CRISPR Therapeutics Reports Promising Phase 1 Cardiovascular Trial Results

CRISPR Therapeutics has reported promising Phase 1 clinical trial data for its in vivo gene-editing candidate CTX310, targeting the ANGPTL3 gene to reduce triglycerides and LDL cholesterol, showing peak reductions of up to 82% and 86%, respectively, with a favorable safety profile. The company plans to present full Phase 1 data for CTX310 in the second half of 2025. Concurrently, CRISPR is progressing with its CTX320 program targeting the LPA gene, with updated data expected in the first half of 2026, and advancing its preclinical CTX340 program aimed at refractory hypertension. The delay in CTX320 data release reflects a strategic decision to incorporate new insights from the evolving Lp(a) treatment landscape. With over 40 million patients in the U.S. affected by elevated LDL or triglycerides, these developments highlight CRISPR's potential to address significant unmet medical needs in cardiovascular disease through gene editing. Analyst sentiment is mixed, ranging from 'Hold' to 'Outperform,' reflecting both optimism about the technology's promise and concerns about financial and valuation risks.