Modality
Gene Editing
MOA
BiTE
Target
RET
Pathway
PD-1/PD-L1
SMA
Development Pipeline
Preclinical
~Jan 2018
→ ~Apr 2019
Phase 1
~Jul 2019
→ ~Oct 2020
Phase 2
Jan 2021
→ Oct 2030
Phase 2Current
NCT05220733
991 pts·SMA
2021-01→2030-10·Not yet recruiting
991 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-10-184.6y awayPh2 Data· SMA
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P2
Not yet…
Catalysts
Ph2 Data
2030-10-18 · 4.6y away
SMA
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05220733 | Phase 2 | SMA | Not yet recr... | 991 | MRD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| RHH-5389 | Roche | Preclinical | RET | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| BII-8315 | Biogen | NDA/BLA | CDK2 | |
| ARG-3458 | Argenx | Preclinical | RET |