Award Data

SummaryThis Phase I program will develop TempO Splicea highly multiplexed targeted sequencing assay monitoring RNA Binding ProteinRBPsplicing expression and alternatively splicedASmRNA representatives of coregulated splicing modulesa novel variation of the TempO Seqassay used for high throughput screeningdesigned to address the hypothesis that it is possible to provide a surrogate assay of the who ...

DESCRIPTION (provided by applicant): Congenital heart diseases (CHD) are the most common serious congenital anomalies and the leading cause of death due to birth defects. Clinical practice relying on physical examination of the newborn before discharge from the nursery often misses newborns with critical CHD (CCHD), which can lead to death or long-term morbidities. Using pulse oximetry (PO) can im ...

DESCRIPTION (provided by applicant): The liver is the site of many metabolic processes, including metabolism of xenobiotics such as pharmaceutical compounds. Drug metabolism is highly species specific and can vary significantly between individuals of the same species. To date no reliable experimental system capable of predicting the human-specific metabolic conversion of candidate small molecules ...

DESCRIPTION provided by applicant Peptide conjugated phosphorodiamidate morpholino oligomers PPMO are single stranded nucleic acid analogs able to modulate gene expression through steric blocking of complementary RNA PPMO are composed of two components an antisense morpholino oligomer cargo covalently conjugated to a cell penetrating delivery peptide PPMO are completely nuclease resistant ...

DESCRIPTION (provided by applicant): Activation of hepatic stellate cells (HSC) is responsible for the development of liver fibrosis in chronic liver diseases of all causes and remarkably, HSC clearance by apoptosis may allow recovery from liver injury andreversal of liver fibrosis. There is full agreement among liver fibrosis experts that inhibiting o reversing HS activation (the therapeutic targ ...

DESCRIPTION (provided by applicant): The proposed project is a Phase I STTR collaboration between Vala Sciences Inc, a small business that specializes in the development of cell-based high content/high throughput assays, and the Sanford Burnham Medical Research Institute, a worldwide leader in biomedical research. The project will develop an assay (Kinetic Image Cytometer-Neuron, or KIC-Neuron) to ...

DESCRIPTION provided by applicant Glioblastoma multiforme GBM the most common and malignant of all gliomas has a median survival time of months Standard of care chemotherapy using temozolomide TMZ is effective initially but the GBM inevitably recurs and these recurrent tumors are resistant to TMZ There are currently no effective treatment options for patients with TMZ resistant ...

Project Summary Linking infectious agent diagnostics to clinical decision making at the point of care requires a fast highly sensitive and simple to use method that takes into account the infrastructure and workflow in clinical settings of both developing and developed regions In addition being able to distinguish among multiple potential infectious pathogens in a single test is of great impor ...

Antiretroviral therapy has become a reality in resource limited settings thanks to entities such as PEFAR and the Global Fund However contrary to the therapeutic choice flexibility in developed countries all patients go on reverse transcriptase inhibitor RTI based first line regimens with more expensive protease and integrase inhibitor based regimens being reserved as second line therapy for ...

PROJECT SUMMARY More thanpatients are diagnosed each year with glioblastomathe most common primary brain tumorCurrent treatments are inadequate and there have been no major therapeutic breakthroughs in decadesHencethere is an urgent need to develop novel strategies to address this devastating diseaseHoweverdevelopment of an effective treatment for glioblastoma is severely hampered bythe blood brai ...