness of cell therapy products will be introduced through the review of scientific data and selected case studies. This data will support cumulative yield improvements of source material and manufactured cell therapy products using BioLife Solutions’ HypoThermosol® and CryoStor™ products. Speaker Ian B. Nicoud, Ph.D., Director of Technology & Business Development Bio Dr. Nicoud served as an intellectual property and technology consultant for BioLife and joined the Company in September 2007. He has a strong background in transplantation medicine and preservation biology and holds a Ph.D. in Cancer Biology from Vanderbilt University and a B.S. in Biology from Saint Norbert College. He is a co-author of several of the Company’s recent patent applications and manages BioLife’s intellectual property portfolio and technology licensing activities.

Wednesday May 6, 2009 Time: 12:00pm – 1:00pm / Grand Ballroom B STEMCELL Technologies Inc.

Topics and Speakers Expansion of Mesenchymal Progenitor Cells (MPCs) in a Novel Serum- and Animal Component-Free Culture Medium ………………………………………………………………………………………………………………. Ravenska Wagey, PhD Senior Scientist, STEMCELL Technologies Inc. To address the need for serum-free MPC culture, STEMCELL Technologies has developed a novel serum- and animal component-free culture medium which promotes superior clonogenic growth and long-term expansion whilst maintaining multi-lineage differentiation potential of human bone marrow-derived MPCs. Dr. Ravenska Wagey will present performance characteristics of the medium as well as optimal procedures for the isolation, characterization and expansion of MPCs. A Rapid Hematopoietic Colony Assay for Measuring the Potency of Umbilical Cord Blood Grafts ………………………………………………………………………………………………………………. Michelle Bowie, PhD Global Product Manager, STEMCELL Technologies Inc. To meet the need for rapid and easy-to-use potency assays, STEMCELL Technologies has developed a new hematopoietic colony assay that can be completed in 7 days, is easier to score than standard CFU-assays and provides accurate measurement of hematopoietic progenitor cell frequencies in cord blood units that correlate well with results of standard CFU-assay formats. Dr. Michelle Bowie will present an overview of the hematopoietic CFU assay as well as the performance characteristics of STEMCELL’s 7-day CFU-assay.

Somatic Cell Therapy for Cardiac Disease: FDA issues a new draft guidance

FDA has issued a new draft guidance entitled "Somatic Cell Therapy for Cardiac Disease".

The guidance is intended to provide sponsors who are developing cellular therapies for the treatment of cardiac disease, with recommendations on the design of preclinical and clinical studies, and on the chemistry, manufacturing, and controls (CMC) information to include in an investigational new drug application (IND) for cardiac cellular therapy.

This guidance provides to sponsors developing cellular therapies for the treatment of cardiac disease with recommendations including, but not limited to, the following: (1) Design of preclinical and clinical studies; (2) information to submit on the product delivery system; and (3) the chemistry, manufacturing and controls information to include in an IND for cardiac cellular therapy. This guidance also includes regulatory considerations for the use of intravascular catheter delivery systems including recommendations regarding the information that sponsors should submit on the product’s delivery system.

The Background section of the guidance states as follows:

Cellular therapies for the treatment of cardiac disease generally meet the definition of “biological product” in section 351(i) of the Public Health Service Act (42 U.S.C. 262(i)), and are regulated by the Center for Biologics Evaluation and Research (CBER). Intravascular catheters and other drug and biologic delivery systems generally meet the definition of “device” under section 201(h) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 321(h)).

FDA expects that these cellular therapies and their dedicated delivery systems would meet the definition of a combination product under 21 CFR 3.2(e), either by being co-packaged or, if marketed separately, by using labeling that indicates they are intended for use together. Based on available information, the somatic cellular therapy generally would provide the primary mode of action of the combination product and the lead center typically would be CBER in accordance with 21 CFR 3.4.

For a combination product, a single investigational application is generally sufficient. For cellular therapies and their dedicated delivery systems, this generally would be an IND. The IND application should contain the information as described in this guidance on the biological product and on the delivery system, along with any other information as required by the applicable regulations. CBER and CDRH intend to consult or collaborate on the IND review, as appropriate for the science and technology of the combination product.

For a combination product, one marketing application is also generally sufficient for FDA review and marketing authorization. However, sometimes two marketing applications may be necessary.

There has been much debate about how to interpret differing data and theories on the mechanism of action of cell therapies in cardiovascular disease. Clearly the agency is looking to set some standards in the design of these trials. I'll let colleagues more qualified than I on the subject comment on what they've accomplished with the draft guidance.

Regenmedguru perhaps?

Cell Therapy Industry HiLites 2009-04-10

Here we are in April 2009. The much newly anticipated date when we expect the Dendreon saga to head toward a climax. Few people know the Dendreon story better then Xconomy journalist, Luke Timmerman who does an outstanding job of summarizing the saga and stakes in Dendreon's immiment Provenge gamble. Indeed tomorrow is the beginning of what we now anticipate will be the final chapter in this epic sage. Tomorrow (at 9am EDT), Dendreon will discuss the final results from their phase III IMPACT trial of Provenge for prostate cancer (call details here).

