drugset / Trial / NCT07657780

CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia

NCT07657780 ↗

Non-randomizedSequentialOpen-labelTreatment

Summary

This is an open-label, single-arm, dose-escalation IIT clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid nanoparticles targeting APOC3, in adult participants (18-65 years) with Severe Hypertriglyceridemia(sHTG).

Timeline

Start
2026-06-15
Primary completion
2028-03-15
Completion
2028-07-31

Drugs

EvaluationDrugModalityDoseRoute
Subject CS-121 CRISPR / gene editing 0.5 mg/kg —
Subject CS-121 CRISPR / gene editing 0.7 mg/kg —
Subject CS-121 CRISPR / gene editing 1 mg/kg —

Indications