RGNX
- REGENXBIO Inc.
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Overview
Company Summary
REGENXBIO Inc. is a biotechnology company focused on developing and commercializing gene therapy treatments for various genetic disorders. The company uses its proprietary adeno-associated virus (AAV) gene delivery platform to develop therapies that can potentially address a wide range of diseases.
REGENXBIO's AAV gene delivery platform is designed to deliver therapeutic genes into targeted cells, enabling the production of functional proteins to correct the underlying genetic condition. This approach has the potential to provide long-term or even permanent treatment options for patients suffering from genetic disorders.
The company has a diverse pipeline of product candidates targeting various diseases, including retinal disorders, metabolic disorders, neurodegenerative diseases, muscular dystrophy, and others. One of their most advanced programs is RGX-314, a potential one-time treatment for wet age-related macular degeneration (AMD), a leading cause of blindness.
In addition to developing its own product candidates, REGENXBIO also collaborates with leading biopharmaceutical companies to license its AAV gene delivery technology. This enables other companies to develop their own gene therapies using REGENXBIO's platform.
Overall, REGENXBIO Inc. is focused on harnessing the potential of gene therapy to provide innovative treatments for patients with genetic disorders, with the aim of improving their quality of life and potentially curing debilitating conditions.