Speaker Profile
MD, PhD, Professor of Pediatrics, Stanford
Biography
Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the coDirector for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it.
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
