Drug Type AAV based gene therapy |
Synonyms- |
Target |
Mechanism GALNS replacements(galactosamine (N-acetyl)-6-sulfatase replacements) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Mucopolysaccharidosis IV | Preclinical | ES | 09 Sep 2021 |