World's First CRISPR-Based Gene Therapy for Blood Disorders

Nobel Prize-winning inventors of CRISPR, awarded in 2020, have seen their gene-editing tool gain approval in the UK for the first-ever therapy targeting blood disorders, specifically sickle-cell disease and thalassemia ().

So far, a bone marrow transplant -- which must come from a closely matched donor and carries a risk of rejection -- has been the only permanent treatment option.

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‘UK has approved the inaugural gene therapy for blood disorders using the CRISPR gene-editing tool, developed by the Nobel Prize-winning inventors of CRISPR in 2020. #genetherapy, #crispr, #blooddisorders, #sickle-celldisease, #thalassemia ’

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The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) authorized the new treatment called Casgevy for patients with sickle-cell disease and transfusion-dependent beta-thalassemia aged 12 and over.

Both sickle cell disease and beta-thalassemia are genetic conditions caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body.

Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning hemoglobin. To do this, stem cells are taken out of bone marrow, edited in a laboratory, and then infused back into the patient after which the results have the potential to be life-long.

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In people with sickle cell disease, the genetic error can lead to attacks of very severe pain, serious and life-threatening infections, and anemia (whereby your body has difficulty carrying oxygen).

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Among beta-thalassemia patients, it can lead to severe anemia. Patients often need a blood transfusion every 3 to 5 weeks and injections and medicines throughout their lives.

“Both sickle cell disease and beta-thalassemia are painful, life-long conditions that in some cases can be fatal,” said Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA, in a statement.

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“We have authorized an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy hemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent beta-thalassemia, relieving the symptoms of disease,” she added.

Casgevy’s approval for sickle-cell disease was based on a clinical trial of 29 patients, of which 28 (97 percent) were free of severe pain crises for at least 12 months after treatment.

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Of the 42 patients in the clinical trial for transfusion-dependent beta-thalassemia, 39 (93 percent) did not need a red blood cell transfusion for at least 12 months after treatment.

The remaining three had more than a 70 percent reduction in the need for red cell transfusions. Side effects from treatment were similar to those associated with autologous (from a person’s cells) stem cell transplants, including (but not limited to) nausea, fatigue, fever, and increased risk of infection.

No significant safety concerns were identified during the trials, the MHRA said, adding that the safety of the treatment will be analyzed further. Casgevy is currently being evaluated by the US Food and Drug Administration (FDA) and is expected to receive the agency’s approval next month.

Reference:

UK first to approve CRISPR treatment for diseases: what you need to know - (https://www.nature.com/articles/d41586-023-03590-6)

Source-IANS