In the field of drug discovery, so called ‘undruggable’ targets are like the stubborn children of the protein world – notoriously difficult to find, and even harder to target. For undruggable protein misfolding diseases such as Alzheimer’s and Parkinson’s, traditional drug discovery and biological approaches have, to date, proved largely ineffective. However, following a wave of scientific advancement and investment from biotech Treventis, Origami and Wren Therapeutics, we are starting to see some promising new research emerge. Most recently, DeepMind’s ground-breaking application of AI has allowed researchers to accurately predict protein structures, vastly accelerating efforts to understand the building blocks of cells. Perhaps these elusive targets are not so ‘undruggable’ after all?
In a recent webinar with pharmacological chaperone company Cantabio’s CEO, Gergely Toth, Toth explained the background behind Cantabio’s small molecule pharmacological chaperones, including the use of computational structure-based and biophysics based high-throughput screening approaches to find small molecule binders. With data also showing how selected small molecules binding to full-length monomeric Tau were able to rescue Tau-overexpression-caused phenotypes in Drosophila, this helped to demonstrate how targeting the monomeric form of alpha synuclein and tau by small molecules may be a feasible drug discovery strategy for Parkinson’s and Alzheimer’s. Being able to modulate and stabilize aberrant proteins through targeted small molecule therapeutics in this way will help enormously with various neurodegenerative diseases where protein misfolding is one of the main drivers for disease progression.
You can register to watch this webinar for free on-demand here
The key to exploring these unchartered territories and cutting-edge insights? Partnership. Often the best way to break new ground in the way that DeepMind and Cantabio are doing is to join with other institutions and pool expertise. This is one of the primary focuses of the upcoming Targeting Protein Misfolding Congress – an event designed to bring together academic and industry leaders to share their research around identifying and validating targets in protein misfolding diseases and intrinsically disordered proteins.
Three reasons why you should attend:
- Understand what delivery mechanisms are revolutionising the delivery of biologics across the blood-brain-barrier, and discuss targeted ASO delivery in CNS disorders with Ionis Pharmaceuticals. Also hear from Laura Ranum, Professor at University of Florida, on how she delivered antibodies across the blood brain barrier to rescue ALS phenotypes.
- Discover what appropriate disease models, physiological assays and blood biomarkers are best to evaluate translational aspects and target engagement for your R&D programs/projects with Chris Missling, CEO at Anavex Corporation and Marcia Taylor, VP of Research at Treventis.
- Rationalise your drug discovery efforts by learning how ProMIS Neurosciences neutralise toxicity and propagation in disease through antibodies directed against misfolding-selective epitopes, and learn how to reengineer chaperone networks to provide therapeutic strategies against neuro-degenerative disorders with Judith Frydman, Professor at Stanford.
Use Drug Discovery World’s exclusive discount code, DDW10, for an additional 10% discount on your booking. Book here