Modality
Gene Editing
MOA
CAR-T CD19
Target
CGRP
Pathway
NF-κB
DMD
Development Pipeline
Preclinical
~Apr 2016
→ ~Jul 2017
Phase 1
~Oct 2017
→ ~Jan 2019
Phase 2
~Apr 2019
→ ~Jul 2020
Phase 3
~Oct 2020
→ ~Jan 2022
NDA/BLA
Apr 2022
→ Sep 2027
NDA/BLACurrent
NCT05837485
1,499 pts·DMD
2022-04→2027-09·Not yet recruiting
NCT07594530
2,915 pts·DMD
2022-05→2026-06·Recruiting
4,414 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-06-203mo awayPh3 Readout· DMD
2027-09-091.4y awayPh3 Readout· DMD
Trial Timeline
Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q4
NDA/BLA
Not yet…
NDA/BLA
Recruit…
Catalysts
Ph3 Readout
2026-06-20 · 3mo away
DMD
Ph3 Readout
2027-09-09 · 1.4y away
DMD
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05837485 | NDA/BLA | DMD | Not yet recr... | 1499 | Safety |
| NCT07594530 | NDA/BLA | DMD | Recruiting | 2915 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| TAK-9344 | Takeda | Phase 3 | CGRP | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| Zenotinib | BioNTech | Phase 1 | CGRP | |
| Capiglumide | BioNTech | Preclinical | CGRP | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| LEG-9870 | Legend Biotech | Phase 1 | CGRP | |
| Talasotorasib | Exelixis | Preclinical | APOC3 | |
| ASN-4879 | Ascendis Pharma | Phase 3 | CGRP |