FDA approves first gene therapies to treat patients with sickle cell disease

FDA has approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older with vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a novel genome editing technology. Patients’ hematopoietic stem cells are modified using CRISPR/Cas9 and transplanted back into the patient. With Lyfgenia, blood stem cells are genetically modified to produce a gene-therapy derived hemoglobin delivered to the patient that functions similarly to hemoglobin A.