- CRISPR Therapeutics (CRSP, Financial) reports significant lipid reductions in Phase 1 trial of CTX310, targeting ANGPTL3.
- The company plans to present complete Phase 1 data in the second half of 2025.
- Updates on additional cardiovascular programs, with CTX320 data expected in H1 2026.
CRISPR Therapeutics (CRSP), a leader in gene-based medicines, has announced promising additional Phase 1 trial data for its cardiovascular drug candidate CTX310, which targets the ANGPTL3 gene. The trial results show dose-dependent reductions of up to 82% in triglycerides and 86% in LDL cholesterol, with a favorable safety profile. These results solidify the potential of CTX310 as a groundbreaking treatment for cardiovascular disease.
The complete Phase 1 trial data for CTX310 is anticipated to be released in the second half of 2025, allowing for further insights into its efficacy and safety. This development forms part of CRISPR Therapeutics' broader strategy to tackle cardiovascular disease through genetic approaches.
In addition to CTX310, CRISPR Therapeutics provided updates on other cardiovascular projects. Notably, CTX320, which targets the LPA gene, is progressing with data now expected in the first half of 2026. Meanwhile, CTX340, aimed at treating refractory hypertension by targeting angiotensinogen (AGT), is advancing towards IND/CTA filings.
The addressable market for CTX310 is considerable, with over 40 million patients in the U.S. alone impacted by elevated LDL and triglycerides. CRISPR's innovative approach targets high-risk groups with significant unmet needs and limited treatment options, including homozygous familial hypercholesterolemia (HoFH) and severe hypertriglyceridemia.
