Revolution Reservoir is a self-powered, rotating catheter system to prevent shunt failure in patients with hydrocephalus. Revolution Reservoir is developed by Dr. Eric Leuthardt, one of the most prolific inventors at Washington University in St. Louis and the world with 628 Issued US patents and thousands of pending applications. He is the co-founder of 4 startup companies and is the director of the Center for Innovation in Neuroscience and Technology (CINT), which is sponsored by Stryker Corporation.
New biomarker for novel CD8 T cell subset that drives systemic sclerosis immunopathology, including monoclonal antibodies anti-human C10orf128 and new targets for small molecule inhibitor therapy. Monoclonal antibodies recognized 5-10% of circulating T cells. Humanized version of them are likely therapeutic agent for systemic sclerosis after humanization. Also so likely have a monoclonal antibody specific for mouse homolog of C10orf128 to enable an animal preclinical model for T cell depletion studies. There is no effective treatment for systemic sclerosis; 50,000 individuals have Ssc; fatal disease. Potential applications include pulmonary fibrosis, asthma with scarring among others.
Human Immunodeficiency Virus (HIV) is a deleterious virus that attacks white blood cells (Specifically CD4 T cells) that fight infection, compromising the immune system and it's ability to fight infection. Untreated, HIV reduces the number of CD4 cells (T cells) in the body - the damage to the immune system makes it increasing difficult for the body to fight off infections and other diseases. Eventually, those infected with HIV develop Acquired Immunodeficiency Syndrome (AIDS), where infections or cancers take advantage of a very weak immune system, including infections that could otherwise be adequately defended against. There is no effective cure for HIV; it can only be managed by antiretroviral therapy (ART). Thus, there is a large unmet clinical need for a cure for HIV.
Pattern Therapeutics is a developing a novel TLR (toll-like receptor) antagonist to treat NASH (non-alcoholic steatohepatitis) and potentially other indications. NASH is the most common liver disease in the United States, with an estimated prevalence of 4-6% and predicted prevalence growth of 15-50% in the next 15 years. A critical finding is that TLRs mediate the Sterile Inflammation (SI) response and exacerbates metabolic stress. The candidate antagonist - PT-7314 - is a novel compound with excellent Phase 1 safety data, and strong in vitro and in vivo efficacy in mouse models of NASH. Pattern Therapeutics is ready for Phase 2 trails and is looking for an entrepreneur to raise funds to file an IND, manufacture the drug for the clinic, and to lead the trial.
Cancer drug efficacy is limited by dosage restructions based on side effects of cancer drugs on healthy cells. Targeting drugs to tumors specifically can substantially improve the efficacy of existing drugs, and reduce the unpleasant side effects of cancer treatment. Cytosolix is using the high acidity characteristic of tumors to preferentially target drugs to these cells. The platform is applicable to 95% of cancers and 90% of those therapies, giving Cytosolix the opportunity to revolutionize drug design in oncology.
DNA damage can be detrimental to cells as it can lead to mutagenesis, genotoxicity and tumorigenesis. One of the most frequent forms of DNA damage is 8-oxoguanine (8-OG), which is repaired by OGG1. Bioactive inhibitors of OGG1 and the GO pathway can be useful tools to investigate the connections of this enzyme activity to disease. Yoma Biosciences has developed potent, selective, small-molecule inhibitors of OGG1 and other enzyme targets in the GO pathway. These inhibitors provide useful tools to study the biological roles of OGG1 and also have therapeutic potential for cancer and inflammation. With multiple enzyme targets in the pathway (OGG-1, MTH1, MutYH, …) - currently un-drugged, Yoma has multiple chances for 1st in class candidate discovery.
Treating The Root Cause Of Periodontal Disease Through Cementum Regeneration. 50 million U.S. adults suffer from moderate to severe periodontal disease, a condition with no effective treatment beyond those with limited efficacy such as deep cleaning and local antibiotic therapy. We have identified a molecular target that regenerates cementum in both animal models and humans, and propose to treat the root cause of periodontisis with a first in class therapeutic.
