On Friday, the Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease (SCD), including the first medical treatment to utilize CRISPR technology.
ICYMI: The FDA approved Vertex’s Casgevy (exa-cel) and bluebird bio’s Lyfgenia (lovo-cel) for sickle cell patients aged 12 and up. They’re the first FDA-approved cell-based gene therapies for treating SCD. Both companies are BIO members.
Why it matters: CRISPR-Cas9 allows for more precise gene editing, first described in 2012, has been employed successfully to improve plants. Researchers have been investigating CRISPR’s huge potential for human medicine, and Casgevy shows these drugs can meet FDA standards.
The disease: SCD affects about 100,000 people in the United States, mostly people of African descent. It’s caused by a mutation in the gene that makes hemoglobin, a protein in red blood cells that delivers oxygen to the body. SCD has historically been under-researched.
The treatments: Casgevy and Lyfgenia involve taking stem cells from a patient, genetically altering those cells, then putting them back in the patient’s body. Lyfgenia employs a previously used technique, lentiviral vector, for genetic modification, while Casgevy utilizes CRISPR.
What they’re saying: “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., Director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.
Read more about the historic approvals and listen to the I am BIO Podcast about the dawning age of CRISPR.
