Speaker Profile
MD, President, Co-Founder, KCNT1 Foundation
Biography
Collaborating with multiple academic and industry partners around the world. Current programs include drug repurposing, de novo small molecules, ASOs, RNAi, and gene editing. Building a clinical trial ready community through various initiatives including patient registry, digital natural history study, biobank, blood and EEG biomarker discovery and patient education.
Session Abstract – PMWC 2024 Silicon Valley
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
