First UK Rett syndrome gene therapy trial site open 

Today, the first UK Rett syndrome gene therapy clinical trial site has officially opened. The clinical trial of Neurogene’s NGN-401 is listed on the clinicaltrials.gov website. The addition of the UK clinical trial site today marks the formal initiation of the UK clinical trial.

This first clinical trial site is at the Royal Manchester Children’s Hospital under Primary Investigator, Dr Aimee Donald and Professor Siddharth Banka. 

This clinical trial is part of a larger international clinical trial of the Neurogene gene therapy NGN-401.

There are three trial sites in the USA and one in Australia as well. 

The age group for the clinical trial is currently 4>10 (over four years old and before the child’s 11th birthday).

The number of patients needed for the trial worldwide is currently 16.  

The number of patients needed in the UK at this time is therefore likely to be very small. 

At the moment, the hospital are recruiting from their own list of patients.  

However, as the number of patients needed may increase, it is likely that doctors involved with this and other UK trials will request patients from the Rett Registry UK which you can sign up to here  

You also can contact the Manchester Children’s Hospital directly here:
[email protected]

This news about the first UK gene therapy clinical trial for Rett syndrome has been a long time coming for the UK Rett community. 

At Reverse Rett, we are grateful to everyone who has supported our work to: 

• Fund the work of Professor Stuart Cobb at the Universities of Glasgow and Edinburgh, which led to this program 

• Develop the Rett Registry UK demonstrating UK infrastructure for successful UK clinical trials 

• Implement clinical trial support services at trial sites across the UK including Manchester in order to develop clinical expertise and experience in Rett syndrome  

• Demonstrate the burden of disease and advocate for the need for treatments with UK regulators  

Without all this work and more, we would not be where we are today. Our infinite thanks goes to all our supporters, donors and fundraisers.

More information about the study including inclusion and exclusion criteria can be found here

Learn more about gene therapy trials for Rett syndrome on the Reverse Rett YouTube channel here.