2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| hemophilia A | Adeno-associated viruses 8 (AAV8)-based gene therapy vector that expresses B domain-deleted human factor VIII under the control of human liver-specific promoters | Orphan Designation | - |
| phenylketonuria | recombinant adeno-associated virus serotype 8-based gene therapy that utilizes a dual mechanism of action under the control of human liver-specific promoters, expressing an artificial microRNA to silence endogenous pathogenic variants of phenylalanine hydroxylase (PAH), while simultaneously delivering a functional, RNA interference-resistant PAH gene | Orphan Designation | - |