Gaucher disease (GD) is an inherited disorder where the body does not make enough glucocerebrosidase (GCase), an enzyme that breaks down fatty chemicals in the body.

Many pathogenic cell states (e.g., tumor cells or activated immune cells) depend on anti-apoptotic proteins for their survival, which makes these proteins effective therapeutic targets, as evidenced by the success in pharmacologic targeting of Bcl-2.

A critical component of normal esophageal function is a protective epithelial barrier that prevents damage to the underlying tissue due to outside environment exposure such as foreign antigens and microorganisms.

To avoid antibody-dependent enhancement (ADE), researches at Colorado State University in collaboration with North Carolina State University have developed an oral vaccine targeting the COVID-19 virus-host cell interaction which depends on protease cleavage of viral spike proteins. Preventing cleavage has been shown to reduce virus infectivity by preventing exposure of viral fusion peptides.

Researchers at Colorado State University have developed a vaccine for COVID-19 produced through a novel method (SolaVAX™) for inactivation of a whole virion particle. The vaccine has been tested in a sensitive hamster animal model for its ability to prevent infection upon challenge with SARS-CoV-2 virus and has been demonstrated to be effective in providing protection against COVID-19 disease.

Researchers at Kansas State University have developed a structure-based multiepitope fusion antigen (MEFA) technology for vaccines against Enterotoxigenic Escherichia coli (ETEC) bacteria.

Mayo Clinic researchers have developed a small molecule modulator for use in treating and preventing a variety of Wnt-related conditions such as Alzheimer’s Disease (AD) and other neurodegenerative disorders, traumatic and ischemic brain injury, as well as bone loss disorders.

Mayo Clinic researchers have developed a novel regimen by which oncolytic virotherapy can be used as a potent immunological adjuvant to improve the efficacy of CAR T cell therapy against solid tumors.

The TTK kinase is critical for proper function of the spindle assembly checkpoint, and its upregulation is associated with poor clinical outcomes in a variety of cancer types.

The estrogen receptors, alpha and beta, are both responsive to endogenous estrogen, but modulate different biological processes.

Dihydroorotate dehydrogenase (DHODH) is a key enzyme in the de novo pyrimidine synthesis pathway.

Researchers at Purdue University developed antimicrobial agents against intracellular human pathogens with improved efficacy compared to the group’s previous-generation compounds.

Researchers at Purdue University have developed a targeted therapeutic option for treating metastatic prostate cancer, the second leading cause of cancer death for men in the United States.

Researchers at Purdue University have developed an immunotherapy approach combining cell therapy and a small molecule inhibitor to treat glioblastoma.

Using C. elegans as an animal model to study radiation-induced bystander effects (RIBE), researchers at the University of Colorado Boulder have identified a highly conserved cysteine protease, cathepsin B, as the first RIBE factor.

The researchers of the University of Colorado developed a series of hydroxamic acid derivatives as HDAC6 selective inhibitors.

Inventors at the University of Iowa have developed a respiratory syncytial virus vaccine. It is a nanovaccine consisting of viral proteins encapsulated by biodegradable polyanhydride polymers.

Endometrial cancer is the most frequent gynecological cancer and is often treated using platinum-based therapies such as cisplatin.

Post-traumatic osteoarthritis weakens the regulation of lubricin gene and increases shear stress within the joints.

It has been proposed that prostaglandin E2 (PGE2), known as the principal proinflammatory prostaglandin, is implicated in pain response and also plays a role in inflammatory diseases.

Novel neuroprotective and restorative peptides that possess unique mitochondrial bioactivity offer the promise of restoring function and preventing further damage caused by neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease.

SerpinB13 is a protease inhibitor linked to autoimmune inflammation and Type 1 diabetes (T1D).

The search for better analgesics has focused on non-addictive alternatives. Agmatine, the decarboxylated product of arginine, is a potential candidate for novel therapeutics.

A dual diagnostic and therapeutic compound binds GRPr and PSMA with high specificity and sensitivity.

Novel dual- and tri-AAV hybrid vector systems increase the upper size limit of gene constructs from <5kb to <15kb.

Researchers at the University of Missouri have developed novel inhibitors of beta-lactamase, a bacterial enzyme that provides resistance to β-lactam antibiotics.

ProGel is a platform technology with a diverse spectrum of drug payloads, such as steroids, statins, antioxidants, opioids, chemotherapeutics, and hormones.  Ensign Pharmaceutical’s initial product focuses on managing osteoarthritis pain, incorporating a potent steroid, dexamethasone, into the formulation.

Researchers at the University of Oklahoma have discovered a class of antitoxin peptides that inhibit a critical clostridium difficile virulence factor.

Persephacin, a novel fungal isolate, strongly inhibits a broad range of pathogenic fungi while exhibiting low cytotoxicity to mammalian cells.

Bioactive synthetic peptides derived from neutrophil proteins, cathepsin G and CAP37, can inhibit and reverse the formation of toxic amyloid beta aggregates.

Microparticles based on hydroxyapatite, calcium carbonate, or calcium citrate and cross-linked gels loaded with a drug such as eugenol or arginine are able to occlude dentinal tubules and release on-demand a cargo drug to the oral cavity and to the pulp of the tooth, providing both desensitizing, antibacterial or any other effect depending on the drug cargo.

A new hybrid nanocapsule uses a silica, metal and organic framework designed to deliver protein, nucleic acid or hydrophilic drugs.

A new formulation of paclitaxel fuses the drug to an oligo-lactic acid (oLA) encapsulated in a PEG-PLA micelle.

This technology uses 2-deoxy-D-glucose (2DG) as a novel treatment for Status Epilepticus (SE).