Statement on FDA Draft Guidance to Leverage Prior Knowledge for Gene Therapy Development

This week, the FDA released draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. The Institute for Gene Therapies (IGT) Chairman Congressman Erik Paulsen issued the following statement:  

“This is a welcome and practical step forward for patients living with rare and serious diseases who cannot afford to wait. By showing developers how to build on existing CMC, nonclinical, and clinical data – rather than starting from scratch with every new product – FDA is helping to lower a significant barrier that has historically slowed access to potentially life-changing treatments.

"We applaud CBER's recognition that reducing redundant testing does not mean reducing standards. Raising collective efficiency while maintaining rigorous scientific review is exactly the kind of regulatory modernization that the gene therapy field needs – and that patients deserve. This guidance, alongside the Plausible Mechanism Framework and the recently issued guidance on next-generation sequencing safety standards, reflects a coherent and science-driven approach to supporting cell and gene therapies.

"We encourage the FDA to finalize this guidance swiftly and to continue engaging the gene therapy community throughout the comment period. Regulatory frameworks must keep pace with the science, and IGT stands ready to support every effort to ensure that these innovations reach the patients and families counting on them."