Modality
Gene Therapy
MOA
Menini
Target
HER2
Pathway
Incretin
Fabry
Development Pipeline
Preclinical
~Sep 2019
→ ~Dec 2020
Phase 1
~Mar 2021
→ ~Jun 2022
Phase 2
~Sep 2022
→ ~Dec 2023
Phase 3
~Mar 2024
→ ~Jun 2025
NDA/BLA
Sep 2025
→ Oct 2030
NDA/BLACurrent
NCT03281160
2,020 pts·Fabry
2025-09→2030-10·Recruiting
2,020 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-10-074.5y awayPh3 Readout· Fabry
Trial Timeline
Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2030-10-07 · 4.5y away
Fabry
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03281160 | NDA/BLA | Fabry | Recruiting | 2020 | 6MWD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Mavutenlimab | Johnson & Johnson | Phase 3 | Cl18.2 | |
| Ivorelsin | Eli Lilly | Approved | JAK1 | |
| Gozesotorasib | Bristol-Myers Squibb | NDA/BLA | HER2 | |
| NVO-2974 | Novo Nordisk | NDA/BLA | PARP | |
| VRT-4938 | Vertex Pharma | Preclinical | HER2 | |
| ROI-9675 | Roivant Sciences | NDA/BLA | HER2 | |
| Olpafutibatinib | Immunocore | Approved | SMN2 | |
| ELV-1411 | Enliven | NDA/BLA | HER2 | |
| TXG-6755 | 10x Genomics | Approved | CD123 | |
| Zoricapivasertib | CSL Limited | Phase 2 | HER2 |