Key points from article :
Vertex pharmaceuticals and CRISPR therapeutics are jointly developing gene-editing treatment, CTX001.
Sickle cell and beta-thalassemia are caused by mutations in the gene responsible for production of oxygen-carrying hemoglobin.
Vertex/CRISPR cure both disorders by increasing the production of healthy, oxygen-carrying form of hemoglobin.
Patients undergo treatment with busulfan, a chemotherapy drug.
Then receive an infusion of CRISPR-edited cells (CTX001), start producing healthy, normal red blood cells.
Vertex/CRISPR clinical trial, CLIMB-111, second paired study, called CLIMB-121.
All patients have been free from symptoms and have not needed blood transfusions.
“These data are preliminary, but they’re also nothing short of great” - Haydar Frangoul, pediatric hematologist and oncologist.
“CRISPR envision not only for patients with sickle cell and beta-thalassemia, but for many more patients in the future” - David Altshuler, Vertex’s chief scientific officer.
