Modality
Gene Therapy
MOA
PRMT5i
Target
B7-H3
Pathway
Tau
Fabry
Development Pipeline
Preclinical
~Jun 2023
→ ~Sep 2024
Phase 1
Dec 2024
→ Oct 2025
Phase 1Current
NCT08335998
2,658 pts·Fabry
2024-12→2025-10·Completed
2,658 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-10-076mo agoPh2 Data· Fabry
Trial Timeline
2025Q2Q3Q4
P1/2
Complet…
Catalysts
Ph2 Data
2025-10-07 · 6mo ago
Fabry
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08335998 | Phase 1/2 | Fabry | Completed | 2658 | UPDRS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-6079 | Eli Lilly | Phase 2 | MDM2 | |
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| RHH-8550 | Roche | Approved | BCL-2 | |
| RHH-682 | Roche | Phase 2 | FXIa | |
| Ceviglumide | Roche | NDA/BLA | B7-H3 | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-8628 | Sanofi | NDA/BLA | B7-H3 | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| Talainavolisib | Novo Nordisk | Preclinical | KRASG12D |