NGN-401

N/A

Report date: 2026/02/28 12:01:13

Base Information

Drug NameNGN-401

Drug Alias NameRecombinant 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

ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector

Mechanisms of ActionMethyl-CpG-binding protein 2 gene replacement

TargetsMethyl-CpG-binding protein 2

Therapy Area/IndicationsRett Syndrome

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryUnited StatesAustraliaUnited Kingdom

CombinationsNo

Global Highest StatusPhase 3 Clinical

Highest Status in ChinaN/A

Originator CompaniesNeurogene Inc

China Registration ClassN/A

Develop Information

Active CompaniesNeurogene Inc

Inactive CompaniesN/A

Active IndicationsRett Syndrome

Inactive IndicationsN/A

Other Information

ATC CodeN/A