Neurogene completes registration for their EMBOLDEN trial 

Neurogene announced yesterday that they have recruited and enrolled all patients in their EMBOLDEN phase ½ trial, and dosed half of them.  

They say gene therapy, NGN-401, has been well tolerated, and that they will share more data later this year.  

What is the EMBOLDEN trial? 

The EMBOLDEN clinical trial tests NGN-401 gene therapy on patients with Rett. The patients are divided into two cohorts; 3 to 11 (pediatric) and 11 and up (adolescent/adult).   

What happens next?  

• Neurogene expects to complete dosing all patients in the EMBOLDEN trial in the second quarter of this year.  

• They will present more data on the safety and efficacy of the pediatric and adolescent/adult cohort in mid-2026, which will show how the drug has been tolerated after a year.  

A message from us:  

 
Whilst NGN-401 is still in clinical trials, this is another positive step. It shows the programme is moving forward and that treatments are getting closer.  

For families, what matters most is Momentum. 

Every step that moves a therapy closer to clinical trials and through them brings us closer to treatments reaching those who need them. 

At Reverse Rett, our focus remains clear: 

• It does not matter where trials take place 

• It matters that they happen quickly, safely, and effectively 

• And that they lead to real-world access for as many people as possible 

You can read the full update here