It has been just revealed the fact that the first CRISPR gene-editing therapy has been approved in the US. Check out the latest reports about this below.
Gene-editing therapy in the US
The U.S. Food and Drug Administration (FDA) has recently approved two gene therapies for sickle cell disease in patients aged 12 years or older. The therapies are called Casgevy and Lyfgenia, and they have been manufactured by Vertex and Bluebird Bio respectively. Casgevy uses CRISPR technology, while Lyfgenia uses lentiviral vectors to edit genes.
Both therapies have undergone rigorous evaluations of scientific and clinical data to obtain approval.
Clinical trial findings have shown that they can reduce pain from vaso-occlusive events, which are a common occurrence in sickle cell disease.
These events happen when blood vessels become blocked, leading to organ damage and severe pain.
“We know they transform people’s lives; they significantly—if not completely—eradicate and get rid of these horrible, painful events that take over people’s lives with sickle cell … What we don’t know is what happens with organ function,” Dr. Julie Kanter, co-director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama, said in an interview with the university.
Sickle cell disease is an inherited blood disorder.
Mutations in a gene that codes for hemoglobin cause the body to produce abnormal forms of hemoglobin, which are proteins in red blood cells responsible for carrying oxygen throughout the body. When hemoglobin is deformed, it becomes sticky and unable to carry oxygen effectively.
As a result, red blood cells become misshapen, taking on a crescent or “sickle” shape.
“So normally, red blood cells aren’t sticky. They easily flow through the blood vessels like water through pipes. But in people with sickle cell disease, in the tiny, tiny pipes, they can get stuck,” Dr. Kanter said in the interview.
“That’s one of the main reasons people end up having pain or organ damage associated with sickle cell disease.”
Individuals with sickle cell disease have inherited the mutation in both of their genes.
They usually live up to an average of 52 years, but their life is often plagued by anemia, episodes of pain, infections, blood transfusions, and other complications.
Stem cell transplants from a donor have shown successful results in treating sickle cell disease in children, with some studies reporting a success rate of over 80 percent. However, the risks associated with the operation increase as the patient gets older.