Modality
Gene Therapy
MOA
SGLT2i
Target
FLT3
Pathway
Tau
Hemophilia A
Development Pipeline
Preclinical
~Aug 2013
→ ~Nov 2014
Phase 1
~Feb 2015
→ ~May 2016
Phase 2
~Aug 2016
→ ~Nov 2017
Phase 3
~Feb 2018
→ ~May 2019
NDA/BLA
~Aug 2019
→ ~Nov 2020
Approved
Feb 2021
→ Mar 2026
ApprovedCurrent
NCT03957354
1,424 pts·Hemophilia A
2021-02→2026-03·Active
1,424 total pts1 indication
CompletedCurrentUpcoming
Catalysts (2)
2026-03-083w agoPh3 Readout· Hemophilia A
2026-09-085mo awayConference· Hemophilia A
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
Approved
Active
Catalysts
Ph3 Readout
2026-03-08 · 3w ago
Hemophilia A
Conference
2026-09-08 · 5mo away
Hemophilia A
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03957354 | Approved | Hemophilia A | Active | 1424 | VA |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8168 | Johnson & Johnson | Phase 2/3 | FLT3 | |
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| Bemalucimab | Bristol-Myers Squibb | Phase 1/2 | Aβ | |
| BAY-3308 | Bayer | Phase 1 | ALK | |
| Sotorapivir | Moderna | Approved | FLT3 | |
| MRN-8225 | Moderna | Preclinical | TNFα | |
| Motazanubrutinib | Biogen | Preclinical | CD38 | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| Nidaratamab | Exelixis | Phase 3 | FLT3 | |
| Zanumavacamten | Iovance | Phase 2 | FLT3 |