Modality
Gene Editing
MOA
CAR-T CD19
Target
GPRC5D
Pathway
Wnt
Hemophilia ALGSParkinson's
Development Pipeline
Preclinical
~Mar 2015
→ ~Jun 2016
Phase 1
~Sep 2016
→ ~Dec 2017
Phase 2
Mar 2018
→ Jun 2030
Phase 2Current
NCT04105039
1,436 pts·LGS
2018-03→2025-02·Active
NCT03471954
554 pts·Hemophilia A
2025-04→2030-06·Recruiting
1,990 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-02-151.1y agoPh2 Data· LGS
2030-06-154.2y awayPh2 Data· Hemophilia A
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
P2
Active
P2
Recruit…
Catalysts
Ph2 Data
2025-02-15 · 1.1y ago
LGS
Ph2 Data
2030-06-15 · 4.2y away
Hemophilia A
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04105039 | Phase 2 | LGS | Active | 1436 | Safety |
| NCT03471954 | Phase 2 | Hemophilia A | Recruiting | 554 | ORR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Pexatenlimab | Novartis | Phase 1/2 | GPRC5D | |
| Ribotuximab | Merck & Co | Phase 2 | GPRC5D | |
| Datoglumide | AbbVie | Approved | CFTR | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| AMG-9654 | Amgen | Phase 2 | GPRC5D | |
| GIL-9227 | Gilead Sciences | Phase 2/3 | GPRC5D | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| BII-4342 | Biogen | Phase 2/3 | CGRP |