methylmalonic acidemia gene therapy (NIH)

N/A

Report date: 2025/07/29 20:00:11

Base Information

Drug Namemethylmalonic acidemia gene therapy (NIH)
Drug Alias NameAAV8-Alb-A2.synMUTAAV8-EF1alpha-MMUT-HPRErAAV8-CBA-MUTAAV8-hAAT-MMUT
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionMethylmalonyl-CoA Mutase gene transference
TargetsMethylmalonyl-CoA Mutase
Therapy Area/IndicationsMethylmalonic acidaemiaalpha 1-Antitrypsin Deficiency
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPhase 1 Clinical
Highest Status in ChinaN/A
Originator CompaniesNational Institutes Of Health
China Registration ClassN/A

Develop Information

Active CompaniesWeill Medical College of Cornell University
Inactive CompaniesNational Institutes Of HealthUniversity Of Pennsylvania
Active Indicationsalpha 1-Antitrypsin Deficiency
Inactive IndicationsMethylmalonic acidaemia

Other Information

ATC CodeN/A
Related LinksN/A