Modality
Gene Therapy
MOA
STINGag
Target
CFTR
Pathway
RNA Splicing
FSGSRSV
Development Pipeline
Preclinical
~Mar 2013
→ ~Jun 2014
Phase 1
~Sep 2014
→ ~Dec 2015
Phase 2
~Mar 2016
→ ~Jun 2017
Phase 3
~Sep 2017
→ ~Dec 2018
NDA/BLA
~Mar 2019
→ ~Jun 2020
Approved
Sep 2020
→ Jun 2026
ApprovedCurrent
NCT07214747
57 pts·FSGS
2020-09→2026-06·Active
57 total pts1 indication
CompletedCurrentUpcoming
Catalysts (1)
2026-06-183mo awayPh3 Readout· FSGS
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3
Approved
Active
Catalysts
Ph3 Readout
2026-06-18 · 3mo away
FSGS
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07214747 | Approved | FSGS | Active | 57 | PANSS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| RHH-5389 | Roche | Preclinical | RET | |
| Datoglumide | AbbVie | Approved | CFTR | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-4496 | Sanofi | Phase 2/3 | CD3 | |
| SNY-5783 | Sanofi | Phase 1/2 | MET | |
| DSN-1421 | Daiichi Sankyo | Phase 3 | Nectin-4 | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| INC-5849 | Incyte | Phase 2 | PCSK9 |