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The Promises and Pitfalls of Gene Therapy for Rare Diseases

April 30, 2021
On-Demand Webinar
YouTube video

The state of gene therapy faces much uncertainty as recent regulatory and clinical setbacks have raised questions about its promise. With only two gene therapies FDA-approved for rare diseases to date, the path to developing these medicines is still a difficult one.

This presentation exploreed how the biopharma industry can overcome development challenges in the pursuit of the promise of gene therapy.