Speaker Profile

RAC, Head of Regulatory and Policy, Co-Director of ABOM*, FAST

Biography
Jennifer has worked in drug development for over 20 years, primarily focused on advancing global development programs for the treatment of rare neurological diseases. Her background and experience working with global regulatory agencies to support diverse drug and biological therapy development programs in both the development and postmarketing strategies will greatly benefit the FAST community. Jennifer has a niece, Samantha who lives with Angelman syndrome.


 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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