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