New CRISPR Phase 1 trial hints at a first-of-its-kind heart treatment on horizon

Drug Discovery News

A gene therapy targeting ANTIGPTL3 reduced cholesterol and triglyceride levels simultaneously.

Elevated low-density lipoprotein (LDL) cholesterol and high triglycerides each affect about one in four adults worldwide. But a new CRISPR-Cas9 therapy may reduce the risk of major heart events caused by cholesterol and triglycerides.

LDL and triglycerides are risk factors for plaque buildup in heart disease. On Saturday morning, a team of researchers from Cleveland Clinic, CRISPR Therapeutics, Victorian Heart Institute, New Zealand Clinical Research, and Royal Adelaide Hospital published results in the New England Journal of Medicine from a 15-person Phase 1 trial of CTX310, which cut levels of both by about half.

Read the full story in Drug Discovery News

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