RegenXBio Inc. (RGNX) announced an update on their ongoing clinical study.
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RegenXBio Inc. is conducting a Phase 3 clinical study titled ‘A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)’. The study aims to assess the safety, efficacy, and pharmacodynamics of RGX-121, a gene therapy designed to treat Hunter Syndrome by delivering a functional copy of the iduronate-2-sulfatase gene to the central nervous system. This research is significant as it could provide a new therapeutic option for this rare genetic disorder.
The intervention being tested is RGX-121-3102, a genetic therapy involving a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a human iduronate-2-sulfatase expression cassette. The purpose of this intervention is to address the underlying genetic cause of Hunter Syndrome by delivering a functional gene to affected patients.
The study follows an interventional design with a single-group, open-label model, meaning all participants receive the same treatment without a placebo group. The primary purpose is treatment, focusing on dose escalation to determine the optimal dosing for efficacy and safety.
The study began on November 5, 2025, with its primary completion date yet to be determined. The latest update was submitted on November 24, 2025. These dates are crucial as they mark the study’s progress and provide timelines for potential results and regulatory milestones.
This clinical update could positively impact RegenXBio’s stock performance and investor sentiment, as successful results may lead to a new treatment option for Hunter Syndrome, potentially increasing the company’s market value. In the competitive landscape of gene therapy, advancements in this study could position RegenXBio favorably against its peers.
The study is currently ongoing, with further details available on the ClinicalTrials portal.
To learn more about RGNX’s potential, visit the RegenXBio Inc. drug pipeline page.