Agenda

The Stem Cell Meeting on the Mesa is a three-day conference combining discussions between key opinion leaders, senior executives and top academic researchers. The program also includes 50 presentations by the field’s most promising companies and poster abstracts by 40 leading scientists from around the globe.


DAY 1 – PARTNERING FORUM
TUESDAY – OCTOBER 7, 2014

ESTANCIA LA JOLLA HOTEL & SPA
 

7:30am REGISTRATION & BREAKFAST
8:00am WELCOME REMARKS
Speaker:
Geoff MacKay, Chair, Alliance for Regenerative Medicine; President & CEO, Organogenesis Inc.
8:15am PLENARY SESSION: CELL THERAPY PRODUCT DEVELOPMENT
Maintaining Momentum in the Face of Expected Clinical Challenges
As more products head into later stage clinical development inevitably there will be successes and setbacks along the way. How do we educate key stakeholder groups so that expectations are realistic and informed, thus avoiding volatility and overreaction to positive and negative data events? What do we see as the key clinical issues we need to address in some of the larger indications so we can help prospective partners and investors better understand the commercial opportunities around cell therapy products? This panel of industry and clinical experts will examine these issues and offer insight into how these challenges will be addressed over the next 3-5 years.
Chair:
Mark Stevenson, EVP & President, Life Science Solutions, Life Technologies, A Thermo Fisher Brand
Panelists:
Gwendolyn Binder-Scholl, Ph.D., EVP, Head of Clinical & Regulatory Development, Adaptimmune
Christian Homsy, M.D., CEO, Cardio3 BioSciences
Jay Siegel, M.D., Chief Biotechnology Officer, Head of Scientific Strategy & Policy, Johnson & Johnson
Donna Skerrett, M.D., Chief Medical Officer, Mesoblast
Gil Van Bokkelen, Ph.D., Chairman & CEO, Athersys
9:15am FROM FIRST PRODUCT TO ROBUST THERAPEUTIC PORTFOLIO
How to Effectively Capture the Value of a Biomaterials Platform
Because biomaterials can often be developed for a wide variety of applications, this session will offer perspective from two regenerative medicine companies each commercializing a tissue engineering platform. It will focus broadly on how to leverage the role of platform matrices in the development of therapeutic applications. In addition, the session will address the strategies for portfolio expansion including the differences in building a therapeutic pipeline when the first product is intended for research use versus therapeutic indication.
Speakers:
David Lightbody, CEO, Biogelx
Adam Gridley, President & CEO, Histogenics
9:45am MORNING BREAK
10:15am COMPANY PRESENTATIONS
Track 1 Track 2
10:15 am Aastrom Biosciences Histogen
10:30 am Mesoblast Stanford University
10:45 am Capricor Therapeutics MEDIPOST America
11:00 am Asterias Biotherapeutics Cyfuse Biomedical
11:15 am NeoStem ISTO Technologies
11:30 am Adaptimmune DiscGenics
11:45 am Athersys RepliCel
12:00pm LUNCH
1:15pm PANEL I: PAYER PERSPECTIVES ON REGENERATIVE MEDICINE & ADVANCED THERAPIES
This session will explore payer views of advanced therapies such as cell therapy, gene therapy and tissue engineered products. The panel is comprised of representatives from payer organizations who will discuss issues including what is the best way to demonstrate clinical effectiveness? How should companies demonstrate the value of these new therapies? And how will new treatment paradigms fit into the reimbursement model?
Chair:
Curt Herberts, Senior Director, Corporate Development, Sangamo BioSciences
Panelists:
Nicholas Anderson, Medical Technology Analyst & HEOR Consultant
Stephen Crawford, M.D., Medical Senior Director, Cigna LifeSOURCE Transplant Network
Matthew Durdy, Chief Business Officer, Cell Therapy Catapult
Stephen O’Malley, VP, Practice Head US Payer Engagement, PriceSpective
2:15pm COMPANY PRESENTATIONS
Track 1 Track 2
2:15 pm ReNeuron ViaCyte
2:30 pm Fibrocell Science Cesca Therapeutics
2:45 pm Organovo TC Biopharm
3:00 pm InVivo Therapeutics Regeneus
3:15 pm StemCells, Inc. UCLA
3:30pm AFTERNOON BREAK
4:00pm PANEL II: COMING OF AGE: HOW DO WE SUSTAIN INVESTOR INTEREST IN ADVANCED THERAPIES OVER THE LONG TERM?
It’s not enough to simply say: “It’s all about the data, stupid.” This is no different from any new drug or therapeutic that has been developed over the last thirty years. While positive clinical results will help sustain momentum, the sector also needs to continue educating investors about the technology, demonstrating mastery or at least a sensible approach to pricing and reimbursement issues, and also showing an understanding about how the healthcare infrastructure will continue to evolve to support next-generation therapies. This panel of investors representing a cross-section of the investor community will examine these issues and speak candidly about what it will take to finance the sector over the next 5-10 years.
Chair:
Reni Benjamin, Ph.D., Managing Director & Head of Equity Research, H.C. Wainwright & Co.
Panelists:
Bong Koh, M.D., Partner, Venrock
Daniel Menichella, VP & Chief Business Officer, AGTC
Dennis Purcell, Founder & Senior Advisor, Aisling Capital
Brock Reeve, Executive Director, Harvard Stem Cell Institute; Portfolio Manager, Poliwogg
5:00pm COMPANY PRESENTATIONS
Track 1 Track 2
5:00 pm MiMedx Group Promethera Biosciences
5:15 pm Osiris Therapeutics Bell Biosystems
5:30 pm TiGenix jCyte
5:45 pm Cytomedix Regenerative Patch Technologies
6:00pm PARTNERING CLOSES
6:30pm GALA RECEPTION
Open to All Regen Med Partnering Forum Attendees

