Betibeglogene autotemcel

贝格基干赛

Report date: 2026/03/09 20:02:04

Base Information

Drug NameBetibeglogene autotemcel
Drug Alias NameBeta-thalassaemia gene therapyLentiviral beta AT87Q-Globin vectorLentiviral β-A(T87Q)-Globin VectorBetibeglogene darolentivecbeti-cel +[11] 
ClassificationCell and gene therapy > Cell therapy > Donor > AutologousCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vectorCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy +[4] 
Mechanisms of ActionGene transferenceHemoglobin beta chain stimulants
TargetsHemoglobin beta chain
Therapy Area/Indicationsbeta-ThalassemiaAnemia, Sickle Cell
Global Approved Indicationsbeta-Thalassemia
Approved Indications in ChinaN/A
Original Approved Country/TerritoryEU
Original Approved Date2019/05/29
Country/TerritoryUnited StatesThailandItaly +[9] 
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesGenetix Biotherapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesGenetix Biotherapeutics Inc
Inactive CompaniesBluebird Bio (Netherlands) Bv
Active Indicationsbeta-ThalassemiaAnemia, Sickle Cell
Inactive IndicationsN/A

Other Information

ATC CodeB06AX
Related LinksN/A