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NGN-401 · 1 trial · 1 indication
Responders will be defined as participants who: * Attain a CGI-I score of ≤ 3 ("minimally improved"); * and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters.
| Arm | Type | Description |
|---|---|---|
| Pediatric 1e15 vg dose (fully enrolled) | EXPERIMENTAL | Dose Level 1 for ages 4-10 years |
| Adolescent/Adult 1e15 vg Dose (fully enrolled) | EXPERIMENTAL | Dose Level 1 for ages 11 years \& above |
| Pediatric 3e15 vg dose (discontinued) | EXPERIMENTAL | Dose Level 2 for ages 4-10 years (discontinued) |
| Pivotal Cohort | EXPERIMENTAL | Dose Level 1 for ages 3 and above |
| Name | Type | Description |
|---|---|---|
| NGN-401 | GENETIC | NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene. |
Inclusion Criteria: * Females who are between the ages of ≥4 and ≤10 years for Arms 1 and 2 (Arms closed). Females who are ≥11 years of age or older for Arm 3 (Arm closed). Females who are ≥3 for Arm 4, the pivotal cohort. * Diagnosis of typical Rett syndrome with a documented disease-causing mutat...
NGN-401 is an investigational gene therapy being developed for the treatment of Rett syndrome in females. It is designed to address the underlying genetic cause of the condition. The therapy is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities.
NGN-401 is a gene therapy intended to deliver a functional copy of a gene to compensate for the genetic defect that causes Rett syndrome. The therapy is designed to provide the missing or faulty protein that is essential for normal neurological function, thereby targeting the root cause of the disease.
NGN-401 is being developed by Neurogene Inc., a biopharmaceutical company focused on rare neurological diseases. Neurogene is listed on the stock exchange under the ticker symbol NGNE. The company is conducting clinical trials to evaluate the safety and efficacy of NGN-401 in patients with Rett syndrome.
NGN-401 is currently in Phase 3 clinical development. It is an investigational therapy, meaning it has not yet received regulatory approval for commercial use. The Phase 3 trial is designed to further assess its safety and effectiveness in females with Rett syndrome.
NGN-401 is being studied in a Phase 3 clinical trial with the identifier NCT05898620. This trial is titled 'A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome' and is currently active but not recruiting. The study is enrolling 33 female participants aged 3 years and older across the United States, Australia, and the United Kingdom.
NGN-401 is not FDA approved. It is an investigational gene therapy currently in Phase 3 clinical trials. However, it has received Breakthrough Therapy designation from the FDA, which is intended to expedite the development and review of therapies that show promise for serious conditions like Rett syndrome.