Tuesday, June 8

11:00–11:45 AM
Bonus Session: A Primer on Regenerative Medicine Science and Recent Breakthroughs
Heather C. Hatcher, Regulatory Scientist, Womble Bond Dickinson (US) LLP
Collin Stabler, Senior Associate, Exponent, Inc.
Moderated by Barbara A. Binzak Blumenfeld, Shareholder, Buchanan Ingersoll & Rooney PC

This bonus session will provide an introduction to the science of cell and gene therapy. The panelists will also discuss recent therapeutic breakthroughs.

12:00–12:05 PM
FDLI Welcome and Introduction
Amy Comstock Rick, President & CEO, FDLI

12:05–12:50 PM
Keynote Address
Peter W. Marks, Director, Center for Biologics Evaluation and Research, FDA

1:05–1:50 PM
Updates on FDA’s Comprehensive Regenerative Medicine Policy Framework
Wilson W. Bryan, Director, Office of Tissues and Advanced Therapies, CBER, FDA
Lauren A. Miller, Senior Counsel, AbbVie
Moderated by Joanne Hawana, Member, Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C.

During this session, speakers will examine FDA’s Framework for supporting and expediting regenerative medicine product development, including the status of new and upcoming guidances. The panel will also discuss FDA’s inter-Center coordination to advance regenerative medicine therapies.

2:05–2:50 PM
Global Regulatory Collaborations in Search of Cell and Gene Therapy Harmonization
Joan Wilmarth Blair, Senior Advisor for International Affairs, CBER, FDA
Victoria Palmi Reig, Senior Product Lead, Office of Advanced Therapies and Therapies for Immune and Inflammatory Diseases, European Medicines Agency
Moderated by Cristiana Spontoni, Partner, Jones Day

FDA and the European Medicines Agency (EMA) have emphasized the need to internationally harmonize regulatory approaches for cell and gene therapy products in an effort to increase access to patients worldwide. What is the status of this dialogue? The session will tour the landscape of international efforts to advance harmonization for these products, including work in the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, the International Pharmaceutical Regulators Programme, the World Health Organization, and the regulatory discussions within the EMA-FDA “Clusters.” The session will also discuss revisions to the European Union’s blood, tissue, and cell regulation, lessons learned during the approval of Advanced Therapy Medical Products in the EU, and regulatory tools to facilitate development, approval, and access.

2:50–3:20 PM 

3:20–4:20 PM
Gene Therapy Clinical Development Considerations
Mantej (Nimi) Chhina
, Senior Director & Head, Global Regulatory Policy, BioMarin Pharmaceutical Inc.
Ilan Irony, Deputy Director, Division of Clinical Evaluation and Pharmacology/Toxicology, CBER, FDA
Gopa Raychaudhuri, Senior Scientist, Office of the Center Director, CBER, FDA
Moderated by Michael N. Druckman, Partner, Hogan Lovells US LLP

This panel will explore issues associated with the clinical development of gene therapies, including design challenges, good clinical practice considerations, and use of real-world evidence in accelerated approvals. Speakers will also discuss the Bespoke Gene Therapy Consortium, a public/private partnership intended to provide a standardized and efficient approach for development and delivery of “bespoke,” or individualized, gene therapies for patients with rare genetic diseases.

4:35–5:20 PM  
Standardization of Regenerative Therapies
Judy Arcidiacono, Regulatory Affairs Specialist, Office of Tissues and Advanced Therapies, CBER, FDA
Fouad Atouf, Vice President, Global Biologics, U.S. Pharmacopeia
Robert Shaw, Executive Director, Standards Coordinating Body
Moderated by Deborah L. Livornese, Director, Hyman, Phelps & McNamara, PC

Section 3036 of the 21st Century Cures Act requires FDA to work with other stakeholders to coordinate and prioritize the drafting of standards and consensus definitions to support the development, evaluation, and review of regenerative medicine therapies. Since 2017, FDA has been working with the Nexight Group and the Standards Coordinating Body to coordinate the development of regenerative medicine standards. This session will discuss the status of this work, why standards are important in this field, and what standards are currently in place.

Wednesday, June 9

12:00–12:05 PM
FDLI Welcome
Laura A. Brown, Director, Educational Programs, FDLI

12:05–12:50 PM
Featured Speaker
Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania

1:05–2:05 PM
Regulation of Gene and Cell Therapy Manufacturing
Mo Heidaran, Vice President, Technical, Parexel International
Lawrence Starke, Global Regulatory Affairs (CMC) Policy and Intelligence, Cell & Gene Therapy, Novartis Pharmaceutical Company
Moderated by Kathleen M. Sanzo, Partner, Morgan, Lewis & Bockius LLP

This session will discuss regulatory challenges associated with manufacturing of gene and cell therapies. Speakers will discuss similarities and differences in the manufacturing of cell versus gene therapies, use of advanced manufacturing technologies, and good manufacturing and tissue practice considerations. Legal issues associated with commercial supply chain agreements, and inspections and supply chain disruptions during COVID-19, will also be discussed.

2:05–2:35 PM

2:35–3:20 PM
Ensuring Regenerative Therapy Product Compliance with FDA
Allison Fulton, Partner, Sheppard Mullin Richter & Hampton LLP
Melissa Mendoza, Deputy Director, Office of Compliance and Biologics Quality, CBER, FDA
Moderated by Kalah Auchincloss, SVP and Deputy General Counsel, Greenleaf Health, Inc.

Due to their innovative and complex technology, regenerative medicine products can have unique considerations that can result in compliance issues. The panel will explore ways product sponsors can work with the agency on an informal basis at various stages of the approval process to ensure compliance. This session will also discuss recent compliance and enforcement trends. 

3:35–4:20 PM
Gene and Cell Therapy Coverage, Reimbursement, and Pricing
Rochelle Fink, Senior Health Science Project Manager, CDRH, FDA
Michael Werner, Partner, Holland & Knight LLP
Moderated by Catherine A. Brandon, Partner, Arnold & Porter LLP

This session will explore challenges in providing access to expensive but highly beneficial regenerative therapies and best practices for aligning goals between manufacturers and payors to minimize coverage issues. Speakers will also explain how FDA and payors, including the Centers for Medicare and Medicaid Services, work together to expedite patient access to these lifesaving therapies.              

4:20 PM
Closing Remarks and Adjournment