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.