9
Orphan Designations
0
FDA Approvals
20
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ABCA4-related retinopathy | Adeno-Associated Virus Serotype 5 capsid protein containing gene construct encoding human Retinoic Acid Receptor-Related Orphan Receptor Alpha | Orphan Designation | - |
| CEP290-related ciliopathy | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| Joubert syndrome 5 | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| Leber congenital amaurosis | Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) | Orphan Designation | - |
| Leber congenital amaurosis 10 | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| RHO-related retinopathy | adeno-Associated Virus containing human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) gene therapy | Orphan Designation | - |
| congenital stationary night blindness autosomal dominant 1 | adeno-Associated Virus containing human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) gene therapy | Orphan Designation | - |
| dystrophies primarily involving the retinal pigment epithelium | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| enhanced S-cone syndrome | adeno-associated virus serotype 5 capsid containing the gene for human Nuclear Hormone Receptor NR2E3 | Orphan Designation | - |
| gallbladder disease 1 | Adeno-Associated Virus Serotype 5 capsid protein containing gene construct encoding human Retinoic Acid Receptor-Related Orphan Receptor Alpha | Orphan Designation | - |
| ocular cancer | brimonidine | Orphan Designation | - |
| ocular melanoma | brimonidine | Orphan Designation | - |
| ocular tuberculosis | brimonidine | Orphan Designation | - |
| retinal dystrophies primarily involving Bruch's membrane | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| retinitis pigmentosa | Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3)recombinant lens epithelium derived growth factor 1-326 | Orphan Designation | - |
| retinitis pigmentosa 19 | Adeno-Associated Virus Serotype 5 capsid protein containing gene construct encoding human Retinoic Acid Receptor-Related Orphan Receptor Alpha | Orphan Designation | - |
| retinitis pigmentosa 37 | adeno-associated virus serotype 5 capsid containing the gene for human Nuclear Hormone Receptor NR2E3 | Orphan Designation | - |
| retinitis pigmentosa 4 | adeno-Associated Virus containing human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) gene therapy | Orphan Designation | - |
| trichothiodystrophy | Adeno-Associated Virus containing the gene for human Nuclear Hormone Receptor NR2E3 (AAV-hNR2E3) | Orphan Designation | - |
| vitreoretinal degeneration | adeno-associated virus serotype 5 capsid containing the gene for human Nuclear Hormone Receptor NR2E3 | Orphan Designation | - |