Zhishan Weixin Biotechnology Co., Ltd, a China-based biotech company, has announced the launch of the first clinical research project in China focused on a recombinant adeno-associated virus (rAAV) genetic therapy for the treatment of Fabry disease. The study will evaluate the adeno-associated virus vector ZS805 as a potential treatment across six tertiary hospitals in China.
ZS805: An Innovative Gene Drug
ZS805 is an innovative Category 1 biologic and an rAAV gene drug independently developed by Zhishan Weixin, holding fully independent intellectual property rights. The molecule has been optimized through genetic engineering to ensure that the alpha-galactosidase-A protein is specifically expressed and efficiently secreted in liver cells. Zhishan Weixin has also selected AAV carrier serotypes that can cover the vast majority of patients, making ZS805 a potentially inclusive treatment option.
Understanding Fabry Disease
Fabry disease, also known as alpha-galactosidase-A deficiency, is an inherited neurological disorder characterized by the enzyme alpha-galactosidase-A’s inability to efficiently break down lipids in the body. The mutated gene results in the buildup of lipids to harmful levels in the body’s autonomic nervous system, impacting breathing, cardiovascular function, as well as the eyes, and kidneys.
ZS805’s Potential Impact on Treatment
Traditional treatment for Fabry disease involves enzyme replacement therapy, which requires patients to receive an infusion of agarase every two weeks for life. Zhishan Weixin’s ZS805 is expected to provide a “one shot treatment” that could offer a durable and potential lifelong cure, revolutionizing the treatment landscape for Fabry disease.- Flcube.com