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.
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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) Abstract Title: Mechanistic Evaluation of Liver-specific Transgene Repression from AAV Vectors in Non-Human Primates and Minipigs (abstract #249) 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) Abstract Title: In Silico Prediction and In Vivo Testing of Promoters Targeting GABAergic Inhibitory Neurons (abstract #385) 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) Abstract Title: Addition of a Protein Domain from the Dystrophin C-Terminus Improves Functional and Biochemical Properties of AAV-Encoded Microdystrophin (abstract #668) Abstract Title: Optimization of AAV-DARPin Fusions to Redirect Capsid Tropism (abstract #447) 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) 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) 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) Abstract Title: Utility of Cryofluorescence Tomography (CFT) as an Unbiased Method to Describe AAV Biodistribution (abstract #1278) Abstract Title: Development of a Self-Complementary AAV.U7snRNA Vector for Efficient Dystrophin Exon 53 Skipping (abstract #1565) 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) NAV® Technology Licensees: Abstract Title: AAV Vector DNA Carrying Two Concatenated miRNA Stem-Loops is Highly Homogenous and Stable in GLP-Grade AAV Product 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) 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 Investors:
SOURCE REGENXBIO Inc. |
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Company Codes: NASDAQ-NMS:RGNX |
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