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ZS802 is an investigational adeno-associated virus (AAV)-based gene therapy developed for the treatment of Hemophilia A. It delivers an optimized and modified human coagulation factor VIII (F8) gene sequence using a proprietary, liver-specific promoter designed to enhance expression and efficacy. The therapy aims to provide long-term correction of factor VIII deficiency by enabling patients' own liver cells to produce functional factor VIII protein, thereby reducing or eliminating the need for exogenous factor VIII infusions. Clinical studies are ongoing in China to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy in patients with severe Hemophilia A[1][2][4].
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