First Ever Gene-Editing Therapy for Hyperlipidemia Achieves Key Milestone
On November 6, 2025, CorrectSequence Therapeutics Co., Ltd. (Correctseq) announced a groundbreaking milestone in the treatment of hyperlipidemia with their gene-editing therapy, CS-121. This biotechnology company, based in Shanghai, China, has been at the forefront of developing innovative health solutions through transformer Base Editing (tBE) technology. CS-121 is uniquely designed to target the APOC3 gene, which plays a crucial role in triglyceride regulation within the body.
The announcement follows the successful treatment of the first patient diagnosed with chylomicronemia, a severe metabolic disorder characterized by dangerously high levels of triglycerides leading to acute pancreatitis. The patient underwent a single low-dose intravenous treatment of CS-121, following which significant improvements in triglyceride levels were observed within just three days.
Chylomicronemia is particularly challenging to manage due to the limited effectiveness of existing therapies, which often fall short in controlling triglyceride levels below critical thresholds. Patients frequently experience distressing dietary restrictions and face severe health risks tied to their condition. Studies suggest that the APOC3 protein contributes significantly to lipid metabolism, and individuals with genetic mutations leading to reduced levels of this protein have naturally lower triglyceride levels. This discovery has propelled efforts to develop gene-editing therapies aimed at fine-tuning APOC3 expression as a means to combat hyperlipidemia.
CS-121 employs a cutting-edge tBE approach that achieves precise editing without introducing double-strand breaks in the DNA, thereby mitigating potential risks commonly associated with traditional CRISPR methods, such as off-target effects and chromosome damage. Researchers have demonstrated the therapy's safety and long-term efficacy in preclinical studies, further solidifying its potential as a revolutionary treatment option. In particular, the results of animal studies indicated no adverse events and no detectable off-target effects across various organs.
The rise of CS-121 marks a significant progression in gene-editing therapies. The first patient, a 63-year-old male with a long history of high triglyceride levels, exhibited a notable reduction in fasting triglycerides post-treatment, facilitating his timely hospital discharge without any treatment-related complications. This patient's successful experience offers a beacon of hope for those suffering from the complications of chylomicronemia and other forms of severe hypertriglyceridemia.
The principal investigators behind the CS-121 study, Professor Huan Zhou and Dr. Zhili Wu from the First Affiliated Hospital of Anhui Medical University, have underscored the revolutionary potential of this therapy. Correctseq aims to scale this innovation, aspiring to deliver not just an effective treatment for hyperlipidemia but also to pave the way for advancements in gene-editing applications across various severe metabolic disorders.
Moving forward, CorrectSequence’s flagship research initiative underscores their commitment to leveraging advanced gene-editing technologies for transforming patient lives. Their prior experience with CS-101, which focused on treating genetic blood disorders like β-thalassemia and sickle cell disease, sets a robust foundation for the future of CS-121 as it transitions into broader clinical trials and eventual commercialization.
As the field of biotechnology progresses, the success of CS-121 instills optimism about the potential for tailored therapies that offer not only symptom management but also lasting solutions to complex health challenges. CorrectSequence Therapeutics is positioning itself as a pioneer in reshaping therapeutic strategies, advocating for innovative treatments that aspire to activate a new era in genetic medicine. For further insights on this advancement in gene therapy, visit www.correctsequence.com.
The announcement follows the successful treatment of the first patient diagnosed with chylomicronemia, a severe metabolic disorder characterized by dangerously high levels of triglycerides leading to acute pancreatitis. The patient underwent a single low-dose intravenous treatment of CS-121, following which significant improvements in triglyceride levels were observed within just three days.
Chylomicronemia is particularly challenging to manage due to the limited effectiveness of existing therapies, which often fall short in controlling triglyceride levels below critical thresholds. Patients frequently experience distressing dietary restrictions and face severe health risks tied to their condition. Studies suggest that the APOC3 protein contributes significantly to lipid metabolism, and individuals with genetic mutations leading to reduced levels of this protein have naturally lower triglyceride levels. This discovery has propelled efforts to develop gene-editing therapies aimed at fine-tuning APOC3 expression as a means to combat hyperlipidemia.
CS-121 employs a cutting-edge tBE approach that achieves precise editing without introducing double-strand breaks in the DNA, thereby mitigating potential risks commonly associated with traditional CRISPR methods, such as off-target effects and chromosome damage. Researchers have demonstrated the therapy's safety and long-term efficacy in preclinical studies, further solidifying its potential as a revolutionary treatment option. In particular, the results of animal studies indicated no adverse events and no detectable off-target effects across various organs.
The rise of CS-121 marks a significant progression in gene-editing therapies. The first patient, a 63-year-old male with a long history of high triglyceride levels, exhibited a notable reduction in fasting triglycerides post-treatment, facilitating his timely hospital discharge without any treatment-related complications. This patient's successful experience offers a beacon of hope for those suffering from the complications of chylomicronemia and other forms of severe hypertriglyceridemia.
The principal investigators behind the CS-121 study, Professor Huan Zhou and Dr. Zhili Wu from the First Affiliated Hospital of Anhui Medical University, have underscored the revolutionary potential of this therapy. Correctseq aims to scale this innovation, aspiring to deliver not just an effective treatment for hyperlipidemia but also to pave the way for advancements in gene-editing applications across various severe metabolic disorders.
Moving forward, CorrectSequence’s flagship research initiative underscores their commitment to leveraging advanced gene-editing technologies for transforming patient lives. Their prior experience with CS-101, which focused on treating genetic blood disorders like β-thalassemia and sickle cell disease, sets a robust foundation for the future of CS-121 as it transitions into broader clinical trials and eventual commercialization.
As the field of biotechnology progresses, the success of CS-121 instills optimism about the potential for tailored therapies that offer not only symptom management but also lasting solutions to complex health challenges. CorrectSequence Therapeutics is positioning itself as a pioneer in reshaping therapeutic strategies, advocating for innovative treatments that aspire to activate a new era in genetic medicine. For further insights on this advancement in gene therapy, visit www.correctsequence.com.
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