NAV® Technology Platform

REGENXBIO’s foundation is our proprietary recombinant adeno-associated virus (AAV) gene delivery platform – our NAV Technology Platform – which consists of exclusive rights to more than 100 novel AAV serotypes, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its NAV Technology Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. Most of our NAV Technology Licensees have licensed specific vectors (NAV Vectors) from our NAV Technology Platform for the indications they are pursuing.

 

We believe the key benefits of NAV Vectors over earlier generation AAV vectors include: higher gene expression, long-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 (NAV Gene Therapy) have significantly enhanced profiles as potential therapeutics.

Our NAV Vectors have been broadly accepted. Approximately 70 percent of all AAV gene therapy clinical trials relating to new treatment INDs posted on the U.S. government clinical trials database from 2012 through 2014 used our NAV Vectors. We believe that proof-of-concept of our NAV Technology Platform is supported by three separately reported Phase I and II third-party clinical trials using AAV8 for the treatment of hemophilia B, and a Phase I clinical trial using AAV9 for the treatment of spinal muscular atrophy.

REGENXBIO currently has exclusive rights to more than 100 patents and patent applications worldwide covering our NAV Vectors, including composition of matter claims for AAV7, AAV8, AAV9 and AAVrh10, as well as methods for their manufacture and therapeutic uses. This patent portfolio forms a strong foundation for our current programs and, with our ongoing research and development, we expect to continue to expand this substantial portfolio. Our patents not only seek to protect our key assets — our NAV Technology Platform and our internal product candidates — they also form the basis for licensing and partnering arrangements.

We have access to significant expertise, proprietary methods and proven capabilities that we believe will enable us to successfully commercialize NAV Gene Therapy by developing scalable processes to manufacture high-quality NAV Gene Therapies efficiently and at commercial quantity.