Award Data

This grant is for translational development of a scalable, nucleic acid-based formulation of cytomegalovirus-vectored vaccines that can be distributed without a cold chain. HIV candidate vaccines that use cytomegalovirus (CMV) as delivery vector and immunomodulatory adjuvant have shown extraordinary promise. In our preliminary work, for example, a rhesus cytomegalovirus-vectored SIV vaccine lackin ...

RA is one of the most common chronic autoimmune disorders that can lead to complete joint destruction and severe disability if untreated. There is no cure for RA and up to 50% of RA patients do not respond to anti-TNF therapies as circulating Th-17/IL-17 levels are highly elevated subsequent to TNF blockade. For this subset of RA patients, disruption of a novel pathway that impairs the synergy bet ...

Non-obstructive azoospermia (NOA), a lack of sperm in the ejaculate due to defective spermatogenesis, affects as many as 100,000 men in the US, and represents an unmet medical need because many of these men would like to father children, but cannot without surgical intervention. Current therapy for such men is suboptimal, because it relies upon microsurgical testicular sperm extraction (micro-TESE ...

Project Abstract/Summary Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in which the life expectancy of affected children is severely truncated from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration. Treatment of PDCD remains a serious, unmet, challenge. Dichloroacetate (DCA) represents the firs ...

Project Summary Medical imaging technologies such as magnetic resonance imaging (MRI) and positron emission tomography (PET) have been widely used in studying the underlying mechanisms of mental illnesses and neurological disorders such as brain tumors, Alzheimer’s disease, epilepsy, and depression, etc. However, PET and MRI scans are almost always ordered as separate studies on separate machine ...

PROJECT SUMMARY/ABSTRACT This Phase I/II STTR Fast Track proposal responds to the call from the 2018/2019 NCATS SBIR/STTR Research Priorities to develop technologies so that “new treatments and cures for disease can be delivered to patients more quickly”. The production of life-altering gene editing vectors, cancer killing viruses, and life- saving vaccines currently depends on traditional cel ...

Principal Investigators for Small Business: DiscoveryBioMed, Inc. (DBM) and Monell Chemical Senses Center Project Summary Abstract Bitter taste in foods and medicines presents a barrier to overcoming global public health challenges: food insecurity, poor nutritional health, and poor compliance with medication use, particularly among children and the elderly. Sugar and salt, the mainstays to addres ...

Despite important advances in surgical techniques, imagistic modalities and computer-assisted stereotactic delivery of radiation therapy, the prognosis for patients with glioblastoma (GB) remains grim and has not significantly changed in decades. The Stupp protocol—i.e., concurrent temozolomide (TMZ) plus conformal external beam radiotherapy (EBRT) followed by adjuvant TMZ—remains the cornerst ...

Open-angle glaucoma is the second leading cause of blindness worldwide, affecting nearly 70 million individuals. Nonsynonymous mutations in the myocilin gene lead to the most common hereditary form of open-angle glaucoma and account for 3-4% of all cases. Disease-causing mutations, localized to its olfactomedin domain (mOLF), destabilize the myocilin protein, leading to its misfolding and accumula ...

PROJECT SUMMARY There is an increased interest in peptide medicines in pharmaceutical research and development (Randamp;D) because peptides are recognized as highly selective and efficacious, and at the same time relatively safe and well tolerated. Chemo-enzymatic peptide synthesis (CEPS) using peptide ligases features excellent purity and yield, thus becoming an attractive method to replace tradi ...