Speaker Profile
M.D.,Ph.D., CEO, Mahzi Tx
Biography
Yael Weiss is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical proof of concept is established. Yael completed her MD at Hadassah Medical School at the Hebrew University in Jerusalem and her PhD at the Weizmann Institute of Science in Rehovot, Israel. She has over 20 years of industry experience in medical-clinical and business development roles at Genzyme, Merck and Ultragenyx. Yael is a member of the NIH driven Bespoke Gene Therapy (BCTG) consortium, ASGCT translational committee, N1 collaborative and is a 2022 Termeer Fellow Board member and advisor to ADNP and FOXG1 foundations.
Session Abstract – PMWC 2025 Silicon Valley
Track Co-Chairs:
Yael Weiss, Mahzi Tx
Morten Sogaard, Astellas Gene Therapies
- PMWC 2025 Award Ceremony
Pioneer Honoree: James M. Wilson, University of Pennsylvania
Luminary Honoree: Carolyn Bertozzi, Stanford
- Keynote: Bioorthogonal Chemistry, from Basic Science to Clinical Translation
- Carolyn Bertozzi, Stanford
- Keynote: The Promise of Genetic Medicines Unless You're Too Poor or Too Rare
- James M. Wilson, University of Pennsylvania
- Reimbursement for Genetic Medicines in Rare Diseases
Chair:
- Erin Satterwhite, Optum Specialty Pharmacy
- Next-Generation Delivery Systems for Gene Therapies (PANEL)
Chair: Morten Sogaard, Astellas
- Mathieu Nonnenmacher, Voyager Therapeutics
- Kevin Friedman, Kelonia Therapeutics
- Kunwoo Lee, GenEdit
- Haig Aghajanian, Capstan Therapeutics
- James M. Wilson, University of Pennsylvania
- Beyond the Genome: Harnessing Epigenetics in Rare Disease Treatment (PANEL)
Chair: Charles Gersbach, Duke
- Prashant Mali, UCSD
- Nadav Ahituv, UCSF
- Blythe Sather, Tune Therapeutics
- Fyodor D Urnov, Berkely University
- Navigating the Regulatory Landscape for Gene and Cell Therapies (PANEL)
Chair: Yael Weiss, Mahzi Tx
- Courtney Silverthorn, Bespoke Gene Therapy Consortium (BGTC)
- Peter Marks, FDA
- Ben Dewees, Kyverna Therapeutics
- AI-driven Improvements in Gene and Cell Therapy (PANEL)
Chair: Morten Sogaard, Astellas
- Francois Vigneault, Shape Therapeutics
- John Androsavich, Ginkgo Bioworks
- Yogev Debbi, mana.bio
