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Rozwój badań leku na dystrofię miotoniczną
Ostatnia aktualizacja: 24/09/2026
Logo | Firma | Faza badań | Program / lek | Podtyp DM | Modalność / technologia | Cel leku / mechanizm | Aktualizacje |
|---|---|---|---|---|---|---|---|
AMO Pharma | 🟢 P1 → 🟢 P2 → 🔵 P3 → ⚪ | Tideglusib | DM1, CDM | Small Molecule | Glycogen synthase kinase 3 beta (GSK3β) | https://www.youtube.com/watch?v=rvMxPkxiSrk&t=3306s | |
Avidity Biosciences | 🟢 P1 → 🟢 P2 → 🔵 P3 → ⚪ | AOC 1001 del-desiran | DM1 | Antibody Conjugated Oligonucleotide | DMPK | https://www.youtube.com/watch?si=VV9tqCESfwkxtedH&t=3306&v=rvMxPkxiSrk&feature=youtu.be | |
Lupin Neurosciences | 🟢 P1 → 🟢 P2 → 🔵 P3 → ⚪ | Mexiletine | DM1, DM2 | Small Molecule | Sodium Channels | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=3039s | |
Dyne Therapeutics | 🟢 P1 → 🟢 P2 → 🔵 P3 → ⚪ | Dyne 101 z-basivarsen | DM1 | Antibody Fragment Conjugated Antisense Oligonucleotide | DMPK | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=1118s | |
ARTHEx Biotech | 🟢 P1 → 🔵 P2 → ⚪ P3 → ⚪ | ATX-01 | DM1 | Antisense microRNA Oligonucleotide | MBNL via miR-23b | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=1740s | |
Harmony Biosciences | 🟢 P1 → 🔵 P2 → ⚪ P3 → ⚪ | Pitolisant | DM1 | Small Molecule | Histamine-3 receptor (H3R) | https://www.youtube.com/watch?v=l0cRbvCDs5U&t=1510s | |
PepGen | 🟢 P1 → 🔵 P2 → ⚪ P3 → ⚪ | PGN-EDODM1 | DM1 | Peptide Conjugated Antisense Oligonucleotide | DMPK | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=3830s | |
Juvena Therapeutics | 🟢 P1 → 🔵 P2 → ⚪ P3 → ⚪ | JUV-161 | DM1 | Stem Cell-Secreted Proteins | — | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=2439s | |
Vertex | 🟢 P1 → 🔵 P2 → ⚪ P3 → ⚪ | VX-670 | DM1 | Peptide Conjugated Oligonucleotide | DMPK | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=4293s | |
Sanofi | 🔵 P1 → ⚪ P2 → ⚪ P3 → ⚪ | — | — | miRNA Technology in Adeno-Associated Virus | DMPK | https://www.youtube.com/watch?v=oJ3mr2auQiM&t=4723s | |
Sarepta Therapeutics | 🔵 P1 → ⚪ P2 → ⚪ P3 → ⚪ | SRP-1003 | DM1 | Investigational RNA interference (RNAi) Therapeutic | r(CUG) of DMPK | ||
Design Therapeutics | 🔵 P1 → ⚪ P2 → ⚪ P3 → ⚪ | DT818 | DM1 | Gene Targeting Chimera Small Molecule | r(CUG) of DMPK |
Źródło: Myotonic Dystrophy Foundation, https://myotonic.org
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