Award Data

Abstract: The objective of this project is to perform the required (and previously approved) IND-enabling experimental work for E1v1.11, a novel drug candidate for Spinal Muscular Atrophy (SMA). SMA is an autosomal recessive disorder that is the leading genetic cause of infantile death worldwide, occurring in ~1:10,000 live births. The gene responsible for SMA is called survival motor neuron-1 (SM ...

Project Summary: Cancer is a leading cause of death both in the United States and across the world, claiming over 600,000 and 8 million deaths each year, respectively. Delayed detection of cancer progression or recurrence after treatment contributes to increased morbidity and rapid death, but re-biopsy or repeat surgery is usually not possible due to severe risks. The accuracy of conventional imag ...

PROJECT SUMMARY Acute respiratory distress syndrome (ARDS) is a severe life-threatening complication that can be caused by viral infection. The disease is characterized by cytokine-driven hyperinflammation and leukocytes count changes. The innate immune response has been linked to ARDS immunopathogenesis. In this regard, neutrophils have been highlighted as essential effector cells in the developm ...

Development of a PCR assay for quantitative detection of HBV cccDNA There is an urgent and unmet need for an effective method to detect hepatitis B virus (HBV) cccDNA in the blood. HBV infection affects nearly 350 million people worldwide. Chronic HBV infection is a major risk factor for the development of severe liver diseases such as cirrhosis and hepatocellular carcinoma, which has a poor survi ...

PROJECT SUMMARY/ABSTRACT The mission of Antiger Therapeutics Inc. is to develop novel immunotherapies for transplant recipients to improve long-term graft outcomes and minimize adverse effects. Antibody-mediated rejection (AMR) is a leading cause of graft loss, with over 3000 new cases per year in the United States and estimated market size of $90 million. However, this estimate is likely to under ...

Research Summary/Abstract ErythroMer (EM) is a novel biosynthetic blood substitute developed to address the critically unmet need for emergency transfusion in situations where the use of banked red blood cells (RBCs) are either not available or undesirable. EM is a self-assembled lipid-oligomeric hybrid nanoparticle with a high per particle payload of hemoglobin (Hb) and the allosteric modifier, R ...

ABSTRACT The identification and removal of process-related protein impurities (PRPI) from biologic products is a critical step in drug development. Despite recent improvements in the purification and processing of biologics, the presence of immunogenic PRPI continue to raise concerns about drug safety and efficacy. We propose an innovative approach for assessing immunogenicity risk of PRPI using o ...

Abstract. Chagas disease is a neglected tropical disease and has been designated as a research priority by NIAID and an SBIR Research Topic of Interest. Six million individuals are infected and 8,000 deaths were caused by Chagas in 2015 in mostly Central and South America by advanced forms of the disease such as Chagas hemorrhagic fever. The cost associated with Chagas disease treatment globally i ...

Few interventions have proven to be successful in promoting medication adherence and impacting short- and long-term posttransplant outcomes in adolescent heart transplant (HT) recipients. Improving adherence is a persistent challenge with youth experiencing chronic health conditions, especially among adolescent transplant recipients. Adolescent organ transplant recipients experience unique challen ...

ABSTRACT In 2020 malaria caused 241M clinical cases and 627,000 deaths, the greatest numbers of annual deaths since 2012. There were more deaths in Africa from malaria than from COVID-19. There is an urgent unmet medical need for a malaria vaccine that prevents infection and disease in individuals and can be deployed in mass vaccination programs for malaria elimination. The RTS,S malaria vaccine w ...