Here are a couple random bits to kick us off:

• According to a recent statement by the high-profile VC Steve Burrill, $5M in VC money used to buy 30-50% of a company; now it buys 90%.

• GEN's editor in chief, John Sterling has included "Stem Cells: Translating Exciting Preclinical Data into Human Clinical Trials (Translational Medicine)" as one of his top-10 session picks for the upcoming 2009 BIO International Convention.

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Not settling with organizing doctors against the FDA with his "American Stem Cell Therapy Association (ASCTA)" (see my previous blog comments), Dr. Christopher "the renegade" Centeno is now inciting patients to oppose the "FDA's position that the patient's own adult stem cells are drugs and should be regulated as such." (Click here for the press release.)

To facilitate patient activism he has launched a patient web-site, safestemcells.org, to drive the activities and opinions of patients in what he's calling the Safe Stem Cells NOW! movement. The Stem Cells Now! movement's goal? "To inform patients and physicians that their ability to access safe stem cell treatment is being heavily restricted by pharmaceutical industry agendas and by the FDA."

He has also recruited the enthusiasm of Barbara Hanson, co-founder of stemcellpioneers.com, who is quoted as saying:

"Adult stem cells are cells from our own body. They are very safe. There are no moral or ethical issues. They are safer than taking aspirin and yet the FDA has classified our own stem cells as drugs that require regulation. This means that prolonged investigations, including lengthy clinical trials, will be required for each and every disease and application that adult stem cells could be used for. This could take years and years. It smells of big pharma to me and many others."

For my other posts about the work of Dr. Centeno, see Regenexx vs the FDA 2009 and Cell therapy is not the practice of medicine.

FINANCIAL

Some people I talk to in the sector say they feel weirdly immune from the entire economic meltdown because the industry seems to be ticking along just fine in many ways continuing to be fueled by its own internal optimism and external enthusiasm for the promise of cell-based therapies...well that, and the still-growing amount of money being spent on the research side of the sector. Others, are certainly feeling the pinch. The dividing line may well be those who are trying to raise money and those who are not.

Having said that, here we go with reports of money being raised -- all from non-US investors mind you. Proving that its economy is not down and out, the first two report of fundraises come from Down Under.

There's no way I can start this first story any better then The Australian did in its March 12 report (that I only now just picked up now): "From the miracles-do-happen file comes this provident yarn of a $3.4 million market cap tiddler that has secured $12 million of funding for further development of its ovarian cancer drug, CVac."

They're talking, of course, about Prima BioMed Limited (ASX:PRR) which has been busy reinventing itself with new management and money since Biomira took a pass on its option to partner on CVac development more than a year ago. The company has been slowly bleeding money and management since. The company is still clearly on a low burn because I had to mine this information from anywhere but direct sources. The company itself has been very quiet about any of this.

The news is this: Prima Biomed has secured access to a $12-million funding facility from investment bank Fortrend Securities to help commercialize the company’s ovarian cancer treatment. The deal allows Prima to place shares with Fortrend as needed over the next three years.

Prima said it will use the cash to move through a phase IIb trial and towards a pivotal US registration trial of its dendritic cell-based ovarian cancer therapy, which the company says is designed to stimulate the body’s own immune system to attack cancer cells. The company said it now hopes to begin a US trial by mid-year, after the company reported positive feedback on its pre-investigational new drug meeting with the US Food and Drug Administration last year.

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Not to be outdone by the magic tricks of its pennystock sibling, the much more stable Mesoblast Limited (ASX: MSB) which reported on 31 December 2008 having $9.6 million of capital at its disposal announced that it has successfully raised an additional $10.81 million by completing a private placement to existing, as well as new, institutional and sophisticated investors. The new funds will be used to expand the company’s clinical trial programs focusing on bone and cartilage regenerative products for spinal vertebral disc disease. These programs will be pursued in parallel to the Company’s ongoing Phase 2 clinical trial in knee osteoarthritis. The placement of 15.02 million shares was reportedly oversubscribed and was made at a 10% discount to the closing price of the Company’s shares on 25 March 2009.

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NeoStem, Inc. (Amex: NBS) reports they have completed an $11 Million series D private placement financing from three Asia-based investors, including a private equity firm operating in partnership with strategic investors. Investors are RimAsia Capital Partners, LP, a pan-Asia private equity firm operating in partnership with a regional network of strategic investors drawn from leading Asian families and companies, investing $5 million; Enhance Biomedical Holding Corporation based in Shanghai, and investing $5 million and Elancrest Investments Ltd., a Singapore-based firm, investing $1 million.

While NeoStem makes mention of using the funds in various ways including the development of its VSEL (very small embryonic-like stem cells) technology licensed from the University of Louisville, what they really seem focused on is expansion activities in China, including those relating to its pending acquisitions and medical tourism - defined as travel by people whose primary and explicit purpose is to access in a foreign country medical treatment not yet available in their own nation.

