1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Kabuki syndrome 1 | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |
| Smith-Lemli-Opitz syndrome | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |
| gastric leiomyosarcoma | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |
| gastric lymphoma | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |
| gastric ulcer | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |
| gastric volvulus, intrathoracic | ex vivo expanded allogeneic cord blood-derived NK cells that have been genetically modified to express a HER2-directed CAR and secrete IL-15 | Orphan Designation | - |