Submitted by Anonymous (not verified) on 10 June 2025 - 7:36
Orphan designation: Adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene Treatment of ornithine transcarbamylase deficiency, 09/12/2022 Positive
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