News Releases

News Releases

REGENXBIO Licenses Key Technologies from University of Pennsylvania and University of Minnesota for the Development of Treatments for MPS I and MPS II

April 8, 2015 at 11:18 AM EDT

WASHINGTON, DC April 8, 2015 – REGENXBIO Inc., a leading biotechnology company in gene therapy, today announced that it has entered into separate license and research agreements with the University of Pennsylvania (Penn) and University of Minnesota (U of M) related to the delivery of AAV vectors for the treatment of MPS I and MPS II, also known as Hurler syndrome and Hunter syndrome, respectively. These agreements provide REGENXBIO with exclusive access to key technologies and resources that the Company plans to apply in the development of first-in-class treatments for MPS I and MPS II using its proprietary NAV® Technology.

“Patients with Hurler syndrome and Hunter syndrome need better treatment options,” said Ken Mills, President and CEO of REGENXBIO. “We are excited to have entered into these agreements, which support our active development programs for the treatment of the central nervous system symptoms of these diseases, for which there are currently no approved therapies. These agreements also strengthen and extend our important relationships with Penn and U of M in the development of treatments for MPS I and MPS II.”


REGENXBIO Inc. is the leading next-generation AAV gene therapy company, developing a new class of personalized therapies based on its proprietary NAV® Technology platform for a range of severe diseases with serious unmet needs. NAV Technology includes novel AAV vectors AAV7, AAV8, AAV9, and AAVrh10. REGENXBIO has enabled leading global partners including Baxter Healthcare, Fondazione Telethon, Audentes Therapeutics, Lysogene, Esteve, AveXis, AAVLife and Voyager Therapeutics to use its NAV Technology. In addition, together with Fidelity Biosciences, REGENXBIO formed Dimension Therapeutics, a company focused on the development and commercialization of NAV-based gene therapies for liver-directed rare diseases.

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