CorrectSequence Therapeutics’ CS-121 Completed Dosing of First Chylomicronemia Patient, Demonstrating Excellent Safety and Significant Efficacy
SHANGHAI, Nov. 5, 2025 /PRNewswire/ — On November 6, 2025, Shanghai, China, CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, 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.
Picture: The world’s first patient of the gene-editing therapy targeting APOC3 (Correctseq’s CS-121) for hyperlipidemia (the 6th from the right) has successfully completed dosing and been discharged.
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.
This is the world’s first successful clinical treatment of hyperlipidemia with the gene-editing therapy targeting APOC3.
Picture: The world’s first patient of the gene-editing therapy targeting APOC3 (Correctseq’s CS-121) for hyperlipidemia (the 6th from the right) has successfully completed dosing and been discharged.
Chylomicronemia is a metabolic disorder characterized by abnormally elevated chylomicrons in the blood, associated with lipid metabolism dysfunction, leading to extremely high fasting TG levels and potentially life-threatening complications such as acute pancreatitis. It is the most severe subtype of severe hypertriglyceridemia (sHTG), including Familial Chylomicronemia Syndrome (FCS) and Multifactorial Chylomicronemia Syndrome (MCS). FCS is a rare autosomal recessive disorder caused by biallelic mutations in the Lipoprotein Lipase (LPL) gene or other key regulatory genes, with fasting TG ≥10 mmol/L (885 mg/dL) and a global prevalence of 1 in 100,000–1,000,000. MCS results from a complex interaction of genetic, lifestyle, or metabolic disorders with a prevalence as high as 1 in 600 worldwide.
Current treatments for chylomicronemia primarily aim to control fasting TG levels below the acute pancreatitis risk threshold (