Harmful cholesterol levels cut in half with one-time gene editing drug in early trial
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Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the blood, according to new research presented Saturday at the annual meeting of the American Heart Association in New Orleans. Limited time: Save 25% on NBC News subscription Get exclusive reporting, live Q&As and ad-free reading. The Phase 1 clinical trial of 15 people was intended to show whether the experimental gene-editing therapy was safe to use in humans. It was, the researchers said.
AI Summary
Main Idea: A CRISPR Therapeutics gene-editing drug cut harmful cholesterol and triglycerides about in half in a small early trial, raising hopes for a one-time heart disease treatment.
Key Points:
- The Phase 1 study of 15 people was mainly meant to test safety, and researchers said the treatment appeared safe in the short term.
- One infusion of the drug, CTX310, lowered LDL cholesterol and triglycerides by about 50% in the highest-dose group.
- The drug targets the ANGPTL3 gene, which affects how the liver breaks down fats in the blood.
- Doctors said the results are promising, but long-term safety and larger trials are still needed before it can be used widely.
Impact on the People
Negative Impact
Long-term safety is still unknown, and a one-time gene edit could carry liver risks and high costs before broad use.
Positive Impact
The CRISPR drug could one day give people with very high cholesterol a lasting drop in heart disease risk with one treatment.
How did each entity affect people?
Rate how each entity in this article affected the American people.
- Major:CRISPR Therapeuticscompany
Candidate Entities12 candidates · 5 weighted vote min · 70% decision
- CRISPR TherapeuticscompanyMajorEvaluated
Company that makes the experimental drug and helped fund the study.
Include 0 (0%)Ignore 0 (0%)5 to threshold - American Heart Associationnonprofit / NGOMajorAI selected
Organization hosting the annual meeting where the research was presented.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Dr. Steven NissenpersonMajorAI selected
Cleveland Clinic investigator quoted about the trial’s results and significance.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Cleveland Cliniceducational / research institutionContextCandidate
Institution where a lead investigator serves and that is tied to the clinical research.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Dr. Elizabeth McNallypersonContextCandidate
Northwestern Feinberg School of Medicine geneticist and cardiologist quoted on the ANGPTL3 target and comparison to existing therapies.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Dr. Karol WatsonpersonContextCandidate
UCLA Health cardiologist quoted offering a cautious evaluation of the therapy.
Include 0 (0%)Ignore 0 (0%)5 to threshold - Dr. Nishant ShahpersonContextCandidate
Duke University Medical Center cardiologist quoted on why the approach is not ready for routine use.
Include 0 (0%)Ignore 0 (0%)5 to threshold
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