LRRK2-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

Resumen del estudio

and not dose limiting. No serious adverse events related to BIIB094 were reported in either part A or B. Systemic BIIB094 exposure increased with dose. Cerebrospinal fluid (CSF) LRRK2 and phosphorylated Rab10 levels were lowered by up to 59% and up to 50%, respectively, irrespective of LRRK2 variant status. Concomitant reductions in CSF lysosomal protein levels suggested a potential mechanism whereby LRRK2 therapeutics may impact underlying PD pathophysiology. ClinicalTrials.gov identifier, NCT03976349 ; EudraCT number, 2018-002995-42. © 2026. The Author(s), under exclusive licence to Springer Nature America, Inc.