Global Regulatory Issues in Gene Therapy Development

Monday, November 4 | 9 a.m.–5 p.m. | The Westin City Center, Washington, D.C.

The goal of this program is to explore solutions to challenges experienced both by global regulators and by sponsors developing gene therapies across established and emerging markets. Scheduled topics include identifying differences in regulatory requirements and areas of opportunity for greater convergence of regulatory frameworks.

Objectives:

1. To inform attendees about differences in regulatory requirements in established and emerging markets
2. To discuss the challenges of global development and to engage in dialogue about areas of opportunity for harmonization to support development of gene therapy
3. To shape recommendations on potential movement toward convergence of regulatory frameworks

Full Agenda

Meeting Welcome & Introduction
9-9:15 a.m.

• Adora Ndu, PharmD, J.D.
  BioMarin

Developments in Gene Therapy Policy Landscape
9:15-11:15 a.m.

• Renata Miranda Parca—The Brazilian Regulatory Framework on Advanced Therapy Medicinal Products
  Office of Blood, Cells, Tissues and Organs, Brazilian Health Regulatory Agency (ANVISA)
• Martin Hernan Bonamino, Ph.D.—The Role and Contributions of the Brazilian Regulatory Agency’s Technical Advisory Board
  Brazilian Health Regulatory Agency (ANVISA) Advanced Therapy Technical Committee
• Liz Anne Gillham-Eisen—Health Canada: Bringing Innovation to the Regulation of Advanced Therapeutic Products
  Health Products and Food Branch, Health Canada
• Keith Wonnacott, Ph.D.—The Impact of National Regulatory Policy on Global Gene Therapy Development
  Pfizer, Inc.
• John C. Bolzano, J.D.—Regulatory Developments in Cell Therapy in China
  Covington & Burling, LLP
• James Mayne, Ph.D.—Global Approaches Supporting Development in Gene Therapy
  The Pharmaceutical Research and Manufacturers of America (PhRMA)

Moderated Panel: Reflection on Policy Developments and the Need for Continued Advances
11:25 a.m.-12:15 p.m.

• Moderator: Keith Wonnacott, Ph.D.
  Pfizer, Inc.
• Martin Hernan Bonamino, Ph.D.
  National Cancer Institute, member of ANVISA’s Advanced Therapy Technical Committee
• Liz Anne Gillham-Eisen
  Health Products and Food Branch, Health Canada
• John C. Bolzano, J.D.
  Covington & Burling, LLP
• James Mayne, Ph.D.
  The Pharmaceutical Research and Manufacturers of America (PhRMA)

The Relevance of Global Regulatory Convergence to Accelerating the Availability of Gene Therapies
1:15-1:45 p.m.

Keynote Speaker: Peter Marks, M.D., Ph.D.
Director of FDA CBER

Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research (FDA CBER), opens the first day of the 2019 Policy Summit with potential areas for increased convergence of global regulatory policies for gene therapies, and the significance of striving toward this goal, as part of a session on the challenges and opportunities for developing multinational programs.

Identification of Challenges and Opportunities for Multinational Gene Therapy Programs
1:45-3:05 p.m.

Regulators from Europe, Japan, and the United States will provide updates and discuss difficulties and opportunities in enabling sustainable and adaptable regulatory frameworks for gene therapies:

• Rune Kjeken, Ph.D.—Regulatory Considerations from Europe
  Norwegian Medicines Agency, European Medicines Agency’s Committee for Advanced Therapy
• Kimberly Schultz, Ph.D.—FDA Framework for Gene Therapy Development
  Division of Cellular and Gene Therapies, FDA CBER
• Megumu Mori, M.D., Ph.D.—Current Status and Future Direction of the Regulatory Framework for Gene Therapy in Japan
  Medical Device Evaluation Division, Ministry of Health, Labour and Welfare, Japan
• Anne-Virginie Eggimann, MSc.—Challenges and Opportunities in Global Development of a Gene Therapy Production: LentiGlobin for Transfusion-dependent Beta-thalassemia – A Case Study
  bluebird bio

Moderated Panel: Regulatory Convergence—Reflection on the Challenges and Opportunities and Where We Go From Here
3:35-4:25 p.m.

• Moderator: Maritza McIntyre, Ph.D.
  Advanced Therapies Partners, LLC
• Peter Marks, M.D., Ph.D.
  Director of FDA CBER
• Kimberly Schultz, Ph.D.
  Division of Cellular and Gene Therapies, FDA CBER
• Rune Kjeken, Ph.D.
  Norwegian Medicines Agency, European Medicines Agency’s Committee for Advanced Therapy
• Megumu Mori, M.D., Ph.D.
  Medical Device Evaluation Division, Ministry of Health, Labour and Welfare, Japan
• Anne-Virginie Eggimann, MS
  bluebird bio

Closing Remarks
4:25-5 p.m.

• Jeanette Hogan, mother of Jack Hogan, the first post-approval recipient of Luxturna