Speaker Profile

M.D., Ph.D., President & CEO, Ultragenyx

A medical geneticist by training, Dr. Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO at Ultragenyx, a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases. The company has three approved therapies and holds ex-US rights to a fourth approved product, and a deep pipeline of potential treatments across a variety of therapeutic modalities including gene therapy, mRNA therapy, biologics, and small molecule. Before founding Ultragenyx in 2010, Dr. Kakkis guided and contributed to the development of multiple approved rare disease products as chief medical officer at BioMarin Pharmaceutical. Dr. Kakkis also founded and serves on the Board of EveryLife Foundation for Rare Diseases, a non-profit dedicated to accelerating innovation for rare diseases. Dr. Kakkis has been recognized for his leadership by Biotechnology Innovation Organization (BIO), the National MPS Society, WORLDSymposium, and California Life Sciences.

 Session Abstract – PMWC 2023 Silicon Valley

Track 1 - January 25 9.00 A.M.-3.00 P.M.

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
    - Amber Salzman, Epic Bio
    - Ben Oakes, Scribe Therapeutics
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    - Hank Greely, Stanford
    - Hana El-Samad, UCSF