RGX-381

About ReGenX Biosciences

ReGenX Biosciences is a clinical-stage biotechnology company dedicated to improving lives through the development of curative gene therapies. Its core asset is the NAV Technology Platform, a collection of over 100 novel AAV vectors that have become industry standards, powering its own pipeline and over 20 partnered programs, including the commercial therapy Zolgensma®. The company's dual-strategy of internal development and broad licensing has established a significant revenue stream and validates its platform's utility across multiple therapeutic areas.

About REGENXBIO

REGENXBIO is a clinical-stage biotech with a mission to deliver life-changing gene therapies through its foundational NAV Technology Platform. Its achievements include enabling the blockbuster therapy ZOLGENSMA® and building a diverse internal pipeline, most notably in MPS II and Duchenne muscular dystrophy. The company's strategy combines internal development of wholly-owned assets with a high-margin, capital-efficient licensing model that provides recurring revenue and validates its technology.

Therapeutic Areas