RegenxBio Inc - Company Profile
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Business Description
RegenxBio Inc (RegenxBio), formerly ReGenX Biosciences LLC, is an early-stage biotechnology company that develops therapeutic proteins or antibodies for the treatment of retinal diseases, metabolic diseases, and neurodegenerative diseases. The company’s gene therapy product candidates deliver genes to cells for addressing genetic defects and enable cells in the body to produce therapeutic proteins or antibodies that act on the disease.
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R&D Overview
RegenxBio works in collaboration with its strategic partners to conduct research to develop therapeutic antibodies, proteins, and gene editing. In FY2023, the company incurred US$232.3 million on its R&D expenses, which declined 2.8% YoY. Its R&D programs focus on developing treatments for retinal diseases, metabolic diseases, neurodegenerative diseases, mucopolysaccharidosis type I (MPS I) caused by autosomal recessive IDUA mutations, and MPS II caused by X-linked recessive IDS mutations, and to advance its NAV technology platform. As of December 31, 2023, the company had several patents and patent applications spanning its NAV vectors, including the composition of matter claims for AAV7, AAV8, and AAV9, and methods for their manufacture and therapeutic uses. Further, the company intends to expand this robust patent portfolio which includes, its licensed patent portfolio including three European patents and 24 issued US patents associated with the AAV7, AAV8, AAV9, and AAVrh10 vectors and their uses. The company's Investigational AAV Therapeutics division had four issued US patents, one issued European patents, two pending International Patent applications filed pursuant to the Patent Cooperation Treaty (PCTs), and 19 PCTs that have entered the national stage relating to the product candidates.
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