CRISPR Therapeutics developed Casgevy, the first approved therapy using the CRISPR gene-editing technique in 2023, and shares its rights with Vertex Pharmaceuticals (VRTX). The article argues that the investigational CTX310, which targets high LDL cholesterol and triglycerides, could matter more to the company's future as a possible one-time option, since more than 40 million U.S. patients have high LDL or TG levels versus about 60,000 addressable patients for Casgevy. In a Phase 1a trial reported in August, the highest dose cut mean TG by 48% and mean LDL by 53% in patients with certain genetic conditions, and Phase 1b data are due in early November. Risks include clinical and regulatory setbacks, high prices and difficult administration. The author views the company as fairly attractive, but only for investors comfortable with significant risk and volatility.
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