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Gene Therapy for Sickle-Cell Disease & β-Thalassemia Received Authorisation in the UK

Written by : Jayati Dubey

November 17, 2023

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MHRA approves Casgevy, the pioneering CRISPR-based therapy for sickle-cell disease and β-thalassemia. This advancement in gene therapy offers hope and relief for patients aged 12 and above.

Britain's Medicines and Healthcare Products Regulatory Agency (MHRA) has granted authorisation for Casgevy, a gene therapy designed to cure sickle-cell disease and β-thalassemia in patients aged 12 and above.

Notably, this approval marks a significant development as Casgevy is the world's first licensed medicine to utilise the gene-editing tool CRISPR, a technology that earned its inventors the Nobel Prize in 2020.

Sickle-cell disease and β-thalassemia are debilitating genetic conditions resulting from errors in the genes responsible for producing haemoglobin, a crucial component of red blood cells essential for oxygen transport throughout the body. MHRA Interim Director Julian Beach highlighted the severity of these conditions, emphasising their painful and life-threatening nature.

Casgevy, in clinical trials, has demonstrated the ability to restore healthy haemoglobin production in a majority of participants suffering from sickle-cell disease and transfusion-dependent β-thalassemia. This advanced therapy offers relief from the symptoms of these disorders, marking a significant stride towards improving the quality of life for affected individuals.

Crucially, the MHRA reported no significant safety concerns identified during the trials, underscoring the meticulous monitoring of the medicine's safety.

The administration of Casgevy involves extracting stem cells from a patient's bone marrow, editing a specific gene within these cells in a laboratory setting, and subsequently infusing the modified cells back into the patient following conditioning treatment to prepare the bone marrow.

Vertex Pharmaceuticals and CRISPR Therapeutics, both based in the United States, welcomed the MHRA's approval in a joint statement. CRISPR CEO Samarth Kulkarni said, "I hope this represents the first of many applications of this Nobel Prize-winning technology to benefit eligible patients with serious diseases."

The green light for Casgevy opens new doors in the field of gene therapy and holds promise for addressing other genetic disorders in the future. As the first-of-its-kind treatment, its approval marks a significant leap forward in the pursuit of innovative and effective medical solutions for genetic conditions worldwide.

In a similar development in the field of genetics, researchers at Google DeepMind, the tech giant's AI division, have unveiled an AI tool that predicts the potential harm of genetic mutations. This development claims to have the potential to significantly advance research into rare diseases.

The tool, named AlphaMissense, focuses on 'missense' mutations, which involve a single letter of the genetic code. A study published in the journal Science revealed that the tool could predict 89% of these mutations with a 90% accuracy rate. Each mutation received a score indicating its potential to cause disease, referred to as pathogenic.


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