REGENXBIO Announces Orphan Drug Designation Granted to RGX-202, a New Candidate for Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

ROCKVILLE, MD., 22 November 2021 / PRNewswire / –

  • Unique potential gene therapy for the treatment of Duchenne, includes a new optimized microdystrophin transgene and the proprietary NAV of REGENXBIO® AAV8 vector
  • Commercial scale cGMP material for use in clinical development
  • The company is on track to submit an IND by the end of 2021

REGENXBIO Inc. (Nasdaq: RGNX) today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation for RGX-202, a unique potential gene therapy for the treatment of muscular dystrophy of Duchenne (Duchenne). RGX-202 is designed to provide a novel optimized microdystrophin transgene with a unique C-terminal domain and muscle-specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. RGX-202 uses REGENXBIO’s proprietary NAV® AAV8 vector.

“This important designation is an important step in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne,” said Olivier Danos, Ph.D., Scientific Director of REGENXBIO. “The novel microdystrophin transgene in RGX-202 comprises coding regions that retain essential functional elements of natural dystrophin to potentially improve muscle strength and resistance in patients with Duchenne. We look forward to advancing this unique gene therapy in the clinic.

REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021. The cGMP material on a commercial scale has already been produced at a capacity of 1,000 liters per year. using REGENXBIO’s suspension cell culture manufacturing process, and the cGMP facility is expected to produce up to 2,000 liters for clinical development of RGX-202.

The FDA orphan drug designation is granted to investigational therapies that treat rare diseases or medical conditions that affect fewer than 200,000 people in United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, waivers of certain FDA fees, and seven years of market exclusivity. after approval.

About RGX-202

RGX-202 is designed to provide a novel microdystrophin transgene that retains key elements of the dystrophin protein, including an extended coding region of the C-Terminal (CT) domain found in natural dystrophin, as well as other fundamental improvements in the transgene. The presence of the CT domain has been shown to recruit several key proteins into the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, have the potential to improve gene expression, increase translational efficiency, and reduce immunogenicity. RGX-202 is designed to support targeted gene delivery and expression in skeletal and cardiac muscle using the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle targeting promoter (Spc5- 12).

About REGENXBIO inc.

REGENXBIO is a leading clinical-stage biotechnology company that seeks to improve lives through the curative potential of gene therapy. NAV of REGENXBIO® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, includes exclusive rights to over 100 new AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third party licensees of the NAV technology platform apply the NAV technology platform to the development of a broad portfolio of candidates in multiple therapeutic areas.

Forward-looking statements
This press release contains “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 usually accompanied by words that express projected future events or outcomes such as “believe”, “could”, “will”, “estimate”, “continue”, “anticipate”. “,” Assume, “” design “,” intend “,” expect “,” might “,” plan “,” potential “,” predict “,” seek “,” should “,” should “or by variations of such words or by similar expressions. Forward-looking statements include statements relating, among other things, to the future operations and clinical trials of REGENXBIO. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyzes 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 that REGENXBIO deems appropriate in the circumstances. ty of actual results and developments with REGENXBIO’s expectations and forecasts is subject to a number of risks and uncertainties, including the timing of recruitment, start-up and completion and the success of clinical trials conducted by REGENXBIO, its licensees and partners, the timing of start and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the development and timely 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 of the businesses and markets in which REGENXBIO operates, size and the growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of pro candidates, the impact of the COVID-19 pandemic or similar public health crises on REGEN XBIO’s activities and other factors, many of which are beyond REGENXBIO’s control. Refer to “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, 2020 and the comparable “risk factors” sections of REGENXBIO’s Quarterly Reports on Form 10-Q and other documents, which have been filed with the United States Securities and Exchange Commission (SEC) and are available at SEC Web at All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Actual results or anticipated developments may not be realized or, even if they are substantially realized, they may not have the consequences or expected effects on REGENXBIO or its activities or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in forward-looking statements. Readers are cautioned not to place undue reliance on any 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 assumes no obligation, and specifically disclaims any obligation, to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Tricia Truehart
Investor relations and corporate communication
[email protected]

Brendan Burns, 212-600-1902
[email protected]

David Rosen, 212-600-1902
[email protected]

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