Drug Type AAV based gene therapy |
Synonyms AAV SGSH, AAV-SGSH, AAV9 SGSH + [12] |
Target |
Mechanism SGSH stimulants(N-sulphoglucosamine sulphohydrolase stimulants), Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhasePhase 3 |
First Approval Date- |
RegulationFast Track (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Regenerative Medicine Advanced Therapy (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Mucopolysaccharidosis III | Phase 3 | US | 28 Sep 2020 | |
Mucopolysaccharidosis III | Phase 3 | US | 28 Sep 2020 | |
Mucopolysaccharidosis III | Phase 3 | AU | 28 Sep 2020 | |
Mucopolysaccharidosis III | Phase 3 | AU | 28 Sep 2020 | |
Mucopolysaccharidosis III | Phase 3 | ES | 28 Sep 2020 | |
Mucopolysaccharidosis III | Phase 3 | ES | 28 Sep 2020 |
Phase 2/3 | 17 | otkjgkfdol(amfzxoefrb) = The individual EYC in cognitive raw scores showed a positive rate of change indicating either stability or gains from baseline in 16 of the 17 patients during the expected window of plateau into decline, defined as >30 months of age. fumpwdyzqf (wpnhfurmyk ) View more | Positive | 06 Feb 2024 | |||
Phase 1/2 | 5 | hbapfsvlzd(fgckrtaugz) = blrsejfrve ozcjivhmvi (clthcdxnkf, xrpvxqqhxg - zfoyrzyrbg) View more | - | 25 Jul 2023 | |||
Phase 1/2 | - | mxuigghsfw(ssulmyqjss) = brszdimmbo krbppwzpgl (hrfusljvzb ) | Positive | 12 Feb 2021 | |||
Phase 1/2 | Mucopolysaccharidosis III CSF heparin sulfate (HS) | 14 | xrauewicwt(oxmwukgzzo) = Mild transient cellular immune responses have been detected in 8 out of 14 patients as assessed by ELISpot (interferon-gamma) jaatfuokpm (mraygwdzzr ) View more | Positive | 28 Apr 2020 | ||