European Society of Cardiology (ESC) Congress 2026 | News | Updates | iPharmaCenter

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CRISPR Therapeutics’ CTX310 Shows Durable Lipid Control One Year After Treatment

CRISPR Therapeutics presented new one-year follow-up results of investigational gene editing therapy CTX310 produced lasting reductions in a protein linked to cholesterol and triglyceride buildup.

A Gene Editing Approach to Lipid Disorders

CTX310 is an investigational in vivo CRISPR-Cas9 therapy designed to edit a gene called ANGPTL3 inside liver cells.

This gene plays a central role in regulating how the body processes fats in the bloodstream.

By reducing ANGPTL3 activity, the therapy aims to lower both low-density lipoprotein cholesterol and triglycerides, two markers strongly associated with cardiovascular disease.Unlike medicines that must be taken repeatedly, CTX310 is designed to produce its effect through a single intravenous infusion.

One-Year Results Show Lasting Effect

At the highest dose tested, 0.8 milligrams per kilogram based on lean body weight, patients experienced substantial and sustained reductions across all three measures one year after treatment.

• Average ANGPTL3 levels fell by 79% from baseline, with reductions reaching as high as 89% in some patients.

• Triglycerides dropped by an average of 48%, with a maximum reduction of 78%.

• LDL cholesterol decreased by an average of 53%, with some patients seeing reductions as large as 84%.

Researchers said these findings build on earlier results already reported for the therapy and reinforce that the biological effect of a single infusion can remain durable well beyond the initial treatment period.

Trial Design and Patient Population

The Phase 1a portion of the study was an open-label, dose-escalation trial. Researchers tested doses of CTX310 ranging from 0.1 to 0.8 milligrams per kilogram in patients across four different lipid disorder categories.

These included people with homozygous familial hypercholesterolemia, severe hypertriglyceridemia, heterozygous familial hypercholesterolemia, and mixed dyslipidemia involving both elevated triglycerides and LDL cholesterol.

To qualify, participants needed triglyceride levels above 150 milligrams per deciliter or LDL cholesterol above 100 milligrams per deciliter, or above 70 milligrams per deciliter for those with established atherosclerotic cardiovascular disease, despite following standard treatment guidelines.

Most participants were already taking statins, ezetimibe, or both. About 40% were also using PCSK9 inhibitors, reflecting how difficult their lipid levels were to manage using conventional therapies.

The trial’s main goal was to assess safety and tolerability. Changes in ANGPTL3, triglycerides, and LDL cholesterol were tracked as secondary measures.

What the Findings Could Mean

Because CTX310 is designed to work through a single administration rather than ongoing dosing, researchers see the durability of these results as an important signal for its potential role in long-term cardiovascular risk management.

Investigators noted that all participants completed at least one year of follow-up as of the data analysis, and additional long-term safety monitoring is planned to continue for an extended period, consistent with regulatory expectations for gene editing therapies.

While these results come from an early-phase trial with a small number of patients, the sustained reductions in ANGPTL3, triglycerides, and LDL cholesterol suggest that a single treatment could offer a meaningful and lasting option for people whose lipid disorders remain difficult to control with existing medicines.

Camzyos Sustains Symptom and Heart Function Gains Through Five Years, New Data Show

Bristol Myers Squibb presented new long-term and real-world evidence supporting Camzyos, also known as mavacamten, for people living with symptomatic obstructive hypertrophic cardiomyopathy at the European Society of Cardiology Congress 2026 in Munich.

The findings reinforce that treatment benefits with Camzyos can be sustained for up to five years, adding to what the company describes as the most extensive clinical evidence base among cardiac myosin inhibitors.

Longest Follow-Up Study of Its Kind

The centerpiece of the presentation was data from the EXPLORER-LTE cohort within the broader MAVA-LTE study. This research represents the largest and longest evaluation to date of patients with obstructive hypertrophic cardiomyopathy treated with Camzyos.

EXPLORER-LTE is a single-arm, open-label, dose-blinded extension of the earlier Phase 3 EXPLORER-HCM trial. A total of 231 patients from the United States, Europe, and Israel who completed the original study chose to continue into this long-term follow-up.

Sustained Improvements at Five Years

At 252 weeks of continued treatment, patients showed meaningful reductions in the heart obstruction associated with this condition.

Compared with levels recorded when the extension study began, the average resting left ventricular outflow tract gradient fell by 38.7 millimeters of mercury, while the Valsalva gradient decreased by 55.6 millimeters of mercury.

Almost all patients, 97.4%, reached a Valsalva gradient at or below 30 millimeters of mercury, the clinical threshold used to define obstruction in this disease.

More than two-thirds of participants, 69.6%, improved by at least one New York Heart Association functional class, and 59.2% became free of symptoms. Average left ventricular ejection fraction declined slightly by 10.2% but stayed within the normal range, indicating that heart pumping function remained stable.

