Drugs / NGN-401
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 3 | NCT05898620 | Jun 2023 → Dec 2029 expected | Rett syndrome | Neurogene Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2026-06-29 | Neurogene Inc. | Results Neurogene Reports Positive Long-term Clinical Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome neurogene.com ↗
Across 10 participants, 47 total developmental milestones were gained (average of 4.7 per participant), with both pediatric and adolescent/adult participants demonstrating clinical response and continued, progressive improvements through 30 months of follow-up, with no plateau or loss of milestones observed. |
| 2026-05-12 | Neurogene Inc. | Results Neurogene Highlights Data at ASGCT Meeting Supporting Therapeutic Rationale for ICV Delivery Method in CNS-targeted Gene Therapy neurogene.com ↗
Presentation adds to body of evidence that ICV administration results in broad biodistribution of gene therapy to the brain and nervous system |
| 2026-02-26 | Neurogene Inc. | Regulatory Neurogene Announces FDA Breakthrough Therapy Designation for NGN-401 Gene Therapy for Rett Syndrome neurogene.com ↗
Breakthrough Therapy designation granted based on interim NGN-401 Phase 1/2 data demonstrating clinically meaningful, durable and multidomain functional improvements |
All press releases naming this drug 5 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | NGN-401 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05898620 ↗ |
| Target | MECP2 | “NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.” NCT05898620 ↗ |