UK Approves First-Ever CRISPR Therapy, Paving the Way for Genetic Disease Treatment

2023-11-16 21:46:31

UK Authorizes Pioneering CRISPR Gene Editing Therapy

The United Kingdom has taken a historic step by being the first to approve a medical treatment utilizing the groundbreaking CRISPR gene editing technology.

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The Medicines and Healthcare products Regulatory Agency (MHRA) endorsed a therapy dubbed Casgevy, aimed at combating sickle cell disease and beta thalassemia. These genetic disorders, stemming from hemoglobin gene mutations, affect red blood cells' oxygen transport capabilities and are currently without a universally effective cure.

Occurrences of sickle cell disease predominantly arise within individuals of African or Caribbean descent. On the other hand, beta thalassemia majorly impacts people from Mediterranean, South Asian, Southeast Asian, and Middle Eastern backgrounds, according to an MHRA official statement.

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CRISPR-Cas9: A Nobel Prize-Winning Innovation

Sickle cell disease and beta thalassemia are not only painful but potentially life-threatening conditions. The exclusive pipeline for a permanent cure to date has been a bone marrow transplant, which requires a suitable donor match and has a risk of rejection, noted by Julian Beach from the MHRA.

The CRISPR-Cas9 gene editing technique, developed by Emmanuelle Charpentier and Jennifer A. Doudna who clinched the Nobel Prize for it, enables extremely precise DNA alterations. Casgevy, engineered by Vertex Pharmaceuticals, involves extracting a patient's stem cells, gene editing in a lab, followed by a "conditioning treatment" to ready the bone marrow before re-introducing the edited cells to the patient's body.

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Post-treatment, patients may spend up to a month in hospital care for the integration of the treated cells into the bone marrow and to commence the production of red blood cells with stable hemoglobin.

Potential Global Impact and Future Prospects

The US Food and Drug Administration is also reviewing Casgevy, with a decision anticipated by December 8. "This advancement in medical science marks a giant leap towards curing genetic diseases once deemed incurable," remarked Alena Pance of the University of Hertfordshire.

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Pance highlights that modulating a patient’s bone marrow stem cells sidesteps the challenges of donor compatibility and post-transplant immunosuppression, setting the stage for a potential definitive cure over a mere treatment.

While the MHRA has not disclosed Casgevy's cost, it is widely expected to be a premium-priced treatment. Nevertheless, CRISPR-Cas9's profound influence extends beyond biomedicine to agriculture, gaining a reputation as a cornerstone in global laboratories.

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Controversy shrouded CRISPR-Cas9 when Chinese scientist He Jiankui declared the birth of gene-edited infants in 2018. This sparked a scientific consensus that such advanced techniques should not be utilized for editing genes inherited by subsequent generations.

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