Clinical trials targeting alpha-synuclein protein in Parkinson’s disease

Deposition of alpha-synuclein (α-synuclein) in the brain is the pathological hallmark of Parkinson’s disease (PD). There is abundant evidence that α-synuclein is not simply a marker but is essential for PD pathogenesis. Aggregates of α-synuclein, termed “Lewy bodies”, interfere with autolysosomal degradation in brain neurons and contribute to neuronal death. Therefore, α-synuclein has become a major therapeutic target in PD. Multiple clinical trials are ongoing (see below)[1].

Clinical Trial Phase Status Sponsor Intervention/Agent
Phase II Active Biogen BIIB054
Phase II Active Hoffmann-La Roche Prasinezumab
Phase II Active Georgetown University Nilotinib
Phase I Completed Neuropore Therapies, Inc. NPT200-11
Phase I Completed Affiris AFFITOPE PD01A and PD03A
Phase I Active University of Colorado, Denver Phenylbutyrate
Phase I Recruiting H. Lundbeck A/S Lu AF82422
Phase I Recruiting AstraZeneca MEDI1341

GeneTex is proud to introduce a set of active recombinant proteins, including preformed fibrils of human and mouse α-synuclein proteins and active human tau protein in fibrillar and monomeric forms. The structure of these active proteins was assessed by transmission electron microscopy (TEM), and the biological activities were tested using functional assays on cultured cells.