Snowdon

Therapeutic Area: Neurological Diseases

Amyloid diseases represent a large class of incurable human pathologies including Alzheimer's disease (AD), Parkinson's disease (PD), and motor neuron disease. In all of these cases, proteins transform from their proper native structure into an abnormal β-rich structure known as amyloid fibril. Coined the "amyloid hypothesis", this process of amyloid fibril formation is responsible for triggering a cascade of physiological events that contribute directly to the initiation and progression of amyloid diseases.

The key step in amyloid fibril formation involves misfolding of individual proteins which eventually leads to collective protein aggregation. This process is triggered by short peptide segments within the protein called core nucleation motifs that are particularly susceptible to misfolding from α-helix or random coil to β-strand. We refer to this trait as "hidden β-strand propensity" (HβP). A viable therapeutic strategy for slowing, preventing, and possibly reversing the progression of this array of devastating diseases is to design aggregation inhibitors that specifically target these core nucleation motifs. Unfortunately, precisely locating these core nucleating motifs in protein sequences remains a major impediment to the discovery of medical treatments.

To address this need, Snowdon has perfected a computational tool called the HβP method that rapidly pinpoints these β-prone core nucleation motifs in disease-related proteins. To our best knowledge, no other computational method exists that possesses this capability. Scientists at Snowdon use the HβP tool to guide the discovery of drug molecules that prevent the misfolding and aggregation of proteins associated with AD, PD, and many other amyloid diseases.

Recognizing that Snowdon's innovative approach may lead to medical breakthroughs, the National Institutes of Health (NIH) has granted a small business award to Snowdon. This pioneering research may lead to medicines that, for the first time, treat the root cause rather than just the symptoms of these notorious diseases.

Therapeutic Areas: Cancer | Acute and Chronic Pain | Infectious Diseases | Neurological Diseases