FDA Guidance Aims to Advance Targeted Gene Therapy Studies

People with Batten disease and other rare genetic disorders are set to benefit from new FDA guidance on developing and testing individualized drug products for patients who otherwise have limited treatment options.

The two draft guidance documents, released Tuesday, include recommendations for administering investigational drugs and the data that medical researchers should submit from single-subject, or N of 1, clinical studies. The guidance, once finalized, will help researchers construct trials to bring more of these gene therapies—which are designed to be unique to one or two patients—to the market.

“We are optimistic that the development of these individualized drug products ...