Modality
ASO
MOA
CAR-T CD19
Target
C5
Pathway
T-cell
GBMParkinson'sHuntington's
Development Pipeline
Preclinical
~Aug 2009
→ ~Nov 2010
Phase 1
~Feb 2011
→ ~May 2012
Phase 2
~Aug 2012
→ ~Nov 2013
Phase 3
~Feb 2014
→ ~May 2015
NDA/BLA
~Aug 2015
→ ~Nov 2016
Approved
Feb 2017
→ Apr 2031
ApprovedCurrent
NCT08972654
123 pts·Parkinson's
2017-02→TBD·Active
NCT03856516
1,673 pts·GBM
2021-11→2031-02·Not yet recruiting
NCT06191106
1,897 pts·Huntington's
2023-02→2031-04·Active
3,693 total pts3 indications
CompletedCurrentUpcoming
Catalysts (3)
2026-11-248mo awayFast Track· Huntington's
2031-02-274.9y awayPh3 Readout· GBM
2031-04-195.1y awayPh3 Readout· Huntington's
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3
Approved
Active
Approved
Not yet…
Approved
Active
Catalysts
Fast Track
2026-11-24 · 8mo away
Huntington's
Ph3 Readout
2031-02-27 · 4.9y away
GBM
Ph3 Readout
2031-04-19 · 5.1y away
Huntington's
ActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08972654 | Approved | Parkinson's | Active | 123 | NT-proBNP |
| NCT03856516 | Approved | GBM | Not yet recr... | 1673 | NT-proBNP |
| NCT06191106 | Approved | Huntington's | Active | 1897 | MRD |
Competitors (10)