Archive · 10 Nov 2025
New Cholesterol Pills and Gene Therapies Show Promise
Doctors have medicines to help people with high cholesterol, which is like gunk building up in your blood vessels and can lead to heart problems.
The most common medicines are called statins, but they don't always work well enough for everyone.
Now, scientists are creating new ways to fight bad cholesterol.
One is a new pill that works a bit like an injection that's already available; both of these target a specific protein to help your body clear out the bad stuff.
This new pill could lower bad cholesterol by up to 60%.
There's also a new idea using special technology called gene editing.
This technology aims to permanently fix a gene that causes high cholesterol, and early tests show it can significantly lower cholesterol and another type of fat called triglycerides.
While these new treatments are exciting, doctors are still checking to make sure they are safe and can truly prevent heart attacks and strokes.
Some newer options might also be very expensive, and a trial for a similar gene therapy had a serious setback recently.
New experimental drugs and therapies are emerging to combat high cholesterol, offering alternatives for patients not fully responsive to statins.
A Merck pill, enlicitide decanoate, reduced LDL cholesterol by up to 60% in Phase 3 trials.
Amgen's injectable drug Repatha showed a 25% reduction in cardiovascular events in high-risk patients.
A gene-editing therapy, CTX310, cut cholesterol by 49% and triglycerides by 55% in early trials.
While promising, newer treatments face hurdles of cost, further safety evaluations (especially for gene therapies), and proving long-term cardiovascular event prevention.
- Who
- Researchers, cardiologists, and patients participating in clinical trials for new cholesterol-lowering drugs and therapies.
- What
- Emerging cholesterol-fighting drugs and therapies, including a PCSK9 pill (enlicitide), an injectable PCSK9 drug (Repatha), and a gene-editing therapy (CTX310), are showing significant reductions in LDL cholesterol and cardiovascular event risks.
- When
- Latest study results were presented recently at the American Heart Association's annual meeting; some drug approvals are anticipated early next year, with gene therapies possibly by the 2030s.
- Where
- Clinical trials and presentations at the American Heart Association's annual meeting; specific locations for drug development and patient trials vary.
- Why
- To provide more effective options for patients whose LDL cholesterol remains high despite statin treatment, aiming to further reduce the risk of heart disease, stroke, and other cardiovascular events.
Potential Benefits
Existing Concerns
Treatment Options
Potential Benefits
Newer therapies, including pills and gene editing, offer more options for patients with high cholesterol, especially those not adequately treated by statins.
Existing Concerns
Statins, diet, and exercise are sufficient for most patients; newer therapies can be very expensive and are still in early stages of testing for long-term outcomes like preventing heart attacks.
Delivery Method
Potential Benefits
Oral pills and potential one-time gene therapies offer convenience over current twice-yearly or more frequent injections.
Existing Concerns
Injectable PCSK9 inhibitors are already available and proven, while gene therapies carry risks, as evidenced by a recent death in a related study, and require extensive further testing.
- Drug Names Mentioned
- Leqvio, Repatha, enlicitide decanoate, ezetimibe, CTX310
- Cholesterol Reduction (Enlicitide)
- Up to 60% reduction in LDL cholesterol
- Cholesterol Reduction (Repatha)
- Reduced risk of cardiovascular events by 25%
- Cholesterol Reduction (CTX310)
- 49% reduction in cholesterol, 55% reduction in triglycerides
- Mechanism (PCSK9 drugs)
- Blocks PCSK9 protein to help liver clear bad cholesterol
- Mechanism (Gene Editing)
- Knocks out ANGPTL3 gene to lower cholesterol and triglycerides
- Status of Gene Therapy
- Early-stage studies; CTX310 showed promise but related therapy study is on hold due to death