RettEd: Gene Therapy in Rare Disease

RettEd: Gene Therapy in Rare Disease

As a rare disease community, we can learn a lot from others further down the road of gene replacement therapy. In this informative conversation with IRSF’s CSO, Dr. Dominique Pichard, you’ll hear from P.J. Brooks, PhD, Program Director for the Office of Rare Disease Research at the NIH’s Center for Advancing Translational Sciences. We’ll also hear from CureSMA’s Chief Scientific Officer, Jill Jarecki, PhD, who will share what CureSMA learned from their FDA-approved gene therapy clinical trial experience.

Join us Wednesday, July 28 at 1 p.m. EDT.

Takeaways from this RettEd:

– Understand the potential for gene replacement therapy for single-gene disorders like Rett syndrome and learn what is involved in delivering complex treatment molecules to the affected cell.

– Understand what NCATS is doing to accelerate clinical trials in rare diseases by moving beyond “one disease at a time” approaches.

– Get perspective from another rare disease foundation’s gene therapy clinical trial experience.

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