Modality
Gene Editing
MOA
EGFRi
Target
SMN2
Pathway
DDR
ACCSMA
Development Pipeline
Preclinical
~Apr 2019
→ ~Jul 2020
Phase 1
Oct 2020
→ Feb 2028
Phase 1Current
NCT06862610
2,791 pts·SMA
2025-07→2028-02·Terminated
NCT03553872
1,632 pts·ACC
2020-10→2026-04·Completed
4,423 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-04-152w awayInterim· ACC
2028-02-171.9y awayInterim· SMA
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
P1
Complet…
P1
Termina…
Catalysts
Interim
2026-04-15 · 2w away
ACC
Interim
2028-02-17 · 1.9y away
SMA
CompletedTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06862610 | Phase 1 | SMA | Terminated | 2791 | HAM-D |
| NCT03553872 | Phase 1 | ACC | Completed | 1632 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| JNJ-5094 | Johnson & Johnson | Phase 2/3 | GIP-R | |
| NVO-6275 | Novo Nordisk | Phase 1 | SMN2 | |
| GSK-2051 | GSK | Phase 1 | PSMA | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| GIL-8529 | Gilead Sciences | Phase 1/2 | JAK1 | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| REG-647 | Regeneron | Preclinical | SMN2 | |
| 207-2754 | Samsung Biologics | Phase 1/2 | KRASG12D | |
| Mavuratamab | Blueprint Medicines | NDA/BLA | BET |