Gene Replacement and Gene Editing Therapies — 2023 RARE Advocacy Summit

January 19, 2024

As part of the Rare Disease Master Class Science & Tech Innovation track from the 2023 RARE Advocacy Summit, this panel will demonstrate how gene replacement and gene editing therapies work, when they are appropriate, and the barriers that need to be overcome.

Gene Replacement and Gene Editing Therapies

Danny Levine
Life Sciences Writer & RARECast Host, Global Genes Presenters:

* Amy Raymond, PhD, PMP
Therapeutic Strategy Lead, Rare Diseases & Cellular and Genetic Medicines, Worldwide Clinical Trials

* Matthew Fuller, PhD, PMP
Executive Director, Gene Therapy Research, Ultragenyx

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