The company has a NAV technology platform and a proprietary AAV gene delivery platform which consists of exclusive rights to more than 100 novel AAV vectors.

Its technology is used for the treatment of metabolic disorders, muscle diseases, hematologic disorders, ocular diseases, and neurodegenerative disorders; and for clinical trails in therapeutic areas, such as inherited diseases, CNS disorders, heart failure, and vaccines.

Regenxbio's mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need. The company seeks to accomplish this mission through a combination of internal development efforts and third‐party platform licensees.