3
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ornithine carbamoyltransferase deficiency | A replication-deficient adeno-associated virus serotype 9 vector containing the ornithine transcarbamylase gene within the transposon and a lipid nanoparticle containing messenger RNA for the transposase | Orphan Designation | - |
| plasma cell myeloma | genetically modified allogeneic chimeric antigen receptor (CAR)-T cell therapy designed to target cell surface B-Cell Maturation AntigenAutologous T cells transfected with PiggyBac transposon plasmid vector expressing B-cell maturation antigen (CAR-T cells) | Orphan Designation | - |