 

DAY 2 – PARTNERING FORUM
WEDNESDAY – OCTOBER 8, 2014
ESTANCIA LA JOLLA HOTEL & SPA

7:00am REGISTRATION & BREAKFAST
7:30am REIMBURSEMENT WORKSHOP
This “hands-on” reimbursement workshop will feature interaction with a series of experts in coding, coverage and payment who have helped companies navigate the many challenges in reimbursement and provide opportunities for specific discussions about how to address issues related to products. Attendees will learn how to incorporate successful reimbursement strategies into their business and clinical programs. Questions to be discussed will include: what are the key strategic questions for a company? Is there existing Medicare data that could support a company’s position and how can you access that data? What reimbursement is available for drugs used in clinical trials?
Chair:
Faraz Ali, VP, Commercial Development, bluebird bio
Participants:
Candace Gunnarsson, Ed.D., VP, Health Economics & Outcomes Research, CTI Clinical Trial & Consulting
Douglas Paul, Pharm.D., Ph.D., Partner, Medical Marketing Economics (MME)
Jayson Slotnik, Partner, Health Policy Strategies
Michael Werner, Executive Director, Alliance for Regenerative Medicine (ARM)
7:30am MANUFACTURING & SCALE-UP WORKSHOP
We often hear that the field of regenerative medicine is today where the biologics industry was 10-15 years ago. With cell and gene therapy products now in commercial distribution, there is greater clarity around the remaining obstacles to completing the journey of ‘bench to beside’. The regenerative medicine industry continues to face challenges unique to its development in realizing its goals. Successful commercialization of these therapeutics requires high quality products with reasonable cost of goods in a scalable manner that can be sustained throughout the commercial life of the product. With increasing sophistication and experience, the solutions to these challenges are becoming more apparent. What lessons can we learn from those who wrote the previous chapter in medical history? In this session, we will hear experts from the biologics revolution as they provide insight as to how they overcame considerable obstacles to build a sustainable commercial pathway two decades ago. We will ask them to comment on our challenges and advise us on how to face this new frontier with practical suggestions of what pitfalls to avoid and what strengths to build around.
Chair:
Robert Preti, Ph.D., Co-Founder & President, Progenitor Cell Therapy (PCT); Chief Scientific Officer, NeoStem
Participants:
Stewart Abbot, Ph.D., Executive Director, Integrative Research, Celgene Cellular Therapeutics
Dolores Baksh, Ph.D., Innovation Leader, Cell Therapy Technologies, GE Healthcare
Ian Harris, Ph.D., Senior Director, Janssen R&D
Harry Lam, Ph.D., SVP, Technical Operations, KaloBios Pharmaceuticals
Stephen Ward, Ph.D., Chief Operating Officer, Cell Therapy Catapult
9:20am WELCOME REMARKS
Introduction:
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine (ARM)
Speaker:
C. Randal Mills, Ph.D., President & CEO, California Institute for Regenerative Medicine (CIRM)
