Rare Daily Staff

Vanqua Bio raised $85 million in a series B financing round to discover and developing next-generation medicines for neurodegenerative diseases.

Omega Funds led the financing with participation from series A investor OrbiMed and new investors Surveyor Capital (a Citadel company), Avoro Ventures, Casdin Capital, Pontifax, Eli Lilly, Logos Capital, and Osage University Partners.

Proceeds from the financing will be used to accelerate the development of the company’s therapeutic programs, which will focus on rare diseases, such as Gaucher disease (GD) and amyotrophic lateral sclerosis (ALS), and more common conditions such as Parkinson’s disease (PD) and Alzheimer’s disease (AD).

“Vanqua Bio is helping usher in a new era of hope for people living with neurodegenerative disorders,” said Jim Sullivan, co-founder and CEO of Vanqua Bio. “Our mission—to develop effective therapies that slow or stop the progression of PD, AD, ALS, and Gaucher disease—is a very personal one, and we are excited to have the support of world-class investors.”

Vanqua Bio is a patient-founded company with a technology platform based on seminal research conducted by Dimitri Krainc, chair of the Department of Neurology at Northwestern University’s Feinberg School of Medicine.

The company’s drug development approach overcomes 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. The company leverages novel, proprietary research tools and in vitro modeling of disease based on patient-derived neuronal cells to translate these genetic insights into transformative therapies.

Vanqua Bio’s lead program targets glucocerebrosidase (GCase) as a potential treatment for Parkinson’s disease (PD) and all forms of Gaucher disease. The program is focused on developing small-molecule activators of GCase, an enzyme that regulates lipid homeostasis in cells. Reductions in the activity of GCase disrupt the function of the lysosome, the recycling center of the cell, enabling toxic forms of proteins, including alpha synuclein, to accumulate and harm neurons. Alpha synuclein aggregation is a hallmark of multiple neurodegenerative diseases including PD. Mutations in the gene that encodes GCase (GBA1) can cause GD and are strongly associated with a form of Parkinson’s disease called GBA-PD and a subset of Lewy body dementia (GBA-LBD) cases. Vanqua Bio’s series B financing will advance the company’s best-in-class GCase activators into human testing within the next two years, initially focusing on GD and GBA-PD.

In addition to its GCase activator programs, Vanqua Bio will also advance programs targeting the innate immune system, which, when overactivated, can accelerate the progression of several neurological diseases. The company is advancing small molecule and antisense oligonucleotide programs with an initial focus on ALS and AD.

Photo: Jim Sullivan, co-founder and CEO of Vanqua Bio

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