- New interim data from Phase I/I/III trial of RGX-121 for the
treatment of MPS II and Phase I/II trial of RGX-111 for the
treatment of MPS I to be presented
ROCKVILLE, Md., Feb. 17,
2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced that three oral and eight poster presentations will
be presented at the 19th Annual WORLDSymposium™
2023, taking place in Orlando,
Florida from February 22 through 26,
2023. The presentations include new interim results from the
Phase I/II/III clinical trial of RGX-121 for the treatment of
mucopolysaccharidosis type II (MPS II), also known as Hunter
Syndrome, and the Phase I/II trial of RGX-111 for the treatment of
severe mucopolysaccharidosis type I (MPS I).
REGENXBIO will also host a sponsored symposium session, titled
"AAV Gene Therapy as a Potential Treatment Modality for
Neuronopathic MPS II" on Friday, February
24, 2023, at 11:45 a.m.
ET.
The oral presentations will be presented as follows:
Abstract 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 (abstract #191)
Presenter: Raymond Wang,
M.D., Division of Metabolic Disorders, CHOC Children's Hospital,
Department of Pediatrics, University of
California, Irvine, CA
Date/Time: Friday, February 24, 2023, 9:24 a.m. ET
Abstract Title: In vitro pharmacology study using retina
organoids and retina-on-a-chip of CLN2 patient-derived induced
pluripotent stem cells (abstract #311, also available as poster
#201)
Presenter: Kwi Hye Kim,
Ph.D., Principal Scientist, Preclinical Development at REGENXBIO
Inc.
Date/Time: Saturday, February 25, 2023, 2:48 p.m. ET
Abstract Title: RGX-121 gene therapy for the treatment of
neuronopathic mucopolysaccharidosis type II (MPS II): Interim
analysis of data from the first in human study (abstract
#LB-21)
Presenter: Can Ficicioglu, M.D., Ph.D., Professor of
Pediatrics at Perelman School of Medicine at the University of Pennsylvania, Director of the Newborn
Metabolic Screening Program and the Lysosomal Storage Diseases
Program, Clinical Director of the Metabolic Disease Program
Children's Hospital of Philadelphia
Date/Time: Sunday, February 26, 2023, 9 a.m. ET (also available as an ePoster
Wednesday, February 22, 2023,
3 p.m. ET)
The poster presentations will be presented as follows:
Abstract Title: Spontaneous seizures associated with
cortical interneuron loss in Cln2R207X mice are
ameliorated via gene therapy (abstract #350)
Presenter: Keigo Takahashi,
Ph.D. Candidate, Pediatric Storage Disease Lab, Washington University in St. Louis
Date/Time: Thursday, February 23, 2023, 3-4 p.m. ET
Abstract Title: RGX-381 Gene Therapy for the Treatment of
Ocular Manifestations of Late-Infantile Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2 Disease): Overview of Nonclinical
Development Program (abstract #LB-17)
Presenter: Gary Chan, Ph.D.,
Scientist II, Preclinical Development at REGENXBIO Inc.
Date/Time: Thursday, February 23, 2023, 4-5 p.m. ET
Abstract Title: The relationship between the expanded
neuronal ceroid lipofuscinosis 2 (CLN2) clinical rating scale for
motor function (CLN2 CRS-MX) and GAITRite® parameters (abstract
#152)
Presenter: Luca Hagenah,
M.D., University Medical Center Hamburg-Eppendorf, Department of
Pediatrics, Hamburg
Date/Time: Friday, February 24, 2023, 3-5 p.m.
Abstract Title: Daily living skills on the Vineland
Adaptive Behavioral Scale Version 2 (VABS-II) following RGX-121
treatment in participants with neuronopathic Mucopolysaccharidosis
Type II (MPS II) (poster #285)
Presenter: Dawn Phillips
P.T., M.S., Ph.D., Senior Director of Clinical Outcomes
Research at REGENXBIO Inc.
Date/Time: Saturday, February 25, 2023, 3-5 p.m.
Abstract Title: Quantifying and modelling disease
progression trajectory for natural history of MPS II (abstract
#73)
Presenter: Yoonjin Cho,
Ph.D., Senior Director, Biostatistics at REGENXBIO Inc.
Date/Time: Saturday, February 25, 2023, 3-5 p.m.
Abstract Title: RGX-381: First-in-human clinical trial of
an investigational AAV9 gene therapy encoding TPP1 for the
treatment of ocular manifestations of CLN2 Batten disease (abstract
#LB-45)
Presenter: Christina Ohnsman,
M.D., Senior Clinical Development Lead at REGENXBIO Inc.
Date/Time: Saturday, February 25, 2023, 3-5 p.m.
Abstract Title: Characterization of retinal
degeneration phenotype in classic CLN2 disease using OCT biomarkers
and an in vitro retinal model (abstract #261)
Presenter: Christina Ohnsman,
M.D., Senior Clinical Development Lead at REGENXBIO Inc.
Date/Time: Saturday, February 25, 2023, 3-5 p.m.
Abstract Title: Loss of visual function associated with
photoreceptor degeneration in CLN2 disease (abstract #260)
Presenter: Christina Ohnsman,
M.D., Senior Clinical Development Lead at REGENXBIO Inc.
Date/Time: Saturday, February 25, 2023, 3-5 p.m.
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 in multiple therapeutic areas.
Contacts:
Dana
Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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SOURCE REGENXBIO Inc.