Science & Innovation

NAV® Technology Platform

REGENXBIO’s gene therapy product candidates deliver genes to cells using adeno-associated virus (AAV) vectors, which are nonreplicating viral delivery vehicles that are not known to cause disease.

Our product candidates all utilize viral vectors from our proprietary gene delivery platform, which we call our NAV Technology Platform.

Our NAV Technology Platform consists of exclusive rights to AAV7, AAV8, AAV9 and over 100 other novel AAV vectors (NAV Vectors). We currently have exclusive rights to over 100 patents and patent applications worldwide covering our NAV Vectors, including composition of matter claims for AAV7, AAV8, and AAV9, as well as methods for their manufacture and therapeutic uses. We believe 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 robust patent portfolio.

The foundation of our 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). We believe the key benefits of NAV Vectors include:

Broad application across multiple disease states

Delivery of multiple types of genetic material

Simplified manufacturing relative to earlier generation AAV vectors

Less likely to trigger an immune response than earlier generation AAV vectors

Improved gene expression over earlier generation AAV vectors, enabling longer-lasting treatment in a smaller dose

Derived from natural sources with modifications to enable therapeutic use

We believe NAV Technology, which underpins our internal development programs and the programs of our licensees, will enable us and our partners to develop best-in-class gene therapy candidates for a wide range of therapeutic areas.