Consensus Paper Calls for Regulatory Framework Development in Genotoxicity Analysis of Gene Therapy Vectors

In March 2023, the NC3Rs and UKEMS hosted a groundbreaking meeting, originally conceived by TestAVec and Novartis, of top experts in gene therapy carcinogenicity to tackle the pressing issue of vector genotoxicity. The gathering featured leaders from academia, industry, and regulatory bodies, including TestAVec's CSO, Professor Michael Themis.

During this meeting, the urgent need to deepen our understanding of vector-related genotoxicity and its carcinogenic potential was agreed. The delegates achieved a consensus on defining genotoxicity, addressing key uncertainties, identifying various endpoints for genotoxic assessment and outlining future research priorities.

This comprehensive report aims to shape the evolution of regulatory guidelines, emphasising the need for robust non-clinical toxicological assessments of gene and cell therapy products. As the field advances, these insights will be instrumental in ensuring the safety and efficacy of innovative therapies.

This paper has been published in Human Gene Therapy, in July 2024.

Improving the assessment of risk factors relevant to potential carcinogenicity of gene therapies: a consensus paper | Human Gene Therapy (liebertpub.com)