News about CRISPR Therapeutics

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.

Ark Invest founder Cathie Wood holds $379.1 million of CRISPR Therapeutics shares and bought $1.1 million on Oct. 9 and $2.3 million on Oct. 12 through the ARK Genomic Revolution ETF, even though the stock is down more than 25% over the past year. CRISPR's only approved therapy, Casgevy, posted $76 million in Q2 revenue, up 151% year over year, but its $2.2 million list price and the 60% revenue share going to Vertex limit its economics. The company's net loss narrowed to $91.2 million from $208.5 million, and it holds $2.36 billion in cash. The article also points to CTX-310, which cut LDL cholesterol by 53% and triglycerides by 48% after 12 months in a phase 1a trial, and to a phase 2 readout for blood thinner CTX611 expected later this year.