ROCKVILLE, Md., Aug. 23,
2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced presentations at the Society for the Study of
Inborn Errors of Metabolism Annual Symposium, taking place from
August 30 through September 2, 2022,
in Freiburg, Germany. The
presentations will highlight new data from the Phase I/II/III
CAMPSIITE™ trial of RGX-121, an investigational one-time AAV
Therapeutic for the treatment of Mucopolysaccharidosis Type II (MPS
II), also known as Hunter Syndrome, and an encore data presentation
for RGX-111, an investigational one-time AAV Therapeutic for the
treatment of severe Mucopolysaccharidosis Type I (MPS I), or Hurler
syndrome.
Presentations include:
Title: RGX-121 gene therapy for the treatment of severe
mucopolysaccharidosis type II (MPS II): Interim analysis of data
from a Phase 1/2 study
Presenter: Roberto Giugliani, M.D., Ph.D., Professor,
Department of Genetics, UFRGS, Medical Genetics Service, HCPA,
Porto Alegre, Brazil
Date/Time: Wednesday, August 31,
2022, 10:00 – 10:15 a.m.
CEDT
Title: RGX-111 gene therapy for the treatment of severe
mucopolysaccharidosis type I (MPS I): Interim analysis of data from
the first in human study
Presenter: Raymond Wang,
M.D., Division of Metabolic Disorders, CHOC Children's Hospital,
Department of Pediatrics, University of
California, Irvine, CA
Date/Time: Thursday, September 1, 2022, 11:30 –
11:45 a.m. CEDT
Title: Natural History of Neurodevelopment in
Neuronopathic Mucopolysaccharidosis Type II (MPS II): Mullen Scales
of Early Learning (MSEL) Cognitive, Motor, and Language
Developmental Trajectories
Presenter: Maria Escolar, M.D., M.S., Professor of
Pediatrics, Director, Program for the Study of Neurodevelopment in
Rare Disorders, UPMC Children's Hospital of Pittsburgh
Date/Time: Wednesday, August 31, 2022, 6:45 –
8:15 p.m. CEDT
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:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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