Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene Treatment of recombination-activating gene 1 (RAG1) deficiency, 24/05/2024 Positive
Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene Treatment of recombination-activating gene 1 (RAG1) deficiency, 24/05/2024 Positive