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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...
Top 2 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Vadadustat |
| Rigel Pharmaceuticals, Inc. | RIGL | 1 | PHASE2 | Fostamatinib |
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 that targets the MECP2 gene. Rett Syndrome is caused by mutations in this gene, and the therapy aims to deliver a functional copy of MECP2 to cells. This approach is intended to compensate for the defective gene and potentially alleviate disease symptoms.
NGN-401 is being developed by Neurogene Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol NGNE. The company is focused on developing treatments for rare neurological diseases and is advancing NGN-401 through clinical trials for Rett Syndrome.
NGN-401 is currently in Phase 3 clinical development. It is an investigational therapy, meaning it has not yet received FDA approval. The drug has been granted Breakthrough Therapy designation by the FDA, which is intended to expedite the development and review of therapies for serious conditions.
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 participants. The study is enrolling 33 female patients aged 3 years and older.
NGN-401 is a distinct gene therapy candidate developed by Neurogene Inc. It is designed to deliver the MECP2 gene and is currently in Phase 3 trials. While other gene therapies for Rett Syndrome may exist, NGN-401 is a unique investigational product with its own clinical trial program.