CS-121 APOC3 Base Editing in FCS

EARLY_PHASE1Not yet recruitingINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

September 30, 2025

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions
Familial Chylomicronemia Syndrome (FCS)
Interventions
BIOLOGICAL

CS-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).

Trial Locations (1)

Unknown

The First Affiliated Hospital of Anhui Medical University, Hefei

All Listed Sponsors
collaborator

The First Affiliated Hospital of Anhui Medical University

OTHER

lead

CorrectSequence Therapeutics Co., Ltd

INDUSTRY