Regenxbio plans to resubmit its Hunter syndrome gene therapy RGX-121 after the FDA reversed course, saying existing data are sufficient for accelerated approval review.
Curran Simpson, Regenxbio CEO (Brian Benton Photography)
June 22, 2026 07:14 AM EDT
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Regenxbio to resubmit Hunter syndrome gene therapy as FDA rare disease about-face continues
Lei Lei Wu
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The agency took another step toward clearing a backlog of Makary-era rejections that appeared to contradict earlier agreements…
The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it…
Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA…
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