Pursuing The Promise Of Gene Therapy: Pathways To Successful Clinical Trials

By Jessica Merryfield

The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. To bring these treatments to market, sponsors are tasked with conducting clinical trials that generate robust evidence with appropriate safety oversight. In addition, gene therapy trials are faced with the myriad logistical hurdles of finding the right patients, pairing them with qualified sites, and ensuring that they understand and can fulfill the study protocol requirements.

In this first part of a three-part blog series on gene therapy development, we explore the emerging landscape of gene therapy and discuss high-level considerations for studies of these advanced therapeutics.