NeoStem expects to connect U.S. citizens with advanced therapies not yet available in the U.S., and attract people from other countries to seek regenerative therapies in China. Indeed Eric Wei, Managing Partner of RimAsia Capital Partners, LP is quoted as saying, "We are extremely optimistic about the growth opportunities for NeoStem based on its multi-pronged plan to capitalize on a growing PRC-based network. We are glad to be joined by other investors in Asia who recognize the huge market potential for stem cell therapies in the PRC [People's Republic of China], a field in which breakthroughs are developing at an accelerating rate."

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In a UK report about how stem cell innovation is at risk, the study authors from The University of Nottingham, funded by the Engineering and Physical Sciences Research Council (EPSRC), reports that the global cell therapy industry, reports that the global cell therapy industry:

"...now involves nearly 200 companies developing primary and secondary cell therapies, plus another 180 banking cord blood. In total the global cell therapy industry currently has sales of over $1 billion a year and a steady number of products are now reaching late stage clinical trials."

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Crystal Research Associates, LLC has issued an Executive Informational Overview® (EIO®) on Neuralstem, Inc. (CUR: NYSE-Alt)

CLINICAL

Amorcyte, Inc recently presented data at the American College of Cardiology (ACC) Annual Scientific Session from its Phase I clinical trial of AMR-001, the company's lead product for the treatment of damaged heart muscle following acute myocardial infarction (AMI), that showed a significant relationship between cell dose and biologic effect. According to the company this is the first and only study to prospectively define a dose of a purified and potent autologous stem cell therapy (AMR-001) that resulted in a significant improvement in perfusion, a trend towards improved cardiac function and the potential to reduce subsequent adverse cardiac events following AMI. Emory University - one of the study sites - also issued its own press release on the data.

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At the same meeting, TCA Cellular Therapy’s Medical Director Gabriel Lasala, M.D. presented preliminary results of an adult stem cell treatment for severe limb ischemia from its Phase I safety/efficacy clinical trial using a combination of the patient’s own endothelial progenitor cells (EPCs) and mesenchymyal stem cells (MSCs) obtained through bone marrow aspiration then mixed and infused into damaged veins.

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MolMed S.p.A. (MLM.MI) announced that results obtained in the European multicentric Phase I/II study (TK007) of its TK therapy have been published in the April issue of the prestigious medical journal The Lancet Oncology. The article - Infusion of suicide gene-engineered donor lymphocytes after family haploidentical hemopoietic transplantation for leukemia: the TK007 phase I/II trial - gives insight into the outcome of the trial, conducted on 54 adult patients, that resulted in significant survival improvement of patients affected by high risk acute leukaemia receiving bone marrow transplants from a partially compatible family donor (haplo-transplants). In this context, the introduction of TK therapy allows the use of add-backs of donor T lymphocytes, that promote a rapid and wide immune reconstitution, abating transplant-related mortality and permitting long-term survival.

These trial results were, of course, already communicated to the market and included in MolMed’s Offering Circular and allowed MolMed to get authorisation to begin a Phase III randomised trial that started in Italy in Spring 2008, and will be expanded to other European clinical centres this year. The Phase III trial TK008 is pivotal for the registration of TK therapy, that could become among the very first cell therapies using genetically engineered cells to obtain marketing approval. MolMed’s partner for the Asian markets, Takara Bio Inc. (OTCPK:TKBIF), started clinical development of TK in Japan in October 2008, with a Phase I trial in relapsed leukaemia patients conducted at the National Cancer Center in Tokyo.

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These breasts and slim waist are brought to you by Cytori Therapeutics, Inc. (NASDAQ: CYTX) and GE Healthcare. Available soon to women for only £6,500?

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While Prima BioMed has been closing the deal on its new financing it has also apparently obtained permission from the Australian TGA's Special Access to treat a patient with its CVac ovarian cancer treatment. The injection of the vaccine in ovarian cancer patients works as a postsurgery and post-chemotherapy maintenance therapy to delay relapse and control metastases. While the company is currently not in clinical trial in Australia and has not yet filed its IND for FDA approval in the U.S., the company has sought special exemption for a single-use treatment for what must be a very special patient.

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Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced the treatment of the first patient in a Phase II clinical trial evaluating Prochymal for the treatment of heart attacks.

COMMERCIAL

Further to my speculative post here last week, we've now learned that ULURU Inc. (NYSE Amex: ULU) has signed a non-binding offer letter to acquire York Pharma plc ("York"). York Pharma is a United Kingdom based skin care company with operations or distribution networks throughout Europe with an established revenue base. By way of reminder, the reason this is of interest here is that York has two cell therapy products on the UK market for the treatment of chronic wounds and burns: Myskin™ and Cryoskin™.

