Hypertriglyceridemia is a heterogeneous disorder of triglyceride synthesis or degradation, and its clinical manifestation is the accumulation of triglyceride-rich lipoproteins in the blood. Currently, there is a lot of evidence that hypertriglyceridemia is closely related to atherosclerotic diseases such as coronary heart disease and cerebrovascular disease. According to the cause, hypertriglyceridemia can be divided into two categories: primary and secondary. Primary hypertriglyceridemia includes rare diseases such as familial hypertriglyceridemia, familial mixed hyperlipidemia, and familial abnormal β-lipoproteinemia; the causes of secondary hypertriglyceridemia mainly include metabolic diseases, certain disease states, hormones, and drugs. The common cause of familial hypertriglyceridemia is lipoprotein lipase (LPL) gene functional defects, and the incidence of acute pancreatitis in patients with LPL gene defects is several times or even dozens of times that of the normal population, and the disease recurs, seriously endangering the patient's life.
The gene drug GC304 injection is a recombinant AAV virus vector carrying the LPL gene expression frame. LPL is a key enzyme in the human body that hydrolyzes triglyceride-rich lipoproteins in plasma. GC304 injection can degrade triglycerides in circulating blood for a long time and efficiently through the efficient expression of the artificially optimized LPL natural beneficial mutant LPLS447X gene in the human body, providing a new gene drug for the prevention and/or treatment of severe hypertriglyceridemia. In addition, in the efficacy verification experiment, it was discovered that the expression of GC304 injection can correct severe hypertriglyceridemia caused by defects in another lipid metabolism-related gene GPIHBP1, which expands the indications for GC304 injection and also represents the international innovation of Chinese gene drugs.
About GeneCradle
Beijing GeneCradle Technology Co., Ltd. is a biotechnology platform company focusing on the innovative research and development of gene therapy drugs. It focuses on the research and development of gene therapy products in the fields of hereditary neuromuscular diseases, hereditary metabolic diseases, lysosomal diseases and ophthalmic diseases mediated by AAV vector delivery technology. The GeneCradle team is committed to promoting China's rare disease gene drugs from basic to clinical and market. With the quality policy of "originating from demand, courageous innovation, dedicated to design, strict verification, sophisticated manufacturing, continuous improvement, and benefit to patients", it is committed to turning "no drug to cure" into "one-time administration, lifelong effectiveness", benefiting patients and their families. Through the research and development and clinical application of rare disease gene drugs, we can have a deeper understanding of life and health, and transition to the treatment and rehabilitation of chronic diseases and other major diseases.