SNCA siRNA

SNCA siRNA is an investigational small interfering RNA (siRNA) therapeutic specifically designed to silence the SNCA gene, which encodes the protein alpha-synuclein. Overexpression or accumulation of alpha-synuclein is implicated in the pathogenesis of Parkinson’s disease and other synucleinopathies. SNCA siRNA functions by engaging the RNA interference (RNAi) pathway: once delivered into neurons, the double-stranded siRNA is incorporated into the RNA-induced silencing complex (RISC), which uses the guide strand to bind complementary SNCA mRNA, leading to targeted degradation of SNCA mRNA and a subsequent reduction in alpha-synuclein protein levels. Preclinical studies have shown sustained, sequence-specific knockdown of SNCA in neuronal tissues, both in vitro and in vivo, with associated improvements in disease models. Intrathecal and direct brain infusion approaches have been investigated, with siRNA agents such as LY3962681 currently in phase 1 clinical trials for Parkinson’s disease[4][8][2][6]. Developers are optimizing chemical modification and delivery methods to improve CNS distribution and duration of effect.

Other names

siRNA targeting SNCAalpha-synuclein siRNAanti-SNCA siRNA