autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA | |
---|---|
Trade Name | |
Orphan Indication | Severe combined immunodeficiency |
USA Market Approval | USA |
USA Designation Date | 2014-10-21 00:00:00 |
Sponsor | Donald B. Kohn, MD - Professor, UC-UCLA;Microbiology, Immunology & Molecular Genetics, 610 Charles E. Yound Drive, East;Los Angeles, California, 90095 |