GlimmerX has developed a fast, robust point-of-care diagnostic development platform based on glycoconjugate immunochemistry for common infectious diseases, such as leptospirosis, sepsis, UTI and meningitis that is better than current technologies. To establish proof of principle for our platform, we focus on glycans in leptospirosis, which has a large unmet US and global diagnostics need for veterinary and human disease. In a pilot project, Blavatnik funds would be used for PCT fees, and CRO costs for GLP antigen production, monoclonal antibody production and animal experiments to validate our approach, and they would leverage CT Innovations matching funds and SBIR funding.
Knee cartilage injuries in young active people have unique challenges that do not face older patients. The treatments need to be able to withstand intense activity and last for years. There is currently no gold standard treatment, with all solutions having significant drawbacks in price, recovery time, and/or longevity of treatment. Nanochon has developed a 3D printed synthetic cartilage replacement which is inserted with minimally invasive surgery and encourages new tissue growth, extending the lifetime of the treatment.
A vast majority of drugs and biologics fail to enter the brain for treatment of brain cancers and neurological diseases. Our discovery yielded key insights into how the immune system naturally overcomes the blood brain barrier to fight infections. We leverage this insight to enable safe and transient drug access to the CNS using a simple intranasal peptide delivery approach.
Antibiotic suppression of beneficial microbes in the gut, skin, and mucous membranes leads to numerous undesirable side effects, including antibiotic-associated diarrhea (AAD). Although concurrent probiotic use has shown benefit in preventing infection recurrence and reducing side effects, results are unpredictable, in part because the probiotics are also susceptible to the antibiotic. Armored Probiotics is an inexpensive & temporary coating that helps patients maintain a healthy microbiome during antibiotic treatment. This technology opens up a new market segment for people receiving antibiotic treatment.
We have developed a novel linker technology that can enable potent targeting of nanomedicines for therapeutic and diagnostic applications. We further have a unique clinical pipeline for development of this technology using ex vivo perfusion of non-transplanted human organs. Our primary lab, the Tietjen lab, has developed a robust pipeline for preclinical research on human kidney and liver with the capacity to expand to heart and lung in the future. This provides a direct path to clinical impact in organ transplantation and will enable broad translation for a variety of indications.
Pathogenic fungi are a major public health threat, causing failure of implanted organs and devices, neonatal mortality and much more. Unfortunately, it is difficult to develop specific drugs against these infections because fungal cells are a lot like those in people: we are both eukaryotes that share a similar set of enzymes and pathways. To address this problem, the Pyle lab has specifically targeted the unique RNA metabolism of fungal cells, giving rise to a new generation of nontoxic drugs that are ready for development and implementation. This has received a Blavatnik Award in May 2019.
Graft versus host disease (GVHD) is a severe immune response in patients after transplantation, such as bone marrow transplantation. GvHD is costly to treat and has a high mortality. The current strategy for preventing GvHD include general immuno-suppresent drugs, with limited efficacy and side effects. Therefore, a more targeted approach is needed to prevent GvHD. This technology is a monoclonal antibody which targets cell surface markers on intestinal crypts, which is the primary site of immune attack in GVHD.
Each year millions of people suffer from symptoms of sore throat, laryngitis and cough due to colds and inflammation of the upper aerodigestive tract. Current methods of treatment are suboptimal and rely primarily on throat gargles, sprays, and lozenges, which treat only the mouth and part of the upper throat. They completely miss 50% of the inflamed area. This product is an OTC inhaled liquid mist that is inexpensive, portable, disposable, natural, tastes good, and is more effective because it treats the entire upper aerodigestive tract. The target market would be everyday people with cough, voice and sore throat complaints, as well as singers and voice professionals.
Our technology combines novelty in bioabsorbable stent technology and regenerative medicine. Our team has unrivaled experience in both technologies and have taken the project funded by the European commission to an advanced stage in development. Our initial therapeutic target will be peripheral vascular disease where there is no comparative technology in use or development. Beyond this, there is potential widespread application of the device and concept to the heart, brain, liver and cancer treatments where the combined technology can provide a unique state of the art therapeutic system.
