We at Girl Power 2 Cure and Rett University are pleased to share this exciting news!

Today, the Rett Syndrome Research Trust shared Neurogene’s announcement that its Investigational New Drug (IND) application for a gene replacement clinical trial for Rett syndrome has been accepted by the FDA.

From Monica Coenraad’s RSRT Announcement:

“… Since RSRT was launched in 2008, I’ve shared with you numerous advancements on our road to reversing Rett and changing the lives of 350,000 afflicted children and adults. It would be difficult to rank the significance of these advancements because each advancement is the result of others that preceded it. However, this is perhaps one of the most important announcements we’ve ever made. The Neurogene gene replacement program, an approach championed by RSRT for well over a decade, targets Rett at its genetic core and aims to deliver a full-length healthy MECP2 gene to the brain…” 

And from Neurogene:

“Today we are excited to share the news that the FDA has cleared Neurogene’s Investigational New Drug (IND) application for NGN‐401 for the treatment of children with Rett syndrome. NGN‐401 is an investigational adeno‐associated virus (AAV) gene therapy candidate to be administered to pediatric patients that uses Neurogene’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology. The full press release issued today may be found at .”

Read Monica’s full Blog here:

Read Neurogene’s announcement here:


Many years of hard work, and many, many contributions from the Rett Syndrome community are now coming to fruition in the form of true potential cures for Rett Syndrome. We are so thankful to RSRT, Neurogene, and the entire research community for the work they do.

And thank you – our GP2C/Rett University community – for all of your dedicated support over these many years… Let’s keep the momentum going!

Regards, Kevin