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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