生产企业：Vertex Pharmaceuticals
Exagamglogene autotemcel is being developed by CRISPR Therapeutics and Vertex. It is an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in clinical trials for the treatment of severe sickle cell disease and severe beta-thalassemia. A potential label expansion for Casgevy in patients between the ages of five and 11 is on the horizon.
Exagamglogene autotemcel is being developed by CRISPR Therapeutics and Vertex. It is an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in clinical trials for the treatment of severe sickle cell disease and severe beta-thalassemia. A potential label expansion for Casgevy in patients between the ages of five and 11 is on the horizon.
CASGEVY (exagamglogene autotemcel) is a one-time gene therapy indicated for patients aged 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises (VOCs), or with beta thalassemia (β-thalassemia) who need regular blood transfusions (transfusion-dependent beta thalassemia, TDT).
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