Modality
Gene Editing
MOA
Cl18.2
Target
USP1
Pathway
Fibrosis
GA
Development Pipeline
Preclinical
~Feb 2018
→ ~May 2019
Phase 1
~Aug 2019
→ ~Nov 2020
Phase 2
Feb 2021
→ Feb 2030
Phase 2Current
NCT03087542
458 pts·GA
2021-02→2030-02·Completed
458 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-09-246mo awayNDA· GA
2030-02-083.9y awayPh2 Data· GA
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
P2
Complet…
Catalysts
NDA
2026-09-24 · 6mo away
GA
Ph2 Data
2030-02-08 · 3.9y away
GA
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03087542 | Phase 2 | GA | Completed | 458 | HbA1c |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| ABB-2476 | AbbVie | Phase 1/2 | USP1 | |
| Zorivorutinib | Bristol-Myers Squibb | Approved | DLL3 | |
| Elrarapivir | Regeneron | Phase 1 | GIP-R | |
| HAL-9635 | Halozyme | Phase 2/3 | EZH2 | |
| VKT-6548 | Viking Therapeutics | Phase 2/3 | PD-L1 | |
| Tiratapinarof | Intellia | NDA/BLA | APOC3 | |
| ELV-1411 | Enliven | NDA/BLA | HER2 | |
| Tezesotorasib | Enliven | Phase 2 | USP1 | |
| ETN-5063 | 89bio | Phase 1/2 | USP1 | |
| Gelitapinarof | MacroGenics | Approved | CDK2 |