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FULL LIVE STREAM AGENDA

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7:15am – 8:45am | CONCURRENT WORKSHOPS – LIVE STREAM BEGINS

DOING BUSINESS IN JAPAN WORKSHOP: BEYOND JAPAN’S REGULATORY RENOVATION

Magnolia Room | 7:15am – 8:45am
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

DOING BUSINESS IN JAPAN WORKSHOP
Beyond Japan’s Regulatory Renovation
Japan has the most ambitious regulatory system in the world and is the best country to start your business in the field of regenerative medicine. Industries in Japan, including pharmas, CMOs and CROs are seeking opportunities to collaborate with ARM member companies. This session will address an overview of the industrial progress under the new regulatory framework for a better understanding of the system in Japan.

Workshop Chair:
Kunihiko Suzuki, Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); Vice Chairman and Member of the Board, MEDINET

7:15am – 7:30am | METI’s Roadmap for Industrialization of Regenerative Medicine
Speaker:
Masahiro Uemura, Director, Bio-Industry Division, Commerce and Service Industry Policy Group, Ministry of Economy, Trade and Industry (METI)

7:30am – 7:45am | Regulatory Policy and Strategy of Regenerative Medicine
Speaker:
Daisaku Sato, Ph.D., Director, Pharmaceutical Safety Division, Pharmaceutical Safety and Environmental Health Bureau, Ministry of Health, Labour and Welfare (MHLW)

7:45am – 8:00am | Rolling Submission by Sakigake Designation System
Speaker:
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

8:00am – 8:10am | A Nation-wide Registry Jointly Developed with PMDA
Speaker:
Kyosuke Mano, Secretary General, The Japanese Society for Regenerative Medicine (JSRM)

8:10am – 8:25am | Experiences of an Actual Approved Case
Speaker:
Osamu Honmou, M.D., Ph.D., Chairman and Professor, Department of Neural Regenerative Medicine, Sapporo Medical University School

8:25am – 8:40am | Audience Q&A

8:40am – 8:45am | Closing Remarks
Speaker:
Akihiko Iwai, Ph.D., Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); Divisional SVP, Candidate Discovery Science Labs, Drug Discovery Research, Astellas Pharma

DEVELOPMENT OF CELLULAR THERAPIES WORKSHOP

Ballroom 2 | 7:15am – 8:45am
Sponsored by QuintilesIMS

DEVELOPMENT OF CELLULAR THERAPIES WORKSHOP
Real World Examples for Overcoming the Unique Complexities and Development Challenges Faced in Regenerative Medicine

The development of cellular and gene-based therapies face many unique challenges that preclude a linear application of clinical trial development best practices. Featuring global leaders in clinical trial management and operations, this workshop will highlight the unique challenges faced in the development of these regenerative medicine therapies. Discussion will include the convergence of best practices in translational techniques with clinical trial development practices to incorporate challenges associated with limited patient sets for rare diseases, restricted quantities of clinical trial materials, and adaptive trial designs to improve understanding of dose ranges throughout the clinical trial process. In addition, tested and emerging models for overcoming these challenges will be discussed in a round robin format.

7:15am – 8:00am | Session 1: Development of Cellular Therapies
Pragmatic Solutions for Overcoming the Unique Complexities and Challenges Faced in Regenerative Medicine from Early Clinical Development Through Commercialization
It is an accepted wisdom that a high degree of technical and operational expertise is needed for successful product development in regenerative medicine (RMx), specifically in cell and gene therapies. But what factors are actually at play in RMx therapy development that require this expertise, and what levers can be pulled to solve these challenges? RMx trials layer the challenges of the most complex trials for traditional therapies (i.e., indication-specific challenges) with unique complexities inherent to RMx. The key to executing a successful trial is solving all of these issues at the outset and operationalizing these solutions throughout. Discussion points will include trial design, site selection and trial operation as well as patient recruitment and retention.

