REGENXBIO Announces Presentations at the American Society of Gene and Cell Therapy’s 23rd Annual Meeting

ROCKVILLE, Md., April 28, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV^® Technology Platform, today announced that three oral presentations, and several poster presentations, will be presented at the American Society of Gene and Cell Therapy’s 23^rd Annual Meeting taking place May 12-15, 2020 in virtual format. Presentations and posters will be available at www.asgct.org.

The oral presentations include:

Abstract Title: Quantitative PET/CT based pharmacokinetic study of AAV9 administered to the cerebrospinal fluid of non-human primates (abstract #135)
Session Title: AAV Vectors Preclinical and Proof-of-Concept Studies in Optimizing the Toolbox
Presenter: Mikhail Papisov, Ph.D., Massachusetts General Hospital, Shriners Hospitals for Children, Harvard Medical School
Date/Time: Tuesday, May 12, 2020 from 4:00 - 4:15 p.m. ET

Abstract Title: RGX-314 Ocular Gene Therapy: Overview of Phase I/IIa Ongoing Trial for Neovascular Age-related Macular Degeneration (nAMD) and Future Directions (abstract #1305)
Session Title: AAV Vectors - Clinical Studies
Presenter: Olivier Danos, Ph.D., Senior Vice President and Chief Scientific Officer, REGENXBIO
Date/Time: Friday, May 15, 2020 from 10:15-10:30 a.m. ET

Abstract Title: AAV gene therapy in mucopolysaccharidosis IVA murine models (abstract #1351)
Session Title: Gene Therapy for Inborn Errors of Metabolism: New Approaches
Presenter: Shunji Tomatsu, M.D., Ph.D., Nemours/Alfred I. duPont Hospital for Children
Date/Time: Friday, May 15, 2020 from 11:15-11:30 a.m. ET

The posters include:

Abstract Title: AAV-mediated antibody delivery for hereditary angioedema (abstract #190)
Presenter: Joseph T. Bruder, Ph.D., Senior Director, Target Discovery, REGENXBIO
Session Title: AAV Vectors - Preclinical and Proof-of-Concept Studies
Date/Time: Tuesday, May 12, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Interim data from the first in human RGX-121 gene therapy trial for the treatment of severe MPS II (Hunter syndrome) (abstract #614)
Presenter: Marie-Laure Nevoret, M.D., Senior Clinical Development Lead, REGENXBIO
Session Title: AAV Vectors - Clinical Studies
Date/Time: Wednesday, May 13, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Development of a vectorized antibody platform for liver and skeletal muscle gene transfer (abstract #556)
Presenter: Devin S. McDougald, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO
Session Title: AAV Vectors – Virology & Vectorology
Date/Time: Wednesday, May 13, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Use of Prophylactic Steroids to Mitigate Potential T-Cell Response in AAV8-Mediated hLDLR Gene Transfer in Subjects with Homozygous Familial Hypercholesterolemia (abstract #612)
Presenter: Marina Cuchel, M.D., Ph.D., University of Pennsylvania
Session Title: AAV Vectors - Clinical Studies
Date/Time: Wednesday, May 13, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Process development and scaleup comparisons for transient Production of AAV (abstract #1266)
Presenter: Robert Stadelman, Senior Scientist, REGENXBIO
Session Title: Vector and Cell Engineering, Production or Manufacturing
Date/Time: Thursday, May 14, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Structural and biochemical characterization of potentially under-utilized gene therapy vector AAV7 (abstract #1007)
Presenter: Samantha A. Yost, Ph.D., Scientist II, Gene Transfer Technologies, REGENXBIO
Session Title: AAV Vectors - Virology and Vectorology
Date/Time: Thursday, May 14, 2020 from 5:30-6:30 p.m. ET

Abstract Title: Development of a Sensitive and Robust Cell-Based Assay for Measuring Potency of the NAV AAV8 Vector-Derived RGX-314 Gene Therapy Product for the Treatment of Wet Age-Related Macular Degeneration (abstract #1005)
Presenter: Raza Zaidi, Senior Associate Scientist, REGENXBIO
Session Title: AAV Vectors - Virology and Vectorology
Date/Time: Thursday, May 14, 2020 from 5:30-6:30 p.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:
Heather Savelle, 212-600-1902
heather@argotpartners.com

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

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