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Method for immune tolerance induction in patients receiving enzyme replacement therapy for a variety of metabolic disorders
Unmet Need Genetically based metabolic disorders affect 1 in 2500 births globally and many of these disorders result in an enzyme deficiency. In most cases, a single enzyme is not produced by the body at…
Novel Method for Treating Steatosis-Associated Diseases
Unmet Need Steatosis-associated diseases are marked by the infiltration of the liver with fatty acids. Liver steatosis occurs in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), as well as rare diseases such as…
Gene therapy for Crb1 retinopathies
Value Proposition Current treatments for retinal disease include laser surgeries, injectable corticosteroids, and other medications which may slow the process of retinal degeneration but are not able to stop it. The Crb1 protein has been…
Biomarkers and novel targets for metabolic disease leveraging branched-chain amino acid metabolism
Unmet Need 1 in 3 adults in the United States have a form of metabolic dysregulation, including diabetes, insulin resistance, obesity, non-alcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH). While these metabolic diseases range…
The Fit Together Playbook: a curriculum that fosters community partnerships to fight child obesity
Unmet Need Childhood obesity affects 1 in 5 children resulting in more than 14.7 million obese children ages 2-19 years old in the United States. Many factors can contribute and cause obesity including diet, lack…
RXR modulators to improve the efficacy of immunomodulatory agents in multiple myeloma treatment
Unmet Need Multiple myeloma (MM) is the second most common hematological malignancy in the United States. Although current anti-myeloma therapeutics help patients live with the disease for more than 10 years, MM remains an incurable…
Pancreatic cancer treatment that inhibits the Piezo1/TRPV4 axis
Unmet Need Pancreatic cancer has one of the highest mortality rates of all cancer types in the US. The American Cancer Society predicts that about 64,000 people will be diagnosed with pancreatic cancer and 55,000…
A functionally selective ghrelin receptor (GHSR) ligand for treatment of chemical and food addiction
Unmet Need The number of Americans with addiction-related disorders, including substance use disorders (SUD) and food addiction is increasing rapidly each year. Recent estimates indicate that >20 million people in the U.S. have been diagnosed…
The ubiquitination status of the glucagon receptor determines signal bias
Unmet Need Currently, management of diabetes and other metabolic disorders lacks understanding of how the ubiquitination status of the glucagon receptor (GCGR) impacts its signaling pathways. Conventional approaches in targeting G protein-coupled receptors (GPCRs) for…
Gene therapy for non-alcoholic steatohepatitis and associated liver fibrosis
Unmet Need Driven by the rising tide of obesity and lack of medical treatment, it is estimated that 1 out of 4 adults in the US has non-alcoholic fatty liver disease or non-alcoholic steatohepatitis (NAFLD/NASH).…
Dual agonist proteins for the treatment of type 2 diabetes
Unmet Need Over 37 million people in the United States have diabetes, and approximately 90-95% of them have type 2 diabetes (T2D). Diabetes and its related complications not only cause premature mortality and lower quality…
A method to optimize neural activity blocking therapies
Unmet Need The use of kilohertz frequency (KHF) as neuromodulatory therapies that block unwanted neural activity have been applied to treat chronic pain, phantom limb pain, obesity, and other applications. However, the high energy demands…