Orphan designation: Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene Treatment of X-linked severe combined immunodefi

Orphan designation: Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene Treatment of X-linked severe combined immunodeficiency, 20/05/2021 Withdrawn