Gene-edited humans? First CRISPR-based gene therapy gets approved for clinical use in the U.K. – NaturalNews.com
The exa-cel therapy, known commercially as Casgevy, has become the first CRISPR-based gene therapy to gain regulatory approval for clinical use in the United Kingdom. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, this groundbreaking treatment utilizes the gene-editing tool CRISPR (clustered regularly interspaced short palindromic repeats) to target and potentially cure certain genetic blood disorders.
The exa-cel therapy is specifically designed to treat sickle-cell disease and transfusion-dependent beta-thalassemia in patients aged 12 and older. Sickle-cell disease is an inherited blood disorder that causes the production of faulty hemoglobin, leading to the formation of sickle-shaped red blood cells and a range of debilitating symptoms. Beta-thalassemia is a rarer blood disorder that affects the production of beta-globin, resulting in the need for frequent blood transfusions.
The approval of Casgevy by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) comes after extensive evaluation of the therapy’s efficacy, quality, and safety. Julian Beach, the MHRA’s Interim Executive Director, emphasized the agency’s commitment to monitoring the ongoing safety and effectiveness of Casgevy through real-world data and post-authorization safety studies. The input of patients with lived experiences was also acknowledged as a valuable contribution to the assessment process.
The Casgevy therapy relies heavily on the CRISPR-Cas9 genome editing technology. The process involves three essential steps: recognition, cleavage or slicing apart, and repair. Initially, blood-producing stem cells are collected from the patient’s bone marrow, which are then edited in a laboratory using CRISPR technology. The CRISPR system employs short stretches of guide RNA to direct Cas9 enzymes to precisely target and modify specific genes. Once the modified cells are reinfused into the patient, a conditioning treatment called myeloablative conditioning is typically necessary to prepare the bone marrow.
While the approval of exa-cel therapy marks a significant milestone in the field of gene therapy, concerns have been raised regarding its accessibility, long-term effects, and safety. The high cost of gene therapy and eligibility restrictions pose barriers to widespread adoption. Additionally, experts have cautioned against potential health risks associated with gene therapy, including toxicity, inflammation, and the possibility of cancer.
In the United States, federal regulators are expected to approve exa-cel treatment for sickle-cell disease and thalassemia in December. The Sickle Cell Disease Association of America (SCDAA) has expressed concerns about the accessibility and safety of this treatment. The SCDAA emphasized the need for continued research to assess the long-term effectiveness and potential organ damage reduction attributed to exa-cel therapy. They also highlighted the cost barriers and eligibility restrictions that may limit patients’ access to this groundbreaking gene therapy.
As the field of gene-editing continues to advance, it is crucial to balance the remarkable potential of these therapies with careful consideration of safety, access, and ethical implications. The approval of Casgevy in the U.K. represents a significant step forward in harnessing CRISPR technology to combat debilitating genetic diseases. However, ongoing research and close monitoring of patients’ outcomes will be vital in determining the long-term impact and safety of this groundbreaking gene therapy.
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