9:40am JAPANESE REGULATORY FRAMEWORK & INDUSTRY INITIATIVES
Featured Speaker:
Yuzo Toda, Senior VP, Fujifilm Corporation; Chairman, Forum for Innovative Regenerative Medicine (FIRM)
10:00am STEM CELLS & FDA OVERSIGHT
Featured Speaker:
Celia Witten, M.D., Ph.D., Director, Office of Cellular, Tissue, & Gene Therapy Center for Biologics Evaluation & Research, Food & Drug Administration (FDA)
10:30am MORNING BREAK
11:00am PANEL III: BRINGING INNOVATIVE PRODUCTS TO MARKET IN THE CURRENT REGULATORY ENVIRONMENT
This panel, comprised of regulators and leading experts in the field, will discuss the key US and international regulatory issues confronting advanced therapy companies. Topics to be discussed will include: clinical trial design, expedited approval programs, pre-clinical and clinical issues.
Chair:
Katherine Tsokas, Senior Director, Global Regulatory Affairs, Janssen Research & Development
Panelists:
Anne-Virginie Eggimann, VP, Regulatory Science, bluebird bio
Ellen Feigal, M.D., SVP, Research & Development, California Institute for Regenerative Medicine (CIRM)
Natalie Mount, Ph.D., Chief Clinical Officer, Cell Therapy Catapult
Robert Pietrusko, Pharm.D., SVP, Regulatory Affairs, Voyager Therapeutics
Celia Witten, M.D., Ph.D., Director, Office of Cellular, Tissue, & Gene Therapy Center for Biologics Evaluation & Research, Food & Drug Administration (FDA)
12:00pm LUNCH
1:15pm COMPANY PRESENTATIONS
Track 1 Track 2
1:15 pm Sangamo BioSciences Voyager Therapeutics
1:30 pm bluebird bio MaxCyte
1:45 pm Oxford BioMedica Juventas Therapeutics
2:00 pm Avalanche Biotechnologies Terumo BCT
2:15 pm AGTC BioLife Solutions
2:30 pm NYSCF
2:45pm AFTERNOON BREAK
3:15pm PLENARY SESSION: GENE THERAPY & GENE-MODIFIED CELL THERAPIES
What are the Deals and Infrastructure Requirements to Support Next-Generation Therapies?
This session will bring together several of the leading in-vivo and ex-vivo gene therapy companies in addition to pharma companies making a large bet in gene-modified cell therapy. While the interest in these technologies abounds due to recent clinical and technological advancements, we also need to understand the commercial model and how these therapies can be deployed into the healthcare system through a sustainable and cost-effective process. With single dose administration regimens and the potential for curative therapies, parts of the healthcare system will have to adapt to these new forms of treatment. This panel will discuss these issues and the unique partnerships that will be required for clinical and commercial success.
Chair:
Edward Lanphier, President & CEO, Sangamo BioSciences
Panelists:
Faraz Ali, VP, Commercial Development, bluebird bio
Jeff Goater, VP, Business Development, Voyager Therapeutics
Hans Hull, SVP, Business Operations, Avalanche Biotechnologies
Douglas Kerr, M.D., Ph.D., Senior Medical Director, Clinical Development, Biogen Idec
4:15pm COMPANY PRESENTATIONS
Track 1 Track 2
4:15 pm Celladon Corporation RhinoCyte
4:30 pm Cellular Dynamics Cedars Sinai Medical Center
4:45 pm Cardio3 BioSciences Accurexa
5:00pm CLOSING REMARKS
5:00pm PARTNERING CLOSES

