Landmark 287 · Trial in people
ALN-SNCA (Regeneron siRNA)
The first safety test in people with early Parkinson's.
In short
A first-in-human study of ALN-SNCA, a drug that switches off the SNCA gene's messages, is testing safety and spinal-fluid synuclein in 46 people with early Parkinson's; it ends in mid-2029.
- What it is
- Trial in people
- Outcome
- Result due Jun 2029
- When
- 2025–2029
- Size
- 46 people · Phase 1
- Approach
- Clear the bad protein
- Research lane
- Synuclein lowering
- Trial registry
- NCT07216066
- Last checked
- 19 Oct 2026
The research record
This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".
Who
Regeneron Pharmaceuticals is the registered sponsor. A drug-industry profile lists Alnylam Pharmaceuticals as originator and Regeneron as developer (secondary source).
What happened
- Design (NCT07216066). Phase 1, randomised, placebo-controlled, dose-escalation. Planned 46 adults aged 50 to 80 with Parkinson's for 4 years or less. They are either untreated or on stable dopamine drugs. Started 5 December 2025; recruiting. Estimated completion 7 June 2029.
- Aims. Primary: adverse events and their severity. Secondary: how much alpha-synuclein protein falls in spinal fluid, and how much drug reaches blood, urine and spinal fluid.
- Drug. The registry names ALN-SNCA and says the aim is to lower alpha-synuclein. The company description gives it as an siRNA (small interfering RNA, a short RNA that makes cells destroy one specific messenger RNA). The registry does not state the route of delivery or dose levels.
Why it moved us toward a cure
Having an extra copy of the SNCA gene causes Parkinson's (landmark 14), so cutting the amount of synuclein is the most direct test of the idea. Mice with no synuclein live normally (landmark 201), which supports lowering as safe in principle. This is the third registered lowering programme in patients, after Lilly's (landmark 288) and Ionis's (landmark 289); see the overview in landmark 29.
Outcome
Pending. Safety and spinal-fluid readouts are expected by mid-2029 at the latest. A Phase 1 cannot show that lowering synuclein helps patients.
Sources
- ClinicalTrials.gov NCT07216066 (API read 19 Oct 2026; record updated 21 Sep 2026)
- EU trial record 2025-521431-37-00 (seen in search summary only)
- Synapse drug profile (secondary: originator/developer)
- Not yet verified: route of administration (intrathecal is likely but unconfirmed); the Alnylam–Regeneron agreement; dose levels; any interim data.
How it connects
Built on
- builds on Synuclein knockout mice are viable · 2000
- tests Extra copies of the synuclein gene cause Parkinson's · 2003
Led to
None recorded.