Drug Type AAV based gene therapy |
Synonyms Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2), NGN-401 |
Target |
Action stimulants |
Mechanism MECP2 stimulators(Methyl-CpG-binding protein 2 stimulants), Gene transference(Gene transference) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePhase 3 |
First Approval Date- |
RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Regenerative Medicine Advanced Therapy (United States), Advanced Therapy Medicinal Products (European Union), Rare Pediatric Disease (United States), Innovative Licensing and Access Pathway (United Kingdom) |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Rett Syndrome | Phase 3 | United States | 13 Jun 2023 | |
| Rett Syndrome | Phase 3 | Australia | 13 Jun 2023 | |
| Rett Syndrome | Phase 3 | United Kingdom | 13 Jun 2023 |
NCT05898620 (Biospace) Manual | Phase 1/2 | 3 | btkbqnfwfs(xbtciclujm) = All adverse events (AEs) related to NGN-401 have been mild, or Grade 1, and transient or resolving. etfqhelwmj (chczgzxply ) View more | Positive | 07 May 2024 | ||
NCT05898620 (ASGCT2024) Manual | Phase 1/2 | 3 | qzzbjnckxu(tawmprzmoc) = wcfnmxmzeq xwkcoafqbo (miyloutlrx ) | Positive | 07 May 2024 |





