Novel Small Molecules for Promoting Nerve Regeneration
Scientific Relevance
Unlike most other tissues which have the ability to heal themselves after injury, damaged axons in the central nerve system are unable to regenerate. There are no existing therapies to promote CNS axon regeneration in humans. Identification of novel small molecules that promote nerve growth and nerve regeneration could provide compounds that can be manipulated to yield drugs useful in treatment of various neurodegenerative diseases.
Commercial Opportunity
Health care costs for CNS patients are staggering. For instance, the patient costs are estimated at $13 billion annually for SCI patients in the U.S. alone. Concerns about the specificity and efficacy of agents reported in the literature, researchers at the University of Miami and New York University have synthesized a novel family of small molecules that are potent and selective in promoting axon regeneration.
Competitive Advantage
Potent and selective
novel mechanisms that could lead to new therapy strategies.
Inventors
John Bixby, Vance Lemmon, Lynn Usher (UM), Young-Tae Chang, Jae-Wook Lee, and Jaeki Min (NYU).
Breakthrough Treatment for Asthma and Other Inflammatory Lung Diseases
Scientific Relevance
The main pathological feature of asthma is airway inflammation. Researchers at the University of Miami have identified the molecule that is responsible for triggering a chain of events that induce asthma. Studies in a mouse asthma model have demonstrated that blockage of this pathway prevents lung inflammation.
Commercial Opportunity
Estimated at $14 billion in direct medical expenditures, treatment of asthma remains an important public health problem associated with significant health care costs.
Competitive Advantage
Target specificity unparalleled in conventional treatments; Reduce or eliminate many of the side-effects of current treatments; Could cure asthma and other inflammatory lung diseases.
The invention uses immunomodulating agents that can both stimulate the immune system or have an immunosuppressive effect. TNFR25 agonists and antagonists have pro and anti-inflammatory action, respectively. TNFR25 agonists can support tumor vaccination, while TNFR25 antagonists can treat disease caused by chronic inflammation and autoimmunity (e.g. inflammatory bowel disease, ulcerative colitis and Crohn's Disease).
Commercial Opportunity
Inflammatory bowel disease, ulcerative colitis and Crohn's disease cost between $3-5 billion annually to treat. To have a novel approach to these diseases that would also apply to transplantation autoimmunity is a unique commercial opportunity.
Competitive Advantage
Tumor vaccines that are currently available are often suppressed in the tumor environment. This approach increases the vaccine's ability to act on the tumor due to increased stability. The myriad of biological effects that this immunomodulating agent can achieve is far superior to anything currently in use.
Transplantation of pancreatic Langerhans islets into subcutaneous, neovascularized devices is one of the possibilities currently explored as part of a search for a cure of diabetes as such transplantations could normalize metabolic control in a way that has been virtually impossible to achieve with exogenous insulin. Syngeneic islets transplanted into a subcutaneous, neovascularized device have been recently shown to restore euglycemia and sustain function long-term in a rat model.
Commercial Opportunity
Diabetes is an increasing worldwide problem, and the need for diabetes-focused drug discovery is well underlined by the fact that whereas mortality rates for heart attacks, breast cancer, and stroke have significantly improved in the U.S. since the 1980's due to the introduction of new drugs and therapeutic methods, they increased for diabetes. The incidence of Type 1 Diabetes has also increased while the age of onset has decreased by 3.5 years over the past decade for yet unknown reasons.
Competitive Advantage
The novel approach requires much smaller doses than a traditional systemic immunosuppression since therapeutically active concentration levels have to be maintained only within the device (by local delivery). Therefore, the serious toxic side effects and the susceptibility for opportunistic infections, the main problems associated with systemic therapies, should be avoidable even if long-time treatment is needed to provide protection against rejection and maintain function.
