REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium

PR Newswire

ROCKVILLE, Md.

,

Aug. 23, 2022

/PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, taking place from

August 30 through September 2, 2022

, in Freiburg,

Germany

. The presentations will highlight new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, an investigational one-time AAV Therapeutic for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, and an encore data presentation for RGX-111, an investigational one-time AAV Therapeutic for the treatment of severe Mucopolysaccharidosis Type I (MPS I), or Hurler syndrome.

Presentations include:

Title:

RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from a Phase 1/2 study

Presenter:

Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA,

Porto Alegre, Brazil

Date/Time:

Wednesday, August 31, 2022

, 10:00 –

10:15 a.m.

CEDT

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:

Thursday, September 1, 2022, 11:30 –

11:45 a.m.

CEDT

Title:

Natural History of Neurodevelopment in Neuronopathic Mucopolysaccharidosis Type II (MPS II): Mullen Scales of Early Learning (MSEL) Cognitive, Motor, and Language Developmental Trajectories

Presenter:

Maria Escolar, M.D., M.S., Professor of Pediatrics, Director, Program for the Study of Neurodevelopment in Rare Disorders, UPMC Children’s Hospital of

Pittsburgh

Date/Time:

Wednesday, August 31, 2022, 6:45 –

8:15 p.m.

CEDT

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, 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

Corporate Communications

[email protected]

Investors:

Chris Brinzey

ICR Westwicke

339-970-2843

[email protected]