Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial

Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial

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

We’re excited to share that today Neurogene announced the locations and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. Enrollment at Texas Children’s Hospital begins today, with enrollment at Children’s Hospital Colorado and Boston Children’s Hospital anticipated to open soon. The trial will enroll five (5) females aged 4-10.


First shared with the research community at last week’s IRSF Rett Syndrome Scientific Meeting, we are excited to now share this news with you and our community around the world. These locations are all IRSF-designated Centers of Excellence for both their commitment to best-in-class care and involvement in clinical trials that will continue to deliver treatments including cures. Neurogene also garnered feedback on the design of this clinical trial from IRSF’s Clinical Trial Committee and from families in the Rett syndrome community to enhance this study’s chance for success and approval with the FDA.

We are grateful to the clinicians at these trial sites, and to the families who will participate in this study. It takes research, families, and industry working together to get us to our vision of a world without Rett. Because of you, we are that much closer.  We remain ALL IN for families and a cure.

For more information on this clinical trial and how to enroll in, CLICK HERE to visit the listing on

We are with you every step of the way. Questions? Email us at [email protected].