Mapping endpoints: building gene therapy trials for the future

In 1990, when a four-year-old child received the first successful gene therapy for a rare immunodeficiency, gene therapies were heralded as the future of pharma research and development (R&D). Their development has since been marred by safety concerns, and only in the past decade have there been positive late-stage trials and even FDA approvals. Heading into 2023, the gene therapy field still has a long way to go to fulfill its promised potential.

Experts say the biggest challenge to designing successful gene therapy trials is selecting the right primary endpoint. In some cases, overly ambitious outcome measures can limit gene therapies that...