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Gene Therapy Clinical Trial for Tay-Sachs and Sandhoff Diseases. At Sio, we operate with a sense of urgency and compassion to develop gene therapies that transform the treatment of serious neurodegenerative diseases. We combine cutting-edge science with rigorous testing to fill unmet medical needs for patients with therapies that are intended to
AXO-AAV-GM1 Gene Therapy Clinical Study for GM1 Gangliosidosis A Phase 1/2 clinical trial is being conducted to learn if an experimental gene therapy called AXO- AAV-GM1 (also called AAV9-GLB1) can help treat children with infantile and juvenile -onset GM1
Classical applications of AAV -based gene therapy in monogenic diseases and extension of gene therapy potential into more prevalent indications like Parkinson’s disease
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