Modality
Gene Editing
MOA
TYK2i
Target
WRN
Pathway
Neuroinflam
RASCDPSP
Development Pipeline
Preclinical
~Apr 2015
→ ~Jul 2016
Phase 1
~Oct 2016
→ ~Jan 2018
Phase 2
~Apr 2018
→ ~Jul 2019
Phase 3
~Oct 2019
→ ~Jan 2021
NDA/BLA
Apr 2021
→ Aug 2030
NDA/BLACurrent
NCT05725524
704 pts·SCD
2025-12→2028-09·Recruiting
NCT07622788
1,211 pts·RA
2021-04→2030-08·Recruiting
1,915 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-09-062.4y awayPh3 Readout· SCD
2030-08-014.3y awayPh3 Readout· RA
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
NDA/BLA
Recruit…
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2028-09-06 · 2.4y away
SCD
Ph3 Readout
2030-08-01 · 4.3y away
RA
Recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05725524 | NDA/BLA | SCD | Recruiting | 704 | Biomarker |
| NCT07622788 | NDA/BLA | RA | Recruiting | 1211 | NT-proBNP |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Lisonaritide | Eli Lilly | Phase 3 | IL-13 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| MRK-2473 | Merck & Co | Phase 1/2 | Nectin-4 | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| Voxacagene | Gilead Sciences | Phase 2/3 | SOS1 |