An Experimental Single-Time Treatment Slashed Cholesterol for a Year

Live updates

1. 14d ago

   CRISPR Gene Editing Shows Promise in Reducing Cholesterol

   CRISPR Therapeutics' CTX310, a single-time gene-editing treatment targeting the ANGPTL3 gene, has shown to safely and continuously lower cholesterol and triglycerides for one year in a Phase 1a trial. This approach aims to provide a permanent solution for high cholesterol by editing a specific gene, eliminating the need for daily medication. The treatment has yielded promising results, prompting the company to pursue the highest dose of the lipid-lowering treatment.

   Why it matters

   High cholesterol is a significant risk factor for cardiovascular disease, which is a leading cause of death worldwide. Current treatments often require daily medication, and their effectiveness can vary. A single-time gene-editing treatment could revolutionize the management of high cholesterol and cardiovascular disease. The ANGPTL3 gene plays a crucial role in lipid metabolism, making it a promising target for gene editing.

   What is confirmed

   • CRISPR Therapeutics' CTX310 targets the ANGPTL3 gene to lower cholesterol and triglycerides.
   • CTX310 has shown to safely and continuously lower cholesterol and triglycerides for one year in a Phase 1a trial.
   • The treatment aims to provide a permanent solution for high cholesterol by editing a specific gene.
   • Immediate initiation of evolocumab after a heart attack significantly improved LDL-cholesterol goal achievement.

   What to watch next

   • CRISPR Therapeutics' plans for pursuing the highest dose of CTX310
   • Results of further clinical trials to confirm the efficacy and safety of CTX310
   • Potential FDA approval and market availability of CTX310
   Sources used for this update (8)

   1. oncodaily.com — Mel Mann: A 1-time CRISPR Shot, that Keeps Lipids Down for a Year
   2. jen.jiji.com — Serie A, today Lecce-Roma - Live
   3. jen.jiji.com — Political poll, Fratelli d'Italia drops and Pd grows. Vannacci overtakes Forza Italia
   4. medicalxpress.com — Immediate evolocumab after a heart attack improves LDL-cholesterol goals but not early cardiovascular outcomes
   5. medicalxpress.com — LDL-cholesterol lowering medication linked to lower mortality in high-risk patients
   6. jen.jiji.com — Bergamo, motorcycle against a truck: a child dead, father serious
   7. jen.jiji.com — Tupac Shakur, the truth after 30 years: Duane Davis found guilty of murder
   8. www.news-medical.net — GLP-1 drugs have benefits beyond weight loss. Could microdoses preserve them?
   confidence 90%
2. 16d ago

   CRISPR Gene-Editing Therapy CTX310 Lowers Cholesterol for One Year

   CRISPR Therapeutics reported Phase 1a trial data for CTX310, a single-time gene-editing treatment that safely and continuously lowered cholesterol and triglycerides for one year. The therapy targets the ANGPTL3 gene to provide deep and durable editing. Following these promising results presented at the ESC Congress 2026, CRISPR Therapeutics intends to pursue the highest dose of the lipid-lowering treatment. This approach aims to eliminate high cholesterol permanently by snipping a specific gene rather than relying on daily or periodic medication.

   Why it matters

   Traditional cholesterol management often requires lifelong adherence to medication. This experimental therapy uses CRISPR technology to modify genetic drivers of high lipids. Successful long-term results could shift the treatment paradigm from chronic management to a one-time cure.

   What is confirmed

   • The CTX310 gene-editing therapy safely and continuously lowered cholesterol and triglycerides after one year in a first-in-human trial.
   • CTX310 demonstrates deep and durable ANGPTL3 editing and LDL lowering.
   • CRISPR Therapeutics plans to pursue the highest dose of the lipid-lowering therapy.

   What to watch next

   • Results from higher-dose CTX310 trials
   • Long-term safety data beyond the one-year mark
   • Regulatory filing timelines for CTX310
   Sources used for this update (11)

   1. The New York Times — An Experimental Single-Time Treatment Slashed Cholesterol for a Year
   2. KCRA — With the snip of a gene, scientists hope to erase high cholesterol for life
   3. Cleveland Clinic Newsroom — Cleveland Clinic First-In-Human Trial of CRISPR Gene-Editing Therapy Shown to Safely and Continuously Lower Cholesterol and Triglycerides After One Year
   4. GuruFocus — CRSP Shares React to Promising CTX310 Trial Data Amid High Price-to-Sales Valuation
   5. Investing.com Canada — CRISPR Therapeutics reports one-year data for gene therapy CTX310 By Investing.com
   6. CNN — Snipping a gene to reduce bad cholesterol for life looks possible
   7. Time Magazine — A One-Time Treatment for High Cholesterol Shows Promising Results
   8. Endpoints News — #ESC26: CRISPR Therapeutics to pursue highest dose of its lipid lowering gene editing therapy
   9. Yahoo Finance — CRISPR Therapeutics Presents Phase 1a Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and LDL Lowering at ESC Congress 2026
   10. www.news-medical.net — Immediate evolocumab use after heart attack improves cholesterol goals but not survival
   11. www.news-medical.net — Cholesterol-Lowering Medication Reduces Major Cardiovascular Events By 30% In Older People Without Known Cardiovascular Disease
   confidence 100%