3
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bietti crystalline corneoretinal dystrophy | Recombinant adeno-associated virus vector carrying human CYP4V2 gene | Orphan Designation | - |
| inherited retinal dystrophy | adeno-associated virus serotype 8 expressing Cas9 gene and single guide RNA targeting R135W mutation in Rhodopsin gene | Orphan Designation | - |
| pseudo-TORCH syndrome | Recombinant adeno-associated virus vector carrying human CYP4V2 gene | Orphan Designation | - |
| retinitis pigmentosa 1 | AAV2/8-SaCas9-sgRNA RHO-T17M | Orphan Designation | - |
| retinitis pigmentosa 12 | AAV2/8-SaCas9-sgRNA RHO-T17M | Orphan Designation | - |
| retinitis pigmentosa 7 | AAV2/8-SaCas9-sgRNA RHO-T17M | Orphan Designation | - |