PR Newswire | Feb 1, 2021 07:05AM EST

02/01 06:05 CST

REGENXBIO Announces Presentations at the 17th Annual WORLDSymposium(tm) 2021 ROCKVILLE, Md., Feb. 1, 2021

ROCKVILLE, Md., Feb. 1, 2021 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that three oral and nine poster presentations will be presented at the 17th Annual WORLDSymposium(tm), taking place virtually from February 8 through 12, 2021. The oral presentations include interim results from the Phase I/II clinical trial of RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II).

The oral presentations will be presented as follows:

Abstract Title Comparative systemic and neurologic effectiveness of intravenous and intrathecal AAV9 delivered individually or combined in a murine model of mucopolysaccharidosis type IPresenter Lalitha Belur, Ph.D., Assistant Professor Genetics, Cell Bio at University of MinnesotaDate/Time Monday, February 8, 2021, 10:06 a.m. ET

Abstract Title Intracisternal administration of AAV9 gene therapies to target the central nervous systemPresenter Bryan A. Pukenas, M.D., Assistant Professor of Radiology at Hospital of the University of PennsylvaniaDate/Time Tuesday, February 9, 2021, 11:36 a.m. ET

Abstract Title RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trialPresenter Marie-Laure Nevoret, M.D., Senior Clinical Development Lead at REGENXBIO Date/Time Thursday, February 11, 2021, 9:42 a.m. ET

The poster presentations will be presented as follows:

Abstract Title Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye (poster #24)Presenter Nicholas Buss, Ph.D., Senior Director, Preclinical Development at REGENXBIO Date/Time Tuesday, February 9, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title Intracisternal administration of AAV9 gene therapies to target the central nervous system (poster #207)Presenter Bryan A. Pukenas, M.D., Assistant Professor of Radiology at Hospital of the University of PennsylvaniaDate/Time Tuesday, February 9, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human -Liduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up (poster #264)Presenter Raymond Wang, M.D., Biochemical Genetics Specialist at CHOC Children's HospitalDate/Time Tuesday, February 9, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestations (poster #45)Presenter Kirsten Cowley, Director, Clinical Operations at REGENXBIO Inc.Date/Time Wednesday, February 10, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title Clinical and numerical presentation of neurocognitive assessments for MPS II patients using the Bayley Scales of Infant Development - version 3 (BSID-III) (poster #34)Presenter Yoonjin Cho, Ph.D., Director, Biostatistics at REGENXBIO Inc.Date/Time Thursday, February 11, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial (poster #171)Presenter Marie-Laure Nevoret, M.D., Senior Clinical Development Lead at REGENXBIO Inc.Date/Time Thursday, February 11, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title Characterizing expressive language skills in children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): The caregiver perspective (poster #198)Presenter Dawn Phillips, Ph.D., Director, Clinical Scientist, Outcomes Research at REGENXBIO Inc.Date/Time Thursday, February 11, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title Characterizing visual function in children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): The caregiver perspective (poster #199)Presenter Dawn Phillips, Ph.D., Director, Clinical Scientist, Outcomes Research at REGENXBIO Inc.Date/Time Thursday, February 11, 2021, from 2:30 to 3:30 p.m. ET

Abstract Title New research examines the evolution of data sharing practices in natural history studies and patient registries among patient advocate leaders, industry and academia (poster #LB-12)Presenter Vivian Fernandez, Senior Director of Patient Advocacy at REGENXBIO Inc.Date/Time Friday, February 12, 2021, from 2:30 to 3:30 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(r) Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Contacts:Tricia TruehartInvestor Relations and Corporate Communications347-926-7709ttruehart@regenxbio.com

Investors:Brendan Burns, 212-600-1902 brendan@argotpartners.com

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

View original content to download multimedia: http://www.prnewswire.com/news-releases/regenxbio-announces-presentations-at-the-17th-annual-worldsymposium-2021-301218589.html

SOURCE REGENXBIO Inc.