2020 Policy Summit Agenda
(Subject to change - all times are CDT)
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Wednesday, September 23
Ethical and regulatory issues in gene and cell therapy
8:30 – 9 a.m. (CDT) Keynote speaker: George Q. Daley, M.D., Ph.D., Dean of the Faculty of Medicine & Caroline Shields Walker Professor of Medicine, Harvard Medical School
Science Advocacy: From Stem Cells to COVID
9 – 10:30 a.m. (CDT) Responsible development and use of technology
- Eric Juengst, Ph.D., Director of the UNC Center for Bioethics & Professor of Social Medicine and Genetics, University of North Carolina at Chapel Hill
Anticipating preventive somatic cell gene editing: “incidental enhancements” and their governance implications
- R. Alta Charo, J.D., Warren P. Knowles Professor of Law and Bioethics, University of Wisconsin Law School
Global law and policy on germline editing
- Sandy Macrae, M.B., Ch.B, Ph.D., Chief Executive Officer of Sangamo Therapeutics
Addressing bioethical challenges in genomic medicine
- Panel discussion moderated by Tim Hunt, J.D., formerly Chief Corporate Affairs Officer, Editas Medicine
10:30 – 11 a.m. Break
11 a.m. – 12:10 p.m. (CDT) Nontherapeutic uses of gene editing
- Renee Wegrzyn, Ph.D., Vice President, Business Development, Ginkgo Bioworks; formerly Program Manager, Defense Advanced Research Projects Agency (DARPA)
Transient gene therapies for prophylactic use
- David A Relman, M. D., Thomas C. and Joan M. Merigan Professor in Medicine, Stanford University
Security implications of genome engineering
- Panel discussion moderated by Pilar Ossorio, Ph.D., J.D., Professor of Law and Bioethics, University of Wisconsin-Madison
12:10 – 1 p.m. Lunch break
1 – 2:40 p.m. (CDT) Challenges and solutions in large-scale manufacturing
- Andrew Byrnes, Ph.D., Chief, Gene Transfer and Immunogenicity Branch, Division of Cellular and Gene Therapies, FDA
Standardization in vector manufacturing
- Christopher K. Murphy, Vice President and General Manager of Viral Vector Services in Thermo Fisher Scientific’s Pharma Services Group
Developing manufacturing facility readiness: CMO perspective
- Robert A. Baffi, Ph.D., Special Advisor to the Chairman and Chief Executive Officer, BioMarin
Strong Process Development for Gene Therapy Products
- Panel discussion moderated by Joanne S. Eglovitch, Senior Editor, Pink Sheet
2:40 – 3:10 p.m. Break
3:10 – 5 p.m. (CDT) CMC and regulatory issues
- Herb Runnels, Ph.D., BioTx Pharmaceutical Sciences, Analytical R&D, Pfizer
Identifying critical quality attributes for AAV vector products
- Katie Miller, Ph.D., Principal, Dark Horse Consulting
Identifying critical quality attributes for gene-modified cell therapies
- Denise Gavin, Ph.D., Chief, Gene Therapies Branch, FDA
Global CMC Convergence: an FDA Perspective
- Panel discussion moderated by Jan Thirkettle, Ph.D., Chief Development Officer, Freeline Therapeutics Limited
Thursday, September 24
Regulatory, legislative, and access issues in gene therapy
8:30 – 9 a.m. (CDT) Keynote speaker: Peter Marks, M.D., Ph.D., Director of FDA CBER
Streamlining Development by Leveraging Applications on File
9 – 10:10 a.m. (CDT) Trial design considerations for gene therapy for rare diseases
- Brad Glasscock, PharmD, Group Vice President, Head of Global Regulatory Affairs, BioMarin
Utilizing increased regulatory flexibilities in gene therapy trial design
- Daniel Chung, D.O., Medical Strategy Lead- Ophthalmology, Spark Therapeutics
Significance of patient-focused trial design: A case study
- Panel discussion moderated by Andrea Spezzi, M.D., Chief Medical Officer and Co-Founder, Orchard Therapeutics
10:10 – 10:30 a.m. Break
10:30 a.m. – 12 p.m. (CDT) Overcoming regulatory challenges in gene therapy trials
- Anne-Virginie Eggimann, Senior Vice-President, Regulatory Science, bluebird bio
Regulatory considerations to improve gene therapy development throughout the life cycle
- Ying Huang, Ph.D., Pharmacologist, Pharmacology/Toxicology Branch, FDA
Selecting appropriate animal models to inform clinical development
- Becky Schweighardt, Ph.D., Executive Director, Translational Sciences, BioMarin
Addressing immunogenicity in clinical trials
- Panel discussion moderated by Nancy Bradish Myers, J.D., President/Founder, Catalyst Healthcare Consulting, Inc.
