Modality
Gene Editing
MOA
BiTE
Target
PD-L1
Pathway
mTOR
Hemophilia A
Development Pipeline
Preclinical
~Nov 2017
→ ~Feb 2019
Phase 1
~May 2019
→ ~Aug 2020
Phase 2
~Nov 2020
→ ~Feb 2022
Phase 3
~May 2022
→ ~Aug 2023
NDA/BLA
~Nov 2023
→ ~Feb 2025
Approved
May 2025
→ Jan 2031
ApprovedCurrent
NCT04216758
698 pts·Hemophilia A
2025-05→2031-01·Terminated
698 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2031-01-234.8y awayPh3 Readout· Hemophilia A
Trial Timeline
Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031
Approved
Termina…
Catalysts
Ph3 Readout
2031-01-23 · 4.8y away
Hemophilia A
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04216758 | Approved | Hemophilia A | Terminated | 698 | NT-proBNP |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| ABB-7516 | AbbVie | Phase 3 | PD-L1 | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| Olpatinib | GSK | Preclinical | PD-L1 | |
| BAY-3684 | Bayer | Approved | PD-L1 | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| BII-8315 | Biogen | NDA/BLA | CDK2 | |
| Geliglumide | Samsung Biologics | Phase 1 | Aβ | |
| Polatuximab | Nuvalent | Approved | PD-L1 |