drugset / Trial / NCT07176923
CS-121 APOC3 Base Editing in FCS
Phase 0
Recruiting
15 enrolled
CorrectSequence Therapeutics Co., Ltd
The First Affiliated Hospital of Anhui Medical University · collab
Non-randomizedSequentialOpen-labelTreatment
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).
Timeline
- Start
- 2025-10-15
- Primary completion
- 2026-12
- Completion
- 2026-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CS-121 | CRISPR / gene editing | — | Intravenous |