While the companies have not yet executed definitive documentation relating to any acquisition transaction and there's no guarantee they will, Uluru is hoping potential benefits will significantly accelerate the maturation of ULURU into a commercially based company with a strong revenue base and positively position the company for more rapid growth both in the United States and Europe.ULURU Inc. is a specialty pharmaceutical company focused on the development of a portfolio of wound management and oral care products to provide patients and consumers with improved clinical outcomes through controlled delivery utilizing its innovative Nanoflex(TM) Aggregate technology and transmucosal delivery system

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Novocell, Inc. announced that it has received U.S. Patent 7,510,876 with claims covering human definitive endoderm cells, an essential cell for generating not only pancreatic type cells, which Novocell is developing for use as a cell therapy for diabetes, but also other endoderm lineage-derived tissues and organs such as lungs, intestine, liver, thymus and thyroid.

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Progenitor Cell Therapy LLC board member Robert A. Hamm promoted as COO of drug maker Biogen Idec.

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Looks like WuXi PharmaTech Incorporated has some serious buyer's remorse of it $162.7 million purchase of AppTec Laboratory Services Inc.

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Advanced Cell Technology, Inc. (OTCBB: ACTC.PK) could receive up to $1.9 million in fees as a result of a new licensing agreement with its Korean partner. ACT has agreed to license certain retinal pigment stem cell technology to CHA Bio & Diostech Co. Ltd. for development and commercialization exclusively in Korea. The two companies have a recently formed joint venture called "Stem Cell & Regenerative Medicine International" located in Worcester, MA and focused on the development of human blood cells. It is majority owned by CHA Bio and benefits from ACT exclusively licensing all of its hemangioblast technology to the joint venture.

If all milestones are met in this new collaboration focused on stem cell technology to treat a variety of eye diseases, ACT stands to earn $1.9 million in fees. CHA will pay all fees associated with clinical trials expected to take place in Korea. Meanwhile, ACT has applied to the U.S. Food and Drug Administration to begin clinical testing of its retinal pigment stem cell technology during the second half of the year.

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Presumably to help soften the financial blow of its interrupted phase III trial of Prochymal for Crohn's disease, Osiris Therapeutics, Inc. (NASDAQ:OSIR) has announced that it has agreed with NuVasive, Inc. (NASDAQ:NUVA) to accelerate transfer of the Osteocel business. Under the terms of amendments to the existing agreements between the parties, NuVasive will assume responsibility for Osteocel processing by April 10, 2009 and has removed contingencies applicable to $30 million of the $45 million in remaining milestone payments.

Osiris is now scheduled to receive $30 million in cash or NuVasive common stock in 3 schedule payments all before the end of September. The terms applicable to the remaining $15 million milestone payment are unchanged. This additional payment becomes due and payable when NuVasive achieves $35 million in cumulative Osteocel sales. NuVasis has projected $29 million in Osteocel sales in 2009.

To date Osiris has received from NuVasive $40 million in upfront and milestone payments. Effective April 10, Osiris will cease all operations associated with Osteocel processing. Concurrent with the amendments to the Asset Purchase and Manufacturing agreements, Osiris will transfer its Osteocel inventory to AlloSource, of Centennial, Co., which had been processing quantities of Osteocel for Osiris and has now agreed to produce Osteocel solely for NuVasive. NuVasive has entered into a separate outsourcing supply agreement with AlloSource for the continued manufacture of Osteocel.

Because of that deal, they are now able to layoff 80 people.

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It looks like the ever well-tanned Burton Feinerman is back in control of his company Stem Cell Biotherapy which he at least appeared to lose temporarily to his former partner Casey Nabavi until Nabavi abandoned ship late last year to start a competing stemcell transplant company, Cellulogix. Now it looks like things are going to bet rough between the former partners. Stem Cell Biotherapy ("SCB") has filed suit in Los Angeles County Superior Court against the Company's Co-Founder and former President Casey Nabavi of Calabasas, California, for among other things conduct by Nabavi giving rise to patient claims against Nabavi that he had misrepresented himself as a medical doctor, practiced medicine without a license, supervised the injection of stem cells which caused some patients to become ill and personally advised patients how to manage these side effects. Nabavi/Cellulogix have offices/clinics in Los Angeles, Tampa, Montreal, Zona Rio (Mexico) and Istanbul. The SCB lawsuit also seeks to recover SCB funds in excess of $1.65 million that Navabi paid himself before leaving SCB, to recover assets including intellectual property belonging to SCB which Navabi took and used to start-up the new Cellulogix business, to prevent Nabavi from continuing to compete with SCB in contravention of the parties' non-competition agreement and for SCB to recover ownership, the business and profits of Cellulogix as belonging to SCB not to Navabi as a result of Nabavi's wrongful conduct and activities. A copy of the SCB lawsuit against Nabavi and Cellulogix can be viewed at the Company's web site www.stemcellbiotherapy.com.

SCB is in the business of "assisting persons who desire to try stem cell therapy treatment for serious medical problems which have not responded to traditional medical treatments." SCB uses medical clinics and specialized doctors in locations around the world where stem cell therapy is authorized to treat medical conditions such as spinal cord injury, ALS, Parkinsonism, Alzheimer's, multiple sclerosis, autism, cerebral palsy, brain damage, heart disease, COPD, kidney diseases, eye diseases, diabetes, transverse myelitis, etc.

