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ROCKVILLE, Md.,Sept. 29, 2025/PRNewswire/ -- REGENXBIO Inc. (Nasdaq:RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society taking place in Vienna, Austria, October 7-11, 2025.
The podium presentation will share new analysis of previously presented 12-month functional data from the Phase I/II trial of RGX-202, including individual patient improvement on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model from the Collaborative Trajectory Analysis Project. As reported, RGX-202 demonstrated a favorable safety profile with no serious adverse events or adverse events of special interest observed in the Phase I/II study. Pivotal dose participants exceeded baseline-matched external natural history controls on all functional measures.
Using multiple models of natural history disease progression, these results further demonstrate the potential of RGX-202 to serve as a differentiated, best-in-class gene therapy for the treatment of Duchenne muscular dystrophy.
Presentation:RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical DataPresenter:StevePakola, M.D., Chief Medical Officer,REGENXBIOSession:Short Oral Presentations 1: Updates onSMAandDMDTrials- Accompanying Poster: P425Date/Time:October 8, 3:30 CET
The presentation will be available on the Publications page of REGENXBIO's website,www.REGENXBIO.com.
About RGX-202RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain.
Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV®AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress®suspension-based platform process.
ABOUT REGENXBIO Inc.REGENXBIOis a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009,REGENXBIOhas pioneered the field ofAAVgene therapy.REGENXBIOis advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment ofDuchenne;clemidsogene lanparvovec(RGX-121) for the treatment ofMPSII and RGX-111 for the treatment ofMPSI, both in partnership withNippon Shinyaku; andsurabgene lomparvovec(ABBV-RGX-314) for the treatment of wetAMDand diabetic retinopathy, in collaboration withAbbVie. Thousands of patients have been treated withREGENXBIO's AAVplatform, including those receivingNovartis'ZOLGENSMA®.REGENXBIO'sinvestigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visitwww.REGENXBIO.com.
FORWARD-LOOKING STATEMENTSThis 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'sfuture operations and clinical trials.REGENXBIOhas based these forward-looking statements on its current expectations and assumptions and analyses made byREGENXBIOin light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factorsREGENXBIObelieves are appropriate under the circumstances. However, whether actual results and developments will conform withREGENXBIO'sexpectations 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 byREGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted byREGENXBIOand its development partners, the timing or likelihood of payments fromAbbVieorNippon Shinyaku, 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 whichREGENXBIOoperates, 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 ofREGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections ofREGENXBIO'sAnnual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections ofREGENXBIO'sQuarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website atWWW.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 onREGENXBIOor 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,REGENXBIOdoes 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 Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
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