AnelleO is a medical device company that is developing a 3D printed intravaginal ring that can be used as a platform for treating a wide range of women's health conditions. Their first product, AnelleO PRO involves the only single administration of progesterone for infertility. Their technology enables controlled drug-release kinetics with up to 100% drug release, as well as rapid manufacturing and custom sizing. The manufacturing method allows for formulation of biologics and drug combinations not possible with traditional technologies. AnelleO was founded by Rahima Benhabbour, professor at the Eshelman School of Pharmacy/UNC_NCSU Joint Biomedical Engineering Department, and Post Doctoral Fellow Rima Janusziewicz.
Idiopathic pulmonary fibrosis (IPF) is a deadly chronic lung disease with median survival of 3 years and with a worse prognosis than lung cancer. 6 million people worldwide are affected - 200,000 in North America are affected with 45,000 dying each year. The progressive decline of lung function characterizing it is interspersed with unpredictable disease flares called acute exacerbations of IPF (AE-IPF) that accelerate lung function loss and increase morbidity and mortality. The annual incidence of AE is up to 20% with a mortality ranging from 35-90%, demonstrating the severity of IPF disease progression and the importance for active disease monitoring (ER visits and hospital stays can amount to >$11,500 per case). Current therapeutics are unable to predict how an individual patient will progress and whether they will respond to available interventions. The market size for biomarker chip detection of PIF is ~$3B, underscoring the need for a more robust treatment for PIF.
Veramorph is developing a polymer-based pre-formed oral dosage technology as a more effective drug delivery vehicle for poorly soluble small molecule drugs. Our technology is capable of improving oral drug delivery performance of a much broader range of small molecule drugs the existing formulation methods. Our product, disintegrating polymer oral dosages (DPODs), can enable more effective drugs to be brought to clinical trials that will improve success rates and reduce the overall cost of successfully commercializing a drug product. Veramorph is developing an internal pipeline of reformulated products and is also seeking product development partnerships through licensing agreements with pharmaceutical companies.
Cache is a MIT startup from Prof. Mark Bathe’s lab is seeking an entrepreneur to help lead the next phase of customer discovery/hypothesis validation. Their technology enables massive and low-energy storage, up to millennium timescales, and random access of nucleic acid samples from broad sources including ecological, forensic, or archival DNA data storage. They are seeking a passionate, adventurous, and creative entrepreneur to join their early-stage venture to help launch this technology into the 21st-century biotechnology space. Their immediate aim is to recruit an entrepreneur who will be excited to mature their business plan using market discovery and research on product-market fit. They are ready to test the market’s appetite for this technology, developing partnerships along the way.
Getting immediate medical attention is critical after a patient has experienced a stroke. However, stroke symptoms are often not identified by the patient or family members until it is too late to administer treatment. There is currently no wearable-linked mobile app which specifically detects a stroke incident. AlvaHealth is pairing wearable devices with machine learning to detect a stroke incident immediately based on the activity in both arms of the patient. Early detection of stroke can improve the outlook for patient recovery.
The use of radioisotope labels is ubiquitous in biochemistry, cell biology, drug discovery and numerous other fields, yet the approaches for detecting radioisotopes are more than four decades old. However, there is an unmet need for radioisotope detection materials that are more compatible with biological samples and that provide better temporal and spatial resolution. Scintillation Nanotechnologies develops and manufactures composite nanomaterials for detection of radioisotopes in biochemical and drug discovery applications. The proprietary composite nanomaterials provide a key enabling tool for established and new biochemical investigations that will directly impact human health. Scintillation Nanotechnologies has four products ready for immediate release to the market.
Existing brain perfusion systems are focused on preserving immediately harvested organs until transplantation. These systems are not applicable to research and development applications where the organs may not be immediately obtained postmortem. OrganEx has developed an effective method to salvage organs ex-vivo to advance clinical research and transplantation studies.
Understanding the immune response to disease, injury and therapy can have a substantial impact on healthcare. Cellintec will provide a novel, scalable, economic approach using the DNA methylation status of immune cells to derive quantitative, easily standardized immune cell profiles that can be tracked through time and compared between patients and across clinical sites. This capability will enable healthcare providers to choose a cancer treatment based on the level of a patient’s immune suppression, cohort patients for clinical trials based on their immune status, assess an individual’s responsiveness to treatments (especially immune therapies), capture and characterize an autoimmune or allergic response, and monitor a person’s general immune health.
