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THURSDAY, MARCH 27

8:25am Chairperson's Remarks

8:30 Stemming Vision Loss with Stem Cells
Martin Friedlander, M.D., Ph.D., Professor, Department of Cell Biology, The Scripps Research Institute; Chief, Retina Section, Division of Ophthalmology, Scripps Clinic La Jolla
Recent advances in the field of vascular biology strongly suggests that specific molecules already identified as critical to normal angiogenesis (e.g., adhesion receptors, their ligands and ECM components) will have utility in preventing the abnormal growth of new blood vessels in the eye. While this �anti-angiogenic�Eapproach is currently the basis for a number of treatments and human clinical trials, we are hopeful that a new therapeutic paradigm, one in which it may be possible to �mature�Eor stabilize immature, abnormal vessels, will be of far greater benefit to patients suffering from ischemic retinopathies. This may be possible through the use of autologous bone marrow or cord blood derived hematopoietic stem cells that selectively target sites of neovascularization and gliosis where they provide vasculo- and neurotrophic effects. Such a therapeutic approach would obviate the need to employ destructive treatment modalities and would facilitate vascularization of ischemic and otherwise damaged retinal tissue. Such treatments would have application to ischemic retinopathies such as ROP and diabetes as well as degenerative retinopathies such as ARMD and retinitis pigmentosa.

9:00 Transplantation of CNS Progenitor Cells to the Mammalian Retina
Henry Klassen, M.D., Ph.D., Assistant Professor, Ophthalmology, University of California, Irvine
Immature, multipotent progenitor cells have been cultured from the brain and neural retina of a number of mammalian species. Following transplantation, these cells demonstrate the ability to survive as allografts for extended periods without immune suppression and integrate morphologically into areas of retinal disease. In addition, progenitors derived from the neural retinal are able to partially repopulate the outer nuclear layer and express photoreceptor markers in abundance. Studies also indicate measurable augmentation of selected basic visual functions, perhaps secondary to rescue of host cones. The full regenerative potential of these cells remains an area of active investigation.

9:30 Panel of Experts
Clinical View: Martin Friedlander, The Scripps Research Institute
Biological View: Henry Klassen, University of California, Irvine
Culture and Scale-up: Scheffer Tseng, TissueTech, Inc. 
Reimbursements: Naomi Aronson, Blue Cross and Blue Shield
Legal: Thomas Quinlan, Reed Smith, IP
Patient Advocacy Group: Stephen M. Rose, Foundation Fighting Blindness 

10:00 Technology Spotlight (Sponsorship Available)

10:15 Technology Spotlight (Sponsorship Available)

10:30 Poster Competition & Refreshment Break in the Exhibit Hall 

11:30 MMTC Break-Out Groups Hosted by the Panel of Experts

Motivating Meetings for the Therapeutic Community
Join the focused discussion tables hosted by each of the Ocular Panel of Experts

MMTC Group One
Martin Friedlander, M.D., Ph.D., Professor, Department of Cell Biology, The Scripps Research Institute; Chief, Retina Section, Division of Ophthalmology, Scripps Clinic La Jolla

MMTC Group Two
Current understanding of cultured stem/progenitor cell populations 
Henry Klassen, M.D., Ph.D., Assistant Professor, Ophthalmology, University of California, Irvine
Proliferation: Do we have a handle on the double-edged sword?
Long term survival and immunological compatibility: Of mice and men
Cellular immaturity versus tissue integration: Time for a closer look?

MMTC Group Three
Amniotic Membrane As a Novel Strategy in Expanding Limbal Stem Cells In Vivo and Ex Vivo
Scheffer C. G. Tseng, M.D., Ph.D., Director, Ocular Surface Center, Medical Director, Ocular Surface Research & Education Foundation Director, R&D Department, TissueTech, Inc.

• Identification of components in amniotic membrane responsible for anti-inflammatory, anti-scarring and anti-angiogenic actions

• Expand the use of amniotic membrane matrix to facilitate regeneration in wound healing

• Consider the use of amniotic membrane matrix as a carrier to support ex vivo expansion of other progenitor cells

MMTC Group Four
View from the Blue Cross Blue Shield Technology Evaluation Center (TEC)
Host: Naomi Aronson, Ph.D., Executive Director, Technology Evaluation Center, Blue Cross and Blue Shield

• What is important to Blue Cross and Blue Shield Plan medical decision makers? 

