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BIO Comments on ICH Draft Guidance S12 Nonclinical Biodistribution Considerations for Gene Therapy Products

November 8, 2021

On Monday, November 8th, BIO submitted comments to the FDA’s draft guidance on Nonclinical Biodistribution Considerations for Gene Therapy Products. In the comment letter, BIO expressed appreciation for the Agency’s decision to put forth a draft guidance that was developed under the auspices of the International Council for Harmonisation (ICH) so that requirements for biodistribution studies are harmonized across regions. BIO further emphasized the importance of global convergence in regard to gene therapy products as most products in development focus on small patient populations. Amongst the constructive feedback provided, it was advised that risk-based approaches be used when designing non-clinical biodistribution studies for gene therapy products. Additionally, BIO recommended the scope of the draft ICH S12 Guidance be modified so that it’s clear that in vivo GT products are the focus; thus, other types of gene therapies, particularly ex vivo genetically modified cells, should be removed from the scope. The comment letter also included a number of specific line edits ranging from requests for broader language to be used in certain sections of the Guidance so as to increase flexibility in application, to requests for further clarification on the circumstances that the various provisions contained in the Guidance would apply to.

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BIO Comments on ICH Draft Guidance S12 Nonclinical Biodistribution Considerations for Gene Therapy Products
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