REGENXBIO

REGENXBIO Inc. is a premier, clinical-stage biotechnology company dedicated to improving lives through the transformative potential of gene therapy. Established in 2009, the company has been a trailblazer in the development of adeno-associated virus (AAV) therapeutics, a sophisticated and innovative class of gene therapy medicines. REGENXBIO's core mission is to address severe diseases with significant unmet medical needs by developing and commercializing gene therapy products that can be administered directly into the body, or in vivo. The foundation of the company's pioneering work is its proprietary NAV® Technology Platform. This platform encompasses exclusive rights to a vast portfolio of over 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. These vectors are engineered to be non-replicating viral delivery vehicles that are not known to cause disease, making them an ideal and safe mechanism for delivering functional genes to target cells and correcting underlying genetic defects.

The company's robust pipeline of investigational AAV Therapeutics is focused on a range of debilitating conditions, with a primary emphasis on retinal, metabolic, and neurodegenerative diseases. Key programs include ABBV-RGX-314, which is being developed in collaboration with AbbVie for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy. Another significant candidate is RGX-202, aimed at treating Duchenne muscular dystrophy. Furthermore, REGENXBIO is advancing treatments for rare metabolic disorders such as Mucopolysaccharidosis Type II (MPS II) with RGX-121 and Mucopolysaccharidosis Type I (MPS I) with RGX-111. The company's comprehensive approach integrates in-house research and development with strategic licensing of its NAV® Technology Platform to a network of third-party licensees. This dual strategy allows REGENXBIO to expand the application of its technology across multiple therapeutic areas while advancing its own promising candidates through clinical trials and towards commercialization, with the ultimate goal of delivering one-time, curative treatments to patients worldwide.