Potency Development For Cell & Gene Therapy Products

By Deb Bhattacharya, Ph.D. Vice President, Analytical CMC

This document outlines the development of potency assays for cell and gene therapy (CGT) products, with a focus on investigational therapies for Huntington's disease (HD). Potency assays are crucial for assessing biological activity and ensuring regulatory compliance, including lot release and stability evaluations.

The document highlights a phased approach for potency development, from early clinical stages to commercial filing. Huntington's disease, caused by mutations in the HTT gene, leads to neurotoxicity and neuronal loss, primarily in the striatum. LETI-101, an investigational gene therapy for HD, utilizes AAV encoding guide RNA to induce double-stranded breaks in the HTT gene, leading to the degradation of the mutant protein.

The potency development strategy involves measuring editing potency using ddPCR-based assays, with a focus on identifying suitable cell lines for transduction and INDEL detection. Potency assays confirmed transduction efficiency and validated the ddPCR method for AAV-delivered gene editing therapy. To learn more, access the full presentation below.