1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| BAP1-related tumor predisposition syndrome | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |
| Hermansky-Pudlak syndrome | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |
| Hinman syndrome | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |
| acute myeloid leukemia with CEBPA somatic mutations | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |
| ataxia, early-onset, with oculomotor apraxia and hypoalbuminemia | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |
| has an isolated presentation | autologous logic-gated Tmod chimeric antigen receptor (CAR) T cells expressing a carcinoembryonic antigen targeted CAR activator and a human leukocyte antigen serotype A*02 (HLA-A*02) targeted blocker | Orphan Designation | - |