Synuclein lowering
The lane
- Status
- Active. Trials are running and the idea is still being tested.
- Verdict
- Too much normal synuclein causes disease; the dedicated lowering drugs (antisense and siRNA) are all Phase 1 with no efficacy data; buntanetap, a translation inhibitor, missed its Phase 3 primary measure.
- Family
- Synuclein
- Landmarks
- 15 in this lane, 1 more tagged with it. Advanced 5 · Open 6 · Pending 2 · Dead end 2
The verdict and status come from the project's branches.yaml and use research wording.
Landmarks in this lane
| Synuclein knockout mice are viable | 2000 | Lab study (cells or animals) | Confirmed and built on | – | – | 19 Oct 2026 | 3 |
| Virus-driven synuclein rat and marmoset models | 2002–2003 | Research model | Confirmed and built on | – | – | 19 Oct 2026 | 4 |
| Extra copies of the synuclein gene cause Parkinson's | 2003 | Discovery | Confirmed and built on | – | – | 19 Oct 2026 | 5 |
| SNCA duplications: ordinary-looking Parkinson's, partial penetrance | 2004–2009 | Discovery | Confirmed and built on | – | – | 19 Oct 2026 | 5 |
| SNCA Rep1 promoter variant raises risk | 2006 | Discovery | Confirmed and built on | – | – | 19 Oct 2026 | 2 |
| Synuclein helps assemble synapse machinery | 2010 | Discovery | Still being studied | – | – | 19 Oct 2026 | 5 |
| Too little synuclein harms neurons | 2010–2018 | Discovery | Still being studied | – | – | 19 Oct 2026 | 3 |
| A risk variant that regulates SNCA | 2016 | Discovery | Still being studied | – | – | 20 Oct 2026 | 2 |
| β2 agonists lower SNCA | 2017 | Discovery | Still being studied | – | – | 20 Oct 2026 | 1 |
| Gene editing of SNCA | 2018–2023 | Lab study (cells or animals) | Still being studied | – | – | 20 Oct 2026 | 3 |
| Synuclein-lowering drugs (overview) | 2022–2026 | Trial in people | Mixed or early results | – | – | 19 Oct 2026 | 3 |
| ION464 / BIIB101 (synuclein antisense) | 2022–2027 | Trial in people | Did not work | 1 | 40 | 22 Oct 2026 | 3 |
| Buntanetap Phase 3 | 2024 | Trial in people | Did not work | 3 | 523 | 22 Oct 2026 | 4 |
| LY3962681 (Lilly siRNA) | 2024–2030 | Trial in people | Result due Jul 2030 | 1 | 124 | 19 Oct 2026 | 2 |
| ALN-SNCA (Regeneron siRNA) | 2025–2029 | Trial in people | Result due Jun 2029 | 1 | 46 | 19 Oct 2026 | 4 |
Also tagged with this lane
| First Parkinson's gene: SNCA | 1997 | Discovery | Confirmed and built on | – | – | 19 Oct 2026 | 4 |
Links with other lanes
Built on work from
- Synuclein misfolding & spread: 4 links
- Gene therapy & delivery: 3 links
- Biomarkers: 1 link
- Models: 1 link
Led to work in
- Lysosome & GBA: 1 link
- Trial methods & regulation: 1 link
- Models: 1 link
- Synuclein misfolding & spread: 1 link