4. Mentesana PE, et al. Characterization of halted T7 RNA polymerase elongation complexes reveals multiple factors that contribute to stability. J Mol Biol. 2000
Intellectual Property: HHS Reference No. E-119-2013/0--US Provisional Patent Application No. 61/843,864 filed
Blood-Based Assay for the Diagnosis and Monitoring of Hyposialylation Disorders
Description of Technology: Sialic acid, a monosaccharide widely distributed in glycoproteins and glycolipids, plays an important role in biological processes such as cellular adhesion, cellular communication and signal transduction. Reduced levels of sialic acid in tissues (also known as hyposialylation) affect the function of muscle, kidney, and other organ systems, and are found in a number of disorders, such as hereditary inclusion body myopathy (HIBM, also known as GNE myopathy), renal hyposialylation disorders, and congenital disorders of glycosylation.
The inventors have developed a sensitive, reliable assay for the diagnosis of hyposialylation disorders that detects a novel glycoprotein biomarker in a patient blood sample. This assay has been validated using samples from patients with GNE myopathy and other hyposialylation disorders. A distinct advantage of this assay is that it is minimally invasive, unlike many currently-available methods for diagnosing hyposialylation disorders, which typically require a tissue biopsy. In particular, this biomarker represents the first non-invasive method for diagnosis of renal hyposialylation.
Potential Commercial Applications:
* Diagnostic assay to detect hyposialylation
* Monitoring tool to track patient response to sialylation-increasing therapy
Competitive Advantages: A blood-based assay based on this technology would be less invasive, time-consuming, and costly than a tissue biopsy, which is the current diagnostic standard for hyposialylation disorders, particularly kidney disorders.
* In vitro data available
Intellectual Property: HHS Reference No. E-056-2013/0--U.S. Application No. 61/785,094 filed
* HHS Reference No. E-217-2007/0--N-Acetyl Mannosamine as a Therapeutic Agent
* HHS Reference No. E-270-2011/0--Encapsulated N-Acetylmannosamine or N-Acetylneuraminic Acid to Increase Sialylation
Vaccine Adjuvant for Inducing Th17 Focused Response
Description of Technology: Adjuvant selection can be critical to a vaccine's effectiveness. Ideally, an adjuvant will target and activate specific immune pathways to increase the magnitude of a response to the vaccine. A limited range of adjuvants are presently available for human clinical use; these primarily affect T helper cells 1 and 2 (Th1 and Th2). Currently, no adjuvants are approved for human use which primarily affect IL-17-producing T helper cells (Th17) cells. Th17 focused adjuvants may prove critical for developing operative vaccines against pathogens where Th17 activity is essential for protection. This technology relates to novel adjuvants activating either caspase-associated recruitment domain protein 9 (CARD9) or caspase 1 pathways, or a combination of the two; and methods for using these adjuvants for stimulating an immune response. These adjuvants induce Th17 focused stimulation, which may prove essential to development of effective vaccines against a range of pathogens including bacteria and fungi.
Potential Commercial Applications: Vaccine
Competitive Advantages: Th17 skewing adjuvant
Development Stage: Early-stage
Publication: Shenderov K, et al. Cord factor and peptidoglycan recapitulate the Th17-promoting adjuvant activity of mycobacteria through mincle/CARD9 signaling and the inflammasome. J Immunol. 2013
Intellectual Property: HHS Reference No. E-089-2012/0--U.S. Provisional Patent Application No. 61/709,713 filed
Collaborative Research Opportunity:
[FR Doc. 2013-20888 Filed 8-26-13;
BILLING CODE 4140-01-P
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