REGENXBIO is a clinical-stage biotechnology company developing curative gene therapies using its proprietary NAV Technology Platform, which consists of exclusive rights to over 100 adeno-associated virus (AAV) vectors that deliver functional genes to address genetic defects. The company generates revenue through upfront payments, milestone achievements, and royalties from partnerships with major pharmaceutical companies including AbbVie, Nippon Shinyaku, and Novartis, while advancing internal clinical pipelines in retinal diseases, neuromuscular disorders, and neurodegenerative conditions. With integrated manufacturing capabilities including a U.S.-based GMP facility, REGENXBIO aims to develop single-administration gene therapies with long-lasting effects for rare and prevalent diseases with significant unmet medical needs.