Modality
Gene Therapy
MOA
BCL-2i
Target
BCL-2
Pathway
Angiogenesis
PompeHuntington's
Development Pipeline
Preclinical
~Oct 2019
→ ~Jan 2021
Phase 1
Apr 2021
→ Nov 2030
Phase 1Current
NCT06458462
626 pts·Huntington's
2021-04→TBD·Active
NCT07813900
2,433 pts·Huntington's
2025-02→2030-11·Active
3,059 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2030-11-274.7y awayPh2 Data· Huntington's
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
P1/2
Active
P1/2
Active
Catalysts
Ph2 Data
2030-11-27 · 4.7y away
Huntington's
Active|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06458462 | Phase 1/2 | Huntington's | Active | 626 | EDSS |
| NCT07813900 | Phase 1/2 | Huntington's | Active | 2433 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5501 | Pfizer | Phase 1 | BTK | |
| RHH-8550 | Roche | Approved | BCL-2 | |
| RHH-3592 | Roche | Phase 1/2 | GIP-R | |
| Geliderotide | Novartis | NDA/BLA | IL-17A | |
| Tezecilimab | Takeda | NDA/BLA | Tau | |
| Rimaosocimab | Amgen | Preclinical | FXIa | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| Capifutibatinib | Incyte | Phase 3 | PCSK9 |