The FDA has released a draft guidance, Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing (June 2026), with comments due by September 1, 2026.
This draft guidance discusses the use of public and platform knowledge to support the development of gene therapy products that incorporate ex vivo and in vivo genome editing, with the goal of improving review efficiency and accelerating development.
Topics of interest include:
• Leveraging prior knowledge across CMC, including shared editing nucleases, delivery systems, vectors, and manufacturing processes
• Applying existing nonclinical data to support genome-edited products
• Utilizing prior clinical experience and platform knowledge across programs
• Platform-based development and regulatory review efficiency
We are seeking volunteers to review the draft guidance and participate in the AAPS response team.
Guidance document:
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/leveraging-prior-knowledge-development-human-gene-therapy-products-incorporating-genome-editing
If you are interested in participating, please contact Joseph Thomas ([email protected]), Kim Huynh-Ba, or me directly.
Thank you!
------------------------------
Subathra Ramamoorthy
Associate Principal Scientist
Veranova
Westford MA
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
------------------------------