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REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023
ROCKVILLE, Md., Aug. 23, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the Society for the Study of Inborn Errors of
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[{"type":"text","content":"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.\nPresentations:\nTitle: 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 studyPresenter: Paul Harmatz, M.D., UCSF Benioff Children's Hospital, USADate/Time: Wednesday, August 30, 2023, 2:00 pm Israeli Daylight Times (IDT)\nTitle: RGX-111: An investigational gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis data from first-in-human studyPresenter: Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, USADate/Time: Wednesday, August 30, 2023, 9:15 am IDT\nPosters:\nTitle: 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 upAuthors: Carolina Fischinger De Souza, M.D., Ph.D., Hospital de Clinicas de Porto Alegre, BrazilDate/Time: Wednesday, August 30, 2023, 6:35 pm - 8:30 pm IDT\nTitle: 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\nAbout REGENXBIO Inc.\nREGENXBIO 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,...