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
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
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
