In humans, there are two nearly identical copies of the Survival Motor Neuron (SMN) gene, SMN1 and SMN2.  Loss of SMN1 in conjunction with skipping of SMN2 exon 7 in pre-mRNA splicing—which results in a truncated, unstable SMN—leads to spinal muscular atrophy. 

Metformin, a naturally-occurring biguanide, is an FDA-approved high-safety profile diabetes drug that has been used extensively for decades.

The fyn kinase inhibitor, AZD0503 has demonstrated excellent clinical tolerability and oral bioavailability in humans.

Therapeutic peptides to combat excessive inflammation and tissue damage in response to ischemia/reperfusion (IR).

Researchers at Kansas State University have identified two synthetic compounds that show
strong activity against various species of C. albicans including C. krusei, C. glabarata and azole
resistant clinical isolates of C. albicans, Aspergillus nidulans, Cryptococcus spp and
dermatophytes.

Kansas State University researchers, in collaboration with researchers at University of Alabama-Birmingham Medical Center, have discovered novel compounds that are highly effective against MRSA, Methicillin-resistant Staphylococcus aureus,  and other gram-positive strains.

An effective method of treatment for chronic and inflammatory pain by inhibiting selective adenylyl cyclases, especially adenylyl cyclase 1. Compounds may also prevent and/or reduce opioid dependence.

Fragment-based drug discovery is a popular method used to design molecules that bind to protein targets. In this method, many small compounds are screened for their binding to a target protein.

The United States has about 220,000 cases a year of acute respiratory distress syndrome, or ARDS, which deactivates the naturally occurring lung surfactant in patients. That causes a decreased ability to inflate the tiny air sacs in lungs called alveoli. When inadequately inflated, the alveoli cannot totally oxygenate a patient’s blood stream, which can lead to organ failure and other health problems.

Nearly 40 percent of active pharmaceutical ingredients under development are poorly soluble in aqueous media leading to low bioavailability and erratic absorption. In an attempt to solve this issue, researchers began to develop nanocrystals of these insoluble drugs.

InnoBioPharma has developed several novel drug candidates (two in pre-clinical trials) that are particularly effective against aggressive cancers that lack effective drug therapy.

A therapeutic vaccine for patients with recurrent prostate cancer that effectively destroys expressing tumor cells while leaving normal cells unharmed.

Viewpoint Molecular Targeting, Inc. develops radiopharmaceuticals for cancer therapy and diagnostic agents.

Orion BioScience is a preclinical stage biotechnology company focused on developing innovative therapies to intercept disease and prevent development of symptoms in autoimmune conditions such as type-1 diabetes and neuromyelitis optica.

Antibody-drug conjugates are an increasingly important class of cancer therapeutics.

PVX is a broad spectrum antiviral therapeutics that could have profound implications on animal health, and subsequently on human health.

Combination of Proton Pump Inhibitors (PPIs) Omeprazole and Lansoprazole in a buffered composition for 24-hour gastric pH control to treat a variety of acid-related disorders such as Barrett’s Esophagus, Gastroesophageal Reflux Disease, and others. 

Combination treatment of Ig-GAD2 and stem cells to cure Type I Diabetes (TID).  Data demonstrate a functional cure in a mouse model for TID through restoration of immune tolerance coupled with regeneration of endothelial cells in the pancreatic islets. Patents issued and pending.

Cell-specific non-toxic reagent for the treatment of hematological and other cancers. Data using micelles composed of peptide amphiphiles targeted using aptamers demonstrate efficacy against a wide range of human and canine cancers, as well as low toxicity in vivo, and 2-3x longer median survival.  Patents issued and pending.

Sphingosine 1-phospate metabolizing enzymes are involved in modulating cellular responses to a variety of viruses in human and veterinary applications. 

A proprietary high-throughput screening method to identify and characterize in-cell based assays compounds that inhibit FATP2 mediated fatty acid uptake.

A substrate mediated delivery system for highly controlled local and sustained release of any hydrophobic or hydrophilic cargo.

University of Nebraska-Lincoln researchers are developing a novel protein based delivery technology that targets sensory neurons to stop pain signals at the source to avoid systemic toxicity and addiction potential.

A molecule capable of specifically inhibiting the NF-κB pathway, a key target involved in a variety of cancers and other diseases.

The Mps1 kinase is critical for centrosome duplication and proper function of the spindle assembly checkpoint (SAC). Its overexpression is associated with poor outcomes in triple-negative breast cancer and other solid tumors. 

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

Titanium dioxide nanoparticles are doped or co-doped with elements such as nitrogen to produce novel multifunctional nanoparticles that are capable of addressing the current need for biomaterials that have non-leaching and long-lasting antibacterial properties.

OU researchers have identified a novel, selective PPAR-alpha agonist with potential therapeutic effect on diabetic retinopathy and age-related macular degeneration. 

Millions are affected by the influenza virus each year. Although vaccines are available, they must be updated on a yearly basis to match the circulating viruses; these updates are based on what epidemiologists predict will be circulating.

A biocompatible and biodegradable delivery vehicle decorated with anti-CD38 monoclonal antibodies for the treatment of MM.

The TR3 molecule is a unique bioactive trimer of TNF superfamily TRAIL (TNF-related apoptosis-inducing ligand, designated TR3) that has been developed with a highly targeted affinity for cancer cells. This unique fused TR3 overcomes historical challenges faced by TRAIL therapies.

Viosera targets under-served deadly infectious disease markets. We use existing FDA-approved and off-patent drugs and combine them in ways that enhance their effectiveness against the deadliest infections.

Accuronix Therapeutics is a leader in discovering and developing a new class of drugs that work by selectively targeting the σ-2 (sigma-2) receptor on cancer cells to deliver cytotoxic payloads.

Each year, in the United State alone, 160,000 people develop heart failure from acute heart damage. This results in more than $17 billion dollars in direct healthcare costs and more than 80,000 deaths within five years from heart damage.