REGENXBIO Announces Appointment of Faraz Ali as Chief Business Officer

ROCKVILLE, Md., March 01, 2016 -- REGENXBIO Inc. (Nasdaq:RGNX), a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV® Technology Platform, today announced that Faraz Ali has been appointed as its Chief Business Officer. As a member of the leadership team, Mr. Ali is expected to oversee a wide range of functional responsibilities, including commercial and portfolio planning, program management, corporate development, patient advocacy and corporate communications.

Mr. Ali joins REGENXBIO from bluebird bio where he was vice president, head of global commercial development and external affairs. At bluebird bio, he led all commercial planning efforts, including engagement with payers in the U.S. and Europe. Mr. Ali provided significant early input into bluebird bio’s portfolio strategy, and provided critical cross-functional leadership during the company’s initial growth phase by establishing new functions, processes and external relationships. He was a co-leader on a team that successfully navigated the first wave of E.U. “Adaptive Pathways” pilot engagements involving the European Medicines Agency, health technology assessment agencies and patient advocacy groups. Mr. Ali is also Chair of the Reimbursement Committee at the Alliance for Regenerative Medicine where he is developing policy proposals for innovative reimbursement and financing models for potentially curative gene and cell therapies.

“Faraz is highly regarded within the biotech industry as a thoughtful and experienced leader in gene therapy and orphan diseases. We believe his expertise with launching and commercializing therapies for severe genetic disorders will be invaluable to our strategic business and product development plans as we advance our lead gene therapies in the clinic and continue to grow the NAV Technology Platform pipeline internally and through our licensees,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We are excited to welcome Faraz to our leadership team and look forward to leveraging his broad strategic and operational experience to develop gene therapy franchises in areas of unmet patient need.”

“REGENXBIO is a leader in the gene therapy space and has an opportunity to create unparalleled value. The robust NAV Technology Platform enables the company to both rapidly advance its internal gene therapy candidates as well as to expand its pipeline through strategic sublicensing,” said Mr. Ali. “I am thrilled to be joining the REGENXBIO team at this exciting time as it continues to transition to becoming a clinical-stage gene therapy company.”

Mr. Ali brings more than sixteen years of healthcare experience to REGENXBIO. Prior to bluebird bio, he held roles of increasing global commercial responsibility at Genzyme Corporation, including Head of U.S. Marketing and Strategic Planning for the rare disease business unit, where he led marketing functions for Cerezyme®, Fabrazyme®, Myozyme® / Lumizyme® and Aldurazyme®. Earlier in his career, Mr. Ali served in leadership roles at GE Healthcare. Mr. Ali holds an MBA with distinction from Harvard Business School and a B.S. in Electrical Engineering from Stanford University.

About REGENXBIO

REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO's mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO's NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third-party NAV Technology Platform licensees. As of December 31, 2015, REGENXBIO's NAV Technology Platform is being applied in the development of 28 product candidates for a variety of diseases, including five internally developed candidates and 23 partnered candidates developed by REGENXBIO's licensees.

Forward Looking Statements

This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIO’s research, development and regulatory plans for RGX-111, RGX-314, RGX-501 and other gene therapies. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the ability to obtain and maintain regulatory approval of REGENXBIO's product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIO's product candidates; REGENXBIO's ability to obtain and maintain intellectual property protection for our product candidates; REGENXBIO's ability to establish and maintain development partnerships; REGENXBIO's expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and foreign countries, as well as other factors discussed in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Quarterly Report on Form 10-Q for the quarter ended September 30, 2015, which is available on the SEC's website at www.sec.gov. Additional factors may be described in those sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2015, to be filed with the SEC in the first quarter of 2016. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect REGENXBIO's results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.

All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). REGENXBIO undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

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