Human Stem-Cell Based Development of a Potent Alzheimer’s Drug Candidate
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been…
A Phase I dose escalation and expansion clinical trial of the novel first-in-class Polo-like Kinase 4 (PLK4) inhibitor, CFI-400945 in patients with advanced solid tumors
Cancer is a major cause of morbidity and mortality worldwide. Many believe that progress in drug development has not been as rapid as one would have predicted based on the…
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) affects 1 in every 3,500 boys worldwide. DMD is caused by mutations in the gene encoding dystrophin, a protein key to muscle health. DMD patients are…
Improving Existing Drugs for Long QT Syndrome type 3 (LQT3) by hiPSC Disease-in-Dish Model
This project uses patient hiPSC-derived cardiomyocytes to develop a safe and effective drug to treat a serious heart health condition. This research and product development will provide a novel method…
Use of human iPSC-derived neurons from Huntington’s Disease patients to develop novel, disease-modifying small molecule structural corrector drug candidates targeting the unique, neurotoxic conformation of mutant huntingtin
The long-term objective of this project is to develop a drug to treat Huntington’s disease (HD), the most common inherited neurodegenerative disorder. Characterized by involuntary movements, personality changes and dementia,…
A drug-screening platform for autism spectrum disorders using human astrocytes
Autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect about 1% of children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social…
Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases
ALS is a progressive neurodegenerative disease that primarily affects motor neurons (MNs). It results in paralysis and loss of control of vital functions, such as breathing, leading to premature death.…
Human pluripotent stem cell-based therapeutics for preeclampsia
Preeclampsia (PE) is a pregnancy complication, characterized by high blood pressure and abnormal kidney function, which affects 5-8% of all pregnancies. It is responsible for a significant proportion of maternal…
Combination Therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy and one of the most common fatal genetic disorders. Approximately one in every 3,500 boys worldwide is affected with DMD.…
Treatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cells
Although most individuals are aware that osteoporosis is disease of increased bone fragility that results from estrogen deficiency and aging, most are unaware of the high risk and cost of…
