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MHRA : Approved Casgevy for Sickle Cell & β-thalassemia - 0 views

www.pharmacy.biz/...e-cell-disease-and-thalassemia

Casgevy-gene-editing-treatment CRISPR-based-medicine MHRA-authorization-for-sickle-cell-disease Gene-therapy-for-β-thalassemia Casgevy-treatment-for-β-thalassemia

shared by pharmacybiz on 20 Nov 23 - No Cached
  • pharmacybiz on 20 Nov 23
     
    The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia. Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over. To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal. The MHRA's authorisation came after a rigorous assessment of its safety, quality and effectiveness.
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