Modality
ERT
MOA
CAR-T CD19
Target
TNFα
Pathway
Amyloid
Hemophilia A
Development Pipeline
Preclinical
~Sep 2022
→ ~Dec 2023
Phase 1
Mar 2024
→ May 2029
Phase 1Current
NCT06660978
35 pts·Hemophilia A
2024-03→2029-05·Not yet recruiting
35 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-05-083.1y awayInterim· Hemophilia A
Trial Timeline
Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
P1
Not yet…
Catalysts
Interim
2029-05-08 · 3.1y away
Hemophilia A
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06660978 | Phase 1 | Hemophilia A | Not yet recr... | 35 | BodyWt |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| ABB-3951 | AbbVie | Phase 2/3 | TNFα | |
| Datoglumide | AbbVie | Approved | CFTR | |
| GIL-6239 | Gilead Sciences | Phase 2 | TNFα | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| MRN-8225 | Moderna | Preclinical | TNFα | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| Sovanaritide | Argenx | Phase 3 | TNFα | |
| Talasotorasib | Exelixis | Preclinical | APOC3 | |
| 369-4107 | Hansoh Pharma | Phase 2 | TNFα |