The U.S. Food and Drug Administration approved two **sickle cell disease genes** on the 8th, which is the first time that cell-based genes have been approved in the United States for sickle cell disease patients aged 12 years and older**.
The FDA said in a statement that the two genes were "milestones." The two ** are called CASGEVY and LYFGENIA, of which CASGEVY is the first ** approved by the US Food and Drug Administration to use CRISPR Cas9 gene editing technology**, marking the innovation and progress in the field of genetics**.
Sickle cell disease is an inherited blood disorder caused by a genetic mutation in hemoglobin (the oxygen-carrying protein in red blood cells) that is characterized by sickle-shaped red blood cells, which block blood flow in the blood vessels, resulting in a restricted supply of oxygen to the body's tissues, causing severe pain and organ damage. About 100,000 people in the United States are affected by sickle cell disease.
According to the U.S. Food and Drug Administration, these two ** drugs are made from the patient's own hematopoietic stem cells, which are genetically modified and returned to the patient through a single injection by hematopoietic stem cell transplantation.
According to reports, the most common *** of casgevy include low platelet and white blood cell levels, mouth ulcers, nausea, musculoskeletal pain, abdominal pain and vomiting, etc.;The most common*** lyfgenia includes low levels of platelets, white blood cells and red blood cells, inflammation of the mouth, and fever, among others.
The FDA said it will conduct long-term follow-up studies in patients who received both genes** to evaluate the safety and efficacy of the drug.
Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said the approval of these two genes marks an important advance in medicine, using innovative cell-based genes to potentially damaging diseases and improve public health.
Nicole Verdun, director of the Product Office at the FDA's Center for Biologics Evaluation and Research, said that genetics are expected to provide more targeted and effective products, especially for patients with rare diseases who currently have limited options.
*: Xinhua News Agency).
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