REGENXBIO and University of Pennsylvania File NAV® Technology Patent Infringement Lawsuit

ROCKVILLE, Md., June 20, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it has filed a second complaint for patent infringement against Sarepta Therapeutics, Inc. (Sarepta) arising from Sarepta's manufacture, use and imminent commercial launch of SRP-9001 for the treatment of Duchenne muscular dystrophy.

The complaint asserted U.S. Patent No. 11,680,274, which covers Sarepta's AAVrh74-based gene therapy vector products, including SRP-9001. REGENXBIO exclusively licensed the newly issued patent from the University of Pennsylvania (Penn), which is a joint plaintiff in the lawsuit. The term of the patent-in-suit extends to October 2027 and damages are being sought to compensate REGENXBIO and its licensor, Penn, which originated the adeno-associated virus (AAV) gene therapy technology.

"Through our clinical programs and the licensing to other biopharmaceutical companies with disease-specific expertise, thousands of patients have been treated with AAV gene therapies built on REGENXBIO's NAV^® Technology Platform," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are proud of our technology and its potential to enable life-changing medicines. Our NAV Technology Platform is being applied over a broad range of disease indications by our NAV Technology Licensees and public-private partnerships such as the NIH/FDA Bespoke Gene Therapy Consortium, all with a focus on ensuring patient access."

"We believe that Sarepta unlawfully exploited groundbreaking inventions and has continued to use them without a license," said Patrick Christmas, Chief Legal Officer of REGENXBIO. "We have taken the appropriate steps to vigorously defend our intellectual property rights. We are not attempting to halt production of gene therapies, we are seeking compensation for the defendants' deliberate infringement."

The complaint was filed June 20, 2023 in the federal district court of Delaware, where a separate patent infringement action previously brought by REGENXBIO and Penn against Sarepta is pending, arising from Sarepta's manufacture and use of cultured host cell technology covered by another Penn patent, U.S. Patent No. 10,526,617, that Sarepta uses to make the SRP-9001 product. That lawsuit was brought September 30, 2020 and the trial is scheduled for January 2024. 

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 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," "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:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com 

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

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SOURCE REGENXBIO Inc.