Speaker Profile

DVM, DACVIM, Chief Science Officer, FAST, NPO

Dr. Berent is a veterinary internal medicine specialist/interventionalist who graduated from Cornell University and completed her residency at the University of Pennsylvania, where she served as an Adjunct Assistant Professor before joining the Animal Medical Center in NYC. She is the Director of Interventional Endoscopy, focusing on clinical trials researching medical devices particularly for ureteral and biliary obstructions. In 2014 Dr. Berent’s daughter, Quincy, was diagnosed with a rare neurogenetic disorder called Angelman syndrome. In 2015 she joined the Board of Directors for the Foundation for Angelman Syndrome Therapeutics (FAST), becoming the Chief Science Officer. Dr. Berent serves as the co-director of the Angelman Syndrome Biomarker and Outcome Measure Consortium. Dr. Berent co-founded GeneTx Biotherapeutics, a company focused on advancing an antisense oligonucleotide therapy, where she was the Chief Operating Officer. GeneTx was acquired in 2022 by Ultragenyx Pharmaceuticals, after launching the Phase 1/2 clinical trial.

The Parents' Journey Through Drug Development
After her little girl was diagnosed with AS Dr. Berent saw the impossible become possible. Through passion, scientific, and medical understanding, this incredible foundation (FAST) and a bold scientific team, advanced numerous treatment options for children with AS, paving the way for what so many other rare disease organizations are working so hard to do.

 Session Abstract – PMWC 2023 Silicon Valley

Track 1, January 25

Track Co-Chairs:
Yael Weiss, Mahzi
Peter Marks, FDA

  • Welcome and Opening by Track Chairs: Yael Weiss, Mahzi & Peter Marks, FDA
  • Patient Driven Therapies: The Challenges and Successes (PANEL)
    Chair: Yael Weiss, Mahzi
    - Allyson Berent, Angelman Foundation
    - Terry Pirovolakis, CureSPG50
  • From Bench to Bedside - Biomarkers, Natural History Studies, Animal Models (PANEL)
    Chair: Peter Marks, FDA
    - Emil Kakkis, Ultragenyx
    - Alysson Muotri, UCSD
    - Nasha Fitter, Invitae
  • Commercialization - Moving from Rare to Common (PANEL)
    Chair: John Maraganore, Alnylam Pharmaceuticals
    - Shankar Ramaswamy, Kriya
    - Rahul Singhvi, Resilience
    - Andrew Lo, MIT
    - Mark Harrison, University of British Colombia
  • Future Modalities (PANEL)
    Chair: Shannon Muir, CIAPM
    - Nadav Ahituv, UCSF
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    - Hank Greely, Stanford