Modality
Cell Therapy
MOA
CAR-T CD19
Target
CD123
Pathway
RNA Splicing
Rett
Development Pipeline
Preclinical
~Oct 2014
→ ~Jan 2016
Phase 1
~Apr 2016
→ ~Jul 2017
Phase 2
Oct 2017
→ Jan 2029
Phase 2Current
NCT05425786
1,104 pts·Rett
2017-10→2029-01·Recruiting
1,104 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2029-01-022.8y awayPh2 Data· Rett
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029
P2
Recruit…
Catalysts
Ph2 Data
2029-01-02 · 2.8y away
Rett
Recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05425786 | Phase 2 | Rett | Recruiting | 1104 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| Talasotorasib | Exelixis | Preclinical | APOC3 | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| Elracapivasertib | Kymera | Phase 3 | PRMT5 | |
| SND-4562 | Syndax | NDA/BLA | AuroraA | |
| Zorizumab | Beam | Approved | CD123 |