PR Newswire
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
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
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