Rapid, reliable, and widespread testing is required to curtail the ongoing COVID-19 pandemic. Current gold standard diagnostic assays are hampered by supply shortages in critical reagents including nasal swabs, RNA extraction kits, personal protective equipment (PPE), instrumentation, and labor. This WUSTL team lead by Jeffrey Milbrandt and William Buchser overcome these challenges with the development of a rapid colorimetric assay using reverse-transcription loop-mediated isothermal amplification (RT-LAMP) optimized on human saliva samples without an RNA purification step. This optimized reaction has been prototyped and tested and can provide for an affordable point-of-care diagnostic of SARS-CoV-2.
There is a growing aging population and there is no cure or treatment for Alzheimer's. The only current drugs are palliative. The Synapse Enhancer enhances the integrity of the synapse which improves cognitive function and can delay/halt neurodegeneration. It could be utilized for a many diseases such as, Alzheimer's, ALS, Parkinson's, Huntington's, Traumatic Brain Injury, Stroke, Epilepsy, Depression, Neuropathic Pain Disorders, and Addiction.
Cancer patients, after the first diagnosis often ask, “how bad is my tumor and what should I do if it is bad?” In about half of all cancer patients, the first line of chemotherapy fails, disease recurs, and patients die. Episteme has succeeded in targeting a specific epigenetic signature of pancreatic cancer patients, which could potentially be reverted by epigenetic drugs making the tumors amenable to chemotherapy. Episteme invented a novel microarray-based platform technology called “ATAC-Array”. This proprietary technology avoids the time and cost of next-generation ATAC library sequencing – the only microarray that reads chromatin accessibility. No other test provides such a comprehensive, cost-effective and clinically useful epigenetic summary. Episteme offers a personalized theragnostic test utilizing ATAC-array, histopathology and immunohistochemistry, to predict chemotherapy response and stratifying cancer patients to epigenetic (reprograming) therapy for better outcome and better quality of life.
Over 95% of rare disease have no treatment or cure: there are over 7,000 known rare diseases collectively affectiting 30 million Americans. Many of these disease are genetic disorders, in which there can be considerable genetic variation between patients. Current drug development pipeline does not cater to rare mutations, as drug development often takes more than 10 years and over $2B to get a drug to market, making the small patient populations inhibiting. Even if a therapy is developed, genetic variation of the disease means not all patients in the already-small patient population may be able to be treated, limiting their impact and incentive to develop these therapies.
Bacchus Therapeutics is a biotech company that exploits cancer’s hypermetabolic state by targeting specific cancer metabolic pathways. The aberrant expression of MYC is virtually unmatched, making MYC one of the most frequently deregulated oncogenes in human tumors, including, liver, kidney, CNS, hematopoietic, breast, prostate, lung and GI tumors. Human studies and animal models have established that dysregulation of MYC underlies the pathogenesis and aggressiveness of numerous cancers. Our founders, Drs. Gouw and Felsher, discovered that MYC regulates the aberrant lipid metabolism of tumors and that inactivation of key lipid enzymes results in regression of tumors.
Multidrug-resistant Gram-negative bacilli (MDRGNB) have emerged as a challenging cause of hospital-acquired infections and present a critical need for innovative antibacterial development. Two new oxopyrazole agents targeting penicillin binding proteins (PBPs) based on a non-beta-lactam core have superior MIC50 values to current billion-dollar last resort antibiotics like Ceftazidime/Avibactam or Meropenem. One shows broad Gr- efficacy while the second oxopyrazole is selective for Acenitobacter baumannii. On target, good in vivo PK, no mammalian toxicity, no off-target liability. Seeking funding for definitive in vivo efficacy studies.
