Shares of Crispr Therapeutics
and Vertex Pharmaceuticals Inc.
gained ground premarket on Thursday after the companies got the world’s first regulatory authorization for a medicine based on CRISPR, the transformative gene-editing technology. A U.K. regulator approved Casgevy, a gene-edited therapy, for the treatment of sickle cell disease, an inherited disorder affecting red blood cells, and transfusion-dependent beta thalassemia, a condition that causes severe anemia and dependence on red blood cell transfusions.
“Today is a historic day in science and medicine,” given the groundbreaking regulatory action, Dr. Reshma Kewalramani, president and CEO of Vertex, said in a statement. Casgevy works by editing a faulty gene in patients’ bone-marrow stem cells, prompting the body to produce functioning hemoglobin, the protein in red blood cells that delivers oxygen to tissues. To accomplish that, the stem cells are taken out of bone marrow, edited in a lab and then infused back into the patient, according to the U.K.’s Medicines and Healthcare Products Regulatory Agency. Patients may need to spend at least a month in the hospital while the treated cells get established in the bone marrow, the regulator said in a statement Tuesday. The therapy, also known as exa-cel, is also under …