REGENXBIO : Reports Continued Progress across Programs in Year-End 2019 Corporate Update

ROCKVILLE - 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 provided a year-end 2019 corporate update and anticipated milestones for 2020.

'2019 was an important year for the advancement and broadening of our internal gene therapy programs using NAV vectors for AAV-mediated antibody delivery and monogenic gene replacement. Importantly, we now have extensive clinical data from our RGX-314 program, which supports further development of our promising ophthalmology platform, with additional studies planned in 2020 to investigate subretinal and suprachoroidal approaches to treating wet AMD and diabetic retinopathy. Our key objective is to make RGX-314 available to patients as quickly as possible,' said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. 'We also recently announced interim data from our RGX-121 program for the treatment of MPS II, which showed encouraging signs of enzyme activity in the central nervous system, and we began construction of our new headquarters, which will include additional cGMP manufacturing facilities to support late-stage development and commercial scale needs.'

'Our strategic partnerships with other leading gene therapy developers continue to validate our NAV Technology Platform,' Mr. Mills continued. 'Zolgensma became the first FDA approved gene therapy based on our proprietary NAV technology last year, a significant milestone, and other strategic partners continue to advance their clinical programs into later stages. We have a strong team and resources in place to drive our business forward to help realize the transformative potential that gene therapy holds for patients.'

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes 'forward-looking statements,' within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as 'believe,' 'may,' 'will,' 'estimate,' 'continue,' 'anticipate,' 'design,' 'intend,' 'expect,' 'could,' 'plan,' 'potential,' 'predict,' 'seek,' 'should,' 'would' or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, research and development activities, preclinical studies, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations' sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2018, and comparable 'risk factors' sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Zolgensma is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.

One patient died 4.5 months after the administration of RGX-314 as a result of the subject's underlying disease, which was assessed to be unrelated to RGX-314. At the time of the death, the subject was free of anti-VEGF injections.

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