A Drug Screening Platform to Increase Protein Expression Levels for Treatment of Neurological Disorders

Challenge: Neurological and psychiatric diseases and disorders affect more than half the population, causing a huge burden on health care costs, lost productivity, and reductions in quality of life. These disorders of the nervous system can span a range of illnesses, including autism spectrum disorders, manic disorders, Parkinson’s disease, stress, depression, and anxiety. Many of these…

First-In-Class Melatonin MT2 Receptor Agonist for Neuropathic Pain: Investigational New Drug (IND) – Enabling Studies

Challenge: Neuropathic pain is a chronic pain disorder that can develop as a result of nerve damage caused by various medical conditions. Available treatments are very limited, and patients resort to using opioids to manage their pain, leading to dependence, tolerance and the so-called opioid-induced hyperalgesia, which is characterized by an increased pain after prolonged use…

Discovery of analgesic drugs targeting the acid-sensing ion channel family

Challenge: Chronic pain affects at least 20% of the population and its societal costs exceed those of cancer, cardiovascular disease and diabetes combined. Most chronic pain patients report many significant side effects from the current opiate-based treatments, including opioid addiction, and these treatments are not highly effective. New, non-opioid therapeutics are therefore needed. Solution: Professor Séguéla,…

Development of a high throughput 3D microphysiological platform for rapid automated assessment of human brain organoids response to drugs targeting neurological disorders

Challenge: Showing architectural similarities with the real human brain, brain organoids offer a promising avenue for understanding human biology, disease processes and for the development and validation of drugs to treat brain-related diseases. By engineering personalized organoids from the reprogrammed cells of patients, scientists can study disease in a very individualized way. However, brain organoids are…

Biomarkers Discovery in Diabetes: New Biomarkers for Measurement of Early Stage Diabetes Disease and for Prediction of Response to Therapy

Challenge: Pancreatic islets play a critical role in diabetes development with about 50% of their function being lost by the time pre-diabetic patients are diagnosed. Levels of pancreatic ß-cells are currently approximated through measurements of blood glucose, C-peptide, and insulin levels. There is therefore an urgent need to develop biomarkers that more accurately monitor the physiological…

Development of a new class of oral and highly potent non-statin LDL-cholesterol lowering therapeutics for patients at high cardiovascular risk

Challenge: Blood level of cholesterol-rich low-density lipoproteins (LDLc) is directly correlated with the incidence of atherosclerosis and cardiovascular risk. Statins, currently the most prescribed class of lipid-lowering drugs, reduce LDLc by increasing LDL receptor (LDLR) gene expression. However, while being on maximally tolerated lipid-lowering therapy, a majority of high-risk patients still have uncontrolled LDLc and remain…

A genetic test to predict diabetic nephropathy in Type 2 diabetes patients

Competition:SynergiQc 2017Funding:$683,665 / 2 yearsBeginning:July 2017 Challenge: Type 2 diabetes (T2D) affects 1 in 11 individuals worldwide and leads to severe complications such as diabetic nephropathy (DN) that is present in about 40% of T2D patients. DN progresses from subclinical disease (microalbuminuria) to overt nephropathy, end-stage renal disease requiring dialysis or transplantation. Identifying T2D patients at…