By Daniel Eisenman, Director of Biosafety Services
The gene therapy field is experiencing explosive growth in today’s competitive research environment. Gene therapy entails the transfer of engineered genetic materials to human research subjects. These studies were previously considered to be highly experimental and limited to early phase trials at a handful of highly specialized academic medical centers. However, well-established safety profiles, promising research results, and the FDA issuing the first gene therapy approvals in recent years have led to dramatic growth.
Searching clinicaltrials.gov for gene therapy studies results in 4070 hits with 1067 studies currently recruiting or enrolling research subjects. As of September 2019, 295 phase III studies are listed, representing a growing pipeline of gene therapy products are preparing to undergo consideration for FDA approval.
This blog summarizes the growth, risks, and regulatory requirements for gene therapy research.
- Risks and regulatory requirements
- Efficiencies from a centralized review