CRISPR Therapeutics announced updates on its in vivo cardiovascular disease programs, including new Phase 1 data for CTX310 targeting ANGPTL3, progress on CTX320 targeting the •LPA• gene, and advancements in the preclinical CTX340 program for refractory hypertension. These programs aim to address significant unmet needs in cardiovascular health by targeting genes involved in lipid regulation and blood pressure control. The company’s focus on gene editing therapies for cardiovascular diseases underscores its commitment to developing innovative treatments for complex conditions.
These developments are important because they represent potential breakthroughs in the treatment of cardiovascular diseases, a leading cause of death worldwide. CTX310’s positive data suggests a potential new treatment option for patients with high cholesterol and triglycerides, while CTX320 and CTX340 offer hope for addressing currently intractable forms of cardiovascular disease like elevated lipoprotein(a) and refractory hypertension. Successfully developing these therapies could significantly improve patient outcomes and reduce the burden of cardiovascular disease.
CTX310 continues to demonstrate dose-dependent reductions in triglycerides and LDL cholesterol, with peak reductions of 82% and 86%, respectively, in early clinical trials. The safety profile remains well-tolerated, and complete Phase 1 data is expected in the second half of 2025. Data for CTX320, targeting the LPA gene for elevated lipoprotein(a), is now expected in the first half of 2026. CTX340, a preclinical program targeting the AGT gene for refractory hypertension, is advancing toward IND/CTA filings.
The continued progress of these programs positions CRISPR Therapeutics at the forefront of developing gene-editing therapies for cardiovascular diseases. Positive clinical results and advancements toward regulatory filings suggest the potential for new treatment options in areas with high unmet need. The coming data readouts for CTX310 and CTX320 will be crucial for determining the next steps in the clinical development of these promising therapies.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
