Recent Updates
Recently added Catalysts

CTX310

Phase 1

Cardiovascular | Small molecule | Cardiovascular |CRISPR Therapeutics AG|Last Updated: Jul 23, 2026

Target and mechanism

Molecular targetANGPTL3
Target classProtein
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment90

FDA Designations

No designations recorded

Clinical trial landscape

CTX310 · 1 trial · 10 indications

Phase 1 1
NCT07491172A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory DyslipidemiasCardiovascular
RECRUITING90 Analytics
PHASE1RECRUITING
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
CardiovascularUnlock trial analytics

Study Endpoints

Primary Endpoints

To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months

Incidence of dose-limiting toxicities and frequency of adverse events

Secondary Endpoints

To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Over 12 months, compared to baseline
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CTX310EXPERIMENTALSubjects will receive an intravenous (IV) infusion.

Interventions

NameTypeDescription
CTX310DRUGCTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites19

Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL i...

Countries:United StatesAustraliaNew ZealandUnited Kingdom
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

LOWJul 24, 2026NCT07491172lastUpdatePostDate: changed
LOWJul 24, 2026NCT07491172lastUpdatePostDate: changed
LOWJul 20, 2026NCT07491172lastUpdatePostDate: changed
LOWJul 20, 2026NCT07491172lastUpdatePostDate: changed

Frequently asked questions about CTX310

What is CTX310 used for?

CTX310 is an investigational small molecule being developed for cardiovascular and metabolic conditions, specifically refractory dyslipidemias. It is being studied in participants with lipid disorders including hypertriglyceridemia, heterozygous and homozygous familial hypercholesterolemia, severe hypertriglyceridemia, mixed hyperlipemia, and hypercholesterolaemia.

What does CTX310 target?

CTX310 targets ANGPTL3, a protein involved in lipid metabolism. By targeting ANGPTL3, the drug is being evaluated for its potential to address refractory dyslipidemias, which are characterized by abnormal levels of lipids in the blood.

Who is developing CTX310?

CTX310 is being developed by CRISPR Therapeutics AG, a biopharmaceutical company traded on the stock exchange under the ticker CRSP. The company is conducting clinical research to evaluate the safety and tolerability of CTX310 in patients with refractory dyslipidemias.

What phase is CTX310 in?

CTX310 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials to assess its safety and tolerability in humans.

What clinical trials is CTX310 in?

CTX310 is being evaluated in a Phase 1 clinical trial with the identifier NCT07491172. This trial is titled 'A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias' and is currently recruiting participants. The trial is an uncontrolled, open-label study enrolling approximately 90 participants.

Where is the CTX310 clinical trial being conducted?

The Phase 1 clinical trial of CTX310 is being conducted in multiple countries, including the United States, Australia, New Zealand, and the United Kingdom. The trial enrolls adults aged 18 years and older, and it is open to all sexes. The study does not include healthy volunteers.