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- FDA places hold on REGENXBIO's Hunter syndrome gene therapy after spinal nodules found, company pauses resubmissionThe FDA placed a clinical hold on RGX-121, REGENXBIO's gene therapy for Hunter syndrome, after MRI scans showed small nodules or cystic masses in the spines of five trial participants. The finding…
- US lawmakers push FDA to tighten rules on Chinese clinical data after three gene therapy deathsThree recent deaths in separate gene‑therapy trials in China have prompted two members of the U.S. House of Representatives to call on the FDA for new safeguards. Representatives John Moolenaar an…