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REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the appointment of Steve Pakola, M.D., as its Senior Vice President and Chief Medical Officer. Dr. Pakola will report to Kenneth T. Mills, REGENXBIO’s President and Chief Executive Officer.

Dr. Pakola joins REGENXBIO from Aerpio Pharmaceuticals, Inc., where he was Chief Medical Officer. He has held key leadership roles in preclinical and clinical development, regulatory affairs and medical affairs. While Dr. Pakola’s therapeutic area experience encompasses multiple indications, his predominant focus has been the development of treatments for retinal disorders, including clinical development experience in diabetic retinopathy, diabetic macular edema and age-related macular degeneration (AMD). He was the lead inventor and program lead for the Jetrea® (ocriplasmin) program, from inception in 2002 through the therapy’s United States and European regulatory submissions in 2012.

“In this role, Steve will leverage his rich industry experience and expertise in the discovery and development of novel treatments for retinal disease,” said Mr. Mills. “Steve’s background as an innovator in drug development is particularly relevant as we leverage our breakthrough science to advance five current proprietary therapeutic programs that focus on retinal, neurodegenerative and metabolic diseases. We are excited to welcome Steve to the team.”

“I am thrilled to join REGENXBIO at this time of exciting momentum for the company as it advances the development of gene therapies,” said Dr. Pakola. “REGENXBIO’s NAV Technology Platform has the potential to transform the course of treatment for patients with rare diseases as well as in broader applications such as wet AMD. I look forward to working with my colleagues, investigators and the regulatory authorities to bring new and innovative treatments to patients.”

Prior to Aerpio, Dr. Pakola served as Chief Medical Officer at Amakem NV and as Senior Vice President and Chief Medical Officer at ThromboGenics NV. Earlier in his career, Dr. Pakola served as Associate Director of Cardiovascular Clinical Research at Boehringer Ingelheim, where he was Global Medical Lead on the Lipid-Lowering Development Programme and the USA Medical Lead for the Direct Thrombin Inhibitor Development Programme. Prior to that, Dr. Pakola served in senior-level clinical development positions at Quintiles Cardiovascular Therapeutics and at Organon.

Dr. Pakola earned a B.A., summa cum laude, and an M.D. from the University of Pennsylvania, where he was elected to the Alpha Omega Alpha Honor Medical Society.





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