Chair:
Avi Kulkarni, Ph.D., SVP and Managing Director, Consulting Services; Director, Stem Cell Center, QuintilesIMS

Speakers:
Stewart Abbot, Ph.D., Chief Development Officer, Fate Therapeutics
Deborah Ascheim, M.D., Chief Medical Officer, Capricor Therapeutics
Robert Deans, Ph.D., Chief Technology Officer, BlueRock Therapeutics
Adrian McKemey, Ph.D., SVP and Managing Director, Consulting Services; Global Head, Research and Development Strategy Solutions, QuintilesIMS
Timothy Schroeder, CEO, CTI Clinical Trial and Consulting Services

8:00am – 8:45am | Session 2: Informing Clinical Design with an Eye Towards Commercial Success
Despite their curative potential, cell and gene therapies continue to face immense barriers to commercialization. Successfully demonstrating product value upon launch and justifying price points across stakeholders with differing needs and priorities requires an informed trial design with an eye towards commercialization. This panel will explore the ways in which cell and gene companies can leverages clinical insights for optimized value demonstration at launch. Discussion points will include trial design with the end in mind, ways of leveraging secondary endpoints beyond safety and efficacy as a critical component of trial design and anticipating operational and manufacturing limitations.

Chair:
John Doyle, Dr.P.H., SVP and Managing Director, Enterprise Solutions, Real World Insights, QuintilesIMS

Speakers:
Eduardo Bravo, CEO, TiGenix
Paul Bresge, CEO, jCyte
Anne Dupraz Poiseau, Ph.D., Chief Regulatory Officer, Orchard Therapeutics
Kimberly Freeman, VP, Commercial Strategy and Planning, Adaptimmune
Avi Kulkarni, Ph.D., SVP and Managing Director, Consulting Services; Director, Stem Cell Center, QuintilesIMS

MANUFACTURING GENE MEDICINES WORKSHOP

Learning Theater | 7:15am – 8:45am
Sponsored by Aldevron, Brammer Bio and MaxCyte

MANUFACTURING GENE MEDICINES WORKSHOP

7:15am – 8:00am | Session 1: Viral Vector Manufacturing
The manufacture of gene transfer vectors used for in vivo and ex vivo delivery is complex. The panelists will address technologies and platforms used to manufacture viral vector systems, critical raw materials, analytical technologies used to characterize viral vectors and accessing clinical and commercial manufacturing capacity.

Chair:
Richard Snyder, Ph.D., Chief Scientific Officer, Brammer Bio

Speakers:
James Brown, Ph.D., VP, Corporate Development, Aldevron
Robert Kotin, Ph.D., Adjunct Professor, Department of Microbiology and Physiological Systems, Gene Therapy Center, University of Massachusetts Medical School
Mark Plavsic, Ph.D., D.V.M., Chief Technical Officer, Lysogene

8:00am – 8:45am | Session 2: Manufacturing Gene-Modified Cell Therapies
The experts on this panel will discuss challenges associated with the delivery of genes via cells. Topics will include viral vectors as ancillary materials, expanding draggable targets to include neo-antigens, challenges in process development, and moving toward commercial-scale autologous cell manufacturing.

Chair:
Jessica Carmen, Ph.D., Director of Business Development, Cellular Therapy Partnerships, MaxCyte

Speakers:
Allan Dietz, Ph.D., Director, Immune, Progenitor and Cellular Therapeutics (IMPACT); Associate Professor, Department of Laboratory Medicine and Pathology, Transfusion Medicine, Mayo Clinic
Natalie Mount, Ph.D., Chief Science Officer, GammaDelta Therapeutics
Stefanos Theoharis, Ph.D., SVP, Corporate Development and Partnering, Cell Medica

