Cincinnati Children’s investigators have generated several potent non-toxic small molecules that directly bind to a specific Rac activator and inhibit Rac signaling in vitro and in vivo.

Tyrosine phospates (PTPs) are up-regulated in cancers, and its inhibition has emerged as a cancer therapy but with limited success due to its ubiquitous presence.

Systemic Lupus Erythematosus (SLE) is one of more than 80 disease states that evolve from immune system dysregulation.

While there are many successes in protease inhibitor drug development including antivirals, anti-cancer and others in preclinical stages, most developments have failed due to weak PK and oral bioavailability of the inhibitors.

NleB1 is an important virulence factor expressed by multiple bacteria including enterohemorrhagic Escherichia coli (EHEC), enteropathogenic E. coli (EPEC), and Salmonella enterica. These bacteria are of significance to human health as they all cause infectious diarrhea.

Mayo Clinic researchers have developed a fibrin-based hydrogel that can support an induced pluripotent stem cell (iPSC) derived retinal pigment epithelial (RPE) monolayer during differentiation but can be degraded in a matter of hours “on demand”.

Mayo Clinic researchers have developed a small molecule inhibitor of Cyclophilin B (CypB) and PIKfyve kinase for use in treating a variety of cancers, including lymphoblastic leukemia and lymphoma.

Mayo Clinic researchers have developed a novel technology to define new repertoires of tumor antigen(s) which work alone or in combination to generate anti-tumor immune responses.

Traditional CAR T therapy requires specialized production facilities to isolate, activate, modify, and to expand in vitro the engineered cells before transfusion back into patients. 

Idiopathic pulmonary fibrosis is a rare fatal disease with no cure. The mechanism of this disease is unknown.

The Mps1 kinase is critical for centrosome duplication and proper function of the spindle assembly checkpoint (SAC).

The activation of ER-Beta signaling may be an effective strategy for treating multiple precancerous and cancerous conditions.

Recent proof of concept research has re-kindled interest in targeting cancers through inhibition of dihydroorotate dehydrogenase (DHODH).

Vancomycin-resistant enterococcus (VRE) is a leading cause of hospital-acquired infections in the U.S.

Researchers at Purdue University have identified compounds with potent antibacterial activities.

Fatty acids are essential components of overall cellular metabolism.

A University of Colorado research group led by Dr. Paco Herson has generated peptides and peptide constructs that are fused to a cell-permeable sequence, and specifically inhibits TRPM2 channel activity.

Founded by a fetal surgeon who has studied the mechanisms leading to scar-free healing in the fetus for 20+ years, Ceria Therapeutics is dedicated to applying those insights to improve healing in children and adults.

Allander Biotechnologies, LLC is a seed-stage company developing fusion protein biologics to treat inflammatory diseases and disorders with unmet medical needs including oral mucositis (extensive oral ulceration), radio-dermatitis, and chronic wounds.

CartilaGen’s technology is an intra-articular injection of drug suspended in a hydrogel vehicle.

Lymphoma immunotherapy can result in durable immune and clinical responses.

Diabetes mellitus (DM) impairs the healing of fractures and there is a need for improved therapeutics to stimulate bone regeneration in diabetic patients.

Novel KOR antagonists can be developed as a therapeutic treatment for mental conditions such as depression and anxiety related disorders.

An extracted form of purified crocetinic acid from saffron has demonstrated significant anti-tumor activity against pancreatic cancer cells in a mouse model with low toxicity.

The development of Ewing Sarcoma therapeutics revolves around the natural product anticancer agent mithramycin (MTM), which was recently shown to be a potent inhibitor of Ewing sarcoma cells in culture and mice, by disrupting the activity of the aberrant transcription factor EWS-FLI1.

A tumor suppressor protein, Par-4 (Prostate apoptosis response-4), was first identified in 1994. This protein induces apoptosis in cancer cells but not in normal cells, by functioning intracellularly and extracellularly.

A new family of fast-acting and quickly reversible boron based neuromuscular blockers can enable tracheal intubation and maintain muscle paralysis during surgery.

Novel inhibitors of nicotinamide phosphoribosyltransferase (NAMPT) can treat cancer, and also has potential in other NAMPT-related conditions like Alzheimer’s disease, diabetes and arthritis.

The loss of a single gene causes spinal muscular atrophy, a devastating disease that is among the leading genetic causes of infant death.

A class of novel small molecule compounds to attenuate cell and tissue damage due to diabetes was developed at UMKC, and preclinical data shows highly significant reversal of hyperglycemia mediated cell and tissue damage in late-stage diabetes.

Immunotherapy using checkpoint inhibitors, especially PD-1/PD-L1 inhibitors, has now evolved into the most promising therapy for cancer patients.

Prostate-specific membrane antigen (PSMA) is the most validated marker for prostate cancer.

New drugs help treat multiple myeloma and other types of cancer by targeting a protein called geranylgeranyl diphosphate synthase, also known as GGDPS.

Shannon Pharmaceuticals, Inc. is an Omaha, Nebraska, start-up company focusing on the development of therapeutic interventions for nephrotic diseases.

A UNMC chemist has developed new compounds for the treatment of inflammatory diseases.

Bacteriocins, antimicrobial peptides produced by certain bacteria, exhibit significant potency against other bacteria, including antibiotic-resistant strains.

Certus Therapeutics provide a novel nanoparticle drug delivery platform (Lypos) for cancer chemotherapies with enhanced tumor-targeting properties.

Novel cardioprotective vasodilator peptides with enhanced affinity for AM and CGRP receptors have been developed.

SHetA2 is a novel, orally bioavailable, small molecule drug with low toxicity being developed for clinical trials based on its induction of apoptosis in cancer cells.

A high-throughput drug screen followed by biochemical analysis showed Veratridine (VTD), a non-steroidal alkaloid derived from the Liliaceae plant, induces a potent anti-growth and pro-apoptotic effects in colon cancer cells.

Researchers at the University of Toledo have developed pancreatic beta-cell-specific chimeric antigen receptor (CAR) Tregs.

Researchers at the University of Toledo have designed and synthesized novel furoxans. These compounds are orally bioavailable in the brain and may be capable of enhancing in vivo cognition.