Thusday, August 23

There is no denying that cell therapy has the potential to be one of the most powerful therapeutic options available. Cell therapy can take several forms and serve many purposes including altering normal cell response, stimulating native signaling cascades, performing missing metabolic functions, restoring lost tissue, or changing the normal course of repair into true regeneration. Designing and implementing a plan for commercialization takes many paths, each with their own set of biological, technical and regulatory considerations. CHI’s Inaugural Cell Therapies – The Path to Clinical Success and Commercialization continues the communication required for both clinical and commercial success. Learn from savvy seasoned experts as they share their experiences and achievements. 

Scientific Advisory Committee:

Lee Buckler, BED, LLB, Progenitor Cell Therapy

Rosemarie Hunziker, Ph.D., Program Director, Tissue Engineering and Regenerative Medicine, National Institute of Biomedical Imaging and Bioengineering, National Institutes of Health

Alan K. Smith, Ph.D., President, Cognate BioServices, Inc. 

Bruce M. Wentworth, Ph.D., Director, Cardiovascular Research, Genzyme Corp.

8:00 am Registration and Morning Coffee

8:30 Chairperson’s Remarks

8:35 Challenges of Translational Cell Therapy
Leslie E. Silberstein, M.D., Director, Center for Human Cell Therapy, Principal Investigator, CBR Institute for Biomedical Research; Program Head, Translational Research, Harvard Stem Cell Institute; and Professor of Pathology, Harvard Medical School
The presentation will give a brief overview of the current projects at the Center for Human Cell Therapy and the individual challenges that each of them face. Touching on communication with the FDA, project equipment needs and costs (such as nucleofection), and research development. In addition, I will also address consulting/collaborating with local biotech companies.

9:05 Bridging the Gap between Research and Manufacturing: The Role of Product and Process Development
Katie Faria, Director, Process Development, Research and Development, Organogenesis Inc. 
Using the Organogenesis experience we will discuss the process flow from research and development, process optimization, validation, FDA registration and through to implementation into manufacturing. Specific steps that will be reviewed; identifying and defining potential process issues early on to develop strategies to avoid production, regulatory and/or GMP compliance issues. Researching and evaluating potential solutions, selecting the process solution that best fits operational and quality requirements, designing the process validations and completing the technical transfer into cGMP manufacturing.

9:35 Major Company Perspective
Geoff Symonds, Ph.D., Senior Research Director, Johnson & Johnson Research
Cell Therapy represents a different treatment paradigm and the presentation will address how Big Pharma develops and views Cell Therapy. Similarities and differences to small molecule and biologics development will be discussed, as well as the means by which Cell Therapy can be 'incubated' to a point that it can stand alone.

10:05 Coffee Break, Poster and Exhibit Viewing

10:45 Regulatory Perspective
Joyce L. Frey-Vasconcells, Ph.D., Executive Director, Pharmanet Consulting
Cell therapies as market products is very new. There is currently only one approved cell therapy in the United States. As a result there is very little precedent to gain an understanding of the regulatory requirements. This presentation will discuss some of the regulatory considerations that should be considered as one advances a cell therapy thru the development phases towards marketing.

11:15 Technology Spotlight (Sponsorships Available)

11:45 Cell Characterization Using DNA Methylation Analysis in Cell Therapy
Ulrich Hoffmueller, Ph.D. MBA, Chief Business Officer and Founder, Epiontis GmbH 
Specific DNA methylation patterns correlate with differentiation states, cell type and long-term cell functions and therefore, offer a large potential for cell characterization. Upon genome wide marker discovery and validation DNA methylation markers are applicable to determine identity, purity and potency of cellular therapeutics. The application of the DNA methylation technology is presented on two examples: (1) The results of the collaboration between Genzyme and Epiontis, demonstrating how DNA methylation analysis can be applied for testing the chondrocyte product Carticel®. (2) Marker discovery for a panel of primary human cells comprising 12 cell types from mesenchymal and hematopoietic lineages. These results indicate that DNA methylation analysis qualifies as a suitable technique for cell characterization and routine release quality control tests for products in tissue engineering and regenerative medicine.

12:15 Lunch on Your Own 
(Luncheon Technology Workshop Sponsorships Available)

1:30 Chairperson’s Remarks

1:35 Allogeneic Bone Marrow Repairs Human Islet Damage: A New Approach for Cell Therapy in ex Vivo
LuGuang Luo, Assistant Professor, Research, Roger Williams Hospital
The main roadblock for human islet transplantation is the gradual diminishing of islet function initiated by islet cell death in the short term which results from the islet isolation process. Developing a method to repair islet injury would overcome this problem. In our group, we utilized allogeneic bone marrow cells to co-culture with human islets and showed that human islets are able to survive and function in the long term. The mechanism studies indicate that bone marrow interacts with human islets in the culture condition (time lapse microscope tracing cell-cell interaction), suppresses islet inflammatory factor production (IL-1 beta) and reduces cell apoptosis while stimulating islet growth. This novel concept and method established by this group will provide a new direction for cell therapy.

