This is a Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and Adolescents (4-18 years) With Hyperchylomicronemia
Male or female participants aged 4 years ≤ age < 18 years.
Severe hypertriglyceridemia (sHTG), defined as a triglyceride (TG) level ≥ 500 mg/dL.
Confirmed diagnosis of genetically inherited FCS via genetic testing, or clinically diagnosed FCS plus persistent chylomicronemia.
Failure to achieve adequate TG control, For participants under 8 years of age, the investigator determine at their discretion whether prior lipidlowering therapy has been administered.
Currently participating in other interventional clinical studies, or having an insufficient washout period of less than 5 half-lives or 30 days (whichever is longer) since the last administration of other investigational drugs.
Used antisense oligonucleotide (ASO)-based or small interfering RNA (siRNA)-based lipid-lowering drugs targeting APOC3 within 3 months prior to study drug administration.
History of acute pancreatitis within 1 month before dosing.
Patients who underwent major surgery within 3 months prior to study drug administration and are judged by the investigator as unsuitable for receiving the study drug, due to potential intolerance to adverse events such as cytokine release storm.
Sequential
CS-121
Biological/VaccineCS-121 is a in vivo base editing therapy formulated in lipid nanoparticles for targeted editing of the APOC3 gene in hepatocytes.
Dose-limiting toxicities (DLTs)
The incidence and severity of treatment-emergent adverse events (TEAEs)
Changes in serum triglyceride (TG) levels
Changes in serum ApoC3 levels from baseline
Concentrations of the active components of CS-121 (sgRNA and mRNA)
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Shanghai Jiao Tong University School of Medicine
Lead sponsor
CorrectSequence Therapeutics Co., Ltd
Collaborator
