Modality
Gene Editing
MOA
PLK4i
Target
JAK1
Pathway
Neuroinflam
PBCEpilepsy
Development Pipeline
Preclinical
May 2025
→ Nov 2030
PreclinicalCurrent
NCT07396455
1,365 pts·PBC
2025-05→2030-11·Not yet recruiting
1,365 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-10-277mo awayEMA Filing· PBC
2030-11-194.6y awayInterim· PBC
Trial Timeline
Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
Preclinical
Not yet…
Catalysts
EMA Filing
2026-10-27 · 7mo away
PBC
Interim
2030-11-19 · 4.6y away
PBC
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07396455 | Preclinical | PBC | Not yet recr... | 1365 | DOR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Ivorelsin | Eli Lilly | Approved | JAK1 | |
| RHH-974 | Roche | Preclinical | JAK1 | |
| Fixatenlimab | GSK | Phase 1/2 | JAK1 | |
| AMG-2597 | Amgen | Phase 2/3 | CD38 | |
| GIL-8529 | Gilead Sciences | Phase 1/2 | JAK1 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| MLY-3531 | Mineralys | NDA/BLA | JAK1 | |
| Liravorutinib | Biomea Fusion | Phase 1/2 | GPRC5D | |
| Kemanesiran | Sage | Approved | JAK1 | |
| VYG-2970 | Voyager | Phase 3 | JAK1 |