Overcoming Barriers to Gene Therapy: ASGCT's Outlook on the NCATS Strategic Vision

The American Society of Gene and Cell Therapy (ASGCT) recently submitted comments to the National Center for Advancing Translational Sciences (NCATS) on their draft strategic plan for 2024-2029. As the leading professional organization representing scientists, physicians, patient advocates, and others in the cell and gene therapy field, ASGCT provided perspectives on how NCATS can help accelerate the translation of these transformative therapies to patients in need. 

In the response, ASGCT commended NCATS for addressing crucial challenges with the unique nature of cell and gene therapy development and commercialization. This includes tackling the limited number of diseases with approved therapies, increasing diversity and inclusion in translational science, and addressing operational inefficiencies slowing therapeutic development. 

Key Points from ASGCT's Response: 

• Platform Technologies: ASGCT supports NCATS' work on platform technologies that could streamline therapy development across multiple products. The Society encouraged collaboration with the FDA on the new Platform Technology Designation program. 

• Natural History Data: Greater flexibility is needed in utilizing natural history and real-world data, which are critical for small patient populations common with rare diseases targeted by gene therapies. 

• Patient Engagement: ASGCT emphasized the importance of engaging diverse patient communities, overcoming historic mistrust, and facilitating early diagnosis through expanded genetic screening and education. 

• Manufacturing: New innovations in advanced manufacturing technologies will be vital for meeting anticipated patient demand as more therapies are approved. ASGCT advised coordinating with the FDA's new Advanced Manufacturing Technology program. 

The comments also highlighted ASGCT's expertise in developing patient education resources on gene therapies. The Society's Patient Education Program exemplifies successful multi-stakeholder collaboration for creating reliable, accessible content tailored to patient needs. 

ASGCT is committed to promoting translational science breakthroughs that transform the development and accessibility of curative cell and gene therapies. The Society's full response provides additional insights on key priorities and areas for continued partnership between ASGCT, NCATS, FDA and others across HHS. 

By proactively engaging with initiatives like the NCATS strategic plan, ASGCT aims to ensure the patient voice remains central to efforts overcoming scientific hurdles and facilitating broad access to safe, effective cell and gene therapies.  

Caitlin is ASGCT's senior advocacy program specialist.

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