Nasdaq: RGNX

REGENXBIO

REGENXBIO was formed from a successful collaboration that began in February 2009 between FoxKiser LLP, the University of Pennsylvania and gene-therapy pioneer James M. Wilson, M.D., Ph.D.

The foundation of their NAV Technology Platform was discovered in an effort to identify next-generation AAV vectors that could overcome the limitations of earlier generation AAV vectors (AAV1 through AAV6). They believe the key benefits of NAV Vectors over earlier generation AAV vectors include: higher gene expression, longer-term gene expression, broad and novel tissue selectivity, lower immune response and improved manufacturability. REGENXBIO believes AAV gene therapies that incorporate the proprietary advances from our NAV Technology Platform have significantly enhanced profiles as potential therapeutics.

Their mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on our NAV Technology Platform. They seek to accomplish this mission through a combination of their internal development efforts and the efforts of their NAV Technology Licensees.