Diseases affecting the brain and central nervous system represent one of the largest healthcare challenges and greatest unmet medical needs in the world today. This week, a renowned biotech leader Tony Coles, M.D. launched Yumanity Therapeutics, a startup that will use discoveries about yeast to develop new treatments for Parkinson’s, Alzheimer’s, and ALS, also known as Lou Gehrig’s disease.

In each of these neurodegenerative diseases, proteins that play important roles in the brain become mis-folded. Yumanity has hired, and will focus on technologies developed by, Dr. Susan Lindquist, the former MIT biomedical researcher who won the National Medal of Science for her work in how proteins fold in the brain.

When these misfolded proteins are put into yeast cells, they die. There begins the research, which the company hopes will lead to new treatments and drug discoveries.

The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and the team will begin work immediately on advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

“We believe the time is now to translate the remarkable advances in protein folding science achieved by Sue and her colleagues into a drug discovery engine that we believe can have a rapid and transformational impact on neurodegenerative diseases,” said Dr. Coles in the announcement Monday. “While no cures exist and currently available therapies only address the symptoms of these devastating illnesses, our unique approach overcomes the fundamental limitations of today’s target-based drug discovery by exploiting the power of phenotypic screening in yeast and human stem cell-derived neurons. This approach is the Yumanity advantage and enables us to identify potential new therapies to modify the cause of these diseases at the cellular level.”

DNA, the foundational code for all proteins, is initially decoded into long, linear strands of amino acids. These simple strands must fold into precise and highly distinct shapes to form functional proteins. When folding goes awry, the consequences can be disastrous, causing disruption of basic cellular processes. Current research and drug discovery efforts have been stymied by a lack of adequate tools to study the protein folding defects that are at the heart of these diseases and discover new drugs that will correct them.

Yumanity will use three discovery platforms, which formed the basis of Dr. Lindquist’s research originating in the lab at the Whitehead Institute and the Howard Hughes Medical Institute, have been documented in three peer-reviewed publications.

Dr. Coles is an esteemed leader in the biotech industry who brings more than 20 years of experience in drug discovery and development to the company. Prior to his role as founding chairman and CEO of Yumanity, Dr. Coles was chairman and CEO of Onyx Pharmaceuticals, Inc., which was acquired by Amgen in late 2013 for $10.5 billion. Under his leadership, Onyx introduced two new innovative cancer medicines to patients.


Story tip from Joel Arellano


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