Drug Type ASO |
Synonyms Enhanced delivery peptide-oligonucleotide conjugate for Duchenne Muscular Dystrophy Exon 51, EDO-51, PGN EDO51 + [1] |
Target |
Action- |
Mechanism- |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhaseIND Approval |
First Approval Date- |
RegulationOrphan Drug (United States), Rare Pediatric Disease (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Muscular Dystrophy, Duchenne | Phase 2 | Canada | 18 May 2023 |
Phase 2 | Muscular Dystrophy, Duchenne exon 51-skipping | 3 | PGN-EDO51 5 mg/kg | tmphtuvwat(byecpfsbrn) = gpqlywxqbd xodrftljhj (nfdkgvshlz ) View more | Positive | 31 Jul 2024 | |
Phase 2 | - | PGN-EDO23 | xsugxlspht(oqjaxodujb) = joqpnbdnhh eadgtmylkm (xwrfdjzfxr ) View more | Positive | 03 Mar 2024 | ||
Phase 1 | - | bevefmwsmq(snxhiyeqcu) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae heippetiiv (wbnxfgtger ) | Positive | 01 May 2023 | |||
Phase 1 | - | kkzfcsbgwg(iywhncejvi) = transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses; with no significant clinical sequelae. At 15 mg/kg, one HV received IV hydration after increases in kidney biomarkers with complete resolution. xsijekxdwm (xdxmhxjnpa ) View more | - | 25 Apr 2023 | |||
Phase 1 | Muscular Dystrophy, Duchenne kidney biomarkers | hypomagnesemia | - | ziofezfaaz(odzkaruacx) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae mklkiohtja (snihfojdhv ) | Positive | 19 Mar 2023 | ||
GlobeNewswire Manual | Phase 1 | Muscular Dystrophy, Duchenne Exon 51 | 32 | kqozyysqmq(zrdxwkbbgu) = mild and resolved without any intervention dxqcxrhlpz (yubybodkkw ) View more | Positive | 28 Sep 2022 | |
Placebo |