REGENXBIO Announces Presentations at the 18th Annual WORLDSymposium™ 2022

Rhea-AI Summary

REGENXBIO Inc. (Nasdaq: RGNX) announced presentations at the 18th Annual WORLD Symposium™ in San Diego from February 7-11, 2022. The company will showcase interim data from Phase I/II clinical trials for RGX-121 and RGX-111, targeting mucopolysaccharidosis types I and II. Notably, five oral and seven poster presentations are scheduled, featuring key researchers and significant advancements in treatment methodologies. This event aims to highlight the company's progress in gene therapy, showcasing its innovative approaches to serious genetic disorders.

Positive

• Presentation of interim data for RGX-121 and RGX-111 at a prominent symposium.
• Engagement of reputable researchers to present findings, enhancing credibility.

Negative

• None.

ROCKVILLE, Md., Jan. 31, 2022 /PRNewswire/ -- 

• Interim data from Phase I/II trials of RGX-121 for the treatment of MPS II and RGX-111 for the treatment of MPS I to be presented

REGENXBIO Inc. (Nasdaq: RGNX) today announced that five oral and seven poster presentations will be presented at the 18^th Annual WORLDSymposium™, taking place in San Diego, CA from February 7 through 11, 2022. The presentations include interim results from the Phase I/II clinical trials of RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter Syndrome, and RGX-111 for the treatment of mucopolysaccharidosis type I (MPS I), also known as Hurler Syndrome.

The oral presentations will be presented as follows:

Abstract Title: Treatment of cardiac, neurologic, and skeletal manifestations of murine MPS I with AAV9-IDUA: Efficacy study of vector dose and route of administration
Presenter: Lalitha Belur, Ph.D., Research Assistant Professor Genetics, Cell Biology and Development at University of Minnesota
Date/Time: Tuesday, February 8, 2022, 12:12 p.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
Presenter: Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil
Date/Time: Wednesday, February 9, 2022, 11:12 a.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
Presenter: Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, CA
Date/Time: Wednesday, February 9, 2022, 11:36 a.m. ET

Abstract Title: Identification of a biomarker that differentiates neuronopathic forms of MPS I and MPS II
Presenter: Nidal Boulos, Ph.D., Director, Clinical Scientist at REGENXBIO Inc.
Date/Time: Thursday, February 10, 2022, 11:24 a.m. ET

Abstract Title: Establishment of in vitro model of CLN2 retinopathy using human induced pluripotent stem cells
Presenter: Kwi Hye Kim, Ph.D., Senior Scientist, Preclinical Development at REGENXBIO Inc.
Date/Time: Friday, February 11, 2022, 1:40 p.m. ET

The poster presentations will be presented as follows:

Abstract Title: Safety, Pharmacodynamics and Efficacy of AAV9.hCLN2 in Preclinical Studies (poster LB-10)
Presenter: Nicholas Buss, Ph.D., Senior Director, Preclinical Development at REGENXBIO Inc.
Date/Time: Monday, February 7, 2022, 6 p.m. – 8 p.m. ET

Abstract Title: Comparative effectiveness of intravenous and intrathecal AAV9.CB7.hIDS (RGX-121) in a murine model of mucopolysaccharidosis type II (poster #287)
Presenter: Miles C. Smith, University of Minnesota
Date/Time: Tuesday, February 8, 2022, 6 p.m. – 8 p.m. ET

Abstract: Title: The Expanded Neuronal Ceroid Lipofuscinosis 2 (CLN2) Clinical Rating Scale for Motor and Language Function: Development and Inter-Rater Reliability (poster #239)
Presenter: Dawn Philips, Ph.D., Director, Clinical Scientist, Outcomes Research at REGENXBIO Inc.
Date/Time: Thursday, February 10, 2022, 6 p.m. – 8 p.m. ET

Abstract Title: Natural History of Neurodevelopment in Neuronopathic Mucopolysaccharidosis Type II (MPS II): Mullen Scales of Early Learning (MSEL) Cognitive, Motor and Language Developmental Trajectories (poster #238)
Presenter: Dawn Philips, Ph.D., Director, Clinical Scientist, Outcomes Research at REGENXBIO Inc.
Date/Time: Thursday, February 10, 2022, 6 p.m. – 8 p.m. ET

Abstract Title: Beyond the normative data: Understanding the Bayley Scales of Infant Development version 3 (BSID-III) (poster #49)
Presenter: Yoonjin Cho, Ph.D., Director, Biostatistics at REGENXBIO Inc.
Date/Time: Thursday, February 10, 2022, 6 p.m. – 8 p.m. ET

Abstract Title: Beyond the normative data: Understanding the Mullen Scales of Early Learning (MSEL)
Presenter: Yoonjin Cho, Ph.D., Director, Biostatistics at REGENXBIO Inc. (poster #50)
Date/Time: Thursday, February 10, 2022, 6 p.m. – 8 p.m. ET

Abstract Title: Pilot study of novel optokinetic nystagmus-based visual acuity test in children with CLN2 disease (poster #218)
Presenter: Christina Ohnsman, M.D., Senior Clinical Development Lead at REGENXBIO Inc.
Date/Time: Thursday, February 10, 2022, 6 p.m. – 8 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:

Dana Cormack
Corporate Communications
dcormack@regenxbio.com 

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

 

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SOURCE REGENXBIO Inc.

FAQ

What is REGENXBIO presenting at the 18th Annual WORLD Symposium 2022?

REGENXBIO is presenting interim data from Phase I/II clinical trials for RGX-121 and RGX-111.

When is the 18th Annual WORLD Symposium taking place?

The symposium is scheduled from February 7 to February 11, 2022.

What are RGX-121 and RGX-111 used to treat?

RGX-121 is for mucopolysaccharidosis type II, and RGX-111 is for mucopolysaccharidosis type I.

Who are the key presenters for REGENXBIO's trials at the symposium?

Key presenters include researchers from the University of Minnesota and UFRGS, among others.

How many presentations will REGENXBIO have at the WORLD Symposium?

REGENXBIO will have five oral and seven poster presentations.