Modality
Gene Therapy
MOA
USP1i
Target
ALK
Pathway
Neuroinflam
Angelman
Development Pipeline
Preclinical
~Sep 2021
→ ~Dec 2022
Phase 1
Mar 2023
→ Dec 2029
Phase 1Current
NCT08481258
1,261 pts·Angelman
2023-03→2029-12·Not yet recruiting
1,261 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-12-113.7y awayPh2 Data· Angelman
Trial Timeline
Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P1/2
Not yet…
Catalysts
Ph2 Data
2029-12-11 · 3.7y away
Angelman
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08481258 | Phase 1/2 | Angelman | Not yet recr... | 1261 | HbA1c |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| SNY-2934 | Sanofi | Phase 3 | KRASG12D | |
| GSK-6983 | GSK | Phase 2 | ALK | |
| BAY-3308 | Bayer | Phase 1 | ALK | |
| AMG-9052 | Amgen | Phase 2/3 | CDK2 | |
| Datozumab | Regeneron | Phase 1 | B7-H3 | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| VRT-6833 | Vertex Pharma | Preclinical | ALK | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| Niralucimab | Ionis | Phase 1 | ALK |