The three members of the endocrine FGF family, FGF19, FGF21 and FGF23 are important circulating hormones that regulate a variety of critical metabolic processes. Endocrine FGFs mediate their cellular processes by binding to and activating FGF-receptors (FGFR) in complex with Klotho proteins. Based on the crystal structure of ligand occupied Beta-Klotho new potent engineered endocrine molecules were developed for treatment of metabolic disease that will benefit from therapeutic stimulation of FGF21 cellular pathway such as pancreatitis, Nash and obesity. Moreover, also potent inhibitors including small molecules will be developed for treatment of bone disorders (XLH) and liver cancer, respectively.
Activating the innate immune system within tumors is a promising new direction in immunotherapy because it can be used against a broad spectrum of tumor subtypes and it ensures that cells throughout the tumor microenvironment become sensitized, resulting in attack and destruction by T cells. RIG-I is a particularly sensitive trigger of innate immune response in tumors, so agonists of this receptor are promising immunotherapy agents. Through a unique class of RIG-I agonists called Stem-Loop RNAs (SLRs), the scientific team is evaluating the efficacy of SLRs as immunotherapy and antiviral agents.
My Gene Counsel has created scalable, digital technology that pairs specific genetic test results with accurate, continuously updating information for clinicians and consumers. By helping patients and doctors become aware of the implications of genetic testing results, My Gene Counsel can help improve patients' lives and avoid unnecessary risky medical procedures.
Vimentin and Keratin Targeted Therapies for Treating Dermatologic Cancers. The Bunick lab discovered a new mechanism governing proper intermediate filament assembly. We believe topical application of peptide therapeutics can disrupt this mechanism in skin cancer cells, leading to an effective first-in-class anti-cancer therapy.
EPO-VG are implantable grafts composed of cells which release a steady dose of erythropoietin (EPO) to patients with end stage renal disease. These grafts reduce the cardiovascular risks associated with single dose EPO injections, improving cost and patient outcomes.
Macrophage Migration Inhibitory Factor (MIF) plays a key role in inflammatory disease and cancer, being an important regulator of the innate immune response. MIF is an inflammatory cytokine; when bacterial antigens are present, it binds to CD74 on other immune cells to trigger an acute immune response. MIF regulates cell proliferation by binding to receptor CD74 in MAPK (ERK) and AKT signal pathways. It also inhibits apoptosis of cancer and inflamed cells by blocking p53, making it an attractive drug target for cancer therapies.
Therapeutic options for glioma cancers are limited, and the survival rate for glioma patients is poor. Athena is progressing NAMDT inhibitor therapy by applying it to cancers with specific mutations, thus increasing the efficiency and decreasing toxicity of treatment.
Glioblastoma is a deadly disease and usually is untreatable. The team has identified Lassa-VSV, an oncolytic virus, as safe and capable of destroying glioblastoma, melanoma, and other cancers within the brain by direct oncolytic actions. It stands to transform the glioblastoma treatment landscape and save lives.
Myasthenia Gravis (MG) causes weakness and rapid fatigue of muscles under voluntary control and is caused by an antibody-mediated autoimmune response. Current treatment options include acetylcholinesterase inhibitors (with modest efficacy at improving neurotransmission). The prevalence in the U.S. is estimated at 20 cases per 100,000 people. The vaccine utilizes cytoplasmic domains of human AChR subunits and incomplete Freund’s adjuvant.
Gateway is an emerging privately held start - up addressing hearing loss and tinnitus. Incubated with BioGenerator in St. Louis, Gateway builds on research and clinical collaborations across several academic institutions including Washington University, Northeast Ohio Medical University and Harvard University. Its founders are scientific and clinical leaders in the field. Gateway has built a comprehensive research, discovery, and clinical development platform for new therapeutics to treat tinnitus and noise-induced hearing loss (NIHL). The platform is highly leveraged by federal funding, cumulatively some $30m of research by the scientific founder, and $12m in translational funding, including a Phase II hearing loss clinical trial funded by the U.S. Army. The Company has raised no equity capital to date.
Age-related macular degeneration (AMD) is a leading cause of blindness, affecting more than 8 million individuals in the United States alone. Although nutritional supplements are recommended for patients with intermediate risk or advanced AMD, there is still no effective therapy for the 90% of AMD patients with the “dry” or atrophic form. The team at Opti-Peutics used a high-throughput screen to identify novel compounds that protect RPE cells from oxidative damage.
