6
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Fabry disease | adeno-associated virus serotype 2/6 encoding a human alpha-galactosidase A cDNA | Orphan Designation | - |
| Hurler syndrome | adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene | Orphan Designation | - |
| Hurler-Scheie syndrome | adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene | Orphan Designation | - |
| Scheie syndrome | adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human alpha-L-iduronidase (hIDUA) gene | Orphan Designation | - |
| beta-thalassemia HBB/LCRB | autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease messenger RNAs SB-mRENH1 and SB-mRENH2 | Orphan Designation | - |
| hemophilia B | Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX) | Orphan Designation | - |
| mucopolysaccharidosis type 2 | Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human iduronate 2-sulfatase (hIDS) gene | Orphan Designation | - |
| sickle cell disease | Autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease messenger RNAs SB-mRENH1 and SB-mRENH2 | Orphan Designation | - |