This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Aged 30 to 70 years inclusive; both males and females are eligible.
The participant fully understands the purpose, nature, procedures of the study and potential adverse events, voluntarily agrees to take part in the study and signs the informed consent form (ICF).
Participants with clinically diagnosed idiopathic Parkinson's disease (PD) meeting the 2015 Movement Disorder Society (MDS) diagnostic criteria for idiopathic PD.
Participants carrying at least one pathogenic GBA1 variant.
Atypical or secondary parkinsonism (including Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
Patients with clinically confirmed LRRK2 mutation.
Previous history of pallidotomy, Deep Brain Stimulation (DBS) surgery, striatal surgery, extrapyramidal surgery, stereotactic brain surgery or other brain surgeries; or any other surgical history judged by the investigators to interfere with the subject's participation in this study.
Participants with previous head Computed Tomography (CT) / Magnetic Resonance Imaging (MRI) showing neurological diseases such as brain trauma, vascular malformations, hydrocephalus, brain tumors; subjects with hyperintense white matter signals on MRI indicating risk of intracranial hemorrhage; subjects with cerebrovascular lesions or microbleeds on SWI (Susceptibility-Weighted Imaging); subjects with vulnerable plaques or intraplaque hemorrhage of craniocervical arteries on HR-MRI (High-Resolution Magnetic Resonance Imaging); or subjects with imaging abnormalities in the striatum or other brain regions that substantially increase surgical risks.