CS-121 / CorrectSequence Therap - LARVOL DELTA

CS-121 APOC3 Base Editing in Severe Hypertriglyceridemia (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: CorrectSequence Therapeutics Co., Ltd

New P1 trial • Dyslipidemia • Hypertriglyceridemia • Severe Hypertriglyceridemia

Treatment of Persistent Chylomicronemia by editing of the APOC3 gene with transformer Base Editor (ASGCT 2026) - "Methods The transformer base editor (tBE) is an elaborately designed base editing system with high editing efficiency and significantly reduced off-target activity, which has been validated in CS-101, a potential Best-in-Class therapy for β-thalassemia currently in clinical trial. Conclusion CS-121 exhibited favorable safety profile and promising efficacy in PC patients. The IIT results support further evaluation of higher doses and in broader patient populations."

Beta-Thalassemia • Cardiovascular • Familial Chylomicronemia Syndrome • Genetic Disorders • Pancreatitis • APOC3 • LPL

CS-121 APOC3 Base Editing in FCS (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: CorrectSequence Therapeutics Co., Ltd | Not yet recruiting ➔ Recruiting

Enrollment open • First-in-human • Familial Chylomicronemia Syndrome

CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Shanghai Jiao Tong University School of Medicine

New P1 trial

...CorrectSequence Therapeutics…announced that the first patient in its Investigator-Initiated Trial (IIT) of the base-editing therapy CS-121 targeting APOC3 for chylomicronemia / hypertriglyceridemia has successfully completed dosing and been discharged from the hospital (PRNewswire) - "The patient, diagnosed with chylomicronemia, had a long history of fasting triglyceride (TG) levels exceeding 12.5 mmol/L and recurrent acute pancreatitis. In the dose-escalation IIT for CS-121, his fasting TG level dropped significantly within three days after a single low-dose administration, with no adverse events."

Trial status • Familial Chylomicronemia Syndrome

CS-121 APOC3 Base Editing in FCS (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: CorrectSequence Therapeutics Co., Ltd

New P1 trial • Familial Chylomicronemia Syndrome