Science & Innovation

REGENXBIO has leading, in-house end-to-end capabilities in the research, clinical development and commercial-ready manufacturing of AAV gene therapies.

Our team of scientists and engineers are dedicated to expanding the understanding and applications of AAV vectors, applying the differentiated capabilities of the NAV® Technology Platform and exploring the potential to generate new, innovative AAV Therapeutics.

We endeavor to rapidly discover and develop a pipeline of investigational AAV Therapeutics with the potential, through a single administration, to alter the course of disease significantly and deliver improved patient outcomes with long-lasting effects.