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Regenxbio Inc. is a biotechnology company specializing in gene therapy using its proprietary NAV® AAV vector platform. It develops investigational treatments for retinal, neuromuscular, and neurodegenerative diseases, delivering functional genes to address genetic defects. The company partners with industry leaders, licenses its platform to others, and focuses on scalable manufacturing and innovative delivery devices to provide long-lasting, potentially curative therapies.
This description was generated via AI from an annual report. Updated about 1 year ago.