2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| AIPL1-related retinopathy | a replication-incompetent recombinant adeno-associated virus (rAAV) vector carrying a gene cassette that can express human aryl hydrocarbon receptor interacting protein like-1 protein AIPL1 | Orphan Designation | - |
| Leber congenital amaurosis 4 | a replication-incompetent recombinant adeno-associated virus (rAAV) vector carrying a gene cassette that can express human aryl hydrocarbon receptor interacting protein like-1 protein AIPL1 | Orphan Designation | - |
| X-linked retinoschisis | Recombinant adeno-associated virus (rAAV) vector expressing human retinoschisis protein RS1 | Orphan Designation | - |