ASGCT Members Lead Congressional Briefing on Gene Editing

Rare diseases, by definition, affect a small percentage of the population. Collectively, however, they impact millions of individuals worldwide. These diseases often lack effective treatment options, leaving patients and their families grappling with the burdens of chronic illness and disability. New approaches like gene editing technologies offer the potential to address the root cause of rare diseases by precisely modifying the underlying genetic mutations responsible for the condition. By editing or repairing faulty genes, these therapies hold the promise of providing long-lasting or even functionally curative treatments for patients with rare diseases.

On April 4, ASGCT hosted a bicameral Congressional briefing on Gene Editing Technologies: Transforming Rare Disease Care. ASGCT members educated Congressional staff on the potential for gene-edited therapies to address unmet medical needs. Presenters focused on CRISPR technologies for this briefing, first explaining how the “molecular scissors” work in cells and the types of diseases that could be addressed. Presenters then educated on development considerations for gene-edited products and some of the key challenges in the regulatory and access landscape. The briefing finally turned to the clinical trials process and a deep dive into why these technologies can be an important tool for the rare disease patient community and unique considerations for patients considering a gene editing clinical trial.

Download the Full Slide Deck

Much of the conversation centered around the patient experience and associated access challenges. Trial enrollment benchmarks, geographic locations of Centers of Excellence, determining relevant endpoints, and patient perception were all discussed as challenges specific to gene-edited modalities. Speakers spanned academia and industry:

• Jeffrey Chamberlain, PhD, University of Washington
• Fyodor Urnov, PhD, University of California Berkley
• Janice Chen, PhD, Mammoth Biosciences
• Matthew Porteus, MD, PhD, Standford University Medical School
• Amy Raymond, PhD, Worldwide Clinical Trials

The landscape of gene editing therapies is rapidly evolving. With the FDA's approval of a gene-edited therapy for sickle cell disease and an exponential pipeline of therapies in development, ASGCT is fulfilling a critical need to inform decision-makers and legislators about the transformative potential of gene editing.

Watch the Full Briefing Below

 [C1]Link to recording when available

Does it make more sense to have this in-line link be the deck, and then the big shiny button the full recording? Or have them both be the recording, since I assume that's really where we want to drive people (rather than static slides with no view count) [CM2]

 [CM3]GE briefing combined slides 4.4.24.pptx