Modality
Gene Editing
MOA
JAK1i
Target
WRN
Pathway
Innate Imm
Huntington'sUrothelial CaRett
Development Pipeline
Preclinical
~Oct 2017
→ ~Jan 2019
Phase 1
~Apr 2019
→ ~Jul 2020
Phase 2
Oct 2020
→ Nov 2028
Phase 2Current
NCT05198835
158 pts·Huntington's
2020-10→2028-11·Active
158 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2028-11-272.7y awayPh3 Readout· Huntington's
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P2/3
Active
Catalysts
Ph3 Readout
2028-11-27 · 2.7y away
Huntington's
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05198835 | Phase 2/3 | Huntington's | Active | 158 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-7458 | Johnson & Johnson | Phase 1/2 | PD-1 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| Geliderotide | Sanofi | Phase 1/2 | PRMT5 | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| Pexaosocimab | Daiichi Sankyo | Phase 2 | PD-1 | |
| Adagratapinarof | Vertex Pharma | Preclinical | BCMA | |
| ARG-1924 | Argenx | Preclinical | DLL3 |