-REGENXBIO AAV Therapeutics pipeline to be discussed by management and external experts

-Focus on programs for Wet AMD, Diabetic Retinopathy, and Duchenne Muscular Dystrophy

ROCKVILLE, Md., June 27, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a virtual investor day on July 11, 2023. During the event, members of REGENXBIO's management team and external experts will provide an update to the investment community on the Company's pipeline, with a focus on programs with expected interim clinical trial data to be presented in the second half of 2023.

The program will include presentations of the ABBV-RGX-314 eye care program, including plans for interim data updates from the Phase II AAVIATE^® and ALTITUDE^® trials. It will also include an overview of RGX-202, an investigational one-time gene therapy utilizing a novel microdystrophin construct for the treatment of Duchenne Muscular Dystrophy (Duchenne), including the expected plans for interim data updates from the Phase I/II AFFINITY DUCHENNE™ trial.

In addition, management will highlight the features and performance of the fully operational REGENXBIO Manufacturing Innovation Center, which is designed to support commercial readiness. This facility is producing GMP lots to support the ABBV-RGX-314 and RGX-202 programs using the NAVXpress™ platform process, which has demonstrated the ability to increase product purity and yield.

A live question and answer session will follow the program.

The event will feature the following external experts:

• George Dickson, world-class researcher in Duchenne and Professor of Molecular Cell Biology at Royal Holloway – University of London.
  
  
• Dr. Peter Kaiser, MD FASRS, Chair for Ophthalmology Research and a Professor of Ophthalmology at the Cleveland Clinic Lerner College of Medicine.
  
  
• Dr. Veeral Sheth, MD, MBA, FACS, FASRS, board-certified vitreoretinal surgeon and Partner at University Retina and Macula Associates in Chicago.

The virtual event will take place on Tuesday, July 11, 2023 at 8:30 a.m. Eastern Time. The webcast and slides of the presentation can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available in the Investors section of REGENXBIO's website for approximately 30 days following the presentation.

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 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025. 

Forward-Looking Statement  
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," "assume," "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 and clinical trials. 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, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, 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, 2022, 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. Except as required by law, 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.

Contacts:
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com

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