REGENXBIO Announces Presentations at the American Society of Gene and Cell Therapy's 24th Annual Meeting

ROCKVILLE, Md., April 27, 2021 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that four oral and nine poster presentations will be presented at the American Society of Gene and Cell Therapy's 24^th Annual Meeting taking place from May 11 to 14, 2021, in virtual format. Presentations and posters will be available at https://www.asgct.org/.

Oral presentations include:

Abstract Title: Effects of Sexual Dimorphism and Genetic Background on AAV Tissue Transduction in Mice Following Intravenous Administration of a Diverse Capsid Pool (abstract #94)
Presenter: Elad Firnberg, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO
Session Title: Novel Factors in AAV Transduction and AAV Genomes
Date/Time: Wednesday, May 12, 2021 from 6:00 to 6:15 p.m. ET

Abstract Title: Intravenous Administration of a Barcoded and Pooled AAV Library for the Comprehensive Characterization and Comparison of Capsid Tropisms (abstract #189)
Presenter: April Giles, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO
Session Title: Novel AAV Biology and Platform Technologies
Date/Time: Thursday, May 13, 2021 from 6:00 to 6:15 p.m. ET

Abstract Title: High-Throughput Screening of AAV Productivity to Enable Rapid Capsid Characterization (abstract #192)
Presenter: Jenny Egley, Scientist, Gene Transfer Technologies, REGENXBIO
Session Title: Novel AAV Biology and Platform Technologies
Date/Time: Thursday, May 13, 2021 from 6:45 to 7:00 p.m. ET

Abstract Title: RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II: Interim Analysis of the First in Human Study (abstract #215)
Presenter: Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil
Session Title: Gene Therapy for Lysosomal Storage Disorders
Date/Time: Friday, May 14, 2021 from 12:15 to 12:30 p.m. ET

Poster presentations include:

Abstract Title: Identification of Transcriptional Start Sites in a Tandem Promoter by 5'-RACE and NGS (abstract #317)
Presenter: Kirk Elliot, Senior Associate Scientist, REGENXBIO
Session Title: AAV Vectors - Virology and Vectorology
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: Comparative Cardiac, Neurologic, and Skeletal Effectiveness of Intravenous and Intrathecal AAV9-IDUA Delivered Individually or Combined in a Murine Model of Mucopolysaccharidosis Type I (abstract #490)
Presenter: Lalitha Belur, Ph.D., Assistant Professor Genetics, Cell Bio at University of Minnesota
Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: Comprehensive Evaluation of an AAV Library in the Mice and Nonhuman Primate Ocular Tissues Following Intravitreal Delivery (abstract #568)
Presenter: Wei-Hua Lee, Ph.D., Scientist II, Research and Early Development, Target Discovery, REGENXBIO
Session Title: Neurologic Diseases
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: RGX-202, an AAV8 Vector Encoding an Extended Microdystrophin Efficiently Restores Dystrophin Associated Protein Complex and Corrects Satellite Cells in a Dystrophic Mouse Model (abstract #597)
Presenter: Chunping Qiao, Ph.D., Principal Scientist, REGENXBIO
Session Title: Musculo-Skeletal Diseases
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: A Novel AAV8 Vector for Microdystrophin Gene Therapy of Duchenne Muscular Dystrophy: Preclinical Studies in the MDX Mouse (abstract #614)
Presenter: SunJung Kim, Ph.D., Senior Scientist, Pharmacology and Toxicology, Preclinical Development, REGENXBIO
Session Title: Musculo-Skeletal Diseases
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: A Case Study on Viral Clearance for a Downstream AAV Process using a Model Virus Panel and Non-Infectious MVM Surrogate (abstract #877)
Presenter: Shaojie Weng, Scientist II
Session Title: Pharmacology/Toxicology Studies or Assay Development
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: Assessment of Inflammatory and Neurodegenerative Biomarkers in NHPs with DRG Toxicity (abstract #889)
Presenter: Ryan Reddinger, Ph.D., Scientist I, Preclinical Development, REGENXBIO
Session Title: Pharmacology/Toxicology Studies or Assay Development
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: Quantification of Double-Stranded RNAs Produced by AAV Vectors: A Methodological Reassessment (abstract #891)
Presenter: Chunping Qiao, Ph.D., Principal Scientist, REGENXBIO
Session Title: Pharmacology/Toxicology Studies or Assay Development
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

Abstract Title: Identification of a Kidney Specific AAV Following Insertion of Tissue Homing Peptides in Variable Region IV (abstract #292)
Presenter: Samantha A. Yost, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO
Session Title: AAV Vectors - Virology and Vectorology
Date/Time: Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. ET

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:

Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com

Investors:
Brendan Burns, 212-600-1902
brendan@argotpartners.com

Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com

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