Scientists have edited a gene that may reduce high cholesterol permanently
- On Saturday, CRISPR Therapeutics reported one infusion reduced LDL cholesterol and triglycerides by about half in 15 volunteers in a Phase I trial presented at the American Heart Association meeting in New Orleans and published in The New England Journal of Medicine.
- The drug targets the ANGPTL3 gene to switch off its function in the liver, mimicking a protective genetic mutation found in about 1 in 250 people in the U.S.
- Participants received varying doses via an infusion lasting about two and a half hours in the Phase I trial across the U.K., Australia, and New Zealand, with ages 31 and 68 and baseline median LDL 155 mg/dL and triglycerides 192 mg/dL.
- One participant died months after the infusion, and researchers plan Phase 2 clinical trials soon to test if a one-time gene edit can safely prevent heart attacks and strokes.
- CRISPR Therapeutics, maker of Casgevy, cautioned that long‑term safety and costs remain unknown, despite potential to give people a cure, said Dr. Luke Laffin.
51 Articles
51 Articles
With gene modification, doctors could one day permanently reduce dangerously high cholesterol levels, possibly eliminating the need for medication, according to a new pilot study published this Saturday in the New England Journal of Medicine.
Study in New England Journal shows potential for cholesterol drug
A recent study published in the New England Journal of Medicine suggests a new gene editing drug could help lower high cholesterol levels. This small study included 15 people and was designed to test the medication's safety.
Merck, the company that introduced statins in the world nearly 40 years ago, has a new, intensely powerful pill that can reduce dangerous LDL cholesterol levels to almost never seen in adults. The new pill, enlicitide, blocks a liver protein, PCSK9, which slows the body’s ability to eliminate cholesterol. With most blocked PCSK9 levels, LDL levels plummet and rates of heart attacks and strokes in high-risk patients fall by up to 20 percent in th…
CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study
A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half in four people taking the highest dose, raising hope for a one-time treatment.
CRISPR gene-editing works to reduce high cholesterol in a new study
A single infusion of an experimental gene-editing drug appears safe and effective for cutting cholesterol, possibly for life, according to a small early study released Saturday.The study, which involved 15 volunteers, found one infusion of a drug that uses the CRISPR gene-editing technique could safely reduce cholesterol, as well as levels of harmful triglycerides, by about half.“Rather than a lifetime worth of medicine, we have the potential to…
Coverage Details
Bias Distribution
- 69% of the sources are Center
Factuality
To view factuality data please Upgrade to Premium



















