New Rett gene therapy program announced by Neurogene


On May 18th 2022, Neurogene announced NGN-401, a new gene therapy candidate for Rett Syndrome. This work has been developed in collaboration with the University of Edinburgh and is led by none other than Dr Stuart Cobb, Ph.D., who is now Chief Scientific Officer at Neurogene.  

With your help, Reverse Rett has financially supported the work of both the Cobb and Bird labs at the University of Edinburgh for more than a decade via the RSRT MECP2 and Gene Therapy Consortia. 

We are delighted to see this work come to fruition in the form of a commercial product at Neurogene and look forward to further progress towards human trials in due course. Congratulations to all involved!

Read Neurogene’s letter to the Rett community here

Read Neurogene’s press release here

Read a blog post from RSRT’s Monica Coenraads here

Read RSRT’s press release here

About Neurogene 

Neurogene is a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, turning them into treatable conditions and improving the lives of patients and families affected by rare diseases. 

Neurogene can be contacted at: 

Patients and families 

Healthcare providers