Modality
Gene Editing
MOA
EZH2i
Target
CD20
Pathway
Epigenetic
EpilepsyAngelman
Development Pipeline
Preclinical
~Dec 2020
→ ~Mar 2022
Phase 1
Jun 2022
→ Nov 2025
Phase 1Current
NCT04447590
1,222 pts·Angelman
2022-06→2025-11·Active
NCT05034404
2,604 pts·Angelman
2022-11→TBD·Active
3,826 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-11-115mo agoPh2 Data· Angelman
Trial Timeline
Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
P1/2
Active
P1/2
Active
Catalysts
Ph2 Data
2025-11-11 · 5mo ago
Angelman
Active|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04447590 | Phase 1/2 | Angelman | Active | 1222 | BodyWt |
| NCT05034404 | Phase 1/2 | Angelman | Active | 2604 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8637 | Johnson & Johnson | NDA/BLA | CD20 | |
| Lisolucimab | Novartis | Approved | CD20 | |
| Adagrazumab | Novartis | Phase 2/3 | Cl18.2 | |
| Ribotuximab | Merck & Co | Phase 2 | GPRC5D | |
| ABB-8985 | AbbVie | Phase 2 | CD20 | |
| NVO-9615 | Novo Nordisk | Phase 2/3 | PRMT5 | |
| ARG-3265 | Argenx | Phase 1/2 | CD20 | |
| Polalucimab | Jazz Pharma | Preclinical | PLK4 | |
| ASN-7038 | Ascendis Pharma | Phase 3 | VEGF | |
| Liratinib | Krystal Biotech | NDA/BLA | TIM-3 |