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REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting

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REGENXBIO Inc. (Nasdaq: RGNX) will present at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting from May 16-19, 2022, in Washington, D.C. Key presentations include interim analyses of the RGX-121 and RGX-111 gene therapies for MPS II and MPS I, respectively, and insights on novel AAV vectors. Notable speakers include Nina Hunter, Ph.D., and Roberto Giugliani, M.D., among others. The event highlights REGENXBIO's capabilities in gene therapy, emphasizing its NAV Technology Platform with over 100 AAV vectors developed for various therapeutic areas.

Positive
  • REGENXBIO showcases its NAV Technology Platform with over 100 AAV vectors.
  • Presentations focus on significant gene therapy advancements for MPS II and MPS I.
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  • None.

ROCKVILLE, Md., May 3, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting, taking place virtually and in Washington, D.C. from May 16 through 19, 2022. The presentations highlight the Company's end-to-end capabilities across research and early development, clinical development and manufacturing.  

The presentations will be presented as follows:

Presenter: Nina Hunter, Ph.D., VP, Regulatory and Science Policy at REGENXBIO, Pathway Development Consortium
Session: Accelerated Approval for Gene Therapies
Date/Time: Monday, May 16, 2022, 8:00 a.m. ET

Abstract Title: RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study (abstract #52)
Presenter: Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil
Session: Gene and Cell Therapy Trials in Progress
Date/Time: Monday, May 16, 2022, 1:30 p.m. ET

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 #802)
Presenter: Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, CA
Session: Gene and Cell Therapy Trials in Progress 
Date/Time: Tuesday, May 17, 2022, 5:30 p.m. ET

Abstract Title: VP1 Unique and VP1/2 Shared Region Serotype Swap Hybrids Enhance Desirable AAV Properties
Presenter: Samantha Yost, Ph.D., Senior Scientist, Gene Transfer Technologies at REGENXBIO & Randy Qian, Ph.D., Scientist I, Gene Transfer Technologies at REGENXBIO (abstract #500)
Session: AAV Vectors - Virology and Vectorology II
Date/Time: Tuesday, May 17, 2022, 5:30 p.m. ET

Abstract Title: A Novel Peptide Insertion into VR-IV or VR-VIII of AAV9 Improves Transduction Strength and Penetration Depth Upon Intravitreal Injection (abstract: #521)
Presenter: Wei-Hua Lee, Ph.D., Scientist II, Target Discovery at REGENXBIO & Samantha Yost, Ph.D., Senior Scientist, Gene Transfer Technologies at REGENXBIO (co-first authors)
Session: AAV Vectors - Preclinical and Proof-of-concept Studies II
Date/Time: Tuesday, May 17, 2022, 5:30 p.m. ET

Abstract Title: Adeno-Associated Virus Adsorption on Different Surfaces Relevant to Production of Pre-Clinical and Clinical Material (abstract #765)
Presenter: Amanda Zhang, Scientific Project Manager, Vector Core at REGENXBIO
Session: Vector Product Engineering, Development or Manufacturing II
Date/Time: Tuesday, May 17, 2022, 5:30 p.m. ET

Abstract Title: Gene Expression from AAV Vectors in the Liver-A Comparative Study Across Species, Promoters and AAV Serotypes (abstract #824)
Presenter: Subha Karumuthil-Melethil, Ph.D., Principal Scientist, Target Discovery at REGENXBIO
Session: AAV Developments in Liver, T-Cells and Toxicity
Date/Time: Wednesday May 18th, 2022, 5:00 p.m. ET

