Regenxbio Delays Hunter Syndrome Gene Therapy Plans After FDA Clinical Hold

The FDA has placed a clinical hold on Regenxbio's RGX-121 gene therapy for Hunter syndrome following discovery of small nodules or cystic masses in treated patients' spine MRIs, delaying Regenxbio’s plans to resubmit for approval. Regenxbio will conduct longer-term studies and data analysis before reassessing the gene therapy's benefit-risk profile.

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