PR Newswire | Jan 31, 2022 07:05AM EST

01/31 06:05 CST

REGENXBIO Announces Presentations at the 18th Annual WORLDSymposium(tm) 2022 ROCKVILLE, Md., Jan. 31, 2022

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 18th Annual WORLDSymposium(tm), 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 administrationPresenter:Lalitha Belur, Ph.D., Research Assistant Professor Genetics, Cell Biology and Development at University of MinnesotaDate/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 studyPresenter: Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, BrazilDate/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 studyPresenter:Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, CADate/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 IIPresenter: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 cellsPresenter: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 MinnesotaDate/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 CormackCorporate Communicationsdcormack@regenxbio.com

Investors:Chris BrinzeyICR Westwicke339-970-2843chris.brinzey@westwicke.com

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SOURCE REGENXBIO Inc.