Neurogene Fourth Quarter 2024, Recent Highlights and Anticipated Milestones
On March 24, 2025 Neurogene provided an investor update which included some highlights on their Rett syndrome gene therapy program. Find our key takeaways below.
Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene said:
“We believe 2024 was a year of significant progress for our NGN-401 Phase 1/2 trial in Rett syndrome, as we reported encouraging interim efficacy data and NGN-401 was generally well-tolerated at the 1E15 vg dose in a pediatric cohort. We remain on track to provide an update on the registrational trial plans in the first half of 2025, and as we look ahead to the second half of 2025, we plan to share additional interim clinical data from the Phase 1/2 trial, including from our 11 years and older cohort.”
Our key takeaways:
- The Neurogene Rett syndrome gene therapy clinical trial (NGN-401) will continue at the lower dose (1E15vg) for all participants.
- The company has expanded the age range in the trial to include patients over the age of 11. *
- Neurogene has received Priority Medicines (PRIME) designation by the European Medicines Agency (EMA).
- Neurogene remains on track to provide a regulatory update on registrational** trial plans in the first half of 2025.
- Neurogene expects to report additional interim clinical data from the Phase 1/2 trial in the second half of 2025, including additional participants expected to be dosed during the first half of the year.
Read the full press release here
*Clinical trial eligibility:
The numbers needed for the Neurogene clinical trial worldwide remain very small at this time.
- The age range in the trial now including individuals over 11 years in the older pilot cohort (n=3). There is no upper age limit.
- The pilot cohort is in addition to the 4 to 10 years cohort (n=8). The company have shared publicly that 5 of those 8 participants have been dosed.
**What does ‘registrational trial’ mean?
This refers to the next phase of the clinical trial which will be subject to review by the regulators (FDA, MHRA etc.,) before the trial can move forward or be expanded.
Clinical trial sites:
Neurogene has clinical trial sites in the U.S., U.K. and Australia. Please see the current list of UK enrolling sites below:
- Royal Hospital for Children and Young People, Edinburgh: [email protected]
- Manchester University NHS Foundation Trust, Manchester: [email protected]
If additional sites are added, site contact information will be found here
Interested families should directly contact a clinical trial site that is currently enrolling.
To register your interest in this or future UK clinical trials for Rett syndrome, please also register your loved one on the Rett Registry UK here.
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