UNIVERSIDADE FEDERAL DO RIO GRANDE DO SUL

RGX-121 Gene Therapy Shows Sustained Efficacy in Hunter Syndrome Trial

9 months ago

• RGX-121 gene therapy demonstrates an 85% median reduction in cerebrospinal fluid levels of heparan sulfate D2S6, a key biomarker in Hunter syndrome. • The CAMPSIITE trial data supports RGX-121 as a potential first gene therapy for Hunter syndrome, with sustained effects observed for up to two years. • REGENXBIO plans to initiate a rolling biologics license application (BLA) using the accelerated approval pathway in Q3 2024 based on these results. • A significant number of patients remained free from enzyme replacement therapy (ERT) post-treatment, suggesting systemic activity of RGX-121.