The third AAV gene therapy for hemophilia B has been approved for clinical use
The clinical trial application for "ZS801 injection" submitted by Sichuan Zhishan Weixin Biotechnology Co., Ltd. has obtained clinical implicit permission. The indications are: This product is suitable for Control and prevention of bleeding in male patients aged 18 years and above with severe or moderately severe hemophilia B (congenital coagulation factor IX deficiency, coagulation factor IX <2%). ZS801 is an AAV gene therapy drug and is the core R&D pipeline of Zhishanweixin. ZS801 is a rAAV gene therapy drug with independent intellectual property rights owned by Zhishanweixin. The design of ZS801 adopts the core sequence and high-efficiency gene expression cassette that Zhishanweixin has completely independent intellectual property rights. The core sequence is obtained by Zhishanweixin and a well-known domestic medical treatment company. Original drug targets independently developed by the institution. (BioValley)
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