Transplanted tissues release donor organ-specific exosomes into the bloodstream which can be detected. The tracking of these exosomes allows the monitoring physician to detect early signs of transplant rejection, allowing early treatment and rejection prevention.
Genital herpes affects more than 500 million globally and it is incurable. In the US alone, 1 million people are diagnosed every year with herpes. Herpes causes painful viral outbreaks, significant psychological stress, and social stigma. Even though women are more prone to genital herpes, the available prevention option, condoms, is controlled by the male partner. The solution is a new female-controlled product called HerShield, a vaginally applied, soft and flexible pharmaceutical film that has proven safety in two phase I clinical trials, and provides sufficient quantities of drug for protection from herpes. HerShield is designed to be discrete, exceptionally portable, low cost, biodegradable, and easily self-administered without the need for an applicator.
Pearl Bio is an early-stage venture pioneering design and production of next-generation therapeutics and biomaterials for medical applications and beyond. Biology is constrained to the 20 natural amino acids, limiting the ability to site-specifically modify therapeutic proteins to improve half-life, tissue targeting, or assembly. In contrast, chemical synthesis of therapeutics enables access to greater functional diversity, but template-directed synthesis is challenging. Pearl Bio unites the precision of biology with the unlimited diversity of chemistry in a transformative platform that produces therapeutics and biomaterials with tunable properties for applications in oncology, immunology, and rare disease.
Inflammatory diseases are often treated with immunomodulatory drugs which can result in severe side effects due to their systemic administration. This technology is a vitamin D analog which can be administered topically, therefore reducing systemic side effects. The drug works by triggering release of a regulatory cytokine from the skin and decreasing T cell activation which is involved in many inflammatory disorders.
Ear infections are the number on cause of doctors visits worldwide, affecting 700 million people each year. Chronic ear infections are often treated with the insertion of a tympanostomy tube into the ear drum. While these help to drain fluid, they often come with complications which can result in follow up surgeries and hearing loss. The PionEar tympanostomy tube technology addresses the most common complications by changing the geometry and materials of the tube to enhance fluid transport and reduce the chances of biofilm formation.
Early indication of fluid accumulation to trigger adjustments to medical therapy preventing costly readmissions and avoiding life threatening complications.
ClostraBio is a preclinical-stage pharmaceutical company creating new medicines to treat diseases resulting from intestinal barrier dysfunction, especially those caused by shifts to the microbiome. ClostraBio's first indications are in the treatment and prevention of food allergy, initially peanut allergy, and ClostraBio recently began a program in colitis. ClostraBio has licensed a proprietary drug delivery platform, developed by the ClostraBio co-founders, from the University of Chicago and are using this platform for oral delivery of small molecules and metabolites derived from the microbiome to the GI tract. ClostraBio's founding scientific team are key opinion leaders and world's experts in food allergy, the microbiome, immunology and immuno-engineering, drug delivery, and polymer chemistry. ClostraBio is seeking a Chief Executive Officer and Chief Medical Officer.
Dr. KiBum Lee of Rutgers University is developing a nanoscaffold material and system that would enable effective stem cell therapy and drug delivery. The innovation promises functional recovery in patients with central nervous system injuries--like spinal cord injuries--for which there are no effective treatments today. Features of the nanoscaffold include 3D biomimicry, stem cell/neuronal differentiation, ECM-protein binding affinity, efficient drug loading/sustained delivery, and MRI-based monitoring capability.
Moving Therapeutic Proteins Into the Cytosol and Nucleus. Exolva is using CPMPs (cell-permeant miniature proteins) to deliver therapeutic enzymes and gene-editing tools to correct inborn genetic disease. CPMPs are small, folded proteins that contain a specific array of five Arg residues on an ⍺-helix backbone. CPMPs can reach cytosol and nucleus with efficiencies as high as 75%. CPMPs possess many advantages relative to previous, purported ‘cell-penetrating peptides’, including low toxicity, high and tunable stability, enzyme cargo retains enzymatic activity, among other features.