Regenxbio Inc is a clinical-stage biotechnology company focused on the development of gene therapy treatments. Its product candidates are designed to deliver genes to cells to address genetic defects or enable the production of therapeutic proteins. The company is developing a pipeline of investigational gene therapy treatments using its NAV Technology Platform based on adeno-associated virus (AAV) vectors. Its internal research and development programs are focused on therapies for retinal, neuromuscular, and neurodegenerative diseases.