International – ICH adopts S12 guideline for gene therapies

The International Council for Harmonisation (ICH) last week announced that its S12 guideline on nonclinical biodistribution (BD) considerations for gene therapy products has reached Step 4 of the ICH process, meaning it’s ready for regulators to adopt in their jurisdictions.

The guideline provides considerations for the design, timing and conduct of preclinical BD studies, and offers various recommendations for sponsors on the animal species or model to be used, the group size and sex of study animals, route of administration and dose selection, as well as sample collection.

Specific considerations are provided for assay methodology, measurement of expression, immunological considerations, ex vivo genetically modified cells, the assessment of biodistribution in gonadal tissues, situations where additional BD studies might be required and alternative approaches.

“Characterisation of the BD profile following administration of a [gene therapy] GT product in animals is a critical component of a nonclinical development programme. The nonclinical BD data contribute to the overall interpretation of the study findings by enabling a better understanding of the relationship of various findings (desired and undesired) to the administered GT product,” ICH writes…