New Biotech Company Yumanity Launched to Treat Protein Misfolding Diseases, Like ALS

Yumanity Therapeutics, a new biotechnology company working on the development of drug discoveries for the treatment of conditions caused by protein misfolding, was launched last Monday by well-known leader in the biotech industry Tony Coles, M.D., who is not only the founding investor of the company, but will also serve as its chairman and chief executive officer. In addition, Susan Lindquist, Ph.D., joined the team as scientific founder and award-winning protein folding expert.

The mission of the company will be to identify and develop new disease-modifying treatments able to address several illnesses with critical unmet medical needs, such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and Parkinson’s disease. The primary focus will be on neurodegenerative diseases, but the company is expects to expand into other conditions in the future. Yumanity Therapeutics has already presented its proprietary platforms meant to determine potential new target therapies, and announced that their research team will start working to advance new drugs for treating the three conditions.

“I am thrilled and inspired to be at the forefront of innovation in an area of real, global medical need,” said Coles. “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.”

“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,” the CEO added.

Cole launched the company based on the realization that brain and central nervous system diseases are one of the biggest healthcare challenges in the world. About 50 million people worldwide are affected by neurodegenerative diseases, and it is estimated that the number of patients with diseases such as Alzheimer’s may double every 20 years. In spite of the fact that there are no currently approved disease-modifying therapies available, $650 billion dollars have already been spent on treating these diseases, and $1 trillion willl have been spent by 2030.

ALS, as well as other neurodenegerative diseases, is caused by protein misfolding, and current studies have been stymied by a lack of adequate tools to study protein folding defects. Yumanity aims to improve the lives of ALS patients with three different discovery platforms, which were created at Lindquist’s lab at the Whitehead Institute and the Howard Hughes Medical Institute (HHMI).

The first product is a proprietary ultra high-throughput, phenotypic screening (uHTS) platform, which is designed to model different protein misfolding pathologies in yeast and compounds to correct these discovered pathologies, while the second is a human neuronal platform, meant to produce neurons from patients’ stem cells with disease-causing genetic mutations that are used to validate the molecules discovered in yeast. The third is a drug-target platform for identification that explores the power of yeast genetics and protein network analyses to elucidate mechanisms of action for pathology correcting molecules as new chemical entities are discovered.

In addition to Coles and Lindquist, Kenneth Rhodes, Ph.D., has also joined the company as chief scientific officer. Rhodes will be responsible for supervising the company’s scientific work about the integrated platforms, as well as for the drug discovery research programs. The platforms were designed during a collaborative development of the company’s scientific co-founders Vikram Khurana, M.D., Ph.D., Chee-yeun Chung, Ph.D. and Daniel Tardiff, Ph.D.