Award Data

PROJECT SUMMARY There is an urgent need for the development of new approaches to treat patients suffering from Ulcerative Colitis (UC), which is estimated to impact approximately one million adults in the United States, leading to morbidity, heightened risk of cancer, and mortality. In multiple preclinical studies, we and others have defined the therapeutic potential of low-dose exogenous carbon m ...

AbstractImpaired mitochondrial respiration plays a key role in metabolic, cardiovascular, and aging-related diseases. However, mitochondrial respirometry analysis currently requires processing of the living tissue sample within an hour after being taken from the patient. This requirement makes respirometry analysis largely unfeasible to standard clinical practice and clinical studies. We invented ...

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 ...

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 ...

ABSTRACTUp to 30% of low birth weight preterm infants manifest some form of periventricular white matter injury (PWMI) making it the most common form of brain injury affecting premature infants. PWMI is associated with significant morbidity, as affected individuals may have profound intellectual impairment and cerebral palsy. Highlighting the magnitude of PWMI, each year more than 400,000 infants ...

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 SUMMARY Neonatal abstinence syndrome (NAS) is an opioid withdrawal syndrome that develops shortly after birth to in utero-exposed neonates. The cost of NAS is high: newborns with NAS are typically receive care in the Neonatal Intensive Care Unit (NICU), where the daily cost of care is high. Nearly 22,000 infants are born with NAS each year at a cost of $1.5 billion1. Moreover, medication-b ...

SUMMARYIn 2014 the United States experienced an outbreak of a previously unknown neurological disease with polio-like symptoms later known as acute flaccid myelitis (AFM). A biennial surge in cases of AFM in 2016 and 2018 has alarmed public health officials. Since then, rapidly accumulating clinical, immunological, and epidemiological evidence has pointed to EV-D68 as the major causative agent of ...

Project Abstract/Summary Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in which the life expectancy of affected children is severely truncated from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration. Treatment of PDCD remains a serious, unmet, challenge. Dichloroacetate (DCA) represents the firs ...

Lyme disease (LD) is the most prevalent vector-borne disease in the United States, with up to 300,000 cases a year. LD is caused by several species of the spirochete bacteria Borrelia burgdorferi sensu lato (the Lyme borreliae), which are transmitted by ticks from animal (bird, rodent) reservoirs to human hosts. While a short course of antibiotics is usually effective in eliminating the bacteria a ...