Can CRISPR Build on Casgevy's Success With Its In Vivo Pipeline?

Core Insights - CRISPR Therapeutics (CRSP) is the only company globally with approval for a CRISPR-based gene therapy, developed in collaboration with Vertex Pharmaceuticals, focusing on in vivo candidates after the success of Casgevy [1][4] In Vivo Candidates - The company is evaluating two in vivo candidates, CTX310 and CTX320, targeting ANGPTL3 and lipoprotein(a) [Lp(a)], both linked to atherosclerotic heart disease [2] - Initial data from the phase I study on CTX310 showed significant dose-dependent reductions in low-density lipoprotein (LDL) and triglyceride (TG) levels, with peak reductions of up to 82% in TG and 81% in LDL [3][8] - Results from CTX320 are anticipated in the second quarter of 2025, and CRISPR Therapeutics plans to advance two additional in vivo programs by year-end [4] Competitive Landscape - CRISPR Therapeutics is currently behind Intellia Therapeutics (NTLA), which has multiple clinical-stage in vivo gene editing programs, including NTLA-2002 for hereditary angioedema and NTLA-2001 for transthyretin amyloidosis [5] - Beam Therapeutics (BEAM) is also developing in vivo candidates, with ongoing phase I/II studies for BEAM-302 and BEAM-301, offering a differentiated base-editing approach [6] Financial Performance and Valuation - CRISPR Therapeutics shares have underperformed the industry year to date [7] - The company is trading at a price-to-book (P/B) ratio of 1.80, lower than the industry average of 3.10, and below its five-year mean of 2.41 [10] - Estimates for CRISPR's loss per share for 2025 have widened from $4.96 to $5.54, and for 2026 from $3.65 to $4.11 over the past 60 days [12]