ICH guideline S12 on nonclinical biodistribution considerations for gene therapy products - Step 2b - Scientific guideline

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 guideline 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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