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ALN-SNCA
- Development stage
- Phase 1
- Lead developer
- Regeneron Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
**ALN-SNCA** is an investigational RNAi therapeutic developed by Alnylam Pharmaceuticals in collaboration with Regeneron Pharmaceuticals for the treatment of early Parkinson's disease (PD). It utilizes small interfering RNA (siRNA) technology to silence the SNCA gene, reducing the production of α-synuclein protein, which abnormally accumulates in the brain and drives neurodegeneration in PD. Administered intrathecally, ALN-SNCA aims to lower α-synuclein levels in cerebrospinal fluid (CSF), potentially slowing disease progression. It is currently in a Phase 1 first-in-human trial (NCT07216066) evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics in early-stage PD patients.
02
Targets
Alpha-synuclein messenger RNA
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