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 and applying the differentiated capabilities of the NAV® Technology Platform to generate new, innovative gene therapies for patients.

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