Drug Type AAV based gene therapy |
Synonyms scAAV-∆h IDUA op, Self-complementary adeno-associated virus serotype 9 vector harboring a miniaturized codon-optimized human IDUA gene |
Target |
Mechanism IDUA gene stimulants(Alpha-L-iduronidase gene stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Mucopolysaccharidosis I | Preclinical | US | 07 May 2024 | |
Mucopolysaccharidosis I | Preclinical | US | 07 May 2024 |