Modality
Gene Editing
MOA
GLP-1ag
Target
USP1
Pathway
Sphingolipid
CholangiocarcinomaMDD
Development Pipeline
Preclinical
~Dec 2018
→ ~Mar 2020
Phase 1
Jun 2020
→ Sep 2026
Phase 1Current
NCT07782208
1,459 pts·MDD
2025-07→2026-09·Not yet recruiting
NCT08445010
1,275 pts·Cholangiocarcinoma
2020-06→2025-12·Not yet recruiting
2,734 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-12-203mo agoPh2 Data· Cholangiocarcinoma
2026-09-055mo awayPh2 Data· MDD
Trial Timeline
Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
P1/2
Not yet…
P1/2
Not yet…
Catalysts
Ph2 Data
2025-12-20 · 3mo ago
Cholangiocarcinoma
Ph2 Data
2026-09-05 · 5mo away
MDD
Not yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07782208 | Phase 1/2 | MDD | Not yet recr... | 1459 | CR |
| NCT08445010 | Phase 1/2 | Cholangiocarcinoma | Not yet recr... | 1275 | SeizFreq |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-8662 | Pfizer | NDA/BLA | CD19 | |
| ABB-2476 | AbbVie | Phase 1/2 | USP1 | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| MRN-7601 | Moderna | Phase 2 | IL-13 | |
| ARG-1250 | Argenx | Phase 2 | C5 | |
| ILM-5680 | Illumina | Phase 2 | BET | |
| CRS-184 | CRISPR Therapeutics | Phase 1/2 | AHR | |
| Polazanubrutinib | Tango Ther | Phase 1 | IL-17A | |
| Tezesotorasib | Enliven | Phase 2 | USP1 | |
| ETN-5063 | 89bio | Phase 1/2 | USP1 |