Abstract Title: Intraparenchymal Administration to the Striatum of a Barcoded AAV Library for the Characterization of Capsid Tropisms in Rodents and Non-human Primates (abstract #892)
Presenter: Jared Smith, Ph.D., Principal Scientist, Target Discovery at REGENXBIO
Session: AAV Vectors – Virology and Vectorology III
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: A Longitudinal, Comparative Analysis of Transgene Expression Durability via Different Promoters in the Striatum of Mice Delivered by Intraparenchymal Injection of rAAV9 (abstract #894)
Presenter: Brad Hollidge, Ph.D., Scientist II, Target Discovery at REGENXBIO
Session: AAV Vectors - Virology and Vectorology III
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: Stability of Microdystrophin Proteins Measured by Pulse-Chase Assays in Tissue Culture (abstract # 1065)
Presenter: Kirk Elliott, Scientist I, Gene Transfer Technology at REGENXBIO
Session: Musculo-skeletal Diseases
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: AAV Vectors Consistently Display Higher Transcriptional Activity in MDX Mouse Muscle Versus Normal Mouse Skeletal Muscle (abstract #1063)
Presenter: Randy Qian, Ph.D., Scientist I, Gene Transfer Technologies at REGENXBIO
Session: Musculo-Skeletal Diseases
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: Recruitment of nNOS and Other Dystrophin-Associated Protein Complex Members by Different Microdystrophin Constructs (abstract #1068)
Presenter: Steven Foltz, Ph.D., Scientist II, Target Discovery at REGENXBIO
Session: Musculo-Skeletal Diseases
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: Evaluating the Impact of Transgene-Specific CpG Removal on AAV9-Mediated Gene Transfer and Immune Responses in the Balb/C Mouse Strain Provides Novel Insights of CpG Depletion (abstract #1671)
Presenter: Justin Glenn, Ph.D., Senior Scientist, Target Discovery at REGENXBIO
Session: Immunological Aspects of Gene Therapy and Vaccines II
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: A Novel AAV8-Based Gene Therapy for Duchenne Muscular Dystrophy: Preclinical Studies in the Mdx Mouse (abstract #1067)
Presenter: SunJung Kim, Ph.D., DABT, Senior Scientist, Pharmacology and Toxicology at REGENXBIO
Session: Musculo-skeletal Diseases
Date/Time: Wednesday, May 18, 2022, 5:30 p.m. ET

Abstract Title: Identification and Characterization of an AAV9-Based Engineered Capsid Variant Capable of Mediating Enhanced Transcription in the Central Nervous System of Non-Human Primates and Rodents (abstract #1200)
Presenter: April Giles, Ph.D., Scientist II, Gene Transfer Technologies at REGENXBIO & Samantha Yost, Ph.D., Senior Scientist, Gene Transfer Technologies at REGENXBIO (co-first authors)
Session: Novel AAV Capsids for the Brain, Kidney and Eye
Date/Time: Thursday, May 19, 2022, 11:45 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:

Dana Cormack
Corporate Communications
dcormack@regenxbio.com 

Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com

(PRNewsfoto/REGENXBIO Inc.)

 

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SOURCE REGENXBIO Inc.

FAQ

What is the significance of REGENXBIO's presentations at the ASGCT meeting?

The presentations at the ASGCT meeting highlight REGENXBIO's advancements in gene therapies, particularly for MPS II and MPS I, showcasing its research and clinical development capabilities.

When will REGENXBIO present its interim analysis for RGX-121?

REGENXBIO will present its interim analysis for RGX-121 on May 16, 2022, at 1:30 p.m. ET.

What therapies are being discussed at the ASGCT meeting for REGENXBIO?

The therapies discussed include RGX-121 for MPS II and RGX-111 for MPS I, along with various studies on AAV vectors.

How does REGENXBIO's NAV Technology Platform benefit its gene therapy development?

REGENXBIO's NAV Technology Platform allows the development of a broad pipeline of gene therapies, leveraging exclusive rights to over 100 novel AAV vectors.

Who will present on the RGX-111 gene therapy at the ASGCT meeting?

Raymond Wang, M.D., will present on the RGX-111 gene therapy for MPS I at the ASGCT meeting.

REGENXBIO Inc.

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