The US Food and Drug Administration (FDA) on December 8 approved two gene therapies, including Lyfgenia, which uses a harmless virus to edit genes; and Casgevy therapy, which uses CRISPR technology, which won the 2020 Nobel Prize, according to AFP.
Casgevy is a collaboration between Vertex Pharmaceuticals (USA) and CRISPR Therapeutics (Switzerland). This drug is used to correct problematic genes in bone marrow stem cells of patients aged 12 and older with sickle cell disease and thalassemia.
Both diseases are caused by errors in the genes that carry hemoglobin. Gene therapy treatments can cost millions of dollars and are out of reach for many patients, so Vertex Pharmaceuticals is working to keep the cost as low as possible.
This is the first FDA approval of Casgevy, after the UK's Medicines and Healthcare products Regulatory Agency (MHRA) gave the drug the green light last month.
Technicians work in a laboratory at Cincinnati Children's Hospital (USA), where gene editing therapies are tested.
Cincinnati Children's Hospital
"These treatments represent a major advance in the field of gene therapy for sickle cell disease. The potential for these products to change the lives of patients with sickle cell disease is enormous," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.
More than 100,000 Americans, mostly people of color, suffer from sickle cell disease, a painful and life-threatening disease that medical professionals are struggling to find a solution to, according to AFP.
US President Joe Biden also emphasized that the FDA's announcement on December 8 "demonstrates the power of medical innovation to improve the lives of Americans." Mr. Biden also emphasized: "My administration will continue to work to accelerate the development of treatments for rare diseases, and support the medical research and innovation that has made this breakthrough possible."
Scientists are also developing methods to edit genes in large organs, including the brain, which could pave the way for treatments for diseases such as muscular dystrophy and Huntington's disease, AFP reported.
Source link
Comment (0)