Neurogene press release: NGN-401 Gene Therapy for Rett Syndrome Selected by FDA for START Pilot Program

Purple diamond logo with a sapling growing out of the tip

On June 3rd, Neurogene announced that the FDA has chosen their Rett syndrome gene therapy program NGN-401 for their START Pilot program. 

The START program was recently developed by the FDA to speed the development of rare disease therapies.  

Sponsors whose products are in the START program will be able to obtain frequent advice and regular ad hoc communication with FDA staff to help them advance their program as quickly as possible. 

Neurogene’s NGN-401 program was chosen as one of three. The product had to be a gene or cell therapy intended to address an unmet medical need for a rare disease or serious condition which is likely to lead to significant disability or death within the first decade of life. 

Sponsor participation in the program will be considered finished when the program has reached a significant regulatory milestone, such as initiation of the pivotal clinical study stage or reaching the stage before submitting a marketing application.  

At Reverse Rett, we are delighted by this news which demonstrates both the commitment of the FDA in advancing potentially life changing treatments for Rett syndrome and calibre of the Neurogene Rett syndrome program, NGN-401. 

We thank all our supporters who fundraised intensively for many years to support the work of Professor Stuart Cobb, via the US based Rett Syndrome Research Trust.  

In 2023, the Rett Syndrome Research Trust held a scientific meeting which included Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research (CBER) at the FDA. You can read more detail about the CBER program as well as this meeting, on the link below.  

We encourage you to watch the video below of Neurogene’s CEO, Rachel McMinn, talking about the benefits of these meetings and Neurogene’s imperative to bring forth a therapy that can dramatically improve the disease presentation for people living with Rett syndrome today. 


START stands for Support for clinical Trials Advancing Rare Disease Therapeutics. 

CBER stands for Center for Biologics Evaluation and Research 

FDA Food and Drug Administration (US based regulator)


Neurogene’s press release

Link to RSRT announcement re CBER START for NGN-401 

Video of Rachel McMinn at RSRT Scientific meeting