Marnetegragene autotemcel

玛奈基奥干赛

Report date: 2026/05/20 15:36:46

Base Information

Drug NameMarnetegragene autotemcel
Drug Alias NameLAD-1-gene-therapy(Rocket-Pharmaceuticals)Leukocyte-adhesion-deficiency-1-gene-therapy(Rocket-Pharmaceuticals)KRESLADIRP-L201LV RP-L201
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vectorCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapyCell and gene therapy > Cell therapy > Donor > Autologous +[4] 
Mechanisms of ActionITGB2 gene transference
TargetsIntegrin beta-2
Therapy Area/IndicationsType I Leukocyte Adhesion Defect
Global Approved IndicationsType I Leukocyte Adhesion Defect
Approved Indications in ChinaN/A
Original Approved Country/TerritoryUnited States
Original Approved Date2026/03/26
Country/TerritoryUnited StatesUnited KingdomSpain
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesRocket Pharmaceuticals IncCiemat
China Registration ClassN/A

Develop Information

Active CompaniesRocket Pharmaceuticals Inc
Inactive CompaniesN/A
Active IndicationsType I Leukocyte Adhesion Defect
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksDrugBank