ROCKVILLE, Md., Jan. 30,
2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced data from its RGX-121 (clemidsogene lanparvovec)
program for the treatment of mucopolysaccharidosis type II (MPS
II), also known as Hunter syndrome, will be shared at the
21st Annual WORLDSymposium™ 2025, taking place in
San Diego, CA February 3-7, 2025.
The following presentations include an encore of the topline
results from the pivotal phase of the Phase I/II/III
CAMPSIITE® trial of RGX-121.
Abstract Title: Audiology assessment of participants in
CAMPSIITE®, a phase I/II/III study of RGX-121 in
neuronopathic MPS II (poster 35)
Presenter: Nidal
Boulos, Ph.D., Director, Clinical
Science, REGENXBIO
Date/Time: Wednesday, February 5, 2025; 3:30 p.m. PT
Abstract Title: CAMPSIITE® Phase I/II/III: An
interim clinical study update of RGX121, an investigational gene
therapy for the treatment of neuronopathic mucopolysaccharidosis
type II (MPS II)
Presenter: Paul Harmatz,
M.D., UCSF Benioff Children's Hospital
Date/Time: Thursday, February 6, 2025;
8:30 a.m. PT
ABOUT REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. Since its founding
in 2009, REGENXBIO has pioneered the development
of AAV Therapeutics, an innovative class of gene
therapy medicines. REGENXBIO is advancing a pipeline
of AAV Therapeutics for rare and retinal diseases,
including RGX-202 for the treatment of Duchenne,
ABBV-RGX-314 for the treatment of wet AMD and diabetic
retinopathy, being developed in collaboration
with AbbVie, and RGX 121 for the
treatment of MPS II. Thousands of patients have been
treated with REGENXBIO's AAV Therapeutic platform,
including Novartis' ZOLGENSMA for
children with spinal muscular atrophy. Designed to be one-time
treatments, AAV Therapeutics have the potential to
change the way healthcare is delivered for millions of people. For
more information, please visit
www.regenxbio.com.
Contacts:
Dana
Cormack
Corporate
Communications
dcormack@regenxbio.com
Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com
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SOURCE REGENXBIO Inc.