2:05 Bone Marrow-Derived Stem Cells for CNS Therapy
Casey Case, Ph.D., Vice President Research, SanBio Inc. 
We have developed a scalable method for deriving neural progenitor cells from adult bone marrow aspirates. These cells have been successfully employed in animal models of Parkinson's disease (6-hydroxydopamine lesions in rats), spinal cord injury (contusion injury model in NOD/SCID mice), and stroke (MCA occlusion model in rats). Data from these studies and the experience of transferring the production methods to a contract manufacturer will be shared. A phase I clinical trial in stable stroke is scheduled to initiate early 2008.

2:35 Using Adult Tissues to Generate New Organs: Autologous Cell Replacement Therapy for Diabetes
Sarah Ferber, Ph.D., Director, Molecular Endocrinology, Sheba MDC and Diabetes Research Institute (DRI) Miami 
We were the first to demonstrate the capacity of inducing a functional pancreatic lineage in adult liver in mice and human tissues. This capacity has been confirmed now in many labs around the world (Ferber et al Nat Medicine 2000 and Sapir et al PNAS 2005). We presented the efficacy of inducing functional endocrine pancreas in adult human liver cells by nuclear reprogramming. These developmentally shifted human liver cells produce insulin, process the hormone and secrete it in a glucose regulated manner. The human derived cells ameliorate diabetes when implanted in immunodefficient SCID-NOD mice for long periods. The mechanism of this developmental redirection process will be disclosed. 

3:05 Refreshment Break, Last Chance for Poster and Exhibit Viewing

3:30 TRC Products as an Emerging Therapy for Regenerative Medicine
Ronnda L. Bartel, Ph.D., Vice President, Research and Development, Aastrom Biosciences 
Aastrom's Tissue Repair Cell (TRC) products are derived from a small amount of bone marrow collected from the patient, which is cultured to generate a unique cell mixture containing large numbers of stem and progenitor cells. TRC-based products have been used to treat over 240 patients, and are currently in clinical trials for bone regeneration (osteonecrosis of the femoral head, long bone fractures and spine fusion) and vascular regeneration (critical limb ischemia) applications. Aastrom has reported positive interim clinical trial results for TRC-based products suggesting both the clinical safety and the ability of TRCs to promote healing. The Company is also developing clinical programs for TRC therapies to address cardiac and neural regeneration indications.

4:00 Advancing a Clinical Pipeline of Cellular Medicines
Ed Field, MBA, President & COO, Aldagen, Inc.
ALDAGEN is advancing a clinical pipeline of regenerative therapies focused on vascular disorders and cord blood transplantation. The Company has three product candidates in clinical development. ALDAGEN’s products are based on a proprietary technology platform that selects and delivers potent, adult stem and progenitor cells for therapeutic use. The platform is further differentiated by its ability to obtain a broad range of therapeutic cells that can be used rapidly, without culture or expansion. The presentation will focus on the design of and data from the three clinical trials with particular emphasis on the critical limb ischemia and chronic heart failure trials.

4:30 Panel Discussion: 
Moving Cell Based Therapies into the Market
Cell therapies present a unique and exciting twist in scientific paradigms and clinical modalities.  Equally as true will be the uniqueness of the business models wrapped around these therapies.  While there are many similarities with predecessor biotech sectors, cell therapies present unique challenges in the areas of regulation, product characterization, delivery, reimbursement, cost of goods, etc that will challenge the most creative business executives to create and develop business models which succeed in bringing cell therapies to market.  This panel will explore the various perspectives of stakeholders that will be critical to moving cell therapies into the market:  venture capital, cell therapy business executives, large biotech/pharma, and insurers.  The panel-based discussion will focus on the primary challenges, business models, and strategies for commercializing cell therapies.

Moderator: Lee Buckler, BEd, LLB, Progenitor Cell Therapy LLC

Panel Members:
Ronnda Bartel, Ph.D., Vice President, Research and Development, Aastrom Biosciences
Ed Field, MBA, President & COO, Aldagen, Inc.
Geoff Symonds, Ph.D., Director, Cell Therapy Research, Johnson & Johnson
Paul J. Schmitt, Managing Director, PA Early Stage Partner
Joyce Frey, Ph.D., Executive Director, Pharmanet Consulting
Bruce M. Wentworth, Ph.D., Senior Director, Cardiovascular Research, Genzyme Corp.
Naomi Aronson, Ph.D., Executive Director, Technology Evaluation Center, Blue Cross Blue Shield Association (invited)

5:30 Close of Conference