2nd UK Gene therapy trial site opens in Edinburgh

15/10/2024

Today, the second UK Rett syndrome gene therapy clinical trial site has officially opened. The clinical trial of Neurogene’s NGN-401 is now listed on the clinicaltrials.gov website here.

This second clinical trial site is at the Royal Hospital for Children and Young People in Edinburgh under Primary Investigator, Dr Jay Shetty, following the first Neurogene site in Manchester opening in September.

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

There are now four trial sites in the USA, two in the UK and one in Australia.

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 Royal Hospital for Children and Young People in Edinburgh directly here:
[email protected]

This news about these first UK gene therapy clinical trials 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 Edinburgh and 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. This could not have happened without you.

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.

Below: Dr Shetty on the critical role of Reverse Rett in supporting UK clinical trials for Rett syndrome.