United Kingdom Authorizes Gene Therapy for Blood Disorders

Sat Nov 18 2023
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LONDON: The United Kingdom has granted authorization for a revolutionary gene therapy designed to cure sickle-cell disease and another inherited blood disorder for patients aged 12 and above. This marks a historic milestone as the first-ever use of the gene-editing tool CRISPR in a licensed medicine, according to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

The newly approved gene therapy, named Casgevy, utilizes CRISPR, a gene-editing technology that earned its inventors the Nobel Prize in 2020. Sickle cell disease and β-thalassemia, the targeted genetic conditions, result from errors in the genes responsible for haemoglobin, crucial for transporting oxygen in red blood cells.

Gene Therapy for Blood Disorders

MHRA Interim Director Julian Beach emphasized the significance of this breakthrough, stating that both sickle cell disease and β-thalassemia are painful, lifelong conditions that, in some cases, can be fatal. Clinical trials have demonstrated Casgevy’s ability to restore healthy haemoglobin production in the majority of participants, providing relief from the symptoms of sickle-cell disease and transfusion-dependent β-thalassaemia.

The MHRA noted that the trials revealed no significant safety concerns, and they are closely monitoring the safety of the newly authorized gene therapy. Casgevy involves a process where stem cells are extracted from a patient’s bone marrow, and a gene is edited in the laboratory. The modified cells are then infused back into the patient after conditioning treatment to prepare the bone marrow.

Vertex Pharmaceuticals and CRISPR Therapeutics, both US-based companies, welcomed the UK’s approval in a separate statement. Samarth Kulkarni expressed optimism, stating, “I hope this represents the first of many applications of this Nobel Prize-winning technology to benefit eligible patients with serious diseases.”

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