 

3rd ANNUAL PUBLIC FORUM
SANFORD CONSORTIUM FOR REGENERATIVE MEDICINE

5:30pm PUBLIC FORUM OPENS
5:45pm ENGINEERED T-CELLS FOR CANCER IN THE ERA OF SYNTHETIC BIOLOGY CHALLENGES & OPPORTUNITIES
Introduction By:
Donna Skerrett, M.D., Chief Medical Officer, Mesoblast
Featured Speaker:
Carl June, M.D., Richard W. Vague Professor in Immunotherapy, Perelman School of Medicine, University of Pennsylvania
6:45pm RECEPTION & NETWORKING
Open to All Stem Cell Meeting on the Mesa Attendees
7:30pm PUBLIC FORUM CLOSES

 

DAY 3 – 9th ANNUAL SCIENTIFIC SYMPOSIUM
THURSDAY – OCTOBER 9, 2014
SALK INSTITUTE FOR BIOLOGICAL STUDIES

7:00am REGISTRATION & BREAKFAST
8:00am WELCOME REMARKS
Introduction:
Roger Bingham, Co-Founder & Director, The Science Network; Member, Computational Neurobiology Laboratory, Salk Institute for Biological Studies
Speaker:
C. Randal Mills, Ph.D., President & CEO, California Institute for Regenerative Medicine (CIRM)
8:15am OFF THE SHELF ENGINEERED ARTERIES
Keynote Speaker:
Laura Niklason, M.D., Ph.D., Professor of Anesthesiology & Biomedical Engineering, Yale University; Founder, Humacyte
8:55am PANEL I: ENGINEERING MICROENVIRONMENTS FOR STEM CELLS
This panel will discuss the use of tissue engineering techniques for controlling the stem cell microenvironment with the ultimate goals of improving stem cell culture and tissue generation. The speakers will discuss biomaterial scaffold design, 3D bio-printing and patterning, novel 3D microenvironments and engineered tissues for tissue generation and drug screening.
Chair / Introduction By:
Karen Christman, Ph.D., Associate Professor of Bioengineering, UC San Diego
3D Bioprinting: An Enabling Technology for Stem Cell Engineering
Shaochen Chen, Ph.D., Professor, NanoEngineering & Bioengineering; Co-Director, Biomaterials & Tissue Engineering Center, UC San Diego
Engineering Dynamic Niche
Adam Engler, Ph.D., Associate Professor, Department of Bioengineering, UC San Diego
Building 3D Human Tissues with Additive Manufacturing Platforms
Sharon Presnell, Ph.D., Chief Technology Officer & EVP of Research & Development, Organovo
10:15am MORNING BREAK / POSTER VIEWING
10:40am PANEL II: CLINICAL TRIALS AT THE SANFORD STEM CELL CLINICAL CENTER
The presenters on this panel will provide the audience with scientific background and trial design for the Viacyte VC-01 diabetes study, the Neuralstem spinal cord injury repair study, and the ROR1 antibody study targeting cancer stem cells. These brief, high-level presentations will discuss how the scientific discoveries informed the trial designs, and how medical considerations influenced the scientific products.
Chair / Introduction By:
Lawrence Goldstein, Ph.D., Distinguished Professor, Departments of Cellular and Molecular Medicine and Neurosciences; Director, Sanford Stem Cell Clinical Center; Director, UC San Diego Stem Cell Program; Scientific Director, Sanford Consortium for Regenerative Medicine
Panelists:
Catriona Jamieson, M.D., Ph.D., Associate Professor, Division of Hematology-Oncology; Deputy Director, Sanford Stem Cell Clinical Center; Chief, Division of Regenerative Medicine, UC San Diego; Director, Stem Cell Research at Moores UC San Diego Cancer Center
Thomas Kipps, M.D., Ph.D., Deputy Director for Research, Moores UC San Diego Cancer Center
Martin Marsala, M.D., Professor, Anesthesiology, UC San Diego
Joseph Ciacci, M.D., Clinical Professor of Surgery, UC San Diego; Chief of Neurosurgery, VA San Diego Healthcare System
Robert Henry, M.D., Professor of Medicine‚ UC San Diego; Chief, Section of Endocrinology, Metabolism & Diabetes, VA San Diego Healthcare System
Kevin D’Armour, Ph.D., VP, Research & Chief Scientific Officer, ViaCyte
