2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| alpha 1-antitrypsin deficiency | in vivo target-primed reverse transcription genome editing product consisting of a messenger RNA and a template guide RNA formulated to target the pathogenic PiZ allele in the SERPINA1 gene | Orphan Designation | - |
| phenylketonuria | An in vivo target-primed reverse transcription genome editing product consisting of a messenger RNA and a template guide RNA formulated to target the R408W mutation in the phenylalanine hydroxylase gene | Orphan Designation | - |