RAG1 CRISPR/Cas9 gene therapy (Casebia Therapeutics)

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Report date: 2025/03/20 09:56:42

Base Information

Drug NameRAG1 CRISPR/Cas9 gene therapy (Casebia Therapeutics)
Drug Alias Nameex vivo RAG1 hematopoietic stem cell based gene therapy (Casebia Therapeutics)
ClassificationCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionV(D)J recombination-activating protein 1 inhibitors
TargetsV(D)J recombination-activating protein 1
Therapy Area/IndicationsSevere Combined Immunodeficiency
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesCasebia Therapeutics Llc
China Registration ClassN/A

Develop Information

Active CompaniesCasebia Therapeutics Llc
Inactive CompaniesN/A
Active IndicationsSevere Combined Immunodeficiency
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A