8:45am – 9:00am | SHORT BREAK IN LIVE STREAM
9:00am – 9:15am WELCOME REMARKS {Ballroom 1}
Speakers:
Robert Preti, Ph.D., President and CEO, PCT, A Hitachi Group Company; General Manager, Hitachi Chemical Regenerative Medicine Business Sector
Janet Lynch Lambert, CEO, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am PLENARY SESSION: IMPLICATIONS OF COMMERCIALIZATION: WHAT DOES APPROVAL MEAN? {Ballroom 1}
This is CAR-T cell therapy’s breakthrough year. The first product, Novartis’ Kymriah, is now approved to treat acute lymphoblastic lymphoma, and therapies for other cancers are hot on its heels. This panel will explore the scientific, clinical, policy and business issues surrounding CAR-T approvals, and set the stage for moving these therapies into mainstream medicine.
Chair:
Amy DuRoss, CEO, Vineti
Speakers:
Bob Azelby, EVP, Chief Commercial Officer, Juno Therapeutics
Nick Colangelo, President and CEO, Vericel Corporation
Pascal Touchon, D.V.M., SVP and Global Head, Cell and Gene, Novartis Oncology
Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio
10:15am – 10:45am | MORNING BREAK – NO LIVE STREAM AT THIS TIME
10:45am – 12:00pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
SPOTLIGHT: TISSUE ENGINEERING {Ballroom 1}
10:45am – 11:15am
Advances in cell biology, including the development of new stem cell differentiation protocols, the large-scale in vitro expansion of adult allogeneic and autologous progenitor cells suitable for therapeutic use and the evolution of improved biocompatible scaffolds provide hope that multiple tissue engineered products will advance to clinical trials in the coming years. This panel will use examples from bone and cartilage products as a jumping off point to highlight the future challenges in making this vision a reality.
Chair:
Tom Novak, Ph.D., VP, Life Sciences Business Development, Cellular Dynamics International (CDI)
Speakers:
Miguel Forte, M.D., Ph.D., Chief Medical Officer, Bone Therapeutics
Adam Gridley, President and CEO, Histogenics
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am Renova Therapeutics
11:00am – 11:15am Juventas Therapeutics
11:15am – 11:30am Capricor Therapeutics
11:30am – 11:45am Cesca Therapeutics
11:45am – 12:00pm Lysogene
COMPANY PRESENTATIONS {Ballroom 1}
11:15am – 11:30am AGTC
11:30am – 11:45am Audentes Therapeutics
11:45am – 12:00pm CRISPR Therapeutics
12:00pm – 1:15pm | LUNCH – NO LIVE STREAM AT THIS TIME
1:15pm – 3:30pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
PANEL: NAVIGATING REGENERATIVE MEDICINE TECHNOLOGY ASSESSMENT AND REIMBURSEMENT IN NORTH AMERICA AND EUROPE: CHARTING A PATH TO PATIENT ACCESS {Ballroom 1}
1:15pm – 2:15pm
As 800+ regenerative and advanced therapies are en route to market, global health technology assessment and reimbursement processes represent one of most critical hurdles to patient access and product commercial success. How and in what ways would value assessment/HTA differ for technologies with transformative or curative potential? What are the options if therapies do not fit “neatly” into existing reimbursement structures? How will global health systems address payment for single administration therapies? This panel will present payer panelists from North America and Europe with rare disease and oncology case examples and explore acceptance and uptake drivers spanning HTA evaluation requirements, fit into existing reimbursement systems and payment for complex, single administration therapies.
