3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wiskott-Aldrich syndrome | Autologous CD34+ hematopoietic stem and progenitor cells modified ex vivo with a lentiviral vector that restores endogenously regulated expression of WASp | Orphan Designation | - |
| chronic granulomatous disease | autologous CD34+ hematopoietic stem and progenitor cells modified ex-vivo with a lentiviral vector that restores expression of Gp91phox | Orphan Designation | - |
| immune dysregulation, autoimmunity, and autoinflammation | autologous CD34+ hematopoietic stem and progenitor cells modified ex-vivo with a lentiviral vector which restores endogenously regulated expression of FOXP3 | Orphan Designation | - |
| immune dysregulation-inflammatory bowel disease-arthritis-recurrent infections-lymphopenia syndrome | autologous CD34+ hematopoietic stem and progenitor cells modified ex-vivo with a lentiviral vector which restores endogenously regulated expression of FOXP3 | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | autologous CD34+ hematopoietic stem and progenitor cells modified ex-vivo with a lentiviral vector which restores endogenously regulated expression of FOXP3 | Orphan Designation | - |