12 – 12:30 p.m. Lunch break
12:30 – 1 p.m. (CDT) Keynote speaker: Michelle McMurry-Heath, M.D., Ph.D., President and CEO of the Biotechnology Innovation Organization (BIO)
Science as Social Justice, Scientists as Crusaders for Truth
1 – 2:30 p.m. (CDT) Evidence generation for regulators and payers
- Eric Faulkner, Vice President, Precision and Transformative Medicine, Evidera
Evidence-generation solutions to support regulatory and payer requirements
- Americo Cicchetti, Professor of Business Organization, Faculty of Economics, Universitá Cattolica del Sacro Cuore
Evidence requirements for health technology assessment in Italy
- Jeet Guram, M.D., Senior Advisor in the Office of the Commissioner, FDA
FDA & CMS parallel review process, expedited approvals, and outcome-based payment models
- Panel discussion moderated by Mark Battaglini, J.D., Senior Vice President and Head of Global Government Affairs, Alliance Development & Public Policy, bluebird bio
2:30 – 3 p.m. Break
3 – 5 p.m. (CDT) Policy in the pipeline: Spotlight on sickle cell disease (SCD)
- John Tisdale, M.D., Senior Investigator, Cellular and Molecular Therapeutics Branch, NHLBI
An introduction to genetic approaches to SCD: Scientific researcher perspective
- Beverley Francis-Gibson, President & Chief Executive Officer, Sickle Cell Disease Association of America
Sickle cell disease: Patient perspectives and ethical considerations in gene therapy
- Samarth Kulkarni, Ph.D., Chief Executive Officer, CRISPR Therapeutics
Policy implications of gene editing for SCD: Developer viewpoint
- Nick Leschly, President & Chief Executive Officer, bluebird bio
Policy implications of gene therapy for SCD: Developer viewpoint
- Panel discussion moderated by Beverley Francis-Gibson
Friday, September 25
Access to testing and treatment
8:30 – 8:35 a.m (CDT) Welcome remarks: The Honorable Tim Scott, United States Senator, South Carolina
8:35 – 9 a.m. (CDT) Keynote address: Joe Grogan, former Director of the United States Domestic Policy Council
9 – 10:30 a.m. (CDT) Gene and cell therapy access: Real world experiences
- Dan and Jodi Symes
The value of gene and cell therapy: A patient perspective
- Adam Hutchings, Managing Director, Dolon
An overview of patient and market access to approved gene therapies in the EU
- Andrew Walker, Ph.D., Director, Salus Alba
Addressing cost effectiveness for gene therapies in the pipeline in the EU
- Nicola Heffron, Head of Europe, bluebird bio
Sharing the risks and the rewards - Lessons from taking a partnership approach to launch a novel gene therapy in the EU
- Panel discussion moderated by Francesca Cook, Senior Director of Pricing and Market Access, REGENXBIO
10:30 – 11 a.m. Break
11 a.m. – 12 p.m. (CDT) Newborn screening policies
- Diane Berry, Ph.D., Senior Vice President, Global Health Policy, Government, and Patient Affairs, Sarepta Therapeutics
The significance of newborn screening policies on access to gene therapy
- Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases
Patient advocate perspectives on newborn screening policies
- Panel discussion moderated by Christine Harrison, Vice President, Corporate Affairs, Orchard Therapeutics
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