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.

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.

Vaccines must elicit immune responses of a sufficient magnitude and quality to prevent or treat disease. While existing vaccine technologies are capable of achieving this goal for many diseases, these strategies have thus far proven inadequate against global threats like HIV and cancer.

For patients and doctors battling resistant infections, Validus Cellular Therapeutics is developing VCT-001, a novel preclinical stage anti-infective therapy unlike current small molecule antibiotics, which are susceptible to resistance and are limited accessing infections.

PhotonPharma’s Innocell involves sampling a patient’s tumor cells, inactivating them so that they are unable to replicate, then administering them back to the patient.

Vasculonics is an Indiana-based biotech formed out of technology licensed from Indiana University.

Pancreatic ductal adenocarcinoma (PDAC) has one of the highest mortalities of all malignancies, with aggressive biology, resistance to therapeutic agents, and a mere 5-year survival of 8-9%.

Pulmonary arterial hypertension (PAH) affects 200, 000 people annually and there is no cure for this condition.  Researchers at Indiana University and University of Illinois at Chicago, have identified a potential drug for curing PAH.

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.

Medical College of Wisconsin inventors have developed a novel biotherapeutic for eliminating antibiotic-resistant enterococci from the GI tract.

Medical College of Wisconsin inventors have developed novel small molecule therapeutics that will help treat small cell lung cancer, among other cancers.

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.

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

B cells are primarily known for their robust adaptive immune response through antibody production; however, they are often overlooked among the body’s professional antigen-presenting cell (APC) populations.

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 selective small molecule inhibitors of G protein-coupled receptor kinase 5 (GRK5) for preventing heart failure and treating breast cancer.

Researchers at Purdue University have developed chimeric antigen receptor (CAR) neutrophils for the targeted treatment of cancer.

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.

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

Inventors at the University of Iowa have developed an RNA-based therapeutic that can be used in medical and dental applications.

Remdesivir is currently the only FDA-approved drug for treating COVID-19. While remdesivir improves recovery time, it does not significantly reduce death rates in hospitalized adults. Researchers at the University of Kansas have produced novel antivirals that shows promise as a COVID-19 treatment.

Despite recent therapeutic advances in cancer treatment, metastatic cancer remains the primary cause of cancer deaths.

Therapeutic proteins have been used to successfully treat a variety of neurodegenerative diseases.

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.

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.

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).