Small Molecules Targeting Neuro Indications

Vanqua Bio is a biopharmaceutical company dedicated to developing innovative therapies for neurodegenerative diseases, with a particular focus on Parkinson's disease and related conditions. The company employs a unique drug development strategy that integrates advanced knowledge of lysosomal biology and the innate immune system. Their lead program targets glucocerebrosidase (GCase), a critical enzyme linked to the genetic risk factor GBA1, which is associated with Parkinson's disease. By utilizing patient-derived induced pluripotent stem cells (iPSCs), Vanqua Bio aims to identify small molecule activators of GCase that can enhance lysosomal function, reduce the accumulation of toxic proteins like alpha-synuclein, and ultimately slow or halt disease progression.

In addition to their work on GCase, Vanqua Bio is also exploring the role of the complement system in neurodegenerative diseases. Their second program focuses on the C5a receptor 1 (C5aR1), which is implicated in neuroinflammation. By developing small molecule antagonists of C5aR1, the company aims to mitigate the harmful effects of overactive complement signaling in the central nervous system, potentially offering new treatment avenues for conditions such as Alzheimer's disease. Through these innovative approaches, Vanqua Bio is committed to delivering first-in-class and best-in-class therapies that can significantly improve the lives of patients suffering from neurodegenerative disorders.

United States

Vanqua Bio is helping usher in a new era of hope for the millions of people living with neurodegenerative diseases. Our unique drug-development approach is designed to overcome longstanding challenges in the neuroscience field by capitalizing on the power of human genetics to identify genes that cause or increase the risk of neurodegenerative disease. We leverage novel, proprietary research tools and in vitro modeling of disease, based on patient-derived neuronal cells, to develop transformative therapies that slow or stop the progression of Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Gaucher disease (GD).

Vanqua Bio

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