Sponsored by Evidera
Chair:
Eric Faulkner, VP, Precision and Transformative Technology Solutions, Value Demonstration, Access and Commercial, Evidera
Speakers:
Jim Cross, M.D., Former VP, National Medical Policy and Operations, Aetna; President, Jim Cross, MD Consulting
John Goldenring, M.D., Medical Director for Care Management, Anthem
Richard Powell, M.D., Chief Medical Officer, MedPOINT Management
Keith Tolley, Director, Tolley Health Economics
COMPANY PRESENTATIONS {Ballroom 2}
1:15pm – 1:30pm Sentien Biotechnologies
1:30pm – 1:45pm Medeor Therapeutics
1:45pm – 2:00pm Promethera Biosciences
2:00pm – 2:15pm Vivet Therapeutics
COMPANY PRESENTATIONS {Ballroom 1}
2:15pm – 2:30pm Fate Therapeutics
2:30pm – 2:45pm Bellicum Pharmaceuticals
2:45pm – 3:00pm Adaptimmune
3:00pm – 3:15pm bluebird bio
3:15pm – 3:30pm MolMed
3:30pm – 3:45pm uniQure
COMPANY PRESENTATIONS {Ballroom 2}
2:15pm – 2:30pm Bone Therapeutics
2:30pm – 2:45pm DiscGenics
2:45pm – 3:00pm MEDIPOST America
3:00pm – 3:15pm Fibrocell
3:15pm – 3:30pm Histogenics
3:30pm – 3:45pm MaxCyte
3:45pm – 4:00pm | AFTERNOON BREAK – NO LIVE STREAM AT THIS TIME
4:00pm – 6:00pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
PANEL: GENE THERAPY: BUILDING THE GENE MEDICINE PIPELINE THROUGH NEXT GENERATION TECHNOLOGIES {Ballroom 1}
4:00pm – 5:00pm
This panel will explore how gene therapy product developers are strategically building the clinical pipeline utilizing next generation technologies. Discovery and design of new highly trophic viral capsids, stronger and shorter synthetic promoters and tightly inducible regulatory elements are examples of the novel components incorporated into the second-generation lead candidates. Topics and discussion will include optimization of the pipeline to enhance tissue specificity, transduction and vector tropism, generate clinically relevant transgene expression levels, solve first generation vector capacity issues, potentially modulate immunity and to enhance safety.
Chair:
Sue Washer, President and CEO, AGTC
Speakers:
Donna Armentano, Ph.D., Executive Director, External Research and Development Innovation, Gene Therapy, Pfizer
David Kirn, M.D., Co-Founder and CEO, 4D Molecular Therapeutics; Co-Founder and CEO, 5D Oncolytic Immunotherapeutics; Professor of Bioengineering, UC Berkeley
Sheila Mikhail, Founder, President and CEO, AskBio; Founder and Former CEO, Bamboo Therapeutics
David Venables, Ph.D., CEO, Synpromics
Thomas Wilton, Chief Business Officer, LogicBio Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
4:00pm – 4:15pm Cynata Therapeutics
4:15pm – 4:30pm BlueRock Therapeutics
4:30pm – 4:45pm Cellular Dynamics International
4:45pm – 5:00pm Healios K.K.
COMPANY PRESENTATIONS {Ballroom 1}
5:00pm – 5:15pm TiGenix
5:15pm – 5:30pm MiMedx
5:30pm – 5:45pm Mesoblast
5:45pm – 6:00pm Vericel
COMPANY PRESENTATIONS {Ballroom 2}
5:00pm – 5:15pm Terumo BCT
5:15pm – 5:30pm TxCell
5:30pm – 5:45pm jCyte
5:45pm – 6:00pm Acucela
6:00pm | LIVE STREAM ENDS FOR THE DAY

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7:15am – 8:45am | CONCURRENT WORKSHOPS – LIVE STREAM BEGINS

HEALTH TECHNOLOGY ASSESSMENT WORKSHOP

Magnolia Room | 7:15am – 8:45am
Sponsored by bluebird bio

HEALTH TECHNOLOGY ASSESSMENT WORKSHOP
Mock Technology Appraisal for Regenerative Medicine: Perspectives from U.S. and EU Health Technology Assessment (HTA) Experts

As a follow-up to the October 5th Technology Assessment Panel, this workshop will take a deeper dive into the challenges HTAs will face when assessing a one-time, potentially transformative therapy. Participants will include experts in HTA from both the U.S. and EU. To stimulate critical thinking, an ‘exemplar’ case study incorporating a hypothetical product will be developed and provided to panel members for their consideration in advance of the meeting. The case study will include target indication and population, a brief summary of current standard of care and what is known about likely comparators, expected evidence generated from trial program and other supporting data. Panel members will present their initial assessment of the product based on the value framework they typically apply. Their feedback is expected to encompass:

  • Strengths and weaknesses of existing evidence base
  • Applicability of comparators
  • Key areas of uncertainty as related to value demonstration

The workshop will then involve participant and audience feedback on creative approaches to fill the identified gaps.

Chair:
Doug Danison, VP Access, Value and Evidence Strategy, bluebird bio

Speakers:
Jim Cross, M.D., Former VP, National Medical Policy and Operations, Aetna; President, Jim Cross, MD Consulting
Bill Dreitlein, Pharm.D., Director of Pharmaceutical Policy, Institute for Clinical and Economic Review (ICER)
Clark Paramore, Head of Value Demonstration, bluebird bio
Keith Tolley, Director, Tolley Health Economics

LOGISTICS, AUTOMATION AND STANDARDS WORKSHOP

Ballroom 2 | 7:15am – 8:45am
Sponsored by Cryoport and World Courier

LOGISTICS, AUTOMATION AND STANDARDS WORKSHOP

7:15am – 8:00am | Session 1: Systems Integration Logistics Management
Systems integration is defined as the process of aggregating component sub-systems into one cooperating system that is able to deliver the overarching functionality and act as a coordinated whole. This integration process includes both physical and informatics process integration, and can include the following functions: Patient Recruitment, Scheduling, Logistics, Analysis, Manufacturing, Distribution, and Reimbursement. This panel will discuss critical process integration recommendations for seamless launch of cell-based therapies.