12:00pm LUNCH / POSTER VIEWING
1:15pm PANEL III: EPIGENETICS & STEM CELLS IN DEVELOPMENT & REGENERATIVE MEDICINE
This panel will discuss the maintenance of pluripotency or commitment to specific cell lineages and further differentiation into specialized cell types determined by specific changes in chromatin structure that lead to unique patterns of gene expression. The speakers will explore the identification and functional characterization of the components of the chromatin-remodeling machinery that underlie the epigenetic control of stem cell fate, a fundamental task for future applications of stem cells in regenerative medicine.
Chair / Introduction By:
Luke Lairson, Ph.D., Assistant Professor, Department of Chemistry, The Scripps Research Institute; Principal Investigator, California Institute for Biomedical Research
Role of L1 Retrotransposon in Neurological Autoimmune Diseases
Alysson Muotri, Ph.D., Assistant Professor, Department of Pediatrics/Cellular & Molecular Medicine, UC San Diego
Developmental Competence Encoded in Enhancer Chromatin
Allen Wang, Ph.D., Postdoctoral Fellow, Department of Pediatrics, UC San Diego
Epigenetic Control of Stem Cell Differentiation into Skeletal Muscle
Pier Lorenzo Puri, M.D., Ph.D., Associate Professor, Development, Aging & Regeneration Program, Sanford-Burnham Medical Research Institute
Dynamic Interplay of 5-Methylcytosine & 5-Hydroxymethylcytosine During Cell Fate Specification 
Michael Boland, Ph.D., Research Associate, The Scripps Research Institute
2:35pm AFTERNOON BREAK / POSTER VIEWING
3:00pm PANEL IV: MODELING HUMAN DISEASE
The increasing ease with which accessible cells (e.g., blood, skin) from a given patient can be reprogrammed and redirected to assume the phenotype of a disease-relevant cell has enabled the in vitro modeling of genetic diseases and even some aspects of idiopathic diseases. Such reprogrammed cells can be expanded and used to improve our understanding of underlying disease mechanisms, as well as to explore, test or suggest interventions by genetic modification, pharmacological intervention and cell-based therapy. This panel explores three different takes on modeling metabolic, cardiac and pulmonary disease and broaches the technical and scientific challenges to be addressed in future research.
Chair / Introduction By:
Evan Snyder, M.D., Ph.D., Professor & Program Director, Center for Stem Cells & Regenerative Medicine, Sanford Burnham Medical Research Institute; Faculty, Biomedical Sciences Graduate Program & Faculty Physician, Department of Pediatrics, UC San Diego; Scientific Steering Committee, Sanford Consortium for Regenerative Medicine
Stem Cells for Cardiac Disease Modeling and Drug Discovery
Joseph Wu, M.D., Ph.D., Director, Stanford Cardiovascular Institute; Professor, Department of Medicine (Cardiology) & Radiology, Stanford University School of Medicine
Preventing Transmission of Mitochondrial Diseases by Germline Heteroplasmy Shift
Juan-Carlos Izpisua Belmonte, Ph.D., Professor, Gene Expression Laboratory, Salk Institute for Biological Studies
Cancer Stem Cells: Reprogramming/Dedifferentiation & Transdifferentiation
Inder Verma, Ph.D., Irwin and Joan Jacobs Chair in Exemplary Life Science, American Cancer Society Professor Molecular Biology, Laboratory of Genetics, Salk Institute for Biological Studies
4:20pm MOLECULAR REGULATION OF STEM CELL QUIESCENCE & ACTIVATION
Keynote Speaker:
Thomas Rando, M.D., Ph.D., Director, Glenn Center for the Biology of Aging, Stanford University School of Medicine
5:00pm CLOSING REMARKS
5:00pm NETWORKING RECEPTION / POSTER VIEWING
6:30pm PROGRAM CLOSES