REGENXBIO Inc. is a clinical-stage biotechnology company focused on the development of gene therapies for patients with serious diseases. The company’s proprietary NAV® (Natural AAV Vector) platform utilizes a collection of adeno-associated viral (AAV) vectors designed to efficiently deliver therapeutic genes to target cells. By harnessing naturally occurring AAVs and employing advanced molecular engineering, REGENXBIO aims to maximize the precision, durability and safety of gene-based treatments across a range of conditions.
The company’s pipeline spans both rare genetic disorders and more prevalent diseases of significant unmet need. Key programs include RGX-314, an investigational therapy for wet age-related macular degeneration; RGX-111 and RGX-121 for mucopolysaccharidosis types I and II respectively; and RGX-501 for the treatment of sickle cell disease. Each program leverages tissue-specific promoters and targeted AAV serotypes to optimize gene expression in the eye, central nervous system or liver, depending on the therapeutic indication.
With its headquarters in Rockville, Maryland, REGENXBIO operates in the United States and collaborates with global partners to advance its product candidates through clinical development and regulatory pathways. The company has established manufacturing capabilities in Maryland and Maryland-based facilities to support Good Manufacturing Practice (GMP) production, while partnering with third-party contract development and manufacturing organizations to scale toward commercial supply. Strategic alliances with industry leaders have furthered development efforts and expanded the company’s access to key expertise and resources.
Founded in 2012 by gene therapy innovators including founders Kenneth A. Simon and Richard Fisher, REGENXBIO has grown under the leadership of President and Chief Executive Officer Kenneth A. Mills. The executive team features seasoned professionals in biopharmaceutical development, regulatory affairs and manufacturing. Through continued investment in its NAV platform and disciplined clinical execution, REGENXBIO is positioned to advance transformative gene therapies that target the root causes of disease.
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