15
Participants
Start Date
September 30, 2025
Primary Completion Date
December 31, 2026
Study Completion Date
December 31, 2026
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).
The First Affiliated Hospital of Anhui Medical University, Hefei
Lead Sponsor
The First Affiliated Hospital of Anhui Medical University
OTHER
CorrectSequence Therapeutics Co., Ltd
INDUSTRY