Chair:
Robert Copeland, VP, Global Logistics, Cryoport

Speakers:
Layne Martin, VP/GM, Specialty Distribution Solutions, McKesson Specialty Health
Tim Valko, VP, Global Supply Chain, Atara Biotherapeutics
Philip Vanek, Ph.D., GM, Cell Therapy Growth Strategy, GE Healthcare
Bob Wynalek, Chief Operating and Commercialization Officer, DiscGenics

8:00am – 8:45am | Session 2: Scale-up and Out: The Role of Transportation and Standards
To ensure integrity of the product, patient safety and successful commercialization, cell therapies call for heightened transport and logistical standards. Planning a supply chain which incorporates these early, during clinical phases, pays dividends as production scales up and out into commercialization. This is particularly true for supply chains which cross international boundaries and where integration with patient services, manufacturing and reimbursement are critical. This panel will discuss best practices and recommendations for building a supply chain aligned to your strategic commercialization goals.

Chair:
James Klingelhoefer, North American Sales Director, World Courier, an AmerisourceBergen company
Speakers:
Robert Jones, Global Director, Advanced Therapies, Fisher Bioservices
Martin Lamb, EVP, Sales and Marketing, TrakCel
Claudia Zylberberg, Ph.D., Founder and CEO, Akron Biotech

HELPING YOUR CELL THERAPY GO GLOBAL WORKSHOP

Learning Theater | 7:15am – 8:45am
Lead Sponsor: PCT, A Hitachi Group Company
Supporting Sponsors: Irvine Scientific and MilliporeSigma

HELPING YOUR CELL THERAPY GO GLOBAL WORKSHOP

With a robust pipeline of cell therapies steadily advancing towards approval and commercialization, developers are increasingly considering and implementing strategies to effectively reach a global patient population. But going global can be fraught with uncertainty and present added risk.

This expert panel will explore a number of important questions including:

  • What are the benefits to going global in terms of cell therapy development and commercialization strategies?
  • How are developers establishing strategies and capacity for global development and manufacturing?
  • What considerations go into the choice to expand geographic reach for a product candidate?
  • Can global strategy wait until the first commercial approval is achieved? What is the optimum timing?
  • What are the pitfalls or challenges of a global strategy and how can they be avoided?

Chair:
Robert Preti, Ph.D., President and CEO, PCT, A Hitachi Group Company; General Manager, Hitachi Chemical Regenerative Medicine Business Sector

Speakers:
Eduardo Bravo, CEO, TiGenix
Marlin Frechette, Director, Quality Systems/Regulatory Affairs and ISO Management Representative, Irvine Scientific
David Mazzo, Ph.D., President and CEO, Caladrius Biosciences
Julie Murrell, Ph.D., Head of Cell Therapy Bioprocessing, MilliporeSigma
Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio

8:45am – 9:00am | SHORT BREAK IN LIVE STREAM
9:00am – 9:15am OVERVIEW OF THE ALLIANCE FOR REGENERATIVE MEDICINE’S INITIATIVES {Ballroom 1}
Speakers:
Janet Lynch Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine (ARM)
Michael Werner, Executive Director, Alliance for Regenerative Medicine (ARM)
PANEL: THE GENE EDITING CHALLENGE: VALIDATING PLATFORM TECHNOLOGIES {Ballroom 1}
9:15am – 10:15am
For any emerging biotech company with a novel platform technology, proving validation and establishing trust in that platform is essential to building momentum and long-term success. Trust must be established with multiple stakeholders – potential licensing partners and investors, regulators and clinicians and foremost with patients. In the gene editing space, these concerns are heightened by the unprecedented manipulation of the body’s basic building blocks. Increased understanding in gene editing technologies across all stakeholders has the ability to open doors for additional discovery, development and commercialization efforts. As companies in this space advance, all parties must maintain discipline in limiting patient risk and validating utility of these platforms in order to enable continued expansion of the field. This panel brings together top leaders representing the most prominent technologies across the gene editing landscape to discuss the applicability of their platforms, potential hurdles to advancement and the implications of pursuing various indications as these technologies move toward commercialization.
Sponsored by ClearView Healthcare Partners
Chair:
Debbie Zhuang, Principal, ClearView Healthcare Partners
Speakers:
Katrine Bosley, CEO, Editas Medicine
Matthew Kane, Co-Founder and CEO, Precision BioSciences
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Albert Seymour, Ph.D., Chief Scientific Officer, Homology Medicines
COMPANY PRESENTATIONS {Ballroom 2}
9:15am – 9:30am Caladrius Biosciences
9:30am – 9:45am Semma Therapeutics
9:45am – 10:00am ViaCyte
10:00am – 10:15am Orbsen Therapeutics
10:15am – 10:45am | MORNING BREAK – NO LIVE STREAM AT THIS TIME
10:45am – 12:00pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
FEATURED TALK: REGENERATIVE MEDICINE ADVANCED THERAPY (RMAT) DESIGNATION {Ballroom 1}
10:45am – 11:00am
Speaker:
Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am American Gene Technologies
11:00am – 11:15am NO LIVE STREAM AT THIS TIME
11:15am – 11:30am Homology Medicines
11:30am – 11:45am Orchard Therapeutics
11:45am – 12:00pm New York Stem Cell Foundation
PANEL: ACCELERATING REGULATORY DEVELOPMENT OF ADVANCED THERAPY MEDICINAL PRODUCTS FOR PATIENTS IN NEED {Ballroom 1}
11:00am – 12:00pm
This panel features distinguished regulators from the U.S., EU and Japan as they share their views on how different expedited programs are supporting innovation and accelerating advanced therapy product developments for patients with significant unmet needs. Panelists will discuss how to improve the regulatory process and lessons learned from recent experience in this rapidly emerging field, including real world evidence generation. Looking to the future, panelists will share ideas on how to bridge the gap with science and the next step in a product life-cycle. Both in terms of process and scientific review and advice, panelists will be asked whether there are things industry could do better, and whether there are tools that could benefit all regions to improve efficiency in the regulatory authority/sponsor dialog.
Sponsored by Cell and Gene Therapy Catapult and Janssen R&D
Chair:
Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell and Gene Therapy Catapult
Speakers:
Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)
Gopalan Narayanan, M.D., VP, Disruptive Biologics, Voisin Consulting Life Sciences (VCLS)
Paula Salmikangas, Ph.D., Director of Biopharmaceuticals and ATMP, NDA Advisory Board
Daisaku Sato, Ph.D., Director, Safety Division, Pharmaceutical Safety and Environmental Health Bureau, Ministry of Health, Labour and Welfare (MHLW)
12:00pm – 1:15pm | LUNCH – NO LIVE STREAM AT THIS TIME
1:15pm – 2:45pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
COMPANY PRESENTATIONS {Ballroom 1}
1:15pm – 1:30pm Precision BioSciences
1:30pm – 1:45pm Cell Medica
1:45pm – 2:00pm Intellia Therapeutics
2:00pm – 2:15pm REGENXBIO
2:15pm – 2:30pm Sangamo Therapeutics
2:30pm – 2:45pm Abeona Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
1:15pm – 1:30pm Sigilon
1:30pm – 1:45pm Verigraft
1:45pm – 2:00pm CellSeed
2:00pm – 2:15pm GammaDelta Therapeutics
2:15pm – 2:30pm Adverum Biotechnologies
2:30pm – 2:45pm WindMIL Therapeutics
2:45pm – 3:15pm | AFTERNOON BREAK – NO LIVE STREAM AT THIS TIME
3:15pm – 5:30pm | CONCURRENT TRACKS – LIVE STREAM RESUMES
PANEL: iPSCs FOR THERAPEUTIC APPLICATIONS  {Ballroom 1}
3:15pm – 4:15pm
This panel will highlight the clinical potential of iPSCs and the technical challenges that companies in this area are facing on the path to commercialization. The discussion will review the transition of new therapies based upon iPSCs from research into the clinic. It will also examine the barriers to the development of the industry and how these can be removed to accelerate growth.
Chair:
Keith Thompson, CEO, Cell and Gene Therapy Catapult
Speakers:
Hardy TS Kagimoto, M.D., Ph.D, President and CEO, Healios K.K.
Ross Macdonald, Ph.D., Managing Director and CEO, Cynata Therapeutics
Emile Nuwaysir, Ph.D., CEO, BlueRock Therapeutics
Yuzo Toda, Chairman, Forum for Innovative Regenerative Medicine (FIRM); Senior EVP and Chief Technical Officer, Director, FUJIFILM Corporation
COMPANY PRESENTATIONS {Ballroom 2}
3:15pm – 3:30pm B-MoGen Biotechnologies
3:30pm – 3:45pm Synpromics
3:45pm – 4:00pm Benitec Biopharma
4:00pm – 4:15pm 4D Molecular Therapeutics
COMPANY PRESENTATIONS {Ballroom 1}
4:15pm – 4:30pm NO LIVE STREAM AT THIS TIME
4:30pm – 4:45pm Athersys
4:45pm – 5:00pm NO LIVE STREAM AT THIS TIME
5:00pm – 5:15pm Asterias Biotherapeutics
5:15pm – 5:30pm ReNeuron
COMPANY PRESENTATIONS {Ballroom 2}
4:15pm – 4:30pm StemBioSys
4:30pm – 4:45pm Universal Cells
4:45pm – 5:00pm Biostage
5:00pm – 5:15pm SCM Lifescience
5:15pm – 5:30pm RoosterBio
5:30pm | LIVE STREAM ENDS FOR THE DAY

