Modality
Gene Editing
MOA
WRNi
Target
USP1
Pathway
Complement
SMA
Development Pipeline
Preclinical
~Jun 2018
→ ~Sep 2019
Phase 1
~Dec 2019
→ ~Mar 2021
Phase 2
Jun 2021
→ May 2030
Phase 2Current
NCT04210036
2,126 pts·SMA
2021-06→2029-12·Recruiting
NCT07208028
1,967 pts·SMA
2024-05→2030-05·Active
4,093 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-12-203.7y awayPh3 Readout· SMA
2030-05-064.1y awayPh3 Readout· SMA
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P2/3
Recruit…
P2/3
Active
Catalysts
Ph3 Readout
2029-12-20 · 3.7y away
SMA
Ph3 Readout
2030-05-06 · 4.1y away
SMA
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04210036 | Phase 2/3 | SMA | Recruiting | 2126 | FEV1 |
| NCT07208028 | Phase 2/3 | SMA | Active | 1967 | eGFR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| ABB-2476 | AbbVie | Phase 1/2 | USP1 | |
| ABB-1817 | AbbVie | Phase 3 | SHP2 | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| GSK-4334 | GSK | Preclinical | CD47 | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| VRT-5853 | Vertex Pharma | Phase 3 | Aβ | |
| Pemitapinarof | BeiGene | Approved | KRASG12C | |
| VKT-4052 | Viking Therapeutics | Phase 2/3 | TNFα | |
| RCK-1666 | Rocket Pharma | Approved | CD38 |