Key Takeaways CRSP's CTX310 showed up to 86% LDL and 82% TG reductions in early-stage study data.CRSP is developing CTX320 in an early-stage study for lipoprotein(a), tied to heart disease.The company plans to advance CTX340 and CTX450 into clinical studies by the end of 2025.CRISPR Therapeutics (CRSP) is the first and only company in the world to market a CRISPR/Cas9-based therapy. The company tasted major success with the approval of its one-shot gene therapy, Casgevy, in late 2023 and early 2024 across t ...

Core Insights - CRISPR Therapeutics' first marketed product, Casgevy, is a one-shot gene therapy approved for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in late 2023 and early 2024 [1] - Initial commercial uptake of Casgevy has been modest, with Vertex Pharmaceuticals reporting only $10 million in product revenues for full-year 2024 [2] - The eligible patient population for Casgevy is estimated to be around 60,000, and Vertex is increasing its commercial efforts to support broader access [3] Product Overview - Casgevy is the first approved CRISPR/Cas9 gene-edited therapy, developed in partnership with Vertex Pharmaceuticals, which leads its global development and commercialization [1] - The therapy involves a complex, multi-step process including stem cell collection, ex vivo gene editing, and reinfusion, contributing to its slow adoption [2][7] Competitive Landscape - Casgevy currently faces competition from other gene-editing treatments, such as Beam Therapeutics' BEAM-101 for SCD, which has shown promising results in clinical trials [4] - Additionally, Casgevy competes with established therapies like Bristol Myers' Reblozyl and Novartis' Adakveo, which are available at significantly lower prices compared to Casgevy's $2.2 million [5] Financial Performance - CRISPR Therapeutics' shares have outperformed the industry year to date, indicating positive market sentiment [6] - The company's shares are trading at a price-to-book (P/B) ratio of 2.10, below the industry average of 3.04 and its five-year mean of 2.39, suggesting a valuation discount [8] Earnings Estimates - Estimates for CRISPR's loss per share for 2025 have widened from $5.06 to $5.54, and for 2026, from $3.76 to $4.30 over the past 60 days [10]