FDA’s Operation Warp Speed for Rare Diseases: First Five Participants Announced

1. START Program Launch: The FDA launched the Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program to accelerate rare disease drug development by leveraging lessons from Operation Warp Speed.
2. First Participants: The first five participants selected for the START program include Denali Therapeutics, Neurogene, Larimar Therapeutics, and Grace Sciences, which are developing gene therapies for various rare neurodegenerative conditions.
3. Eligibility Criteria: To be eligible, companies must have products in clinical trials under an active Investigational New Drug (IND) application, focusing on rare neurodegenerative diseases or gene/cellular therapies for conditions likely to cause significant disability or death within the first decade of life.
4. Program Goals: The START program aims to facilitate more efficient development of rare disease therapies by providing frequent engagement with FDA staff, offering guidance on study design, patient selection, and product characterization.
5. Future Expansion: The FDA plans to evaluate the initial pilot cohort and may consider a second iteration of the program with additional participants.