Speaker Profile

Impactful contributions to the gene therapy field for her groundbreaking work in developing a gene therapy for hemophilia that has paved the way for the development of other gene therapies and has made a significant impact on the field of precision medicine.

MD, Visiting Professor, Rockefeller U.

Katherine A. High is a distinguished physician-scientist and currently a Visiting Professor at Rockefeller University. She is also Professor Emerita at the Perelman School of Medicine at the University of Pennsylvania and previously served as President of Therapeutics at AskBio. Dr. High is renowned for her contributions to the field of gene therapy, particularly in the development of therapies for rare diseases. She was the co-founder, president, and chief scientific officer of Spark Therapeutics, where she led the development of the first FDA-approved gene therapy for genetic disease, a rare form of inherited blindness, and played a pivotal role in the development of gene therapies for hemophilia B and hemophilia A. Dr. High's groundbreaking research has earned her numerous awards and honors, including the E.Donnall Thomas Prize from the American Society of Hematology, the Karl Landsteiner Award from the American Association of Blood Banks, the Outstanding Achievement Award from the American Society of Gene and Cell Therapy, and the Sanford Lorraine Cross $1 Million Medical Innovation Award, shared with Dr. Jean Bennett. She is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, the Royal College of Physicians, and the National Academy of Sciences. Dr. High's ongoing commitment to advancing precision medicine and improving patient outcomes has had a transformative impact on the field of gene therapy and rare disease research. Her work has led to the development of innovative therapies that have the potential to cure or significantly improve the lives of patients with rare diseases.

 Session Abstract – PMWC 2024 Silicon Valley

Track 1 - January 24 9.00 A.M.-4.45 P.M.

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


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