Designing Robust Gene Therapy Studies

By Jessica Merryfield

To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients, pairing them with qualified sites, providing informed consent, and confirming they are capable of — and committed to — fulfilling all protocol requirements.

In this second part of a three-part blog series on gene therapy development, we explore the key considerations for designing gene therapy trials.