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Adding themselves to the growing list of companies looking to leverage their cell-based technologies to get to market early with some type of non-therapeutic low-hanging fuit in order to finance their more serious, therpeutic product development aspirations, Stem Cell Therapy International Inc. (OTCBB: SCII) -- soon to be known as AmStem International after a successful merger with Histostem Ltd, of South Korea -- has decided that its first product will be a "cosmetic face cream made with stem cells" to be sold wholesale to major cosmetic distributors worldwide. This is expected to provide AmStem with an influx of capital to finance "expansion in other areas".

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Proving there may never be an end to the types of stem cell banking models that continue to pop up, StemSave™, Inc. is now encouraging dental patients to bite on their offer to bank stem cells collected from their disposed teeth. StemSave’s patented technology turns routine trips to the dentist into "potentially life-saving experiences".

So we can now pay to save our stem cells from our umbilical cord blood, umbilical cords, amniotic fluid, placenta, adipose (fat) tissue, menstrual blood, bone marrow, blood (after stem cell mobilization or not), and teeth. Hmmm.

RESOURCES, EVENTS & MISCELLANEA

Regenerative Medicine Vol. 4, No. 2, March 2009 is now available online.

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Stem cell scientist Dr. Robert Lanza, Chief Scientific Officer of Advanced Cell Technology has managed to find time away from changing the world of science and modern medicine to pen a book with astronomer Bob Berman on Biocentrism , a subject that challenges our traditional, outdated perceptions on life, time & space, and even death.

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The New York Academy of Sciences, China’s Ministry of Health, the Chinese Academy of Medical Sciences, the Chinese Academy of Sciences and international partners including the Medical Research Council of the UK and the German Research Foundation are collaborating to host a conference entitled ‘Regenerative Medicine’, May 15-16, 2009 in Beijing, China. The meeting is intended to foster collaborations between industry, academia, government, and finance with a world-renowned scientific organizing committee including Nobel Laureate Dr. Torsten Wiesel and the Chinese Minister of Health, Zhu Chen. The scientific program includes Drs. Anthony Atala, John Gearheart, and Stefanie Dimmeler, among many other leaders from industry, academia, and the clinic. A full conference agenda can be found at www.nyas.org/regmed

Sign-off

That's all I got.

Thanks again to lab equipment supplier, Next Advance, for supporting this issue of the Cell Therapy Industry Hilites. They found me on Twitter, you can too. I'm @celltherapy.

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

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.

Cell Therapy Stimulus Opportunities

There are some real, imminent stimulus funding opportunities related to cell therapy that offer up to $1 million in funding over 2 years for up to 200 projects all to be handed out quite quickly. If you're interested, here is some info.

To cut right to the chase here's the link:

Recovery Act Limited Competition: NIH Challenge Grants in Health and Science Research (RC1) — request for applications (RFA) number: RFA-OD-09-0003. Bethesda, MD: National Institutes of Health, March 4, 2009. (http://grants.nih.gov/grants/guide/rfa-files/RFA-OD-09-003.html)

The American Recovery and Reinvestment Act of 2009 (Recovery Act) (H.R. 1, S. 1 - PDF-1MB) is a Federal public law passed by the 111th United States Congress and signed into law by President Barack Obama on February 17, 2009.

As part of the Recovery Act, NIH has designated at least $200 million in FYs 2009 - 2010 for a new initiative called the NIH Challenge Grants in Health and Science Research, to fund 200 or more grants, contingent upon the submission of a sufficient number of scientifically meritorious applications.

The April 9 issue of NEJM (Volume 360:1479-1481) summarizes the program as follows:

The NIH also announced that it had designated "at least" $200 million of the stimulus money to fund 200 or more grants in specific challenge areas (comparative-effectiveness research, clinical research, genomics, information technology for processing health care data, regenerative medicine, stem-cell research, and nine others) that "focus on specific knowledge gaps, scientific opportunities, new technologies, data generation, or research methods that would benefit from an influx of funds."

This program will support research on Challenge Topics which address specific scientific and health research challenges in biomedical and behavioral research that will benefit from significant 2-year jumpstart funds.

Challenge Areas, defined by the NIH, focus on specific knowledge gaps, scientific opportunities, new technologies, data generation, or research methods that would benefit from an influx of funds to quickly advance the area in significant ways. The research in these areas should have a high impact in biomedical or behavioral science and/or public health.

