SNP614

SNP614 is a next‑generation antisense oligonucleotide (ASO) therapeutic candidate developed by SciNeuro Pharmaceuticals to treat Parkinson’s disease by selectively downregulating leucine‑rich repeat kinase 2 (LRRK2) expression.[3][13] Built on a chemically modified locked‑nucleic acid (LNA) scaffold, SNP614 targets LRRK2 mRNA to reduce LRRK2 levels and activity, addressing pathogenic LRRK2 overactivation implicated in both familial and sporadic Parkinson’s disease.[3][1][7] In preclinical in vitro and in vivo models, including non‑human primates, SNP614 has shown broad CNS distribution, potent and durable knockdown of LRRK2 mRNA and protein, and a favorable tolerability and safety profile, supporting its potential as a disease‑modifying therapy rather than purely symptomatic treatment.[1][3][2][9]