Modality
Gene Editing
MOA
DLL3 ADC
Target
CFTR
Pathway
Epigenetic
FabryIgAN
Development Pipeline
Preclinical
~Mar 2019
→ ~Jun 2020
Phase 1
~Sep 2020
→ ~Dec 2021
Phase 2
Mar 2022
→ Aug 2025
Phase 2Current
NCT04572927
506 pts·Fabry
2022-03→2025-08·Completed
506 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-08-088mo agoPh2 Data· Fabry
Trial Timeline
Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3
P2
Complet…
Catalysts
Ph2 Data
2025-08-08 · 8mo ago
Fabry
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04572927 | Phase 2 | Fabry | Completed | 506 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| NVS-4076 | Novartis | Preclinical | TROP-2 | |
| Rimamavacamten | Merck & Co | Phase 1 | PI3Kα | |
| Datoglumide | AbbVie | Approved | CFTR | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| AMG-9654 | Amgen | Phase 2 | GPRC5D | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| Doxasotorasib | Samsung Biologics | Phase 2/3 | CFTR |