ROCKVILLE, Md., May 2, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
presentations at the American Society of Gene & Cell Therapy
(ASGCT) 26th Annual Meeting, taking place in Los Angeles, California from May 16 through 20, 2023. The presentations
highlight the Company's end-to-end capabilities across clinical
development and research and early development.
The presentations will be presented as follows:
Oral Presentations:
Abstract Title: AUF1 Gene Therapy for Duchenne Muscular
Dystrophy Increases Durable Endogenous Utrophin Expression, Muscle
Regeneration and Muscle Function Performance in Pre-clinical Animal
Studies (abstract #135)
Presenter: Dounia Abbadi, Postdoctoral Fellow, NYU
Langone Medical Center
Session: Gene Tx Approaches for Muscle & Skeletal
Diseases
Date/Time: Thursday, May 18, 2023, 2:38-2:55 p.m. PST
Abstract Title: Mechanistic Evaluation of
Liver-specific Transgene Repression from AAV Vectors in Non-Human
Primates and Minipigs (abstract #249)
Presenter: Zhuo Wang, Ph.D., Scientist II, Research
& Early Development at REGENXBIO
Session: AAV Vector Genome Biology & Engineering II
Date/Time: Friday, May 19
3:45-4:00 p.m. PST
Poster Presentations:
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 #807)
Presenter: Paul Harmatz, M.D., Professor in Residence
at University of California, San
Francisco
Session: Wednesday Poster Session
Date/Time: Wednesday, May 17, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: In Silico Prediction and In Vivo Testing
of Promoters Targeting GABAergic Inhibitory Neurons (abstract
#385)
Presenter: Robert Duba-Kiss, Ph.D. Candidate,
Pharmacology/Neuroscience at University of
Toronto University of
Toronto
Session: Wednesday Poster Session
Date/Time: Wednesday, May 17, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: Development of AAV-Expressed C5
Inhibitor to Locally Suppress Complement Pathway Activation in the
Eye as a Potential Treatment for Dry Age-related Macular
Degeneration (abstract #364)
Presenter: Wei-Hua Lee, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Wednesday Poster Session
Date/Time: Wednesday, May 17,
2023 12:00-2:00 p.m. PST &
5:30-7:00 p.m. PST
Abstract Title: Addition of a Protein Domain from the
Dystrophin C-Terminus Improves Functional and Biochemical
Properties of AAV-Encoded Microdystrophin (abstract #668)
Presenter: Steven Foltz, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Wednesday Poster Session
Date/Time: Wednesday, May 17, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: Optimization of AAV-DARPin Fusions to
Redirect Capsid Tropism (abstract #447)
Presenter: Elad Firnberg, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Wednesday Poster Session
Date/Time: Wednesday, May 17, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
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 #932)
Presenter: Laura Pisani-Betancourt, MD, MBA, FACMG,
Senior Medical Director, Clinical Development Lead at REGENXBIO
Session: Thursday Poster Session
Date/Time: Thursday May 18, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: Local Delivery of Vectorized Therapeutics
Targeting a Pro-inflammatory Cytokine Effectively Reduces Disease
Severity in a Mouse Model of Non-Infectious Uveitis (abstract
#860)
Presenter: Jessica Gumerson, Ph.D., Scientist II,
Research & Early Development at REGENXBIO
Session: Thursday Poster Session
Date/Time: Thursday May 18, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: High Resolution Biodistribution Analysis
Following Suprachoroidal Administration of a Pool of AAV3B, AAV8,
and AAV9 Vectors to Non-Human Primates Reveals Spatial and
Cell-Based Tropism Differences (abstract #876)
Presenter: April R. Giles, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Thursday Poster Session
Date/Time: Thursday, May 18, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: Utility of Cryofluorescence Tomography
(CFT) as an Unbiased Method to Describe AAV Biodistribution
(abstract #1278)
Presenter: Samantha A. Yost, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Friday Poster Session
Date/Time: Friday, May 19, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
Abstract Title: Development of a Self-Complementary
AAV.U7snRNA Vector for Efficient Dystrophin Exon 53 Skipping
(abstract #1565)
Presenter: Randolph Qian, Ph.D., Scientist II, Research
& Early Development at REGENXBIO
Session: Friday Poster Session
Date/Time: Friday, May 19,
2023 12:00-2:00 p.m. PST &
5:30-7:00 p.m. PST
Abstract Title: Kinetic Analyses Reveal Very Early
CpG-specific Antagonism to AAV-Mediated Transgene Expression:
Implications for TLR9 Stimulatory Effects Prior to Effector T Cell
Activation Following Gene Transfer (abstract #1487)
Presenter: Justin D. Glenn, Ph.D., Senior Scientist,
Research & Early Development at REGENXBIO
Session: Friday Poster Session
Date/Time: Friday, May 19, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
NAV® Technology Licensees:
Abstract Title: AAV Vector DNA Carrying Two Concatenated
miRNA Stem-Loops is Highly Homogenous and Stable in GLP-Grade AAV
Product
Presenter: Chenxia HE, Ph.D., Director of Gene Therapy
at uniQure, in collaboration with REGENXBIO
Session: AAV Vectors – Product Development Manufacturing:
Analytics & Stability Studies
Date/Time: Thursday, May 18, 2023, 1:45-2:00 p.m. PST
Abstract Title: Preclinical Proof-of-Concept of AMT-260,
a Novel AAV9-Dual microRNA-Based Vector Targeting GRIK2 for the
Treatment of Temporal Lobe Epilepsy (abstract #1159)
Presenter: Nick Pearson, Head of Toxicology &
Translational Safety at uniQure, in collaboration with
REGENXBIO
Session: Thursday Poster Session
Date/Time: Thursday, May 18, 2023 12:00-2:00 p.m. PST & 5:30-7:00 p.m. PST
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 and AAV9. 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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