Links to high priority topics within the broad Challenge Areas that relate particularly to cell therapies include:

(06) Enabling Technologies
(07) Enhancing Clinical Trials
(11) Regenerative Medicine
(13) Smart Biomaterials – Theranostics
(14) Stem Cells
(15) Translational Science

I've scanned through and below are some of the funding high priority topics that I thought might be applicable and of interest here but it is certainly not exhaustive (in other words you should look for yourself):

06-DK-101* Development of cell-specific delivery systems for therapy and imaging. Develop non-viral strategies for cell-specific delivery of pathway-interactors and molecular probes. These new molecular complexes could allow delivery of cell-penetrating agents for the study of disease pathways, the imaging of tissue mass and disease progression, or the development of tissue-specific therapeutics. Contact: Dr. Olivier Blondel, 301-451-7334, blondelol@mail.nih.gov; NIAAA Contact: Dr. Samir Zakhari, 301-443-0799, szakhari@mail.nih.gov; Contact: Dr. Joan McGowan, 301-594-5055,
NIAMShelp-NIHChallengeGrants@mail.nih.gov

06-EB-102* Development of biomedical technologies and systems. Focus areas include: (1) providing immediate diagnostic information for multiple conditions at the point-of-care; (2) a robust, consistently accurate glucose sensor with extended functional lifetime, improved accuracy and low variability of readings; or (3) low cost diagnostic or therapeutic systems. Also, development of such devices engineered to work in low resource settings. Contact: Dr. William Heetderks, 301 451-6771, heetderw@mail.nih.gov

11-DC-101* Hair Cell Regeneration and Maintenance. One common cause of hearing impairment in humans is the progressive loss of the auditory transduction cells, or hair cells, in the inner ear. A similar loss of motion transduction cells in the vestibular organ is a probable cause of balance disorders. Once lost, these cells cannot be spontaneously regenerated in mammals. The Challenge is to develop and validate methods to regenerate and maintain hair cells in animal model systems with the eventual goal of successful translation to human treatments. Contact: Dr. Nancy Freeman, 301-402-3458, freemann@nidcd.nih.gov

11-EB-101* Vascular networks in engineered tissues. Research on the design, optimization, and formation of a complete vascular network capable of delivering oxygen and nutrients and removing waste products in engineered tissues (i.e., vascularization of engineered tissue constructs). Dr. Rosemarie Hunziker, 301-451-1609, hunzikerr@mail.nih.gov

11-HL-101* Develop cell-based therapies for cardiovascular, lung, and blood diseases. Cell-based therapies for cardiovascular, lung, and blood diseases offer a new paradigm for advancing and transforming patient care. Translational and early-phase clinical research has demonstrated that cell-based therapies may improve left ventricular function, reduce myocardial ischemia, and lead to improved lung function. Reconstitution of normal hematopoeisis using modified stem cell graft sources has great potential for treating specific genetic blood disorders. However, a number of significant challenges and barriers must be overcome to move the field forward toward broad clinical application. We encourage further research to determine the characteristics of the most promising target patient population, the best cell type and number of cells to use, the optimal methods and timing of delivery, and other preclinical parameters. Contact: Dr. Sonia Skarlatos, 301-435-0477, skarlats@nhlbi.nih.gov

14-EY-101* Development of stem cell treatment for degenerative diseases of the eye. The restorative properties of stem cells hold the promise in the treatment of degenerative eye diseases such as macular degeneration, diabetic retinopathy, retinitis pigmentosa and glaucoma, and diseases of the ocular surfaces. There is a need for the identification of biomarkers that can define stem cells and the end-stage cells, as well as reproducible protocols for the generation and purification of viable terminally differentiated cells. Contact: Dr. Grace Shen, 301-451-2020, sheng@mail.nih.gov

15-RR-101* Applied translational technology development. This program will support two-year applied translational projects to move advanced technologies from the prototype stage into the clinic. Novel, cost-effective tools for clinical care or clinical research will be modified, hardened, and tested. Interdisciplinary teams of technology developers, basic researchers and clinicians will address scientific and engineering problems associated with clinical applications of new technologies. Contact: Dr. Douglas Sheeley, 301-594-9762, sheeleyd@mail.nih.gov; NIDA Contact: Dr. Kris Bough, 301-443-9800, boughk@mail.nih.gov

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The broader Omnibus of Broad Challenge Areas and Specific Topics has many more challenge topics including the following I thought were potentially applicable:

06-EB-108 Imaging of Drug and Gene Delivery Systems. Three major challenges in the field of drug and gene delivery are: targeting of therapies to tissues, cells, and intracellular compartments; monitoring exactly where the therapies localize after administration; and determining if the agents delivered are doing what they were intended to do. We encourage proposals to develop multifunctional systems that: 1) are capable of targeted delivery of drugs, proteins, genes, or nucleic acids to specific cells, or compartments within cells in vivo; and 2) possess imaging capabilities to track delivery, assess function, and determine therapeutic efficacy. Contact: Dr. Lori Henderson, 301-451-4778, hendersonlori@mail.nih.gov