• What are the critical outcome measures and how good is the evidence? 

• How will value and affordability shape benefit design and the market for new technologies?

MMTC Group Five
The Foundation Fighting Blindness as a Catalyst in Translational Research
Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness 

• Is there a "best balance" of investigator initiated science vs. targeted research? 

• What is needed to translate bench research to clinical trials? 

• Bench scientists are not the best translators- who to bring in to help get to the clinic? 

MMTC Group Six
Legal and Ethical Landscape
Thomas Quinlan, J.D., Partner, Reed Smith, IP

• Requirements for Ethics Oversight

• Conflicts among diiferent US, various national and international regulatory regimes regarding research

• Intellectual Property developments

12:30pm Luncheon Workshop (Sponsorship Available) or Lunch on Your Own

1:30 Break

1:45 Chairperson's Remarks
Joseph Frank, Ph.D., Chief, Experimental and Neuroimaging Section, National Institutes of Health

1:50 Therapeutic Application of Human Embryonic Stem Cells for Parkinson's Cell Therapy 
Stephen Minger, Ph.D., Senior Lecturer, Stem Cell Biology Lab, King's College London
Cell therapy using early gestational-age human foetal tissue has been shown to provide clinical benefit in patients with Parkinson's disease (PD). However this is not practical on a large scale due to ethical concerns and logistical problems procuring enough tissue. Human embryonic stem (hES) cells have the capability of generating every cell type including dopaminergic neurons lost in PD. Our lab is highly focused on the generation of dopaminergic progenitors from hES cells for human transplantation and we have initiated a programm of work to translate this work into Phase I clinical trials within the next five years. We have assembled a team of stem cell biologists, gene therapy experts, PD neurobiologists, neurosurgeons, and experts in cell therapy clinical regulation and cell banking to fulfill this goal.

2:20 Dopamine Cell Transplantation as a Treatment for Parkinson's Disease 
Curt Freed, M.D., Professor of Medicine and Pharmacology, University of Colorado School of Medicine 
Symptoms of Parkinson's disease can be improved by transplantation of fetal dopamine neurons from abortions, but tissue recovery is difficult. Results in patients in our double-blind, placebo-controlled clinical trial have shown that dopamine cell implants can duplicate the best effects of L-DOPA. In some people with a history of L-DOPA-induced dyskinesias, transplants may produce similar excess movements. Patients have come to autopsy as long as 14 years after surgery. Histologic analysis has shown that implanted dopamine neurons can survive indefinitely even without immunosuppression. The fact that fetal tissue is almost impossible to obtain has limited transplants to very few patients worldwide and only two controlled clinical trials, human embryonic stem cells may provide an unlimited cell source if they can be converted to dopamine neurons and survive implantation into brain. A refined source of dopamine cells will make it possible to regulate tissue dose and thereby have more predictable transplant outcome in the individual patient. We and other groups have studied the differentiation of human embryonic stem cells to a dopaminergic phenotype. The general strategy has been to recapitulate normal brain development. Cells are guided toward a midbrain neuroprogenitor fate and thereafter to ventral mesencephalic dopamine neurons. Based on our past clinical experience and the promise of stem cell research, we believe that neurotransplantation will be an important treatment for patients with Parkinson's disease.

2:50 Drug Screening Assays Using Neural Cells Derived from Human Embryonic Stem Cells 
Steven Stice, Ph.D., Professor and GRA Eminent Scholar, Director of the Regenerative Bioscience Center, University of Georgia/Aruna Biomedical, Inc.
We have developed a high-throughput drug screening assay using neural stem cells derived from embryonic stem cells that could be used for neurological diseases ranging from Alzheimer's and Schizophrenia. 
Representative human neural cells can either compliment or potentially replace rodent or transformed cell lines used screening process. Intuitively, human neural cells that have the proper properties will provide human specific data that may lead to more efficacious and safer drugs that enter clinical trials.

