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World's First Gene Therapy for Blood Disorders Approved

Earlier this month, the UK's Medicines and Healthcare Regulatory Agency granted approval for the world's first gene therapy for sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

Casgevy (exagamglogene autotemcel) uses the nobel prize winning CRISPR gene-editing technology to target the problematic gene in bone marrow stem cells, allowing the body to produce properly functioning hemoglobin. It was approved for patients ≥12 years with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.

Its approval was based on two global clinical trials which found that ~97% and ~93% of patients receiving Casgevy achieved the primary endpoint of their trials, reporting no debilitating episodes of pain or need for a red-blood cell transfusion, respectively, for at least 1-year following treatment. The benefits of treatment are expected to be life-long and offer a promising new treatment option for patients with these conditions. However, it’s accessibility may be limited by cost, as gene therapies typically come with a hefty price tag.

The FDA is currently reviewing the treatment with a decision expected early December, emphasizing the global significance of this pioneering step in gene therapy for blood disorders.

Written by Courtney Barker


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