Two Gene Therapy Products for Sickle Cell Anemia Approval by FDA

Two gene therapy products, Casgevy and Lyfgenia, have been approved by the FDA for the treatment of patients 12 years and older with sickle cell anemia. The FDA approved Casgevy to Vertex Pharmaceuticals and approval of Lyfgenia to Bluebird Bio Inc. Casgevy is the first FDA-approved treatment using CRISPR/Cas9 technology, representing an innovative breakthrough in gene therapy. Both products are made from the patient’s hematopoietic stem cells and are returned to the patient’s body with a single dose of injection as part of the hematopoietic stem cell transplant.