P2Y14 Receptor Antagonists Containing A Biaryl Core

The technology discloses composition of compounds that fully antagonize the human P2Y14 receptor, with moderate affinity with insignificant antagonism of other P2Y receptors. Therefore, they are highly selective P2Y14 receptor antagonists. Even though there is no P2Y14 receptor modulators in clinical use currently, selective P2Y14 receptor antagonists are sought as potential therapeutic treatments for asthma, cystic fibrosis, inflammation and possibly diabetes and neurodegeneration.

Reducing Bloodstream Neutrophils as a Treatment for Lung Infection and Inflammation

During lung infection, bloodstream neutrophils (PMNs) responding to infection travel to the airspace lumen. Although successful arrival of microbicidal PMNs to the airspace is essential for host defense against inhaled pathogens, excessive accumulation of PMNs in the lung contributes to the pathogenesis of several prevalent lung disorders, including acute lung injury, bronchiectasis, and COPD. Unfortunately, there is no treatment for controlling PMN accumulation in the lung.

Novel Activators of Pyruvate Kinase for the Treatment of Hemolytic Anemias

This technology includes the development and use of small molecule activators of pyruvate kinase (PK) for the treatment of inherited nonspherocytic hemolytic anemia, including PK deficiency. PK deficiency is caused by an inherited deficiency in an enzyme that reduces the lifespan of red blood cells. More than 150 unique mutations have been identified in the PK gene that lead to decreased activity in this essential enzyme in the glycolytic pathway. The prematurely lysed red blood cells can lead to jaundice, splenomegaly, and a hemolytic anemia.

Vectors for the Treatment of Sickle Cell Disease and Beta Thalassemia

This technology includes lentivirus vectors to be used to treat sickle cell disease and beta thalassemia. (i) Lin28A or Lin28B vectors designed for erythroid-specific expression using EKLF1, SPTA1, or similar erythroid-specific regulatory elements will be used to transduce hematopoietic stem cells isolated from humans with sickle cell disease or beta-thalassemia syndromes.

Live Attenuated Vaccine to Prevent Disease Caused by West Nile Virus

West Nile virus (WNV) has recently emerged in the U.S. and is considered a significant emerging disease that has embedded itself over a considerable region of the U.S. WNV infections have been recorded in humans as well as in different animals. From 1999-2014, WNV killed 1,765 people in the U.S. and caused severe disease in more than 41,762 others. This project is part of NIAID's comprehensive emerging infectious disease program.

Diagnostics and Therapeutics for Hydrocephalus

Congenital hydrocephalus is a significant public health problem, affecting approximately one in 500 live births in the United States. Congenital hydrocephalus has an adverse effect on developing brain and may persist as neurological defects in children and adults. Some of these defects may manifest as mental retardation, cerebral palsy, epilepsy and visual disabilities. Improved diagnostics are needed for assessing the risks of developing this debilitating disease.

Oral Treatment of Hemophilia

This invention portrays a simple method for treatment of antigen-deficiency diseases by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome. This method increases hemostasis in a subject having hemophilia A or B, by orally administering to the hemophiliac a therapeutically effective amount of the appropriate clotting factor, sufficient to induce oral tolerance and supply exogenous clotting factor to the subject.

Enhanced GFP-Expressing Human Metapneumovirus (HMPV): A Versatile Tool for Virology Research and Antiviral Drug Screening

The technology involves genetically engineering Human Metapneumovirus (HMPV) to express enhanced green fluorescent protein (GFP), enabling the monitoring of virus infection and gene expression through GFP fluorescence. This system serves as a sensitive and versatile tool for virology research, antiviral drug screening, and diagnostic applications.

Advanced Human Cell Line Technology for RSV Replication Complex Production and Antiviral Drug Discovery

This technology includes the NeurEx® mobile application, a groundbreaking tool designed for neurologists to conduct and document neurological examinations efficiently. Deployed on iPads, it integrates with a secure, cloud-based database, automating the computation of four key disability scales used in neuroimmunology. The app's robust design enables precise mapping of neurological deficits, blending spatial distribution with quantitative assessments.

Stable Cell Line Technology for Enhanced Production of Varicella-Zoster Virus Vaccines

This technology includes a stable cell line engineered to have reduced expression of the pro-apoptotic protein Bim when exposed to doxycycline. This reduction in Bim expression allows for significantly higher yields of the Varicella-Zoster Virus (VZV), which is used in the production of live, attenuated VZV vaccines. The enhanced viral production makes this cell line particularly useful for vaccine manufacturing.

Subscribe to Pre-Clinical (in vitro)