REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023

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REGENXBIO Inc. presents interim clinical trial data at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023, showcasing progress in gene therapies for neurodegenerative disorders.

ROCKVILLE, Md., Aug. 23, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel (August 29 – September 1). Investigators will deliver encore presentations of interim clinical trial data from REGENXBIO's pipeline of one-time investigational AAV Therapeutics for progressive, neurodegenerative lysosomal storage disorders, as well as initial interim data from the single-patient, investigator-initiated study of RGX-181 for the treatment of CLN2 disease.

Presentations:

Title: RGX-121: An investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II), an interim analysis of data from the first-in-human study
Presenter: Paul Harmatz, M.D., UCSF Benioff Children's Hospital, USA
Date/Time: Wednesday, August 30, 2023, 2:00 pm Israeli Daylight Times (IDT)

Title: RGX-111: An investigational gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis data from first-in-human study
Presenter: Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, USA
Date/Time: Wednesday, August 30, 2023, 9:15 am IDT

Posters:

Title: First-in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6-month follow up
Authors: Carolina Fischinger De Souza, M.D., Ph.D., Hospital de Clinicas de Porto Alegre, Brazil
Date/Time: Wednesday, August 30, 2023, 6:35 pm - 8:30 pm IDT

Title: Daily living skills on the Vineland Adaptive Behavioral Scale version 2 (VABS-II) in neuronopathic mucopolysaccharidosis type II (MPS II)
Authors: Michelle Wood, Great Ormond Street NHS Foundation Trust; Dawn Phillips, Ph.D., Yoonjin Cho, Ph.D., Caroline Mulatya, Catherine Wilson, D.P.T., Joe Hagood,
Paulo Falabella, M.D., Ph.D., REGENXBIO Inc.
Date/Time: Wednesday, August 30, 2023, 6:35 pm - 8:30 pm IDT

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, 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.

Contacts:

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

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