Therapeutic/Technology: Small molecule therapy
Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor
Therapeutic Candidate or Device Rebecsinib is a novel small molecule inhibitor of ADAR1 splicing that selectively eradicates therapy-resistant cancer stem cells in blood cancers. Indication The target indication is relapsed/refractory secondary acute myeloid leukemia (sAML), or int-2 or high-risk myelofibrosis (MF). Therapeutic Mechanism Rebecsinib therapy for patients with relapsed/refractory sAML, or int-2 or HR MF […]
Skin regeneration and wound healing with a topical BRAF inhibitor
Therapeutic Candidate or Device LUT017 gel is a small molecule inhibitor of BRAF Indication Venus leg non-healing ulcerous wounds Therapeutic Mechanism The administration LUT017 gel will regenerate cutaneous stem cells and induce keratinocyte proliferation resulting in an improvement of VLU non-healing wounds. Unmet Medical Need There are no previously approved FDA drugs for this condition […]
Development of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of Osteoarthritis
Therapeutic Candidate or Device The therapeutic candidate is a drug-like small molecule that promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. Indication Osteoarthritis and cartilage injury Therapeutic Mechanism The therapeutic candidate, through intra-articular administration, promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. The newly formed chondrocytes replace the dead chondrocytes, synthesize and secret […]
Development of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis
Osteoarthritis (OA) is the most prevalent musculoskeletal disease affecting nearly 27 million people in the United States, and is the leading cause of chronic disability in the United States. Current therapeutic options are limited to pain or symptom-modifying drugs and joint replacement surgery; no disease-modifying drugs are approved for clinical use. OA is characterized by […]
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 identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
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 significant technological advancements that have led to improved molecular understanding of this disease. There are numerous explanations for the lag in clinical success with new […]
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 typically weaker than normal by age 3, and with progressive muscle weakness most loose the ability to walk by age 11. DMD progresses to complete […]
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 for a human genetic heart disorder characterized by long delay (long Q-T interval) between heart beats caused by mutations in the Na+ channel α subunit. […]
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, HD is a devastatingly progressive disease that results in death 10–20 years after disease onset and diagnosis. No therapy presently exists for HD; therefore, this […]
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 interaction, and limited and repetitive interests and behavior. The causes and best treatments remain uncertain. One of the major impediments to ASD research is the […]