Regenxbio to seek accelerated approval of Hunter syndrome gene therapy using biomarker data
A Hunter syndrome gene therapy developed by Regenxbio significantly cut levels of a key biomarker associated with the disease, according to new results of a clinical trial shared Wednesday. The Rockville, MD biotech plans to file for accelerated approval based on the biomarker data later this year.
In 10 patients who received the pivotal dose of the gene therapy RGX-121, levels of a component (D2S6) of heparan sulfate fell 86% in the cerebrospinal fluid at week 16. After that drop, patients stayed “at a near normal or normal range,” lead investigator Paul Harmatz of the University of California, San Francisco told Endpoints News.
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