The UK became the first country to approve Casgevy’s gene therapy

turning point

com. casgevy The process involves editing genes that code for hemoglobin in blood-producing stem cells. UK medicines regulator Medicines and Healthcare Products Regulatory Agency The MHRA became the first agency to approve this treatment after successful clinical trials. Oxford universityGeneticist in Kay Davies He said: “This is a landmark approval that opens the door to further application of CRISPR therapeutics in the future for the potential treatment of many genetic diseases.”

According to the journal Nature, the treatment is given intravenously. On the other hand, Vertex Pharmaceuticals and CRISPR Therapeutics have developed the treatment. Citing Kasjevi’s success, scientists say the treatment will help patients suffering from sickle cell disease. Saves you from debilitating pain And regularly in Mediterranean anemia (β-thalassemia) Reduces or eliminates the need for blood transfusions open.

The result is incredible

A severe disease whose standard treatment requires monthly blood transfusions Anemia in the Mediterranean In trials for β-thalassemia, 42 of 54 people taking Casgevy were reported to have been in treatment long enough to achieve interim results. While 39 of them did not need a blood transfusion for a year, the remaining three saw a more than 70 percent reduction in their need for a blood transfusion.

The revolutionary aspect of this treatment is that it uses CRISPR-Cas9 technology, the tool that won the 2020 Nobel Prize in Chemistry, to precisely edit genes in blood-producing stem cells. By targeting a specific gene called BCL11A, Casgevy stimulates the production of fetal hemoglobin, which can relieve symptoms by improving oxygen supply to tissues.

Treatment is very expensive

However, in participants in clinical studies Nausea and fever Some side effects have been observed, but no major safety concerns have been identified. On the other hand, scientists believe that CRISPR-Cas9 has potential Unwanted genetic modifications They also expressed concerns about other countries, including the United States and Europe, reviewing Kasgeevi for approval. However, high cost and complex technology may limit access, especially in low- and middle-income countries. Accounts and treatment About $2 million per patient It shows he can be a commodity.

source

https://www.nature.com/articles/d41586-023-03590-6


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