#China #Shanghai #CorrectSequence #Hyperlipidemia #APOC3

Revolutionary Gene-Editing Therapy for Hyperlipidemia

On November 6, 2025, CorrectSequence Therapeutics, a pioneering biotechnology firm, announced a significant breakthrough in the treatment of hyperlipidemia, specifically addressing conditions that stem from the APOC3 gene. This innovative approach focuses on a gene-editing therapy named CS-121, aimed directly at combating hyperlipidemia associated with chylomicronemia.

The Launch of CS-121

The groundbreaking therapy has successfully completed its initial assessment phase with the first patient receiving treatment. This patient, suffering from severe chylomicronemia marked by fasting triglyceride levels exceeding 12.5 mmol/L, participated in an investigator-initiated trial. Remarkably, three days post dosage, the patient exhibited a significant reduction in fasting triglyceride levels with no adverse effects reported.

Chylomicronemia represents a critical metabolic condition where there is an abnormal elevation in chylomicrons—lipoproteins that transport dietary lipids. The ramifications of this disorder can be dire, often leading to acute pancreatitis and other complications. Thus, the CS-121 therapy is being hailed as a pivotal advance towards effective treatments for this serious condition.

How CS-121 Works

CS-121 operates using transformer Base Editing (tBE), a technique that allows for high-precision modifications at the genetic level without causing DNA double-strand breaks. This method is advantageous over traditional CRISPR technologies as it minimizes potential safety risks, such as off-target effects and liver toxicity.

By targeting the APOC3 gene directly, CS-121 aims to emulate the beneficial effects seen in individuals naturally carrying loss-of-function mutations of the APOC3 protein, which are associated with lower triglyceride levels. Populations studied have demonstrated that these genetic mutations correlate with a lower risk of hyperlipidemia and related disorders. Consequently, through the precise modulation of APOC3 expression, CS-121 seeks to deliver a therapy that could achieve lasting effects from a single treatment.

The Research and Development Journey

CorrectSequence Therapeutics, founded as a spin-off from ShanghaiTech University, has developed a robust pipeline encompassing various genetic disorders alongside metabolic diseases. Prior to CS-121, the company rolled out CS-101, a successful ex-vivo gene-editing therapy for patients with β-thalassemia and sickle cell disease.

The latest therapy further establishes CorrectSequence’s commitment to leveraging innovative genetic interventions to improve patient outcomes. CS-121 is presently progressing through IND (Investigational New Drug) clinical trials, with ambitious plans for commercialization aimed at providing long-term therapeutic solutions to patients facing severe hyperlipidemia and metabolic challenges.

The Future of Hyperlipidemia Treatments

The implications of this therapy extend beyond chylomicronemia; they open doors to potential treatments for other metabolic disorders characterized by dysregulated triglyceride levels. By harnessing advanced gene-editing technologies, CorrectSequence is positioning itself at the forefront of a revolution in how hyperlipidemia and related illnesses are understood and treated.

In conclusion, as the first gene-editing therapy specifically targeting APOC3 achieves success in clinical applications, the journey towards comprehensive genetic solutions in treating hyperlipidemia appears promising. With ongoing research and clinical trials, the future holds the potential to transform the lives of those affected by these debilitating conditions.