A single infusion of an experimental CRISPR-Cas9 therapy was reported to safely lower LDL cholesterol by 52.5% and triglycerides by 47.8%, as measured 12 months after treatment.
What if one infusion could keep cholesterol down for a year? An experimental gene-editing treatment called CTX310 has shown early promise in people whose cholesterol and other blood fats remain high despite medication.
In a small Cleveland Clinic trial, patients who received the highest dose still had roughly half their original level of LDL, or “bad,” cholesterol a year later. The treatment works by switching off a gene in the liver.
That gene, called ANGPTL3, helps regulate blood fats. Switching it off can lower both LDL cholesterol and triglycerides, which are linked to heart disease. CTX310 delivers CRISPR-Cas9 editing machinery to the liver to make that change. CRISPR-Cas9 is a technology that allows researchers to modify specific regions of DNA.
Cholesterol reductions last a full year
The Phase 1 study was the first to test CTX310 in humans and included 15 patients with medication-resistant lipid disorders. Participants received a single infusion at doses ranging from 0.1 to 0.8 milligrams per kilogram of body weight, following pretreatment with corticosteroids and antihistamines. Researchers monitored safety and measured ANGPTL3, LDL cholesterol, and triglyceride levels.
Earlier findings had shown reductions in LDL cholesterol and triglycerides after two months. The new results examined whether those effects would last for 12 months. Reductions persisted across all doses through the year of follow-up. At the highest dose, LDL cholesterol was 52.5% below its pretreatment level, and triglycerides were 47.8% lower.
“Building upon the initial data presented in November 2025, the durability of the lipid-lowering effect was impressive,” said Dr. Luke Laffin, a Cleveland Clinic cardiologist and the study’s first author.
Tracking safety over the long term
No serious adverse events related to the therapy occurred during the one-year follow-up. The findings provide an early assessment of the treatment in a small group, with larger studies needed to investigate it further.
“It is encouraging that there were no serious safety events related to CTX310 in the trial and in the year following treatment. We look forward to continuing to investigate this therapy in a larger number of patients,” Laffin said.
Participants will undergo additional long-term safety follow-up planned for the next 15 years, as the FDA recommends for all gene-editing therapies.
Reference: “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310” by Luke J. Laffin, Stephen J. Nicholls, Russell S. Scott, Peter M. Clifton, Renate Koops, Ashish Sarraju, Shweta Singh, Qiuqing Wang, Kathy Wolski, Huansheng Xu, Jen Nielsen, Naimish Patel, Jason M. Duran and Steven E. Nissen, 27 August 2026, New England Journal of Medicine.
DOI: 10.1056/NEJMc2609825
Meeting: 2026 European Society of Cardiology
The study was funded by CRISPR Therapeutics AG; Zug, Switzerland. Dr. Laffin’s institution has received research funding from Crispr Therapeutics.
News – Curated by Amanda Scott, Alias Group Creative
Follow her on Bluesky
Study identifies new targets for antiviral drugs against coronaviruses
A study led by researchers at the University of California San Diego School of Medicine and University of California, Riverside (UCR) School of Medicine has identified new targets for antiviral drugs against coronaviruses, including [...]
“Nearly Everybody Had Given Up” – Scientists Finally Solve a 60-Year Chemistry Mystery
After more than six decades of failed attempts, chemists have achieved a breakthrough involving a class of molecules long considered too unstable to isolate. Chemists have succeeded in isolating a group of molecules that [...]
Depression Symptoms Improved in Just 48 Hours After a Single Psychedelic Dose
A single dose of psilocybin produced rapid antidepressant effects in a small Swedish clinical trial, with improvements lasting for weeks. A single dose of psilocybin, the psychedelic compound found in magic mushrooms, produced rapid [...]
GHCE Concept
From the preface of the book Global Health Care Equivalency in the Age of Nanotechnology, Nanomedicine and Artificial Intelligence, Edited by Frank Boehm: Since the publication of my first book (Nanomedical Device and Systems [...]
Human Bodies Are Producing Two Billion Tons of CO2 a Year
A new study reveals that the amount of carbon dioxide produced by human metabolism has increased dramatically over three decades. The carbon dioxide produced by human bodies as they convert food into energy increased [...]
A Hidden DNA Mechanism Could Explain How 20% of Human Cancers Develop
Researchers have identified an unexpected molecular vulnerability in cancers carrying BAF mutations. Mutations found in roughly 20% of human cancers may promote tumor growth by changing how DNA is organized inside cells. Researchers have [...]
Ebola kills over 5,300 in fastest-growing outbreak on record
Africa’s latest Ebola outbreak has killed nearly 5,400 people, the vast majority of them in the Democratic Republic of Congo (DRC), according to the continent’s top health agency. Africa Centres for Disease Control and [...]
China makes early progress in using sound waves to stimulate damaged brains
Chinese researchers are testing whether focused ultrasound can help restore consciousness in patients with severe brain injuries, adding to a growing international effort to use sound waves to stimulate deep brain structures without surgery. [...]
Micro-drones fly through the body thanks to ultrasound
At the EPFL, micro-drones have been developed that can be moved without a motor or battery.No motors, no batteries, no electronics on board, just small cavities that convert sound waves into thrust: this is [...]
A Single Enzyme Could Hold the Key to Treating a Devastating Brain Disorder
Blocking an enzyme’s production restored brain growth and healthy behaviors in mice engineered to develop Angelman syndrome, pointing to a possible treatment target. Children with Angelman syndrome begin to show difficulties with thinking and [...]
Scientists Develop a Clever New Way To Kill Cancer Cells From the Inside
Scientists have developed an experimental strategy that exploits a chemical byproduct of cancer cells to produce anticancer drugs directly inside them. Scientists in Japan have developed an experimental method that uses a chemical produced [...]
Our books are available worldwide!
Online Sellers other than Amazon, Routledge, and IOPP Indigo Global Health Care Equivalency in the Age of Nanotechnology, Nanomedicine and Artifcial Intelligence Global Health Care Equivalency In The Age Of Nanotechnology, Nanomedicine And Artificial [...]
After only a short training, people in a coma managed to send targeted signals
Researchers have succeeded in communicating with brain-injured patients who are unconscious. They see enormous potential in this for future treatments. Patients who lie motionless in bed after a severe brain injury and do not [...]
MIT Scientists Reprogram Immune Cells Inside the Body To Destroy Cancer
Natural killer cells reprogrammed inside mice eliminated lymphoma tumors and helped damaged livers heal, though whether this approach could help people with cancer or autoimmune disease remains unknown. To make a cancer treatment from [...]
Melanoma mRNA Vaccine Shows Promise in Preventing Disease Recurrence
Key takeaways: Phase 3 trial finds personalized intismeran plus pembrolizumab may better prevent high-risk melanoma recurrence after surgery The trial enrolled 1,137 patients and builds on phase 2 results showing about half the recurrence [...]
Scientists Discover a Powerful New Weapon Against a Dangerous Gut Infection
The promise of an experimental treatment for C. difficile gut infections lies partly in the bacteria it leaves alive, according to early laboratory and mouse research. Broad-spectrum antibiotics can disrupt the gut’s microbial community, [...]
