News about pharmaceuticals

Shares of CRISPR Therapeutics have fallen about 47% since mid-October 2021, while the S&P 500 has gained almost 73% as of writing, despite clinical and regulatory progress over the past five years. Casgevy, developed with Vertex Pharmaceuticals, was approved in 2023 and early 2024 for transfusion-dependent beta-thalassemia and sickle cell disease, making it the company's first commercial product and the first approved CRISPR-based gene-editing therapy. Other candidates include CTX310, aimed at lowering LDL cholesterol and triglycerides, where Phase 1 data showed significant decreases in both; zugo-cel for autoimmune diseases and blood cancers; and CTX611, an anticoagulant that could be given twice a year. Casgevy's label now covers patients as young as two, adding about 5,500 U.S. patients at $2.2 million per treatment course. The article warns of possible clinical failures and the costly, one-time nature of many gene-editing therapies, which makes profitability uncertain.