4D5A0D86872BB8698725867E0076D3C7 Hearing Summary

Scott McGoohan, Senior Director, Policy and Alliance Development, Vertex Pharmaceuticals, introduced the presentation (Attachment B) about genetic therapies. He discussed different examples of gene abnormalities that can be treated by gene replacement therapy. He explained how different gene therapies work, including gene replacement therapy, gene editing, and in vivo and ex vivo modification. He gave a timeline of the past research on gene therapy and how it could progress in the future. He highlighted the challenges to patient access to this type of therapy, including regulatory barriers.