1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bloom syndrome | a fourth generation activating chimeric receptor (ACR) natural killer (NK) cell therapy engineered with an NKG2D ACR, OX40 costimulatory domain, CD3zeta signaling moiety, and membrane bound IL-15 (mb-IL15) | Orphan Designation | - |
| DDX41-related hematologic malignancy predisposition syndrome | a fourth generation activating chimeric receptor (ACR) natural killer (NK) cell therapy engineered with an NKG2D ACR, OX40 costimulatory domain, CD3zeta signaling moiety, and membrane bound IL-15 (mb-IL15) | Orphan Designation | - |
| dyskeratosis congenita, X-linked | a fourth generation activating chimeric receptor (ACR) natural killer (NK) cell therapy engineered with an NKG2D ACR, OX40 costimulatory domain, CD3zeta signaling moiety, and membrane bound IL-15 (mb-IL15) | Orphan Designation | - |