Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

Categories: Blog, Clinical Trials, Clinics, Family Empowerment, Homepage, News, Research

We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in 2023 in pediatric females. This is the second gene therapy clinical study in Rett syndrome currently underway and the very first gene therapy clinical trial for Rett starting in the U.S.


Neurogene garnered feedback from families in the Rett syndrome community and IRSF’s Clinical Trial Committee to design a clinical trial that the FDA found worthy of approval. We’re grateful to each family for participating in their survey and to our Clinical Trial Committee for evaluating Neurogene’s study to enhance its chance for success in Rett syndrome.

IRSF’s continued investment in our Center of Excellence network ensures that we have clinics throughout the country that are ready to fully engage in clinical trial opportunities. While no sites have been announced, IRSF is confident that there are many qualified sites available for the study. Study information will be available on IRSF’s myRett Trial Finder™.

2023 Starts Off Strong!

2023 is starting as a big year for Rett! This year we will hear the FDA’s decision on the possibility of the first approved treatment for Rett syndrome (trofinetide) and the launch of the first pediatric gene therapy trial for Rett in the U.S.

Through IRSF’s Rett Syndrome Center of Excellence network, Clinical Trial Committee, Rett Syndrome Registry™, and myRett Trial Finder, we create a smooth path for promising discoveries to travel from the lab to clinical trials to families. Our Foundation aggressively invests in groundbreaking science aimed at a cure for Rett, but we understand that discoveries need an effective clinical trial infrastructure. Thanks to IRSF donors, we have one to test this promising therapy!

It takes research, families, and industry working together to get us to our vision of a world without Rett. We remain ALL IN for families and a cure.