Award Data

PROJECT SUMMARY Congenital Myasthenic Syndromes (CMS) are a group of clinically similar neuromuscular transmission disorders that differ in their underlying genetic mutation. While some phenotypical variation exists, CMS are commonly characterized by muscle weakness (myasthenia) that worsens with physical exertion. Defects in Collagen Q (ColQ), a multidomain functional protein of the Neuro Muscula ...

For more than a decade, our work has focused on development of therapeutic interventions for viral- and bacterial-induced acute lung injury (ALI) and the more severe acute respiratory distress syndrome (ARDS). Significantly, we identified the Toll-like receptor 4 (TLR4) signaling pathway as key to the host response to influenza and secondary bacterial infection following influenza. We also identif ...

Project Summary There are 24,000 stillbirths per year in the United States, and 2.6 million stillbirths per year worldwide, with no significant decrease in the last decade. Abnormal fetal heart rhythms, such as long QT syndrome, are thought to play an important role in these deaths, and may cause 3- 10% of unexplained stillbirths. This is in addition to known fetal arrhythmias, which affect 1-3% o ...

Project Summary/Abstract for STTR Phase I Application: An immunotherapeutic IgY formulation against norovirus diarrhea Human norovirus (HuNoV) infects millions of people globally each year, causing diarrheal illness resulting in more than 200,000 deaths annually, particularly in the very young, the very old, and the immunocompromised. There is currently neither an effective vaccine nor effective t ...

Summary Lung diseases, such as interstitial lung diseases including idiopathic pulmonary fibrosis (IPF), chronic obstructive pulmonary disease (COPD), and acute viral infection are major leading causes of death worldwide. The recent increase due to air pollution, tobacco smoking is and use of E-cigarettes has grown rapidly in the US contributing to a sharp increase in chronic lung diseases. IPF is ...

ABSTRACTGlobally, more than 71 million people are infected with Hepatitis C virus (HCV), with 1-2 million new infections occurring each year. This major health concern necessitates the development of an effective vaccine. Since HCV rapidly accumulates mutations, vaccines must elicit the production of broadly neutralizing antibodies (bnAbs) in a reproducible fashion. The viral envelope E1E2 glycopr ...

PROJECT SUMMARY Microinjection technologies underlie many research and clinical applications that require gene and cell delivery, including studies and emerging treatments of neurological conditions (e.g., neurodegenerative diseases, traumatic brain injury, and cancer). Unfortunately, challenges associated with the microinjection tools by which the viral vectors and cells inherent in these applica ...

Abstract Nytricx is developing a new class of Nitric Oxide (NO)-releasing intravascular medical catheters to combat infection and thrombosis associated with indwelling vascular catheters. NO gas is a natural anti-infective and anti-thrombotic human product principally released from blood vessels and immune cells. Nytricx NO-releasing technology to the catheter surfaces via a polymer matrix, mimics ...

The following contains proprietary/privileged information that Moonlight Therapeutics requests not be released to persons outside the government, except for purposes of review and evaluation Safety and Tolerability of TASIS-Peanut (Targeted Allergen Specific Immunotherapy within theSkin) patch for the Treatment of Peanut Allergy Food allergies affect 30 million people in the USA and 17 million in ...

Modern pharmaceuticals have greatly improved the quality of life for many patients suffering a variety of illnesses. Unfortunately, among the 17 most commonly prescribed drugs, only 6 are highly effective for most patients, 5 have a modest effect, and 6 show little clinical effect. Worse still, unwanted illness and death from treatment of patients with the incorrect drug or dosage causes over 275, ...