3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CEP290-related ciliopathy | a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs | Orphan Designation | - |
| Leber congenital amaurosis 10 | a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs | Orphan Designation | - |
| Senior-Loken syndrome 6 | a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs | Orphan Designation | - |
| beta thalassemia | autologous CD34+ hematopoietic stem and progenitor cells edited by CRISPR/Cas12a ribonucleoprotein (RNP) at the HBG1 and HBG2 promoters | Orphan Designation | - |
| sickle cell disease | Autologous CD34+ hematopoietic stem and progenitor cells edited by CRISPR/CAS12a at the HBG1 and HBG2 promoters | Orphan Designation | - |