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).
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Treatment-Emergent Adverse Events (TEAEs)
Timeframe: From the start of dosing through approximately 10 months of follow-up
Dose-Limiting Toxicities (DLTs)
Timeframe: Number and type of dose-limiting toxicities (DLTs), defined as CTCAE grade ≥3 adverse events considered possibly, probably, or definitely related to CS-121 and persisting ≥7 days, or other adverse events/laboratory abnormalities that lead to suspension o