Submitted by Anonymous (not verified) on 11 March 2025 - 10:34
Orphan designation: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human ADA2 gene Treatment of adenosine deaminase 2 deficiency (DADA2), 27/02/2025 Positive
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