Exagamglogene autotemcel

艾卡基奥干赛

Report date: 2026/05/12 20:00:17

Base Information

Drug NameExagamglogene autotemcel
Drug Alias NameAutologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cellsAutologous CRISPR-Cas9 modified CD34+ hHSPCsCRISPR-Cas9-based-therapyExa-celCASGEVY +[6] 
ClassificationCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9Cell and gene therapy > Cell therapy > Donor > AutologousCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionStem cell replacementsBCL11A gene inhibitors
TargetsB-cell lymphoma/leukemia 11A
Therapy Area/IndicationsIron deficiencyGenetic Diseases, InbornHemoglobinopathies +[9] 
Global Approved Indicationsbeta-ThalassemiaAnemia, Sickle Cell
Approved Indications in ChinaN/A
Original Approved Country/TerritoryUnited Kingdom
Original Approved Date2023/11/16
Country/TerritoryBelgiumCanadaUnited States +[10] 
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesUmeå UniversityCrispr Therapeutics Ag
China Registration ClassN/A

Develop Information

Active CompaniesCajal Therapeutics IncCrispr Therapeutics IncCrispr Therapeutics Ag +[5] 
Inactive CompaniesN/A
Active IndicationsIron deficiencyGenetic Diseases, InbornHemoglobinopathies +[9] 
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksDrugBankKEGG