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Shanghai Hongji Biotech is a Chinese biotechnology company specializing in the development of next-generation adeno-associated virus (AAV) gene therapies for rare genetic disorders. The company focuses on high-impact indications including spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and hemophilia B, utilizing its proprietary AAV capsid engineering platform to enhance tissue specificity and therapeutic efficacy. Its lead program, HJ-001, is an AAV9-based gene therapy for SMA that has advanced into clinical trials in China. Hongji Biotech aims to address significant unmet medical needs through a robust pipeline of transformative genetic medicines targeting the central nervous system, muscle, and ophthalmic tissues.
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