#United States #San Diego #REGENXBIO #RGX-121 #MPS II

Introduction

In an exciting development for the biotech community, REGENXBIO Inc. has announced that it will present groundbreaking data on its investigational gene therapy, RGX-121 (clemidsogene lanparvovec), at the upcoming 21st Annual WORLDSymposium™ 2025. This event, scheduled for February 3-7, 2025, in San Diego, CA, serves as a crucial platform for discussing advancements in treatments for lysosomal storage disorders, particularly mucopolysaccharidosis type II (MPS II), commonly known as Hunter syndrome.

Presentations Scheduled

REGENXBIO plans to highlight two key presentations during the symposium:

1. Audiology Assessment in CAMPSIITE® Trial
Presenter: Nidal Boulos, Ph.D., Director, Clinical Science, REGENXBIO
Date/Time: Wednesday, February 5, 2025; 3:30 PM PT
This presentation will focus on the audiological assessments of participants in the CAMPSIITE® trial, which evaluates the efficacy of RGX-121 for patients suffering from neuronopathic MPS II.

2. Interim Clinical Study Update
Presenter: Paul Harmatz, M.D., UCSF Benioff Children's Hospital
Date/Time: Thursday, February 6, 2025; 8:30 AM PT
An update regarding the interim results of the Phase I/II/III CAMPSIITE® trial will be covered, providing insights into the investigational gene therapy and its impact on affected children.

About Hunter Syndrome and MPS II

MPS II is a rare genetic disorder caused by the deficiency of the enzyme iduronate-2-sulfatase. This deficiency leads to a buildup of glycosaminoglycans (GAGs) in the body, causing various health complications including developmental delays, hearing loss, and respiratory problems. Hunter syndrome predominantly affects males and has a wide spectrum of clinical manifestations.

REGENXBIO's Commitment

REGENXBIO is at the forefront of gene therapy innovation, particularly in developing AAV (adeno-associated viral) Therapeutics aimed at addressing various genetic disorders. Since its inception in 2009, the company has made significant strides in the realm of gene therapies, including RGX-202 for Duchenne muscular dystrophy and ABBV-RGX-314 for age-related macular degeneration.

The company’s commitment to advancing gene therapies stems from its desire to provide one-time effective treatments that could potentially alter the course of rare diseases. With thousands of patients already benefiting from its AAV Therapeutic platforms, including Novartis' ZOLGENSMA aimed at spinal muscular atrophy, REGENXBIO continues to bring hope to patients and their families.

Looking Ahead

The presentations at the WORLDSymposium are pivotal for REGENXBIO as they seek to expand awareness and understanding of RGX-121's potential to improve the quality of life for children with MPS II. As the biotechnology field advances, REGENXBIO stands out as a dedicated player poised to make a lasting impact through its innovative gene therapy solutions.

For more detailed information about RGX-121 and REGENXBIO’s other projects, visit their official website at www.regenxbio.com.