Speaker Profile
MD, Chief Medical Officer, Ultragenyx
Biography
Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics in November 2017 and is responsible for strategic leadership of the company's clinical development and translational research programs. He also oversees global development functions including Clinical Development, Clinical Operations, BioMetrics, Endpoint Development and Strategy, Regulatory Affairs and Drug SafetyPharmacovigilance. Dr. Crombez is an appointed industry representative on the FDA Cellular, Tissue and Gene Therapies Advisory Committee. Before joining industry, he was assistant professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA). Dr. Crombez is a board certified clinical geneticist and completed residencies in pediatrics and medical genetics and a fellowship in clinical biochemical genetics.
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
