Project Grant R43HL144350

This Project Grant award from the National Center for Advancing Translational Sciences (NCATS) under CFDA 93.350 will provide $463,150 to The Regents of the University of California, San Francisco (UCSF) to establish proof-of-concept for enzyme replacement therapy (ERT) as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome (SPLIS), an ultra-rare and often lethal metabolic disorder. The project aims to demonstrate the efficacy of ERT in preserving kidney function and...

This $815,584 Project Grant award from the National Center for Advancing Translational Sciences (NCATS) CFDA 93.350 Federal Grant Program supports Nostopharma LLC, a small business in Rockville, Maryland, in developing a novel treatment for Fibrodysplasia Ossificans Progressiva (FOP), an ultrarare genetic disease. The objective is to demonstrate the feasibility of a proprietary formulation of the Hedgehog pathway inhibitor arsenic trioxide encapsulated in immunomodulatory nanoparticles for...

This Project Grant award from the National Institute of Allergy and Infectious Diseases (NIAID) under the Allergy and Infectious Diseases Research Federal Grant Program (CFDA 93.855) provides $313,176 to Rarefied Biosciences, Inc. to test MALT1 inhibitors as a treatment for the rare pediatric inborn error of immunity known as CARD11 gain-of-function (GOF). CARD11 GOF is a genetic disorder that causes recurrent infections, severe autoimmunity, and increased lymphoma risk in children, for which...

The National Heart, Lung, and Blood Institute (NHLBI) awarded Novomedix LLC a $306,872 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to develop novel oral small molecule therapeutics for the treatment of progressive pulmonary fibrosis, including idiopathic pulmonary fibrosis (IPF). The goal is to create a safer and more effective therapy compared to the currently approved drugs, which only slow disease progression and have significant side effects. Novomedix will...

This Project Grant award from the National Institute of Allergy and Infectious Diseases (CFDA 93.855 - Allergy and Infectious Diseases Research) provides $284,699 to Aqualung Therapeutics, Corp, a biotechnology company, to optimize the dosage and evaluate the efficacy of their novel ALT-100 monoclonal antibody therapy in preclinical mouse models of lung fibrosis. The goal is to demonstrate the ability of ALT-100 to slow the progression of idiopathic pulmonary fibrosis (IPF), a severe and often...

This Project Grant award of $613,558 from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847 Diabetes, Digestive, and Kidney Diseases Extramural Research) supports Genetobe Inc., a Michigan-based biotechnology company, in developing novel, selective sodium-dependent glucose cotransporter 1 (SGLT1) inhibitor compounds as an adjunctive therapy for cystic fibrosis (CF) associated metabolic disorders, including CF-associated liver disease and CF-related diabetes. The...

This Project Grant award of $230,250 from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865) supports research on reversing disrupted developmental sterol biosynthesis caused by prescription medications. The research aims to find a treatment for Smith-Lemli-Opitz Syndrome (SLOS), a rare genetic disorder that disrupts cholesterol biosynthesis, by reducing toxic 7-DHC levels and counteracting...

This $803,958 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program supports the development of an innovative approach to deliver a more biologically stable form of the enzyme acid β-glucosidase (GCase) through the blood-brain barrier into the central nervous system. The goal is to enable treatment for neuronopathic Gaucher disease, a...

This federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) provides $778,266 to the Oklahoma Medical Research Foundation (OMRF) to conduct comprehensive research on sarcoidosis. The key objectives are to: Distinguish sarcoidosis patients from healthy and other immune-mediated disease controls using high-throughput plasma proteomics on over 2,000 participants. Identify biomarkers to distinguish sarcoidosis patients with...

This Project Grant award from the National Institute on Aging's Aging Research program (CFDA 93.866) provides $2,181,250 in funding to the Seattle Institute For Biomedical And Clinical Research to develop small molecule inhibitors of the MSUT2 protein as a potential therapeutic approach for treating tauopathy disorders such as frontotemporal lobar degeneration, Alzheimer's disease, and related conditions. The project aims to optimize potent and brain-penetrant MSUT2 inhibitors and demonstrate...