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FRIDAY, FEBRUARY 27

THERAPEUTIC THEMES: iPS CELLS AND PERSONALIZED MEDICINE

8:00 am Morning Coffee

8:30 Chairperson’s Remarks
Robert Deans, Ph.D., Senior Vice President, Regenerative Medicine, Athersys, Inc.

8:35 Diagnostic Perspective: iPS Cells for Cardiovascular Diagnostics
Bruce Conklin, M.D., Senior Investigator, Gladstone Institute of Cardiovascular Disease, Professor of Medicine, Division of Medical Genetics, University of California San Francisco 
Our functional genomic experiments focus on GPCR signaling pathways in pluripotent ES cell-derived cardiac myocytes. We use high-throughput gene inactivation methods, including siRNA and gene trapping in ES cells, and then analyze ES cell-derived cardiomyocytes. Our initial signaling studies focused on mouse ES cells, and ES cell-derived mice. More recently we have used human iPS cells for similar signaling studies and to produce models of human cardiac disease, including Long QT syndrome.

9:05 Patient-Specific Pluriopotent Stem Cells for Drug Discovery and Regenerative Medicine
Justin Ichida, Ph.D., Post Doctoral Fellow, Stem Cell and Regenerative Biology, Harvard Stem Cell Institute 
We have created pluripotent stem cell lines from skin cells taken from patients with Amyotrophic Lateral Sclerosis (ALS). We have differentiated these stem cell lines into motor neurons, the cells that degenerate in ALS patients. We aim to use these cells to study the disease in a petri dish and as biologically relevant substrates for drug discovery screening. Furthermore, we are modifying our process for making the pluripotent stem cell lines so that they can be safely used for regenerative medicine.

9:35 iPS Panel of Experts 
Therapeutic Perspective: Justin Ichida, Ph.D., Harvard Stem Cell Institute
Diagnostic Perspective: Bruce Conklin, M.D., Gladstone Institute of Cardiovascular Disease
Commercial Considerations: R. Lee Buckler, LLB, Cell Therapy Group
Screening: Berta Strulovici, Ph.D., VP Research & Discovery, iZumi Bio
IP & Regulatory Landscape: Stacy Taylor, J.D., DLA Piper US, LLP 
Quantitative Analysis: Paul Grayson, President and CEO, Fate Therapeutics



10:20 Coffee Break

11:00 MMTC Break-Out Groups Hosted by the Panel of IPS Experts
Mingle, Meet, Talk & Communicate
Join the focused discussion groups hosted by:

Table 1
Recapitulating Disease Phenotypes in vitro
Host: Berta Strulovici, Ph.D., VP Research & Discovery, iZumi Bio
Discussion topics include:

• What diseases are most likely to be faithfully recapitulated in vitro and why?
• How important is microenvironment in making in vitro models of cellular disease meaningful?
• What value will disease relevant cell type models afford to translational research in academia and in industry?
• How many iPS lines are necessary to represent a particular disease or condition and in which populations?

Table 2
Scientific Progress and Hurdles Regarding the use of iPS Cells for Cell Therapy and Regenerative Medicine
Host: Justin Ichida, Ph.D., Stem Cell and Regenerative Biology, Harvard Stem Cell Institute
This table will discuss questions regarding the current state of scientific research aimed at developing iPS cell therapies including:

• How close are we to making iPS cells that are safe for transplantation into human patients?
• What experiments have been done to test cell therapies in humans or laboratory animals?
• What are the key challenges facing iPS cell therapy?

Table 3
The iPS Patent Frontier: The Yamanaka Patent Claims and Beyond
Host: Stacy Taylor, J.D., Partner, DLA Piper LLP
Discussion topics include:

• The Yamanaka iPS technology, recently issued patent claims and potential conflicts.
• How convincing is the evidence supporting the various claims made for the iPS cells?
• How might the acquisition of broad iPS patent rights stimulate or restrict technical advancement in the field?

Table 4
Generation of Useful Cell Types through iPSC Technology for Drug Development and Toxicity Screening
Host: Paul Grayson, President & CEO, Fate Therapeutics
Topics to be discussed include:

• What approaches lead to reliable and reproducible cell programming (i.e. creation and differentiation of iPSCs or transdifferentiation)?
• What are the appropriate methods to quantitatively analyze cell state and phenotype?
• What processes are needed and what scale is required for iPSC technologies to fulfill industrial level quality, quantity and reproducibility in commercial drug discovery?

12:00 pm Luncheon Presentation (Opportunity Available) or Lunch on Your Own

FIVE N’ FIVE VENTURE CAPITAL AND EMERGING COMPANY FORUM

1:00 Chairperson’s Remarks
Gregory Bonfiglio, J.D., Managing Partner, Proteus Ventures

VENTURE CAPITAL FORUM

1:05 VC Panel of Experts
A panel of venture capital experts present an overview of their VC firm and give helpful tips in building a cell-based technology into a viable business.
Gregory Bonfiglio, J.D., Managing Partner, Proteus Venture Partners 
Beth Seidenberg, M.D., Partner, Kleiner Perkins Caufield & Byers
Ken Aldrich, J.D., Member, Tech Coast Angels, Managing Director, Convergent Ventures, LLC 
Michael Goldberg, M.B.A., General Partner, Mohr Davidow Ventures 

EMERGING COMPANY FORUM

In this unique session, emerging stem cell companies present their technologies, tools, therapies, and business goals. Each has FIVE minutes to present FIVE slides, followed by an interactive discussion with the VC experts who will give valuable feedback to the company on how to better present their technology and plan to receive adequate funding.

1:50 America Stem Cell Inc. 
Stem Cell Homing and Engraftment
Amelia J. Spiliotes, M.B.A., Executive Vice President, Corporate Development

2:05 AuxoCell Laboratories, Inc. 
Umbilical Cord Matrix Mesenchymal Stem Cells: Regenerative Medicine Beyond Umbilical Cord Blood
Kyle Cetrulo, M.D., Chief Operating Officer

2:20 California Stem Cell Inc. 
High-Purity Human Cells for Therapeutic Development and Clinical Application
Chris Airriess, Ph.D., Chief Operating Officer

2:35 Capricor Inc.
Cardiac Stem Cell Therapies for Heart Disease
Oliver Foellmer, M.B.A., President and Chief Executive Officer

2:50 GIRUS Life Sciences, Inc. 
Directed Cell Trafficking for Improved Bone Marrow Transplants
M. Abi Abitorabi, M.A., Ph.D., Founder and President

3:05 Close of Conference

Scientific Advisory Committee
R. Lee Buckler, B.E.d, LLB, Principal, Cell Therapy Group
Robert Deans, Ph.D., Senior Vice President, Regenerative Medicine, Athersys, Inc.
Rosemarie Hunzinker, Ph.D., Program Director, Tissue Engineering & Regenerative Medicine, NIH/NIBIB
Mark E. Levenstein, Ph.D., Senior Scientist, CDI Biosciences
Patricia Olson, Ph.D., Senior Research Scientist, California Institute of Regenerative Medicine
Deepak Srivastava, M.D., Gladstone Institute of Cardiovascular Disease, University of California San Francisco

Overview | Day 1 | Day 2 | Day 3 | Download Brochure | Breakout Discussions