FDA allows the use

The US health authorities approved the first treatment on Friday using the Crispr molecular scissors, which is a technique for genetic editing (rewriting the genetic material) which represents an important achievement in medicine. In this case, it is expected to reduce the suffering of people with rare blood disease. In total, two drugs against sickle cell anemia, a rare genetic disease, were approved, affecting about 100,000 people in the United States. “These treatments are great progress in the field of genetic therapy for patients with sickle cell anemia, which is a rare and exhausted blood disease,” Peter Marx said at a press conference. “The potential of these products to change the lives of patients with enormous sickle cell anemia,” he added. The US agency has confirmed that this disease is affecting Afro -Americans in particular. The Nobel Prize for Chemistry. The development of the French molecular scissors, Emmanuel Sharbantier and American Jennifer Dodna, authorized the Nobel Prize for Chemistry in 2020. This technique has made the treatment of genome a revolution by the accuracy and ease of use compared to previous instruments. Patients with sickle cell anemia suffer from a mutation that affects hemoglobin, a protein found in red blood cells that transport oxygen to the tissue of the body. Due to this mutation, red blood cells become in the form of a sickle, which limit blood flow and connect oxygen. This can lead to a chronic anemia and very painful attacks. The consequences may be related to the occurrence of damage to important organs, dangerous and even deadly. Both remedies, CASEVY and Lyfgenia, are adopted by the US Food and Drug Administration for patients aged 12 and older. US President Joe Biden praised these licenses as a ‘great achievement’. “This important medical progress is very promising to develop other treatments,” he said in a statement. A new era was approved by the British health authorities for the Casge treatment in November, developed by Vertex Pharmaceuticals, but this new license is the first of its kind in the United States. Patient cells are amended using CrisPR technology and then planted again in the individual. During a clinical trial, 29 out of 31 patients suffered for at least 12 consecutive months from a vascular blockage crisis during a 24 -month follow -up period. The most common side effects were oral pain, nausea, abdominal pain and vomiting. The second treatment, called “Body Genia”, developed by Bluebird Bio, works differently as it uses a harmful virus to effect genetic modification. Nicole Verdun, director of the therapeutic products in the US Food and Drug Administration, said two patients in leukemia died during the clinical trials of this treatment, which is why a warning was added to the approved therapy to inform the public of the risks. Derdin explained that pharmaceutical laboratories were agreed to perform follow -up studies for a period of 15 years. Once the stem cells of the patient have been collected, chemotherapy should be subject to the removal of cells from the spinal cord, and it can then be replaced by modified cells. After the injection, in one dose, patients will have to follow up in hospital for a few weeks. The company estimates that about 16,000 patients will be eligible for this. According to Peter Marx of the US Food and Drug Administration, these treatments will be more suitable for patients with regular and serious attacks, or ‘about 20%’ of people with this disease in the country. But according to Marx, the license of these two drugs “that is likely to” transform the path of research in this area shows the current dynamics of this ‘promising new era of medicine’.