An Urgent Need

Proteins are large, exquisitely folded molecules that play essential and diverse roles in the human body. When normal protein folding is disrupted in progressive brain diseases, these misfolded proteins clump together – binding to form toxic aggregates of plaques and tangles that cause inflammation, and eventually, nerve cell death.

Protein misfolding is a well-characterized hallmark of severe neurodegenerative diseases, including Alzheimers’ and Parkinson’s, as well as certain rare systemic amyloidosis diseases.

There is a critical unmet need to develop novel treatments for protein misfolding diseases, for which no approved disease-modifying treatments currently exist.

Neurodegenerative diseases including Alzheimer’s and Parkinson’s place an enormous economic and social burden on patients, caregivers, and society. Alzheimer’s is the most common neurodegenerative disease and the leading cause of dementia, which currently affects approximately 47 million victims worldwide. That number is expected to grow to 75 million by 2030.

Meanwhile, Parkinson’s, the second most common neurodegenerative disease, affects an estimated 7 to 10 million worldwide.