IPRP Details Expectations for Biodistribution Assessments for Gene Therapies
The newly formed International Pharmaceutical Regulators Programme (IPRP) has released a reflection paper laying out expectations for non-clinical biodistribution (BD) assessments for gene therapies (GT).
The reflection paper was developed by the international body's Gene Therapy Working Group (GTWG) in April and approved by the IPRP Management Committee at its first-ever meeting last month.
BD assessments are conducted to determine the distribution, persistence and clearance of gene therapy vectors in the body, which are critical factors for understanding the effects and safety of a gene therapy product.
"The BD profile should be determined for a GT product that has not been previously administered to humans and is proposed for a first-in-human (FIH) clinical trial," IPRP writes, noting that BD studies may be conducted at later stages of development under limited circumstances.
Specifically, the eight-page reflection paper provides considerations for BD studies ahead of FIH clinical trials; recommendations for the design of BD studies; BD assay methods; special considerations for modified vector types; and a discussion of circumstances that would trigger a need for additional BD studies.
According to the paper, the principles discussed in the reflection paper are relevant to various gene therapy products but are not applicable to genetically modified cells.