Understanding Potency In Gene Therapy Development

By Understanding Potency in Gene Therapy Development

Explore the importance of understanding potency in cell and gene therapy (CGT) development and the challenges in developing potency assays for CGT products. The FDA requires all approved biological products to undergo in vivo and/or in vitro tests to measure potency. The article discusses the necessity of measuring transgene expression levels in transduced cells throughout product development and at lot release. It also emphasizes the importance of precise and accurate in vitro assays in the development of treatments, stating that early and rigorous potency testing can lower failure rates and lead to more cost-effective treatments. Compliance with potency tests, FDA regulatory frameworks, and standard operating procedures can ensure product efficacy, purity, and safety.