REGENXBIO Announces Presentations of RGX-121 at the 21st Annual WORLDSymposium 2025

Core Insights - REGENXBIO Inc. announced that data from its RGX-121 program for treating mucopolysaccharidosis type II (MPS II) will be presented at the 21st Annual WORLDSymposium™ 2025 in San Diego, CA from February 3-7, 2025 [1] - The presentations will include topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121 [1] Company Overview - REGENXBIO is a leading clinical-stage biotechnology company focused on gene therapy, founded in 2009 [3] - The company has developed AAV Therapeutics, a class of gene therapy medicines, and is advancing a pipeline for rare and retinal diseases, including RGX-202 for Duchenne and ABBV-RGX-314 for wet AMD and diabetic retinopathy in collaboration with AbbVie [3] - Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, which includes Novartis' ZOLGENSMA for spinal muscular atrophy [3] - AAV Therapeutics are designed as one-time treatments with the potential to transform healthcare delivery for millions [3]