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FDA Approval to Commence Studies in Gene Therapy for CLN5 Disease

16 September 2021

We are excited to hear the announcement overnight that Neurogene’s investigational gene therapy NGN-101 for the treatment of CLN5 Batten disease has received investigational new drug (IND) approval from the FDA.
IND clearance now enables Neurogene to initiate a Phase 1/2 trial which will assess the safety, tolerability, and efficacy of NGN-101 in CLN5 patients. They expect to initiate the study in the first half of 2022.
“FDA clearance of our first IND is an incredibly significant milestone for Neurogene, and moves us one step closer to delivering on our mission to bring transformative treatments to patients living with rare neurologic diseases,” said Rachel McMinn, Ph.D., Founder and CEO of Neurogene.