Gene Therapy for Neurodegenerative Diseases Guidance

FDA has published a draft guidance, Human Gene Therapy for Neurodegenerative Diseases, with recommendations for sponsors developing human gene therapy products for neurodegenerative diseases affecting adult and pediatric patients. The document defines neurogenerative diseases as a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system.

The guidance focuses on considerations for product development, preclinical testing, and clinical trial design. It also discusses marketing approval pathways for investigational gene therapy products.

The document covers Introduction; Considerations for Chemistry, Manufacturing, and Controls; Considerations for Preclinical Studies; Considerations for Clinical Trials; Expedited Programs; Communication with FDA; and References.