RGNX Profile
Regenxbio Inc (RGNX) is a biotechnology company that focuses on developing and commercializing gene therapy treatments for inherited retinal diseases, neurodegenerative diseases, and other genetic disorders. The company uses its proprietary adeno-associated virus (AAV) gene delivery platform to develop its gene therapy treatments.
Regenxbio's lead product candidate, RGX-314, is a gene therapy treatment for wet age-related macular degeneration (AMD). Wet AMD is a leading cause of vision loss in people over 50 years old. RGX-314 is designed to deliver a therapeutic gene to retinal cells using the AAV vector platform, which is expected to reduce the need for frequent injections into the eye.
The company has several other product candidates in its pipeline, including treatments for Huntington's disease, spinal muscular atrophy, and lysosomal storage disorders. Regenxbio has collaborations with several pharmaceutical companies, including Biogen, AstraZeneca, and AbbVie, to develop gene therapies using its AAV vector platform.
Regenxbio was founded in 2008 and is headquartered in Rockville, Maryland.
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