CRISPR Therapeutics’ gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%
Title
Breakthrough:
CRISPR Therapeutics' Gene Editing Therapy Slashes LDL Cholesterol and Triglycerides by 80%
Keywords
- CRISPR
- Gene editing
- LDL cholesterol
- Triglycerides
- Cardiovascular disease
- PCSK9
- In vivo liver editing
- CTX310
- Single-dose therapy
- Clinical trial
- Lipid nanoparticle delivery
Key Facts
- CRISPR Therapeutics has reported early clinical trial results demonstrating that its gene-editing therapy, CTX310, achieved reductions of more than 80% in both LDL cholesterol (“bad” cholesterol) and triglyceride levels following a single dose23.
- The therapy uses in vivo gene editing to target specific genes in the liver—most notably genes like PCSK9, which regulates cholesterol metabolism, allowing the liver to more effectively remove cholesterol from the bloodstream45.
- This approach could represent a major advance over current cholesterol-lowering treatments, which typically require lifelong daily or periodic dosing (e.g., statins or injections)5.
- Early clinical data indicates a dose-dependent response, meaning higher doses led to greater reductions in LDL and triglycerides3.
- The treatment’s goal is to significantly lower the risk of heart disease, especially for patients with genetic forms of high cholesterol who do not fully respond to existing medicines5.
- If further trials confirm safety and efficacy, this one-time gene-editing therapy could potentially replace the need for ongoing cholesterol and triglyceride medication for many patients25.
- Ongoing clinical trials are still needed to establish long-term effects, durability, and safety in a larger and more diverse patient population234.
Summary Table
| Feature | Details |
|--------------------------------|--------------------------------------------|
| Therapy Name | CTX310 |
| Technology | In vivo CRISPR gene editing |
| Target | Genes regulating cholesterol (e.g., PCSK9) |
| Effect on LDL/Triglycerides | >80% reduction (single dose) |
| Clinical Status | Phase 1 (early data) |
| Potential Impact | Major advance over current chronic therapies|
| Future Steps | Dose escalation, expanded trials |
Sources:
2. https://endpts.com/crispr-therapeutics-gene-editing-therapy-reduces-bad-cholesterol-and-triglycerides-by-as-much-as-80/
3. https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-provides-first-quarter-2025-financial
4. https://crisprmedicinenews.com/news/the-latest-updates-from-the-gene-editing-clinical-trials-february-2025/
5. https://accessh.org/from-chairs-desk/progress-in-treating-high-cholesterol-clinical-trial-interim-results/