A single injection capable of permanently lowering “bad” cholesterol: the promise is making the rounds on social networks. Behind the announcement effect, very real clinical trials are exploring CRISPR gene editing, between therapeutic hope and major ethical questions.
In recent months, viral publications have claimed that a single injection would have “permanently” replaced a lifetime of cholesterol medications. The message is seductive, almost revolutionary: no more daily tablets, no more statins, but a targeted genetic correction, carried out once and for all. Behind this simplified story, however, lies a more nuanced scientific reality, based on experimental work still at an early stage.
The technology in question is based on CRISPR-Cas9 gene editing, a tool for precisely modifying DNA. In the case of cholesterol, researchers mainly target the PCSK9 gene, expressed in the liver. This gene plays a key role in the regulation of LDL cholesterol: when active, it limits the elimination of LDL by liver cells. However, for around fifteen years, we have known that certain people carrying natural mutations that inactivate PCSK9 have very low LDL levels, with no apparent deleterious effect, and a greatly reduced cardiovascular risk.
It was on this observation that researchers relied on to first develop anti-PCSK9 antibodies, then, more recently, a radically different approach: deactivating the gene itself. In 2023, an international team published in the New England Journal of Medicine early results from a phase 1 clinical trial using an in vivo CRISPR therapy, called VERVE-101. This therapy is based on a system of base editinga more precise version of CRISPR, administered by intravenous injection and specifically targeting liver cells.
Preliminary results showed a reduction in LDL cholesterol of up to around 50% in some participants, after a single injection, with a persistent effect over several months. These figures immediately generated considerable enthusiasm, as they are comparable, if not superior, to those obtained with regularly administered statins or PCSK9 inhibitors. However, it is crucial to remember that these trials involved a very small number of patients, suffering from severe hypercholesterolemia and at high cardiovascular risk, and that they aimed primarily to evaluate the safety of the treatment, not its long-term effectiveness.
In parallel, another approach, called CTX310, this time targets the ANGPTL3 gene, also involved in the regulation of blood fats. Presented at the end of 2025 at the American Heart Association congresses, this experimental CRISPR-Cas9 type therapy was administered once by intravenous infusion to fifteen volunteers suffering from difficult-to-treat lipid disorders. Preliminary results showed an average reduction in LDL of about 50%, sometimes as much as 70%, as well as a decrease of about 55% in triglycerides, with no serious adverse effects reported. The effects appeared within the first two weeks and were maintained for at least sixty days in the available follow-ups. These data confirm the feasibility of lasting genetic correction of lipid mechanisms, but they remain limited by the size and duration of the study.
This is where the viral narrative becomes misleading. Talking about “permanent replacement” of daily treatments is scientifically premature. Changing the DNA of liver cells is, by definition, irreversible. However, any irreversible therapy imposes extremely high safety requirements. The main risks concern so-called “off-target” effects: unintentional modifications of DNA elsewhere in the genome, likely to lead to unforeseen consequences, notably cancerous. Even if new generations of CRISPR are much more precise, clinical experience remains very limited.
Furthermore, cholesterol is not a simple monogenic pathology in the majority of cases. Common hypercholesterolemia results from complex interactions between genetics, diet, lifestyle and other metabolic diseases. Disabling PCSK9 or ANGPTL3 does not address dietary imbalances, chronic inflammation, or other cardiovascular risk factors such as smoking, hypertension, or diabetes. Gene therapy could therefore concern, at best, very targeted populations.
The researchers themselves remain cautious. In their publications, they emphasize the need for prolonged follow-ups over several years, as well as phase 2 and 3 trials involving a much larger number of patients. Regulatory agencies, such as the FDA or the EMA, also impose particularly strict criteria for any therapy that modifies the genome permanently.
The enthusiasm is nevertheless understandable. If these approaches are confirmed, they could transform cardiovascular prevention in certain very high-risk patients, in particular those who do not tolerate current treatments or present severe genetic forms. More broadly, this work opens the way to preventive medicine based on the biological correction of upstream pathological mechanisms, rather than on the chronic management of symptoms.
But between scientific promise and clinical reality, the road remains long. At this stage, CRISPR has not replaced statins: it has simply opened the door to a medicine where prevention would occur, perhaps one day, by correcting the gene rather than taking the pill. It remains to be seen whether society will accept lasting modification of the genome to prevent a risk rather than to treat a disease. A scientific question, but also an ethical one.
Sources
– Laffin, LJ, Nicholls, SJ et al. CRISPR-Cas9 Gene Editing Targeting ANGPTL3: First-in-Human, Phase 1 Results. New England Journal of Medicine, 2025.
– Musunuru, K. et al. In Vivo Base Editing of PCSK9 in Humans. New England Journal of Medicine, 2023.
– Musunuru, K. et al. Base Editing for Cholesterol Reduction in Monkeys and Human Cells. Nature, 2021.
– Cohen, J.C., Boerwinkle, E., Mosley, T.H. Jr., & Hobbs, H.H. Sequence Variations in PCSK9, Low LDL, and Protection against Coronary Heart Disease. New England Journal of Medicine, 2006.
