The Story Has Moved — Here's Where It Stands Now

When the Cleveland Clinic published its first 15-patient CRISPR cholesterol trial in the New England Journal of Medicine in November 2025, the medical world paid attention. That coverage focused on the promise.

This is the update. And it's more concrete.

According to NBC News, a second, expanded interim analysis — now covering 35 patients — has been published in the New England Journal of Medicine. The trial, which will ultimately enroll up to 85 participants, showed a single infusion of the highest dose cut LDL cholesterol by as much as 62 percent. That's up from the 49-50 percent drop reported in the original 15-patient cohort last November.

One shot. No daily pills. Potentially permanent.

The Patient Nobody's Talking About

NBC News profiled Christos Soteriou, a 51-year-old man from South Australia who needed a quadruple bypass at age 29. His father died of heart disease at 46. His son was diagnosed with elevated cholesterol at 14. He's had two heart attacks since the bypass.

Statins didn't work. Repatha — a newer, expensive injectable drug — didn't work either.

He enrolled in the CRISPR trial. His doctors' reaction afterward, in his words: "Jeez, it's better than it's ever been."

A real patient with a documented, severe family history of cardiovascular disease reported results that surprised his own cardiologist.

What the Numbers Actually Say Now

The original November 2025 data, presented at the American Heart Association's Scientific Sessions in New Orleans and published simultaneously in NEJM, covered 15 patients. According to the American Heart Association's own newsroom, those patients saw:

• LDL cholesterol drop by nearly 50 percent
• Triglycerides drop by about 55 percent
• Both reductions occurring within two weeks of the single infusion
• Effects sustained for at least 60 days of follow-up at that point

The updated 35-patient analysis — reported by the New York Times — extends that timeline significantly. A subgroup treated 18 months ago is still showing sustained LDL reductions. That's not 60 days. That's a year and a half.

The treatment is CTX310, developed by CRISPR Therapeutics — the same company behind the already-approved CRISPR therapy for sickle cell disease and beta thalassemia.

How It Works — In Plain English

Your liver produces a protein called ANGPTL3. That protein blocks the body's ability to clear LDL cholesterol and triglycerides from your blood. People born with a natural mutation that disables ANGPTL3 have low cholesterol and triglycerides their entire lives — and according to the American Heart Association, lower rates of heart disease with no apparent harmful effects.

CTX310 uses CRISPR-Cas9 — molecular scissors — packaged inside a fat particle and injected into the bloodstream. The particle targets liver cells. The scissors cut the ANGPTL3 gene. Gene turned off. Cholesterol drops.

This is not a drug you take repeatedly. The DNA edit is permanent.

What Mainstream Coverage Is Missing

The trial is still Phase 1. That means it was designed primarily to test safety, not effectiveness. The effectiveness numbers are encouraging signals, not confirmed outcomes.

The FDA requires 15 years of long-term safety monitoring for all CRISPR-based therapies, according to the American Heart Association. We have 18 months of data on a small subgroup. The gap between those two numbers is enormous.

Also missing from mainstream coverage: the side effects. According to the American Heart Association, three participants experienced infusion-related reactions — back pain and nausea — that resolved with medication. One participant with already-elevated liver enzymes saw a temporary further rise that lasted a few days before returning to normal without treatment. Minor, possibly. Worth knowing, absolutely.

Dr. Eric Rubin, editor in chief of the New England Journal of Medicine, told the New York Times that it is unusual for NEJM to publish such a preliminary result. He noted it "looks like it works pretty well." That's a respected editor acknowledging the bar was lowered because the potential upside is so large.

Why the Scale of the Problem Changes the Calculus

Heart disease kills nearly 800,000 Americans a year, according to the New York Times. That's not a rare disease. Most CRISPR therapies to date have targeted conditions affecting thousands of people, not hundreds of millions.

Dr. John H.P. Alexander, a cardiologist at Duke University who had no involvement in the study, told the New York Times: "We have these debates and new guidelines that we should be treating people earlier. A curative therapy would change the game."

Dr. Steven Nissen, chair of cardiovascular medicine at Cleveland Clinic and senior investigator on the trial, called it "a very big deal" — telling Time magazine it was "the first time anybody has ever edited a gene related to cholesterol metabolism and published results in a peer-reviewed journal."

Larger trials are already underway, according to NBC News. The next phase involves 200 patients.

What Comes Next

The jump from 15 patients to 35, with 18-month durability data now in hand, is a meaningful update — not just a rehash.

But 35 patients is not drug approval. It is not proof of long-term safety. And a one-time gene edit that permanently alters your DNA is not something to take lightly, regardless of how good the early numbers look.

If the 200-patient trial holds up, this changes cardiovascular medicine entirely. If it doesn't, we'll know soon enough. Either way, the data now is more compelling than it was six months ago.