CTX310: how it works, status and trials
Phase 1Gene editingLowering triglycerides
CTX310 is an investigational one-time infusion that uses CRISPR to cut the ANGPTL3 gene in the liver, imitating natural gene variants that protect people. It is in phase 1.
In technical terms: Cuts the ANGPTL3 gene to create a permanent loss-of-function, mimicking protective natural variants.
The highlighted badge marks where CTX310 acts. Open the full map or compare every therapy.
At a glance
- Acts on
- ANGPTL3 gene · liver
- How it is given
- CRISPR-Cas9 mRNA + guide RNA in lipid nanoparticle · single IV infusion
- What the main trial found
- ANGPTL3 −73% to −80% at 0.7–0.8 mg/kg (phase 1, n = 15)
- Status (curated October 25, 2026)
- Phase 1
Where it fits in the body
Triglyceride-rich lipoproteins
VLDL from the liver and chylomicrons from the gut carry triglyceride. Lipoprotein lipase (LPL) on capillary walls breaks it down. ANGPTL3 and apoC-III act as brakes on this process. Removing either brake (11–15) lowers triglycerides, and ANGPTL3 blockade also lowers LDL-C without needing LDL receptors, which matters in homozygous FH.
RNA and gene-editing therapies
These drugs act before a protein is made. siRNA and antisense drugs are attached to GalNAc, which hepatocytes take up, and they destroy one specific mRNA. A dose lasts weeks to months. Gene editors (10, 13) make a permanent single-dose change to the DNA and are in early trials.
Trials and registry status
From ClinicalTrials.gov, PubMed and openFDA, refreshed monthly. Last run October 27, 2026.
CTX310 from CRISPR Therapeutics
Stage: Phase 1. 1 active of 1 registered trials. 0 new papers in the last 31 days.
- NCT07491172 (NCT07491172): recruiting, primary completion 2027-06
Evidence
- Phase 1, NEJM 2025 (PubMed 41211945)
Common questions
How does CTX310 work?
CTX310 is an investigational one-time infusion that uses CRISPR to cut the ANGPTL3 gene in the liver, imitating natural gene variants that protect people. It is in phase 1. In technical terms: Cuts the ANGPTL3 gene to create a permanent loss-of-function, mimicking protective natural variants. It acts on ANGPTL3 gene · liver.
How much does CTX310 lower cholesterol or other lipids?
ANGPTL3 −73% to −80% at 0.7–0.8 mg/kg (phase 1, n = 15). Individual results differ, and a clinician can say what to expect for one person.
Is CTX310 approved, and what is the status?
Phase 1 (curated October 25, 2026). Registry stage as of October 27, 2026: CTX310, Phase 1.
How is CTX310 taken?
CRISPR-Cas9 mRNA + guide RNA in lipid nanoparticle · single IV infusion.
Read more
Related therapies
- Evinacumab (Evkeeza): Approved 2021 (HoFH)
- Olezarsen (Tryngolza) and volanesorsen: Olezarsen approved 2024; volanesorsen EU only
- Plozasiran (Redemplo): Approved Nov 2025 (FCS)
- Fibrates (fenofibrate): Approved (generic)
Educational, not medical advice. Mechanism and status text curated October 25, 2026; registry data refreshed October 27, 2026. Registries can lag company announcements. Effects are placebo-adjusted results from the main published trial, not head-to-head comparisons. Investigational agents are available only in clinical trials. Preval Health does not practice medicine; licensed clinicians in a partner network provide care. If you have chest pain or signs of a stroke, call 911.