7
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CD4+/CD56+ hematodermic neoplasm | autologous T cells genetically modified to express CD123 specific hinge-optimized CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor | Orphan Designation | - |
| Waldenstrom macroglobulinemia | Autologous T-Cells transduced with a lentiviral vector (LVV) expressing CD20-specific chimeric antigen receptor (CAR) gene (CD20 CAR-T Cells) | Orphan Designation | - |
| acute myeloid leukemia | autologous T-cells genetically modified to express a CD123-specific, hinge-optimized, CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor | Orphan Designation | - |
| malignant glioma | an autologous ex vivo expanded cluster of differentiation 62-enriched naive and memory T-cell investigational product that has been genetically modified using a self-inactivating lentiviral vector to express an IL13Ralpha2-targeted, hinge-optimized, 4-1BB-costimulatory chimeric antigen receptor with a truncated CD19a chimeric tumor-selective replication competent herpes simplex virus type 1, in which both copies of the gamma134.5 gene have been deleted | Orphan Designation | - |
| post 5-alpha-reductase inhibitors treatment syndrome | firolimogene autotemcel | Orphan Designation | - |
| post-treatment Lyme disease syndrome | firolimogene autotemcel | Orphan Designation | - |
| severe combined immunodeficiency | Mobilized peripheral blood-derived autologous CD34+ hematopoietic stem cells, that have been genetically modified, ex vivo, with self-inactivating lentiviral vector CL20-4i-EF1a-hyc-OPT containing a normal version of the coding region of the IL2RG gene. | Orphan Designation | - |
| treatment-refractory schizophrenia | firolimogene autotemcel | Orphan Designation | - |