Two UK gene therapy trials for children with Rett syndrome cleared by UK regulator


by Rachael Stevenson

Last week, gene therapy companies Neurogene Inc and Taysha Gene Therapies have announced that they have received clearance from the UK regulator, the MHRA to start gene therapy trials for children with Rett syndrome right here in the UK. 

This is tremendous news for the UK Rett community and progress which has been hard wrought for Reverse Rett. This is a moment for us to send you, our supporters, our deepest appreciation.  

Without you, we could not have funded the research which led to the development of both these programs. Neither could we have started the Rett Registry UK, facilitated the first ever UK drug trial in 2017 and developed Clinical Trial Support Services for the following 3 drug trials, demonstrating UK capacity for the more complex trials which lay ahead. 

Without you, we wouldn’t now be working to reach patients with Rett who may be undiagnosed so that they too can benefit from emerging treatments. We wouldn’t be funding the CIPP Rett Centre who care for more than 200 Rett patients of all ages from all around the UK. We wouldn’t be working now to ensure that when the time comes for UK payers to determine if these potentially disease-modifying treatments should be funded by the NHS and for whom. 

So thank you, thank you for all your support whether you have been supporting Reverse Rett since we launched in 2010, or whether Rett syndrome has only just landed on your radar. 

Thank you, whether you are a militant and repetitive fundraiser or someone who has donated once but kindly stayed on our mailing list.  

However you have helped us, thank you.  

Here are the results of your support: 


Neurogene announcement: Reverse Rett key takeaways


Taysha announcement: Reverse Rett key takeaways


Find out more about current Rett gene therapy programs by watching our most recent Reverse Rett Bitesize Emerging Gene Therapies sessions below.