RecruitingEarly Phase 1NCT07176923

CS-121 APOC3 Base Editing in FCS

A Clinical Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Adults With Familial Chylomicronemia Syndrome

Sponsor

CorrectSequence Therapeutics Co., Ltd

Enrollment

15 participants

Start Date

Oct 15, 2025

Study Type

INTERVENTIONAL

Conditions

Familial Chylomicronemia Syndrome (FCS)

Summary

This is an open-label, single-arm, dose-escalation Phase I 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 (18-55 years) with familial chylomicronemia syndrome (FCS).

Eligibility

Min Age: 18 YearsMax Age: 55 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called CS-121 for people with familial chylomicronemia syndrome (fcs). The study is currently recruiting participants at 1 location. People eligible for this study include aged 18 Years to 55 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALCS-121

CS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes. In this study, participants receive a single intravenous infusion of CS-121 at escalating dose levels. The investigational product is designed to reduce ApoC3 protein expression and serum triglyceride levels in adults with familial chylomicronemia syndrome (FCS).

Locations(1)

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China

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NCT07176923