The U.S. Food and Drug Administration (FDA) has given the green light to two new gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease in patients 12 years old and older. Sickle cell disease is a genetic blood disorder that affects about 100,000 people in the U.S., mainly African-Americans and Hispanic Americans.
It was reported that Casgevy is the first therapy approved by the FDA that uses CRISPR/Cas9 genome editing technology. It changes patients’ blood stem cells to make more fetal hemoglobin, which stops red blood cells from becoming sickle-shaped.
Lyfgenia, another gene therapy, uses a lentiviral vector for genetic modification. It allows patients’ blood stem cells to create gene-therapy-derived hemoglobin that works like normal adult hemoglobin.
This reduces the chance of blood cells becoming sickle-shaped and blocking blood flow. Both treatments involve a one-time, single-dose infusion as part of a stem cell transplant.
Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said these approvals are a significant step forward in using innovative gene therapies to target serious diseases and improve public health, as reported by the FDA.
The federal agency reported that they approved Casgevy to Vertex Pharmaceuticals Inc. and approval of Lyfgenia to Bluebird Bio Inc.
