Speaker Profile

MD, PhD, Chief Medical Officer, BridgeBio GT

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 clinical stage programs for Canavan disease and congenital adrenal hyperplasia. Prior to joining BridgeBio Adam led clinical stage 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 Israel Deaconess 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 1 - January 24 9.00 A.M.-4.45 P.M.


Track Co-Chairs:
Yael Weiss, Mahzi Tx
Peter Marks, FDA

  • PMWC 2024 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
    - Maria Grazia Roncarolo, Tr1X
    - Katherine A. High, Rockefeller University
    - Jennifer Panagoulias, FAST (Angelman Syndrome Foundation)
    - 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
    - Justin West, KCNT1 Foundation
    - Michael Graglia, Syngap Research Fund
    - Wendy Erler, Alexion Pharmaceuticals
  • Advancing the Frontier: Gene Editing for Rare Diseases (PANEL)
    Chair: Matthew Porteus, Stanford
    - James Chung, Kyverna Therapeutics
    - Lucas Harrington, Mammoth Biosciences
    - Aron Stein, Intellia Therapeutics
  • Advancing the Frontier: Gene and Cell Therapies for Rare Diseases (PANEL)
    Chair: Morten Sogaard, Astellas Gene Therapies
    - Pooja Agarwal, BioMarin Pharmaceutical
    - David Kirn, 4dmt
    - Adrian Veres, Dyno Therapeutics
    - Thomas Wechsler, J&J
  • PMWC Showcase
    - Dominic Borie, Kyverna Therapeutics

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