Drug Type AAV based gene therapy |
Synonyms AAV9 iduronate-2-sulfatase gene therapy - Esteve/UAB, EGT 301, EGT-301 |
Target |
Action- |
Mechanism Iduronate sulfatase replacements, Gene transference(Gene transference) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Mucopolysaccharidosis II | Preclinical | Spain | 30 Jan 2022 |