A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

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An AAV vector genome integration event associated with PLAG1 overexpression was identified in a preliminary analysis of the resected tumor.

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REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I, also ...

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