Biomarkers to Predict Functional Lymphocytes and Immunoglobulins in Humans
Scientific Relevance
A particular set of biomarkers for functional B lymphocyte and immunoglobulin activity in humans has been identified. As part of the immune system, B-lymphocytes are important for making antibodies and helping fight infections. Specifically, antibodies made by B lymphocytes prevent colonization by pathogens and provides immunity against invading pathogens. In the elderly and in patients with weakened immune systems, vaccines and therapeutic agents may not be as effective because the number of functional B lymphocytes and immunoglobulins are fewer than in healthy people. By using the newly identified biomarkers, responses to vaccines and therapeutic agents can be followed more accurately, thus better dosing schedules and/or therapeutic approaches can be developed and followed.
Commercial Opportunity
Infectious pathogenic diseases are fairly common, with influenza alone affecting up to an estimated 50 million people each year in the US. Approximately 40,000 deaths are attributed to the flu, with most of the affected being the elderly and those patients with weak immune systems.
Competitive Advantage
A unique set of biomarkers have been identified in humans. These biomarkers would more accurately track immune responses and activity than the murine models currently used, and would be better indicators of vaccine and therapeutic agent effectiveness in humans.
A Novel Cell/Tissue Culture System to Enhance Cell Proliferation & Induce Cell Differentiation
Scientific Relevance
Conventional cell and tissue culture typically involved cells resting atop (or attaching to) a gas-impermeable plastic bottom, in a given volume of specific medium to maintain viability and function. For immortalized cell lines or cells that are not exquisitely depend on oxygen, this means of culture is sufficient and results in acceptable growth and differentiation for experimental needs. However, these conditions are sub-optimal for tissues with high metabolic requirements. The researchers at the University of Miami have designed a novel culture system whereby three dimensional tissues can receive oxygen both from the top (after diffusion through medium) and the bottom (through direct diffusion across a perfluorohydrocarbon-silicon membrane).
Commercial Opportunity
The Petri dish market is over two billion dollars a year. This novel system can be used to promote both growth and differentiation of stem/progenitor cells where oxygen becomes limiting as is invariably the case in conventional culture systems. Such application is of particular interest for cell types known for their high in vivo oxygen demands. The premise behind this approach is that, unless culture systems meet the physiological requirements of such cells, their in vitro differentiation from stem cells will be severely impaired. Among the tissues with a high metabolic rate whose differentiation may benefit from our invention are: pancreatic islet cells, liver, kidney, cardiac tissue, brain cells and lung epithelium, to name a few. Additionally, this device could also be used to improve the culture of primary or already differentiated tissues.
Competitive Advantage
Provides a more physiological mode of oxygen delivery, preventing hypoxia even in thick cellular aggregates.
Promotes cell viability and function
Enhances differentiation in stem cell systems where oxygen has been proven to act directly as a cell specification agent.
Inventors
Christopher A. Fraker, Juan Dominguez-Bendala, Camillo Ricordi & Luca Inverardi.
Rapid Identification of Ocular Fungal and Amoebic Pathogens
Scientific Relevance
Fungal infection of the eye, or ocular mycosis, is a serious condition that may result in loss of vision if the disease is not diagnosed and treated in time. Despite an increase in the incidence of the disease over the last decades, management remains a clinical challenge due to misdiagnosis and inadequate detection methods, which are often slow.
Commercial Opportunity
Mycotic keratitis is considered a leading cause of ocular morbidity throughout the world. This corneal infection, which is mostly caused by yeast or filamentous fungi, can have devastating and irreversible effects if not treated on time. For instance, if the disease is left untreated, 50% of infected eyes can suffer from visual impairment or loss of sight due to irreversible corneal damage. The incidence of the disease, which has increased over the past four decades, has been the result of overuse of topical steroids and antibacterial agents, the rise in the number of patients with immuno-deficiencies, trauma, chronic ocular diseases and corneal anesthetic abuse. Contact lens wearers are also a growing sector of the population at risk for fungal keratitis
Competitive Advantage
High speed and high sensitivity for rapid and accurate diagnosis
Inexpensive
Versatile as a variety of probes can be added or subtracted to create different arrays that can be employed in a wide variety of applications
Microparticles as a Treatment Method for Bleeding Disorders
Scientific Relevance
Microparticles (MP) derived from blood can be administered to a patient to help stop bleeding and decrease blood clotting time. This would be important in cases involving internal bleeding, trauma, surgery, and chemotherapy, where the patient's own blood prior to treatment could be banked for later use.
