3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| RPGR-related retinopathy | Dual, recombinant adeno-associated viral vector-based Prime Editing product targeting USH2A gene mutations | Orphan Designation | - |
| chronic granulomatous disease | PE CD34+ cell-based drug product targeting the delGT mutation in the NCF1 gene | Orphan Designation | - |
| glycogen storage disease Ib | Lipid nanoparticles containing prime editing components (mRNA, pegRNA and ngRNA) capable of editing genetic mutations in the SLC37A4 gene | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Dual, recombinant adeno-associated viral vector-based Prime Editing product targeting USH2A gene mutations | Orphan Designation | - |
| progressive retinal dystrophy due to retinol transport defect | Dual, recombinant adeno-associated viral vector-based Prime Editing product targeting USH2A gene mutations | Orphan Designation | - |