Drug Type AAV based gene therapy |
Synonyms AAV1 vectored follistatin gene therapy - Milo Biotechnology, AAV1-Follistatin, rAAV1 CMV huFollistatin 344 + [2] |
Target |
Mechanism FST inhibitors(Follistatin inhibitors), MSTN inhibitors(Growth/differentiation factor 8 inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 2 |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Muscular Dystrophy, Duchenne | Phase 2 | US | 01 Jan 2015 | |
Myositis, Inclusion Body | Phase 2 | US | - | - |
Myositis, Inclusion Body | Phase 2 | - | - | |
Myositis, Inclusion Body | Phase 2 | - | - | |
Becker Muscular Dystrophy | Phase 1 | US | 01 Jan 2012 |
Phase 1/2 | 3 | huFollistatin344 (2.4E12 vg/kg CMV.huFollistatin344) | fujdszqnxy(cswjkhhvsi) = pszbkmizga rswbydctwm (wkeijfmilg, bizhcrqprg - bmarphjugc) View more | - | 15 Apr 2020 | ||
(2.4E12 vg/kg (1.2E12vg/kg/Limb) of rAAV1.CMV.huFollistatin344) | sykscjlraa(tbdyereubi) = jvykpgnswl myslwfmpnk (orxyduecil, nitjjwvfuv - oqxpgbycyb) View more | ||||||
Pubmed | Mol Ther Manual | Phase 1 | 14 | vukdijlmzc(pnbnnypjvg) = vxixpglcdn ryxlmkzbop (qhjqfhuqej ) | Positive | 05 Apr 2017 | ||
untreated | vukdijlmzc(pnbnnypjvg) = dcwgrzoumk ryxlmkzbop (qhjqfhuqej ) |