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Vanqua Bio to Advance Investigational Drug Into Parkison's Disease Clinical Trials Next Year

NEW YORK – Vanqua Bio announced on Tuesday that in Q1 2024 it will begin testing the investigational agent VQ-101 as a treatment for Parkinson's disease characterized by GBA1 mutations in clinical trials.

Approximately 10 percent of Parkinson's patients have GBA1 mutations, the most common genetic risk factor for the disease. Mutations in the gene reduce activity of the enzyme glucocerebrosidase, or GCase, which limits the ability of lysosomes to efficiently clear toxic alpha synuclein proteins that harm neurons.

In preclinical studies of patient-derived neuronal cells, VQ-101 has shown it can activate wild-type and mutant forms of GCase, lower lipid substrates, and hinder the buildup of pathogenic forms of alpha synuclein. In whole blood ex vivo studies, VQ-101 has activated GCase in multiple species including humans, and in vivo the agent has enhanced GCase activity in four animal species and demonstrated significant central nervous system exposure with once-daily dosing.

In clinical trials starting next year, Chicago-based Vanqua hopes to establish VQ-101's safety and demonstrate target engagement in healthy volunteers and GBA-mutated Parkinson's patients.

Vanqua also announced the appointments of Jesse Cedarbaum as the firm's chief medical officer and Omer Siddiqui as chief development officer. Cedarbaum is a neurologist and clinical trialist who previously worked at Regeneron Pharmaceuticals and other drug companies, and Siddiqui was head of clinical development operations at neurodegenerative-focused drugmaker Alector. Rand Sutherland, a physician-scientist and former CEO of Seeker Biologics and president of Translate Bio, will also join Vanqua's board of directors.