Bristol Myers Squibb reported that no new safety concerns emerged beyond what was already documented in the original EXPLORER-HCM trial.

Real-World Evidence Reinforces Trial Findings

Beyond the extension study, additional research presented at the meeting examined how Camzyos performs outside controlled clinical trials.

Two analyses from COLLIGO-HCM, a global retrospective study using real-world data, found that the medicine’s effectiveness and safety profile in routine clinical practice were consistent with results seen in earlier studies. Patients in this real-world setting experienced symptom improvement and reduced outflow tract obstruction.

A separate observational registry analysis conducted in Germany produced similar findings, with patients experiencing functional class improvements that mirrored outcomes from other real-world studies. Together, these results suggest that benefits seen in formal clinical trials extend into everyday medical practice across different patient populations.

Understanding the Broader Burden of the Disease

A healthcare resource utilization study conducted in Sweden was also presented, offering insight into the wider impact of hypertrophic cardiomyopathy and its obstructive form on patients and health systems.

The analysis underscored the importance of timely diagnosis and consistent long-term care for people living with this condition, given the disease’s ongoing burden on health resources.

About Camzyos and it's mechanism of action

Camzyos, known generically as mavacamten, is a selective and reversible inhibitor of cardiac myosin. It is approved by regulatory authorities in more than 60 countries and regions worldwide.

In the United States, Camzyos is approved for adults with symptomatic New York Heart Association class II to III obstructive hypertrophic cardiomyopathy to help improve functional capacity and reduce symptoms. In the European Union, it carries a similar approved use for adult patients with symptomatic disease in the same functional classes.

The medicine works by targeting the excessive heart muscle contraction that underlies this form of cardiomyopathy. By reducing that contractility, Camzyos can ease outflow tract obstruction, improve the heart’s energy efficiency, and lower internal cardiac pressures. It may be used with or without other background therapies, including in patients who are newly diagnosed.

Bristol Myers Squibb said Camzyos has now been prescribed by more than 5,000 healthcare providers to over 25,000 patients in the United States alone, reflecting its growing role as a standard treatment option for this rare heart condition.

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Repatha Cuts Death Risk in High-Risk Patients Before a First Heart Attack or Stroke

Amgen has reported new findings showing that Repatha, known generically as evolocumab, reduced the risk of death in high-risk adults who had not yet experienced a heart attack or stroke, when added to statins or other cholesterol-lowering treatments.

A First for PCSK9 Inhibitors in Primary Prevention

The analysis included more than 12,000 patients considered to be at high cardiovascular risk but without a documented history of heart attack or stroke.

Among this group, Repatha reduced the overall risk of death by 20%. The reduction applied to both deaths from any cause and deaths specifically linked to cardiovascular events.

According to Amgen, this marks the first time a PCSK9 inhibitor has demonstrated a reduction in all-cause mortality specifically within this high-risk primary prevention population in a Phase 3 trial.

Benefit Emerged Over Time and Persisted

The mortality benefit did not appear immediately. Researchers observed that the reduction in deaths began to separate from placebo at approximately 1.5 years into treatment.

This benefit continued through a median follow-up period of 4.6 years, suggesting that sustained treatment may be necessary to achieve the full protective effect.

Amgen said the mortality reduction likely reflects the drug’s ability to help patients avoid the serious cardiovascular events, such as heart attacks and strokes, that often lead to long-term health decline and increased risk of death.

Additional Trial Findings Reinforce Early Treatment

Two further analyses from the VESALIUS-CV trial added context to the mortality results.

One analysis found that Repatha reduced the risk of a first cardiovascular event, as well as subsequent and total cardiovascular events over time, indicating a cumulative benefit from continued treatment.

A separate analysis showed that the reduced risk of heart attack associated with Repatha became apparent as early as six months after starting treatment. This early benefit was primarily driven by fewer heart attacks caused by the rupture of atherosclerotic plaque, as well as fewer large-scale infarctions.

Researchers reported that these benefits were consistent across key patient subgroups, reinforcing the broader case for earlier and more aggressive LDL cholesterol lowering in people identified as high risk.

Real-World Data Reveal Treatment Gaps

Alongside the clinical trial results, Amgen presented findings from two global real-world studies examining how lipid management is handled in everyday medical practice.

The first study found that many patients with coronary artery disease, but without a prior heart attack or stroke, were not receiving adequate treatment. Researchers noted low rates of treatment initiation, insufficient escalation of therapy, and inconsistent monitoring of LDL cholesterol levels.

The second study identified a gap in care between men and women.

High-risk women without a prior heart attack or stroke were less likely than men to receive intensive lipid-lowering therapy and were also less likely to reach recommended LDL cholesterol targets, a pattern observed across multiple regions.