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FDA Selects Neurogene Rett Program for START Pilot Program

Neurogene's Rett program was selected as one of three to participate in the FDA's START Pilot Program. NGN-401 was selected based on potential for clinical benefits and clinical development program readiness.

June 3, 2024
FDA Selects Neurogene Rett Program for START Pilot Program
FDA Selects Neurogene Rett Program for START Pilot Program

In the fall of last year the FDA announced a pilot program called START (Support for clinical Trials Advancing Rare disease Therapeutics) with a goal of testing the concept that frequent communication with the FDA speeds the development of rare disease gene therapy programs. The success of the START program provides the FDA with data to request additional resources to help advance more gene therapy programs.

The FDA has two divisions, CBER which oversees biologics programs like gene therapy and CDER which oversees drug programs. FDA invited applications from companies and shared that it would choose three programs submitted to CBER and three from CDER, to participate in START.

Today, Neurogene announced that its Rett gene therapy program, NGN-401, has been selected as one of the three CBER applications. This is terrific news for the Rett community as it means the program will benefit from more access to the FDA so Neurogene can move faster. Receiving timely and comprehensive feedback from FDA on an ad-hoc basis as opposed to waiting for formal meetings is a game changer. Importantly, it also raises the profile of Rett syndrome at CBER.

Peter Marks (FDA) and Andrew Mulberg (Neurogene)

At the global Rett Syndrome Genetic Medicines Summit hosted by RSRT last September, the director of the FDA’s CBER program, Dr. Peter Marks, attended to give a keynote, moderate panels, and learn more about advancements in Rett research. At the summit, Dr. Marks had an opportunity to hear from Neurogene’s Chief Scientific Officer Stuart Cobb and head of regulatory relations Andrew Mulberg as well as meet other Neurogene leadership team members. Dr. Marks also met a child with Rett syndrome, providing an always critical human element. Besides sharing the latest scientific data the Genetic Medicines Summit was important as it provided an opportunity to network and build relationships.

Thanks to the support of our donors, RSRT is able to fund innovative scientists like Stuart Cobb who went on to join Neurogene and advance a gene therapy program.

Monica Coenraads (RSRT) and Peter Marks (FDA)

We are in extraordinary times. Built on decades of basic research, much of it funded by RSRT, gene therapies for Rett are in the clinic. This advancement is coinciding with a remarkable phenomenon at CBER which is embracing gene therapies for rare disease in flexible and creative ways. Dr. Marks is a believer in genetic medicines and is a staunch supporter of rare disease patients.

We sincerely thank Dr. Marks and everyone at CBER for selecting the Neurogene gene therapy program for Rett syndrome and we congratulate the entire Neurogene team on this significant achievement.