Speaker Profile

M.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism

Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics, a liver directed AAV gene therapy company. During his tenure at both companies, Dr. Crombez led the clinical development efforts for four clinical stage programs including gene therapy programs for hemophilia B, hemophilia A (in partnership with Bayer), ornithine transcarbamylase (OTC) deficiency and glycogen storage disorder type Ia (GSDIa). Dr. Crombez is also serving as an Industry Representative on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. Prior to this work in gene therapy, Dr. Crombez worked at Shire in their Human Genetics Therapy business unit for nearly eight years. Prior to joining industry, Dr. Crombez 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).

 Session Abstract – PMWC 2022 Silicon Valley

Track Chair:
Jane Grogan, Graphite Bio


  • Building Future Precision Care in Qatar
    Session Chair: Khalid Fakhro, Sidra Medicine
    - Barbro Friden, Sidra Medicine
    - Ziyad Hijazi, Sidra Medicine
    - Edison Liu, Jackson Labs
  • Advances in Gene & Cell Therapies
    - Peter Nell, Mammoth
    - Laura Sepp-Lorenzino, Intellia Tx
    - Amy Simon, Beam Therapeutics
    - Eric Ostertag, Poseida Therapuetics
    - Jane Grogan, Graphite Bio
  • Translating CGT to the Clinic
    Session Chair: Nicole K. Paulk, UCSF
    - Matthew Porteus, Stanford
    - Eric Crombez, Ultragenyx Gene Therapy
    - Peter Francis, 4D Molecular Therapeutics
    - Erandi De Silva, Forge Biologics
    - Patrice Hugo, Q2 Solutions
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    Session Chair: Ilana Yurkiewicz, Stanford University
  • Past, Present and Future of CRISPR Where will CRISPR Take Us and How is the Technology Evolving to Realize its Full Potential
    Session Chair: Janice Chen, Mammoth
    - Fyodor Urnov, UC Berkeley
    - Kiana Aran, Cardea Bio
    - Laura Sepp-Lorenzino, Intellia Tx