Skip to main content

Neurogene Doses First Two Patients in Pediatric US Trial

November 30, 2023
Neurogene doses first two children in gene therapy trial

Today Neurogene announced via press release that it has sequentially dosed the first two patients in its US pediatric trial with the gene therapy product, NGN-401. The company reports that the gene therapy has been well tolerated in the first two children.

The trial is administering a full length MECP2 gene using a one-time delivery into the ventricles of the brain in an effort to maximize the number of brain cells receiving the gene therapy. NGN-401 employs EXACT, a proprietary technology that controls the amount of MECP2 protein made by the gene. The age range for the trial is 4 to 10 years old and is expected to enroll a total of 5 children. It's being conducted at Rett centers in Houston, Boston and Denver with Houston the only site recruiting thus far.

Neurogene plans to report on clinical data in the fourth quarter of 2024.

This trial is the result of many years of research conducted by Stuart Cobb, PhD, from the University of Edinburgh and funded, in large part, by RSRT. Dr. Cobb is the Chief Scientific Officer of Neurogene. We thank all of our donors who support this critical work.

Congratulations to everyone at Neurogene on this milestone! Enormous gratitude to the courageous families in the trial. The Rett community is holding your daughters close!