Session Chair Profile
M.D., Ph.D., Chief Medical Officer, BridgeBio
Biography
Adam Shaywitz leads the clinical organization for Gene Therapy at BridgeBio Pharma, focused on developing gene therapy treatments for a range of monogenic rare diseases including clinicalstage programs for Canavan disease and congenital adrenal hyperplasia. Prior to joining BridgeBio Adam led clinicalstage programs at BioMarin Pharmaceutical aimed at treating a broad range of rare diseases including Sanfilippo Syndrome (MPS III), achondroplasia, endocrinologic disorders, Morquio A syndrome and other lysosomal storage disorders. Before joining BioMarin Adam spent four years at Amgen in their early clinical development group, focused on advancing promising diabetes and metabolism agents into the clinic. He received his M.D. and Ph.D. degrees from Harvard Medical School, did his internal medicine training at MGH and subsequently went on to pursue a fellowship in endocrinology, diabetes and metabolism at Beth IsraelDeaconess Medical Center and the Joslin Diabetes Center. Adam currently serves on the Board of Directors at Cure Sanfilippo Foundation.
Session Abstract – PMWC 2024 Silicon Valley
Track Co-Chairs:
Yael Weiss, Mahzi Tx
Peter Marks, FDA
- PMWC 2024 CGT Award Ceremony
Pioneer Honoree: Katherine A. High, AskBio
Pioneer Honoree: Timothy Yu, Boston Children's Hospital
- Piloting Interventional Genomics for Orphan Diseases
Keynote: Timothy Yu, Boston Children's Hospital
- Accelerated Approval for Small Populations: Biomarkers & Endpoints (PANEL)
Chair: Peter Marks, FDA
- Jennifer Puck, UCSF
- Katherine A. High, Rockefeller University
- Timothy Yu, Boston Children's Hospital - Novel Clinical Design Approaches in Rare Diseases (PANEL)
Chair: Adam Shaywitz, Bridge Bio
- Salvador Rico, Encoded Therapeutics
- Eric Crombez, Ultragenyx
- Elizabeth Berri Kravis, Rush University
- Empowering Patient Advocacy in Rare Disease Therapies (PANEL)
Chair: Yael Weiss, Mahzi Tx
- Charlene Son Rigby, STXBP1 Foundation
- Michael Graglia, Syngap Research Fund - Advancing the Frontier: Gene and Cell Therapies for Rare Diseases (PANEL)
Chair: Matthew Porteus, Stanford
- Swati Tole, Capsida Biotherapeutics
- Adrian Veres, Dyno Therapeutics
- Thomas Wechsler, J&J
- David Lebwohl, Intellia Therapeutics
- Pooja Agarwal, BioMarin Pharmaceutical
