The UK’s MHRA has greenlit Casgevy, a CRISPR/Cas gene-editing drug, for treating blood disorders. This groundbreaking approval offers hope to patients aged twelve and above suffering from beta-thalassemia and sickle cell anemia. Developed by Nobel Prize winners Emmanuelle Charpentier and Jennifer Doudna, CRISPR/Cas technology has paved the way for targeted gene therapy.
Casgevy, utilizing CRISPR/Cas gene scissors, addresses hereditary blood disorders at their source. Approved by the MHRA, it marks the world’s first gene-scissors-based drug sanctioned by a regulatory body. Clinical studies show its efficacy in restoring proper hemoglobin production, significantly reducing symptoms with no observed side effects
This gene therapy involves modifying faulty genes in stem cells extracted from the patient’s bone marrow. The corrected cells are then infused back into the patient. MHRA’s Julian Beach emphasizes the significance of addressing these lifelong and potentially fatal diseases, heralding Casgevy’s approval as a transformative milestone.
Samarth Kulkarni, CEO of Vertex Pharmaceuticals and Crispr Therapeutics, sees Casgevy’s approval as a promising start for applying this Nobel Prize-winning technology to benefit patients with serious illnesses. This marks a pivotal moment in the intersection of gene therapy and conventional medicine, offering a new avenue of treatment for blood disorders.
Source:
MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia (no date) GOV.UK. Available at: https://www.gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia (Accessed: 18 November 2023).
