Neurogene announcement: Key takeaways


On 5 January 2024, Neurogene announced a business update and 2024 outlook. This news included the announcement of MHRA clearance for a UK clinical trial of Neurogene’s NGN-401. We will share more information as soon as we are able. In the meantime, here are the key takeaways: 

  • The UK regulator, the MHRA has approved Neurogene’s clinical trial application for NGN-401 in children with Rett syndrome.  
  • This means that the company is able to expand the current clinical trial outside the USA to include children with Rett syndrome in the UK. This is part of a wider strategy to expand the clinical trial globally. 
  • The ongoing trial of NGN-401 currently specifies a cohort, or group of five patients who will each receive a lower dose of the gene therapy straight into the brain. 
  • In the first half of 2024, Neurogene plans to expand the trial to include more patients to go into this first group receiving the lower dose of the gene therapy and a higher dose cohort or group so that they can make sure that they are getting the best possible results out of the product whilst still ensuring that they have strong safety data to take to regulators.
  • Neurogene also announced that it has extended its research collaboration with the University of Edinburgh for an additional three years allowing them to continue to advance a number of products for different diseases using the same proprietary technology as they are using in NGN-401. 

2024 key milestones 

Expanding the ongoing phase 1/2 clinical trial for NGN-401 for Rett syndrome in the first half of 2024 to include more patients. 

Reporting of the interim data for NGN-401 for Rett syndrome in the last quarter of 2024. 

Did you know?

Through our longstanding partnership with the US based RSRT, Reverse Rett has provided a significant level of funding for Professor Stuart Cobb, now the Chief Scientific Officer of Neurogene, whose work led to the Neurogene NGN 401 program.  

The Cobb Lab alone has received over £1.4million from Reverse Rett over the last decade enabling this work to move from the laboratory bench to human clinical trials

We are delighted for Stuart and send our congratulations to him, all his colleagues at the University of Edinburgh and all the team at Neurogene!

In the relentless pursuit of life-changing treatments for Rett syndrome, it’s essential that we continue to contribute to research projects focused on developing better, less risky, more effective treatments for everyone with Rett syndrome. You can help by starting a fundraiser today. Email [email protected] to find out how you can help.