Speaker Profile

MD, Professor of Pediatrics, UCSF

Biography
Jennifer Puck served on the faculties of U of Pennsylvania Medical School and the National Human Genome Research Institute before becoming a Professor of Pediatrics at UC San Francisco in 2006. While caring for patients with immune disorders, she has researched the genetic causes and mechanisms for rare human immune deficiencies. She developed the newborn screening test for severe combined immunodeficiency (SCID), now adopted throughout the USA and in many countries, enabling early diagnosis and optimal treatment. She has participated in multiple gene therapy trials for SCID and is currently CoPI of the successful first human Artemis SCID lentiviral gene addition trial at UCSF. While these advanced clinical studies offer health benefits to those with rare diseases, they also illuminate important basic immune pathways that were previously unknown.


 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

×

Add Names/PO on Receipt

Apply to Speak at PMWC Silicon Valley by JUL. 18TH

The PMWC team will reply back to a suitable company/topic with an official request for talk/speaker details.

Contact Us:

Fields marked with an * are required 

Contact Us:

Fields marked with an * are required 

Discover What's Next in Precision Medicine!

Join our community today for the latest news, exclusive interviews, and unique insights from world-renowned speakers and experts


Contact Us:

Fields marked with an * are required 

Discount also depends on group size. The bigger group – the bigger discount.

Trouble Registering

Fields marked with an * are required

Stay Tuned For More Information.

Sign up for occasional updates on upcoming conferences, news, and other information. 

Get Updates:

Submit to Speak in the PMWC Showcase

Fields marked with an * are required