3:20 Plenary Keynote

4:00 Ice Cream Refreshment Break in the Exhibit Hall with BEST OF SHOW AWARDS 

4:45 Panel of Experts
Moderator: Joseph Frank, National Institutes of Health
Biological View: Stephen Minger, King's College London
Clinical View: Curt Freed, University of Colorado School of Medicine
Technical View: Steven Stice, University of Georgia/Aruna Biomedical, Inc.
Commercialization: Stewart Craig, Progenitor Cell Therapy
IP: William Christiansen, Seed IP
Funding: Gregory Bonfiglio, Proteus Ventures, VC

5:15 MMTC Break-Out Groups Hosted by the Panel of Experts

Mingle, Meet, Talk, & Communicate 
Join the focused discussion tables hosted by each of the PD Panel of Experts

MMTC Group One
Stephen Minger, Ph.D., Senior Lecturer, Stem Cell Biology Lab, King's College London

MMTC Group Two
Curt Freed, M.D., Professor of Medicine and Pharmacology, University of Colorado School of Medicine 

MMTC Group Three
What Are the Major Factors to Consider in Using Human Stem Cells in the Drug Discovery Process?
Steven Stice, Ph.D., Professor and GRA Eminent Scholar, Director of the Regenerative Bioscience Center, University of Georgia/Aruna Biomedical, Inc.

• What is the low hanging fruit and where is the biggest need for stem cells 

• What are thresholds for cell purity and functional endpoints 

• Scalability of stem cell production 

• Will they replace steps in the process or be an added screen 

• Convincing the Pharma Industry to use stem cells, other technical and lingering ethical concerns 

MMTC Group Four
Treatment of Parkinson's Disease: Cellular versus Drug Treatment
Joseph Frank, M.D. Chief, Experimental and Neuroimaging Section, National Institutes of Health
The ultimate goal of cell therapy for PD is the reconstitution of nigrostriatal neuronal pathways by endogenous stem cells or grafted Dopaminergic neurons. 

• Which cell or combination of cells should be used in transplantation studies and what are the best outcome measures (i.e., imaging and clinical) for assessing cell engraftment, function and clinical improvement?

• When is the appropriate time to institute cellular therapy for PD?

• How should the micro-environment or niche be assessed prior to transplantation and what needs to be done to prepare area for cell transplantation?

• Can Cellular Therapy succeed for PD when Pharma is using stem cells for drug screening?

• Are there long term alternatives to cellular or drug therapy in treatment of PD?

MMTC Group Five
Applied Solutions for Cell Therapy Product Development & Commercialization
Stewart Craig, Ph.D., Chief Technology Officer & Vice President, Progenitor Cell Therapy, LLC
For cell-based therapies, successful commercialization requires an integrated approach with a cost -effective product development plan to achieve a risk-based balance of:

• The time and resources required for appropriate development and validation of a manufacturing process with associated controls commensurate with commercial-scale operations and an optimized COGs

• The implementation of a Quality Systems and facilities infrastructure required for cGMP and/or cGTP manufacturing operations and product distribution

• A regulatory strategy from preclinical through to commercial launch and beyond, which includes considerations of comparability when introducing changes to the manufacturing process, the product and/or the manufacturing facility 

This discussion group will focus on considerations, approaches and solutions to successfully address the above, including:

• What, where and by when?

• Know your product!

• Regulatory strategy

MMTC Group Six
Intellectual Property Hurdles to Overcome in Developing a Stem Cell Therapy for Parkinson's
William Christiansen, Ph.D., Seed IP

• Current State of IP for stem cells

• The future of stem cell and stem cell therapy IP

• Navigating the stem cell patent minefield

• What do the new USPTO rules mean to current stem cell patent portfolios

MMTC Group Seven
Funding For Stem Cell Companies
Gregory Bonfiglio, J.D., Managing Partner, Proteus Ventures, VC

• Where are we? What is the Current Funding Environment for Stem Cell Companies? 

• Who has the Money? What are the Sources of Funding for Stem Cell Companies?

• What Do They Want? What are the Criteria for Funding? 

• How Do I Get Venture Capital Money? What is the VC Funding Process? 

• How Do I Capture Some of the Value I have Created? What are the Key Exit Strategies? 

5:45 End of Day