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GUIDANCE DOCUMENT

S12 NONCLINICAL BIODISTRIBUTION CONSIDERATIONS FOR GENE THERAPY PRODUCTS Draft Guidance for Industry September 2021

Download the Draft Guidance Document Read the Federal Register Notice
Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

Docket Number:
FDA-2021-D-0875
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research
Center for Biologics Evaluation and Research

The objective of this guideline is to provide harmonised recommendations for the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products. This document provides recommendations for the overall design of nonclinical BD assessments. Considerations for interpretation and application of the BD data to support a nonclinical development programme and the design of clinical trials are also provided. The recommendations in this guideline endeavour to facilitate the development of GT products while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles.

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All written comments should be identified with this document's docket number: FDA-2021-D-0875.

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