06-HL-103 Develop new imaging methodologies to track cells and measure accurately the chemical activities of enzymes and metabolites in intact cells, tissues, and organisms to improve basic understanding of cellular interactions, biological pathways, and their regulation. An improved ability to track cells in vivo will enhance our understanding of homing, engraftment, cell differentiation, and pathogenesis resulting from abnormal cells trafficking. Understanding the components and kinetics involved in biochemical reactions is key to evaluating and predicting the response of intact organisms to physiological and pathophysiological challenges and drug responses. Although our knowledge of the identity and quantity of proteins and complexes associated with reaction pathways in health and disease continues to advance, direct methods for imaging those reactions in intact systems are lacking. Development of appropriate tools to track cells, image the microvasculature, and image chemical activity in intact systems in real time will have broad applicability to many heart diseases, including myocardial ischemia and reperfusion injury, heart failure, and arrhythmias and lung diseases such as COPD, asthma, pulmonary hypertension, and sleep apnea. Similarly, new non-invasive cellular imaging modalities, capable of differentiating between normal and pathological states, would increase our understanding of the role of the microvasculature in sickle cell disease and thrombotic disorders. Contact: Dr. Lisa Schwartz Longacre, 301-402-5826, schwartzlongal@mail.nih.gov

11-DK-104 Use of Hematopoietic Stem Cells (HSC) to regenerate or repair mesenchymal tissues. Examples include: Develop and validate methods and reagents that induce HSCs to develop or trans-differentiate into different mesenchymal cell and tissue types; Develop and validate methods and reagents that allow HSCs to be propagated in vitro without loss of self-renewal potential. Contact: Dr. Terry Bishop, 301-594-7726, bishopt@mail.nih.gov

11-EB-105 Advanced Imaging Systems for Tissue Engineering. The ability to monitor complex cell-cell and cell-matrix interactions in engineered tissues in vitro and in vivo is critically important. The imaging needs to be functional—able to assess meaningful changes non-destructively and non-invasively; intrinsic—using inherent signatures of normal biological processes (e.g. intermediates of energy metabolism, conformationally-based changes in light scattering); and dynamic—monitoring events as they are occurring. Proposals to develop tools for characterizing engineered tissues in vitro and in vivo are encouraged. Contact: Dr. Rosemarie Hunziker, 301-451-1609, hunzikerr@mail.nih.gov

11-EB-106 Technologies for Expanding Stem Cells and Producing Engineered Tissue. Tissue engineering and regenerative medicine is a rapidly evolving field, but current production and manufacturing technologies are confined to the laboratory scale and grossly inadequate to ensure sufficient quantity and quality on an industrial scale. New measurement tools, and engineering methods and design principles that can model, monitor, and influence the interaction of cells and their environment at the molecular and organelle level are urgently needed. Projects are sought to develop scaleable bioreactors to precisely control the chemical and mechanical environment for functional 3D tissue growth or for rapidly expanding stem cells; quantitative, non-invasive tools to monitor structure, composition, quorum sensing, and function of heterogeneous tissues in real time; and technologies for preservation, sterilization, packaging, transport, and ensuring cell and tissue health and phenotypic stability. Contact: Dr. Albert Lee, 301-451-4781, alee@mail.nih.gov

11-GM-101 Establishment of regenerative capabilities. Development of approaches and technologies to establish regenerative capabilities in adult cells to repair or replace damaged tissues and organs in situ and to improve wound healing and reduce scarring in human and animal models. Contacts: Dr. Susan Haynes, 301-594-0943, hayness@nigms.nih.gov, Dr. Richard Ikeda, 301-594-3827, ikedar@nigms.nih.gov

13-DK-104 Islet encapsulation. Development of novel islet encapsulation technologies/biomaterials for the optimization of a bioartificial pancreas. Contact: Dr. Guillermo Arreaza, 301-594-4724, arreazag@mail.nih.gov.

13-EB-102 Non-viral Gene Delivery Systems. The major barrier to success of gene therapy in the clinic is the lack of safe and efficient DNA delivery methods. Although viral delivery systems allow efficient and long-term gene expression, they generally do not permit targeted delivery to particular cells and tissues and pose problems with regard to immune response. Proposals are invited to develop novel, safe, and targeted, synthetic or viral mimetic vectors for gene delivery including quantitative studies that relate their structure and properties to function. Contact: Dr. Lori Henderson, 301-451-4778, hendersonlori@mail.nih.gov

14-DE-103 Enhancing Human Embryonic Stem (ES) Cell Culture Systems. Cell differentiation and tissue morphogenesis during normal development is guided by the highly orchestrated temporal, spatial and combinatorial action of multiple of ligands, signaling pathways, transcription factors, and extracellular matrices. In light of this tremendous complexity, the existing human ES cell in vitro culture systems lack appropriate sophistication thus necessitating the need for strategies to better mimic normal developmental processes. Recent progress in the fields of bioengineering, nanotechnology, biomaterials and bioimaging offer a wealth of tools that can lend tight control of the multiple parameters needed to improve the existing human ES culture systems. Goal: Integration of engineering disciplines with developmental biology and with ES cell technology for deriving a new generation of human ES cell culture protocols that will facilitate the application of ES cell-based therapies for the treatment of a multitude of human tissue degenerative diseases and trauma, including those of oral and craniofacial complex. Contact: Dr. Nadya Lumelsky, 301-594-7703, Nadya.Lumelsky@nih.gov

Finally, here is some other key data and links.

