The two biotechnology companies CRISPR Therapeutics from Zug and Vertex Pharmaceuticals from Boston, Massachusetts, which have already been working together since 2015, have been awarded market approval for their CASGEVY therapy by the Medicines & Healthcare products Regulatory Agency (MHRA) in the UK. This is the world’s first regulatory authorization for a therapy approach based on the CRISPR gene scissors for genome editing.
CASGEVY has been authorized for the treatment of sickle cell anemia and transfusion-dependent beta thalassemia (TDT) in patients aged 12 and above, for whom a hematopoietic stem cell donor is not available. The press release states that there are an estimated 2,000 patients eligible for treatment with CASGEVY in the UK.
According to the press release, the primary result was achieved in two global clinical trials covering both diseases, namely that the patients treated were free of severe vascular occlusion crises or transfusion independent for at least twelve consecutive months. “Once achieved, these benefits are potentially expected to be life-long”, the press release explains.
“Today is a historic day in science and medicine”, explains Dr. Reshma Kewalramani, CEO and President of Vertex, in the press release. Samarth Kulkarni, Chairman and CEO of CRISPR Therapeutics, is hopeful that this will come to represent “the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases”.