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8:00am | LIVE STREAM BEGINS
8:00am – 8:15am WELCOME REMARKS
Speaker:
Alysson Muotri, Ph.D., Chair, Scientific Symposium Steering Committee; Director, Stem Cell Program; Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
8:15am – 8:55am ADVANCED GENETIC ENGINEERING OF HEMATOPOIESIS TO TREAT INHERITED DISEASES AND CANCER
Sponsored by Lonza
Keynote Speaker:
Luigi Naldini, M.D., Ph.D., Director and Head, San Raffaele Telethon Institute for Gene Therapy; Professor, Cell and Tissue Biology, Vita-Salute San Raffaele University; Milan, Italy
8:55am – 10:15am PANEL: TARGETING ANTI-VIRAL IMMUNITY IN CANCER STEM CELLS
The capacity of cancer stem cells to divide without differentiating (self-renew) and alter their cell cycle characteristics in protective niches is predicated, at least in part, on aberrant activation of anti-viral enzymes like ADAR1 that edit self-renewal and cell cycle regulatory transcripts, including primary microRNAs. In addition, ADAR1 edits the DNA mutagenic enzymes, APOBECs. Thus, clonal cancer stem cell evolution may be prevented by targeting ADAR1 or the inflammatory cytokine signaling pathways that activate it.
Chair / Introduction By:
Catriona Jamieson, M.D., Ph.D., Professor of Medicine, Division of Hematology-Oncology; Chief, Division of Regenerative Medicine; Deputy Director, Sanford Stem Cell Clinical Center; Co-Leader, Hematologic Malignancies Program; Director, Stem Cell Research, Moores UC San Diego Cancer Center
Targeting Innate Immune Response Pathways in Cancer Stem Cells
Catriona Jamieson, M.D., Ph.D., Professor of Medicine, Division of Hematology-Oncology; Chief, Division of Regenerative Medicine; Deputy Director, Sanford Stem Cell Clinical Center; Co-Leader, Hematologic Malignancies Program; Director, Stem Cell Research, Moores UC San Diego Cancer Center
Killing Cancer Stem Cells
Jeremy Rich, M.D., Professor, Department of Medicine, Division of Regenerative Medicine; Director, Neuro-Oncology; Director, Brain Tumor Institute, UC San Diego
Chimeric Antigen Receptor Engineered T-Cells for the Treatment of Cancer: From Blood to Brain
Christine Brown, Ph.D., Heritage Provider Network Professor of Immunotherapy; Associate Director, T-Cell Therapeutics Research Laboratory, Department of Hematology and Hematopoietic Cell Transplantation, Beckman Research Institute of City of Hope
Development of Control Systems for CAR-T Cell Therapy
Travis Young, Ph.D., Principal Investigator, Immuno-Oncology, California Institute for Biomedical Research (Calibr)
10:15am – 10:40am | MORNING BREAK – NO LIVE STREAM AT THIS TIME
10:40am – 12:00pm | LIVE STREAM RESUMES
10:40am – 12:00pm PANEL: THE EYES HAVE IT: USING iPSC TO STUDY NEURO-VASCULO-GLIAL CROSS TALK IN THE CNS
Chair / Introduction By:
Martin Friedlander, M.D., Ph.D., Professor, Department of Cell Biology, The Scripps Research Institute; Chief, Retina Services, Department of Ophthalmology, Scripps Clinic; President, The Lowy Medical Research Institute
Reprogramming Approaches to Understand Development and Disease
Kristin Baldwin, Ph.D., Associate Professor of Neuroscience, The Scripps Research Institute; Adjunct Associate Professor, Department of Neuroscience, UC San Diego
Targeting the Brain Vasculature in Aging and Disease
Sean Buchanan, Ph.D., Scientist, Stem Cell and Regenerative Biology, Harvard University
Stemming Vision Loss with Stem Cells: There is More than Meets the Eye
Martin Friedlander, M.D., Ph.D., Professor, Department of Cell Biology, The Scripps Research Institute; Chief, Retina Services, Department of Ophthalmology, Scripps Clinic; President, The Lowy Medical Research Institute
12:00pm – 1:15pm | LUNCH – NO LIVE STREAM AT THIS TIME
1:15pm – 2:35pm | LIVE STREAM RESUMES
1:15pm – 2:35pm PANEL: THE USE OF TISSUE ORGANOIDS IN SEVERAL BIOLOGICAL APPLICATIONS
Tri-dimensional models can best mimic human tissues in a dish. On this panel, the audience will hear how investigators are deriving live human 3D organoids in a dish to understand human development, perform disease modeling and for potential therapeutic applications.
Chair / Introduction By:
Alysson Muotri, Ph.D., Director, Stem Cell Program; Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
The Emergence of Neuroinflammation in Human Brain Organoids
Alysson Muotri, Ph.D., Director, Stem Cell Program; Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
Modeling Neurodevelopmental Disorders with Genetically Engineered Cortical Organoids
Helen Bateup, Ph.D., Assistant Professor of Neurobiology, UC Berkeley
Consider the Lung as a Sensory Organ: An In Vivo Neural-Immune Organoid
Xin Sun, Ph.D., Professor of Pediatrics and Biological Sciences, UC San Diego
3D Retinal Organoids for Modeling Eye Development and Disease
Karl Wahlin, Ph.D., Assistant Professor of Ophthalmology, UC San Diego
2:35pm – 3:00pm | AFTERNOON BREAK – NO LIVE STREAM AT THIS TIME
3:00pm – 5:00pm | LIVE STREAM RESUMES
3:00pm – 4:20pm PANEL: STEM CELLS AND CANCER
This session will focus on how cancer progression and therapy resistance are fundamentally driven by epigenetic and stem cell signals, and how defining these signals will enable development of new strategies for effective cancer control.
Chair / Introduction By:
Tannistha Reya, Ph.D., Professor, Pharmacology and Medicine, UC San Diego
Wnt and Fgf Cooperativity in Mammary Stem Cells and Breast Cancer – NO LIVE STREAM AT THIS TIME
Jeffrey Rosen, Ph.D., Distinguished Service Professor; Vice-Chair and C.C. Bell Professor, Molecular and Cellular Biology and Medicine, Baylor College of Medicine
SWI/SNF Mutations in Cancer
Diana Hargreaves, Ph.D., Assistant Professor, Molecular and Cell Biology Laboratory, Salk Institute for Biological Studies
The Role of the LIF Stem Cell Factor in Pancreatic Cancer
Tony Hunter, Ph.D., American Cancer Society Professor, Molecular and Cell Biology Laboratory; Renato Dulbecco Chair, Salk Institute for Biological Studies
4:20pm – 5:00pm USING A COLLECTION OF iPSC LINES FROM 222 INDIVIDUALS AND DERIVED CELL TYPES TO FUNCTIONALLY ANNOTATE HUMAN GENETIC VARIANTS
Sponsored by Lonza
Keynote Speaker:
Kelly Frazer, Ph.D., Director, Institute for Genomic Medicine; Founding Chief, Division of Genome Information Sciences, Department of Pediatrics, UC San Diego
5:00pm | LIVE STREAM ENDS