Challenge Award Resources

• 2009 Funding Opportunity Announcement (RFA-0D-09-003)
• NIH Institute & Center (IC) Web Sites
• Frequently Asked Questions - NIH Challenge Grants
• Challenge Awards Review Timeline

Some key timeline dates:
Application Due Date(s): April 27, 2009
Peer Review Date(s): June/July 2009
Council Review Date(s): August 2009
Earliest Anticipated Start Date(s): September 30, 2009

Budget requests should be commensurate with project needs up to a two-year project period. The requested budget may not exceed $500,000 total costs per year for a maximum of $1,000,000 total costs over a two-year project period.


Thanks to Bob Speziale from Invetech to pointing this out to me and thus...to you. Good luck to you all.

Will the real regenmed association please stand up?

Further to my post on this topic back in September last year and ongoing discussions I have been having, I have decided to create a quick 4-question poll to get your opinion.

I am not representing anyone or any organization with this survey. I'm out on a limb on my own. I'm curious to see your response to this quick 4-question survey.

Click here to see the survey.

CIRM & friends join efforts for one-day focus on translating stem cells into products

This is my public service announcement for the week. ISCT continues to stack on the value to it's annual meeting this year in San Diego with the recent announcement that ISCT, CIRM, and ISSCR are collaborating to bring a unique cell therapy translational development workshop to California.

Translation of Stem Cell Therapies:
Best Practices and Regulatory Considerations

Sheraton San Diego Hotel and Marina

Saturday, May 2, 2009

DRAFT PROGRAM

7:00am – 7:50am Registration

7:50am – 8:00am Welcome + Introduction

8:00am – 10:00am Introduction to Translation of Stem Cell Therapies
Session chair: EJ Read MD

CIRM’s Initiatives for Translation of Stem Cell Therapies
Marie Csete, MD PhD (CIRM)

FDA’s Approach to Stem Cell Therapies
Kimberly Benton PhD (FDA/CBER/OCTGT)

IND Planning & Preparation for Stem Cell Therapies
EJ Read, MD (BSRI/UCSF-CTSI)

CMC Preparation & Pitfalls for Stem Cell Therapies
Darin Weber, PhD (Biologics Consulting Group)

Q&A/Panel Discussion


10:00am – 10:20am Break


10:20am – 11:50pm Late-Stage Translation: Cell Banking and GMP Manufacturing Models
Session chair: Shelly Heimfeld PhD

Cell Banking: FDA Perspective
Brenton McCright PhD (FDA/CBER/OCTGT)

Banking/Manufacturing Approach for Banked, Off-the-Shelf hESC-Derived Allogeneic Product (Oligodendrocytes, for spinal cord injury)
Anthony Davies, PhD (Geron)

Manufacturing Approach for a Banked, Off-the-Shelf Allogenic Fetal-Derived Product (CNS Stem Cells, for neurology applications)
Stewart Craig, PhD (StemCells Inc)

Manufacturing Approach for a Patient-Specific Allogeneic Expanded Cord Blood Progenitor Cell Product
Shelly Heimfeld, PhD (Fred Hutchinson Cancer Research Center)

Q&A / Panel Discussion


11:50pm – 12:50pm Lunch


12:50pm – 2:20pm Mid-Stage Translation: Assay Development and Preclinical Safety Testing
Session chair: Darin Weber PhD
FDA panelists: Theresa Chen PhD, Thomas Finn PhD

Assay Development for Stem Cell Products
Melissa Carpenter, PhD (Carpenter Group)

Preclinical Safety Program for Myeloid Progenitor Cell Product for Neutropenia and Radiation Injury
Holger Karsunky, PhD (Cellerant)

Preclinical Safety Program for hESC-Derived Products for Neurology Applications
Hans Keirstead, PhD (California Stem Cell and UC-Irvine)

Q&A / Panel Discussion


2:20pm – 3:20pm Mid-Stage Translation: Process Development and Product Characterization
Session Chair: Shelly Heimfeld PhD

FDA Perspective on Development & Characterization Issues
Thomas Finn, PhD (FDA/CBER/OCTGT)

Universal Donor Adult Stem Cell Product (for cardiovascular and other applications): Development and Characterization
Robert Deans, PhD (Athersys)

Gene-Modified Adult Stem Cell Product (for neurology applications): Development and Characterization
Casey Case, PhD (SanBio)

Q&A / Panel Discussion


3:20pm – 3:40pm Break


3:40pm – 4:20pm Early-Stage Translation: Challenges for Developing Combination Products
Session chair: Darin Weber PhD

Case study: Development of a Complex Combination Product (Device + Cells + Genetic manipulation)
B. Lynn Allen-Hoffman. PhD (Stratatech)

Q&A

4:20pm – 5:30pm Early-Stage Translation: Induced Pluripotent Stem Cells
Session chair: Marie Csete MD PhD

IPS cells in Amyotrophic Lateral Sclerosis
Evangelos Kiskinis, PhD (Harvard Stem Cell Institute)

IPS cells in Spinal Muscular Atrophy
Clive Svendsen, PhD (U of Wisconsin)

5:30pm – 5:40pm Wrap Up

5:45pm – 7:15pm Reception