PhoreMost and Cambridge Uni collaborate to identify innovative drug targets for neurodegenerative disease

The studies have the potential to reveal novel insights for previously unknown autophagy signalling components, which could suggest rapid and innovative starting points for new drug treatments.

Professor Rubinsztein is Professor of Molecular Neurogenetics and Deputy Director of the Cambridge Institute for Medical Research. He also holds the appointment of Lead Academic Scientist of the Alzheimer’s Research UK Cambridge Drug Discovery Institute. Professor Rubinsztein’s laboratory is a world leader in cell autophagy, which is a natural pathway that monitors and degrades defective proteins in healthy cells. Therapeutic regulation of this pathway has the promise to reverse or prevent the deposition of aggregation-prone proteins in nerve cells that lead to neurodegenerative diseases.

Unfortunately, few druggable targets have yet been discovered that modulate the autophagy pathway. To address this issue, PhoreMost will work with cutting edge autophagy models from Professor Rubinsztein’s lab. PhoreMost will apply its novel SITESEEKER discovery technology to comprehensively screen for ‘hidden’ targets and drug sites that are the most relevant for treating neurodegenerative disorders. The studies have the potential to reveal novel insights for previously unknown autophagy signalling components, which could suggest rapid and innovative starting points for new drug treatments.

Dr Chris Torrance, CEO of PhoreMost, commented: “We are very excited to be joining forces with Professor Rubinsztein, whose expert insights into neurodegenerative diseases will enable PhoreMost to expand into this important yet therapeutically unmet disease area of ageing, and thereby complement our growing oncology discovery pipeline. This collaboration with the Rubinsztein research team fits our scientific and corporate mantra: find and drug the best disease targets, even if they are currently considered impossible, and engage closely with experts to solve the problem.”

Professor Rubinsztein said: “Autophagy is a fundamental protein quality control process in cells. It has the potential to significantly impact several neurodegenerative disorders, if we can recruit it to degrade intracellular, disease-causing, aggregate-prone proteins in the brain. Combing our cutting-edge research and cell models in this area with innovative techniques like SITESEEKER will enable us to exploit the full depth of targetable disease biology that is present in this pathway.”

Both parties also highlight Professor Rubinsztein’s position at the Alzheimer’s Research UK Cambridge Drug Discovery Institute, a venture focused on early stage drug discovery based on targets identified by the academic community. Dr Torrance added: “The Drug Discovery Institute provides an innovative path for companies to translate early collaborations, like this current one we hold through the University, into later-stage research and development efforts. Like many new drug start-up companies, PhoreMost benefits greatly by working in collaborative networks such Alzheimer’s Research UK’s Drug Discovery Alliance, which is a creative initiative that shares PhoreMost’s primary mission of translational research and rapid new medicine development.”