ICH S12 Guideline on Nonclinical Biodistribution Considerations for Gene Therapy Products

20 April 2023 – the EMA published step 5 of the ICH S12 guideline.

The objective of this guideline is to provide harmonized 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 program and the design of clinical trials are also provided. The recommendations in this guideline endeavor 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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