BCH_BBLCR-shRNA (bluebird bio)

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Report date: 2025/08/08 09:36:22

Base Information

Drug NameBCH_BBLCR-shRNA (bluebird bio)
Drug Alias NameBCL11a shRNA(miR)BCH-BBLCR-shRNA (miR)BCL11A-D12G5-2-shRNA
ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide > short hairpin RNACell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vectorCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionBCL11A gene inhibitorsHemoglobins stimulantsRNA interference
TargetsB-cell lymphoma/leukemia 11AHemoglobins
Therapy Area/IndicationsAnemia, Sickle Cell
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 CompaniesGenetix Biotherapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesBoston Children'S HospitalGenetix Biotherapeutics Inc
Inactive CompaniesN/A
Active IndicationsAnemia, Sickle Cell
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A