Award Data

Abstract Tuberculosis (TB), caused by Mycobacterium tuberculosis (Mtb), is the biggest killer among infectious diseases. TB is also responsible for a quarter of all deaths associated with Antimicrobial Resistance (AMR). It is projected that by 2050, 75 million people, or one person every 12 seconds, will die due to AMR associated with TB. Long treatment times and increasing resistance to TB drugs ...

Non-obstructive azoospermia (NOA), a lack of sperm in the ejaculate due to defective spermatogenesis, affects as many as 100,000 men in the US, and represents an unmet medical need because many of these men would like to father children, but cannot without surgical intervention. Current therapy for such men is suboptimal, because it relies upon microsurgical testicular sperm extraction (micro-TESE ...

PROJECT SUMMARY/ABSTRACT This Phase I/II STTR Fast Track proposal responds to the call from the 2018/2019 NCATS SBIR/STTR Research Priorities to develop technologies so that “new treatments and cures for disease can be delivered to patients more quickly”. The production of life-altering gene editing vectors, cancer killing viruses, and life- saving vaccines currently depends on traditional cel ...

ABSTRACTChronic Obstructive Pulmonary Disease (COPD) is the third leading cause of hospitalization in the US. Exacerbations - a worsening or “flare up” of symptoms - cause most COPD hospitalizations. Since most exacerbations can be treated with changes of inhalers and/or oral medications, at-home detection of lung function deterioration may facilitate earlier intervention and help delay or pre ...

Open-angle glaucoma is the second leading cause of blindness worldwide, affecting nearly 70 million individuals. Nonsynonymous mutations in the myocilin gene lead to the most common hereditary form of open-angle glaucoma and account for 3-4% of all cases. Disease-causing mutations, localized to its olfactomedin domain (mOLF), destabilize the myocilin protein, leading to its misfolding and accumula ...

PROJECT SUMMARY There is an increased interest in peptide medicines in pharmaceutical research and development (Randamp;D) because peptides are recognized as highly selective and efficacious, and at the same time relatively safe and well tolerated. Chemo-enzymatic peptide synthesis (CEPS) using peptide ligases features excellent purity and yield, thus becoming an attractive method to replace tradi ...

PROJECT SUMMARY Thiopurines such as mercaptopurine are S-substituted antimetabolites used as functional analogs to natural nucleobase precursors. They are highly effective against hematologic malignancies (leukemia and lymphoma) and autoimmune diseases (inflammatory bowel diseases [IBD]), with an estimated patient population andgt;350,000 in the US. However, the cytotoxicity of thiopurines depends ...

PROJECT SUMMARY The rising number of Americans currently living with Alzheimer’s disease (AD) demands pressing therapeutic and diagnostic solutions. The consensus is that early detection is critical to delay and better manage the disease. The recent discovery of extracellular vesicles (EVs) and their potentially important cellular functions in neuronal-glial communication, synaptic plasticity, o ...

Project Summary Natural products (NPs) and derivatives thereof provide a rich source of chemical matter for drug discovery and development. Over the past 30 years, NP-derived drugs have accounted for more than 35% of FDA approvals. Despite their demonstrated importance, development of new NPs has slowed over the past 15 years due to the lack of efficient methods for discovery, production, and opti ...

Project Abstract An estimated 5.7 million Americans have Alzheimer’s disease or a related dementia (ADRD) and one in ten people age 65 or older show symptoms of Alzheimer’s disease. As ADRD progresses so does the level of dependency on others. As the disease progresses, higher levels of dependence in people with Alzheimer’s disease are associated with significantly increased burden and costs ...