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CASGEVY (exagamglogene autotemcel/exa-cel)

Sickle Cell Disease (SCD)

ApprovedCommercial

Key Facts

Indication
Sickle Cell Disease (SCD)
Phase
Approved
Status
Commercial
Company

About CRISPR Therapeutics

CRISPR Therapeutics is a pioneering biopharmaceutical company dedicated to discovering, developing, and commercializing curative gene-editing treatments. Founded by CRISPR gene-editing pioneers, the company leverages its proprietary CRISPR/Cas9 platform to create a diverse pipeline targeting hemoglobinopathies, immuno-oncology, regenerative medicine, and in vivo diseases. Its landmark achievement is the approval of CASGEVY, the world's first CRISPR/Cas9 gene-edited therapy, marking a new era in medicine and validating its platform's potential. The company collaborates with Vertex Pharmaceuticals and Bayer to accelerate development and commercialization across multiple therapeutic areas.

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About CRISPR Therapeutics

CRISPR Therapeutics is a pioneering biopharmaceutical company dedicated to discovering, developing, and commercializing curative gene-editing treatments. Founded by CRISPR gene-editing pioneers, the company leverages its proprietary CRISPR/Cas9 platform to create a diverse pipeline targeting hemoglobinopathies, immuno-oncology, regenerative medicine, and in vivo diseases. Its landmark achievement is the approval of CASGEVY, the world's first CRISPR/Cas9 gene-edited therapy, marking a new era in medicine and validating its platform's potential. The company collaborates with Vertex Pharmaceuticals and Bayer to accelerate development and commercialization across multiple therapeutic areas.

View full company profile

Other Sickle Cell Disease (SCD) Drugs

DrugCompanyPhase
Undisclosed In Vivo ProgramEditas MedicineResearch