Modality
Gene Therapy
MOA
AHRant
Target
BCL-2
Pathway
Complement
ALL
Development Pipeline
Preclinical
~Apr 2013
→ ~Jul 2014
Phase 1
~Oct 2014
→ ~Jan 2016
Phase 2
~Apr 2016
→ ~Jul 2017
Phase 3
~Oct 2017
→ ~Jan 2019
NDA/BLA
~Apr 2019
→ ~Jul 2020
Approved
Oct 2020
→ Sep 2026
ApprovedCurrent
NCT07412857
313 pts·ALL
2020-10→2026-09·Active
313 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2026-09-176mo awayPh3 Readout· ALL
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
Approved
Active
Catalysts
Ph3 Readout
2026-09-17 · 6mo away
ALL
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07412857 | Approved | ALL | Active | 313 | UPDRS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-8550 | Roche | Approved | BCL-2 | |
| AZN-5978 | AstraZeneca | Phase 2/3 | GIP-R | |
| BII-5449 | Biogen | Phase 3 | FXIa | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| Elratapinarof | Halozyme | Phase 2 | C5 | |
| HAL-996 | Halozyme | Phase 1/2 | BCL-2 | |
| Nidaratamab | Exelixis | Phase 3 | FLT3 | |
| Bemaderotide | Revolution Medicines | Phase 3 | BCL-2 |