Groundbreaking Gene Editing Therapy Targets APOC3 to Address Hyperlipidemia
On November 6, 2025, in Shanghai, China, CorrectSequence Therapeutics Co., Ltd., a pioneering clinical-stage biotechnology company, made a groundbreaking announcement regarding its gene editing therapy, CS-121. This innovative treatment targets APOC3, which is pivotal in managing lipid metabolism, particularly in patients suffering from severe hyperlipidemia, including chylomicronemia.
The first patient undergoing this therapy experienced significant health improvements following a single dose, demonstrating an impressive reduction in fasting triglyceride levels from dangerously high values without any adverse effects. This event signifies not just a success for the treating medical team but symbolizes a new chapter in the treatment of lipid disorders, transcending conventional approaches.
Chylomicronemia is characterized by abnormally high levels of chylomicrons in the blood, leading to severe complications like acute pancreatitis. The condition represents the most severe form of hypertriglyceridemia and is classified into familial chylomicronemia syndrome and multifactorial chylomicronemia syndrome. The first involves rare recessive genetic mutations, while the second results from a complex interplay of genetic and lifestyle factors.
Currently available treatments fail to adequately manage triglyceride levels, and lifestyle modifications such as stringent low-fat diets can be challenging to maintain. Notably, APOC3 has emerged as a core player in triglyceride regulation, with population studies revealing that individuals with natural mutations leading to APOC3 loss-of-function exhibit significantly lower triglyceride levels.
CorrectSequence's CS-121 is based on advanced transformer Base Editing (tBE) technology, allowing precise genetic modifications without the risk of double-strand breaks associated with conventional CRISPR-based therapies. This method improves safety profiles by minimizing potential adverse events like chromosomal damage and off-target effects.
Administered intravenously, the tBE system uses lipid nanoparticles to deliver the editing tool directly to the liver, where it selectively modifies the APOC3 gene, thereby mimicking beneficial natural variants. The goal is not just to alleviate symptoms but to potentially provide a long-lasting cure by fundamentally altering lipid metabolism at the genetic level.
The initial patient, a 63-year-old male, demonstrated a marked decrease in fasting triglyceride levels just three days post-treatment and was discharged without any reported complications. Such outcomes promise a transformative impact on patients grappling with hyperlipidemia, paving the way for future innovations in gene editing therapies.
Headed by prominent researchers Professor Huan Zhou and Dr. Zhili Wu from the First Affiliated Hospital of Anhui Medical University, the trial aims to validate the potential of CS-121 as a durable solution for metabolic disorders.
Further building on its commitment to revolutionary healthcare solutions, CorrectSequence has previously developed CS-101, an ex vivo gene editing treatment that successfully treated multiple patients with β-thalassemia and sickle cell disease. With CS-121, the company is on the path to becoming a frontrunner in in vivo gene therapy, intending to deliver lasting benefits to patients afflicted with chylomicronemia and related metabolic disorders.
For more information about CorrectSequence Therapeutics and its pioneering work, visit www.correctsequence.com.
The first patient undergoing this therapy experienced significant health improvements following a single dose, demonstrating an impressive reduction in fasting triglyceride levels from dangerously high values without any adverse effects. This event signifies not just a success for the treating medical team but symbolizes a new chapter in the treatment of lipid disorders, transcending conventional approaches.
Chylomicronemia is characterized by abnormally high levels of chylomicrons in the blood, leading to severe complications like acute pancreatitis. The condition represents the most severe form of hypertriglyceridemia and is classified into familial chylomicronemia syndrome and multifactorial chylomicronemia syndrome. The first involves rare recessive genetic mutations, while the second results from a complex interplay of genetic and lifestyle factors.
Currently available treatments fail to adequately manage triglyceride levels, and lifestyle modifications such as stringent low-fat diets can be challenging to maintain. Notably, APOC3 has emerged as a core player in triglyceride regulation, with population studies revealing that individuals with natural mutations leading to APOC3 loss-of-function exhibit significantly lower triglyceride levels.
CorrectSequence's CS-121 is based on advanced transformer Base Editing (tBE) technology, allowing precise genetic modifications without the risk of double-strand breaks associated with conventional CRISPR-based therapies. This method improves safety profiles by minimizing potential adverse events like chromosomal damage and off-target effects.
Administered intravenously, the tBE system uses lipid nanoparticles to deliver the editing tool directly to the liver, where it selectively modifies the APOC3 gene, thereby mimicking beneficial natural variants. The goal is not just to alleviate symptoms but to potentially provide a long-lasting cure by fundamentally altering lipid metabolism at the genetic level.
The initial patient, a 63-year-old male, demonstrated a marked decrease in fasting triglyceride levels just three days post-treatment and was discharged without any reported complications. Such outcomes promise a transformative impact on patients grappling with hyperlipidemia, paving the way for future innovations in gene editing therapies.
Headed by prominent researchers Professor Huan Zhou and Dr. Zhili Wu from the First Affiliated Hospital of Anhui Medical University, the trial aims to validate the potential of CS-121 as a durable solution for metabolic disorders.
Further building on its commitment to revolutionary healthcare solutions, CorrectSequence has previously developed CS-101, an ex vivo gene editing treatment that successfully treated multiple patients with β-thalassemia and sickle cell disease. With CS-121, the company is on the path to becoming a frontrunner in in vivo gene therapy, intending to deliver lasting benefits to patients afflicted with chylomicronemia and related metabolic disorders.
For more information about CorrectSequence Therapeutics and its pioneering work, visit www.correctsequence.com.
Topics Health）
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