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The FDA has placed clinical holds on RegenXBio's investigational gene therapies RGX-111 and another for rare neurodevelopmental disorders, including mucopolysaccharidosis type I (Hurler syndrome). This decision follows the discovery of a neoplasm in a trial participant, impacting ongoing studies.
Read full storyRegenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.
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