Neurogene Announces Interim Results from Gene Therapy Clinical Trial

Today Neurogene hosted a webcast to share preliminary results on their pediatric clinical trial of NGN-401 gene therapy for Rett syndrome. Here are some highlights. Please note the links below.

Safety results on 5 patients who received low dose gene therapy and 2 patients who received high dose.  

• No unexpected safety concerns

• Known side effects of gene therapy (eg. liver function labs) resolved with steroid treatment

• No signs of MECP2 overexpression suggests EXACT regulatory control is working as expected

• No problems with Intracerebroventricular injection (ICV) procedure (direct injection into the ventricle of the brain)

During the webcast Neurogene disclosed an emerging treatment-related serious adverse event consistent with known risks of AAV gene therapy in the third high-dose participant who was recently dosed. This patient was recently treated and was not included in the safety or efficacy analysis presented today.

Efficacy results on 4 patients who received low dose gene therapy (the 5th patient was dosed 1 month ago and was not included).

• All patients gained skills
• No signs that improvements are plateauing
• First ever example of children with Rett gaining skills they would not be expected to gain when compared against the natural history of the disease
• It is not surprising that learning skills takes time as we would not expect the children to gain skills any faster than a typically developing child

Neurogene is adding a pilot adult trial with 3 patients receiving high dose.

It is our hope and expectation that greater improvement will occur with time.
 

The Neurogene program began and was incubated at RSRT and we are delighted to see it advance. We are grateful to all the families and their networks who fundraised and made it possible for this work to flourish.

We extend our gratitude to the brave families and children who are participating in the trial. We thank everyone at Neurogene for their dedication and passion and congratulate them on this initial success.  Upward and onward!

NOTE: We will add the webcast recording link below as soon as it's made available.