USA – FDA offers first thoughts on neurodegenerative disease gene therapies

The US Food and Drug Administration (FDA) has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released on 5 January 2021, also highlights approval pathways for these novel products.

The draft guidance, which applies to products for both adult and pediatric populations, emphasizes the importance of early communication with FDA before the submission of an investigational new drug (IND) application. The agency pointed to INTERACT meetings, which can be used to discuss issues in a product’s early preclinical program, and pre-IND meetings, which occur later in development but prior to the submission of an application…