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Bespoke Gene Therapy ‘Playbook’ Outlines Platform Approach To AAV-Based Treatments

 
• By Grace Moser

Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

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The Foundation for the National Institutes of Health’s Bespoke Gene Therapy Consortium is offering a regulatory guidebook to help investigators navigate the investigational new drug application submission process via a newly published “Regulatory Playbook.”

The playbook, published on 6 February, is the first output of the FNIH-led initiative to support “n of 1” gene therapy development. The public-private partnership launched in 2021 with US Food & Drug Administration Center for Biologics Evaluation & Research Director Peter Marks as a key champion

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