Commercial Opportunity
This technology addresses a critical necessity because of the endless need, and short supply, of blood and its products. The market for a platelet substitute product is significant. It is estimated that over 10,000,000 units of platelets were transfused in the United States last year at an average cost of $50 per unit, which equates to an a market potential of $500 million annually.
Competitive Advantage
At present, no synthetic microparticles or blood cells have emerged as being clinically useful. In addition, since the patient's own blood can be used, the risk of acquiring infectious agents from an external blood supply is decreased substantially.
Inventors
Wenche Jy, Joaquin Jimenez, Lawrence Horstman, Yeon Ahn, and Eugene Ahn
In this invention, the aircraft tires' sidewalls are modified to include an airfoil shaped impeller. The impeller blades are co-extruded from rubber material during tire manufacturing. The airfoils are aligned with the relative flow at an optimum angle of attack in the front portion of the tire to maximize the force and movement generating area, which covers the front half circumference of the tire.
Commercial Opportunity
This invention may capture a part of commercial aircraft tires market.
Competitive Advantage
The present invention increases aircraft tires service life and improves safety by reducing the possibility of tires' failure during landing.
A Set of Small Molecule Inhibitors of Human Galactokinase (GALK)
Scientific Relevance
Galactosemia is a genetic disorder, in which the body becomes deficient in a liver enzyme, galactose 1-phosphate uridyltransferase (GALT), required for the digestion of galactose. The only treatment for galactosemia is eliminating lactose and galactose from the diet. Even with an early diagnosis and a restricted diet, however, some individuals with galactosemia experience long-term complications such as speech difficulties and neurological impairment.
Commercial Opportunity
Pharmaceutical industry has been searching for a way of inhibiting GALK as a therapeutic approach for galactosemia.
Competitive Advantage
Novel and effective treatment for Classic Galactosemia will qualify as Orphan Drug according to the US Federal Orphan Drug Act (amended).
Set of Genes and SNP's that Predict Susceptibility to Mood and Anxiety Disorders (MDD) and Co-morbid Conditions
Scientific Relevance
This invention represents a screening method to aid in the identification of individuals who are suffering from or are at increased risk of developing MDD, and for identifying effective treatment.
Commercial Opportunity
Major Depressive Disorder (MDD) is a common complex disorder of unknown etiology that affects about 15% of the population.
Competitive Advantage
By establishing an association between susceptibility to MDD via genotyping a panel of single nucleotide polymorphisms (SNPs), identification of individuals at increased risk for this disorder can be established and therapy can be effectively begun.
Inventors
Ma-Li Wong, Chuanhui Dong, Jorge Maestre-Mesa and Julio Licinio
Perforin 2 Proteins as a Novel Antibacterial Therapy
Scientific Relevance
This invention relates to the fields of antibiotics, anti-cancer agents and drug discovery. More specifically, it relates to a compound useful in activating the body's natural defenses to both infections and tumors.
Commercial Opportunity
The incidences of serious bacterial infections are increasing at home and in hospitals due to the development of antibiotic resistance. Furthermore, there are certain bacteria such as those causing tuberculosis that are naturally resistant to most antibiotics. For biological reasons alone, there is an urgent need to discover novel antibacterial agents. Antibiotics, many of which have patents that will expire in the next few years, are currently a multi billion dollar industry.
Competitive Advantage
This is a unique antibacterial approach in that it will not lose efficacy due to the development of resistance.
Transgenic Mice Expressing a Real-Time Reporter for Cellular cAMP in a Tissue-Selective and Inducible Manner
Scientific Relevance
Cyclic AMP (cAMP) mediates the response to hormones, neurotransmitters and other molecules in practically every tissue of the body. The inventors have generated a unique transgenic mouse that is able to express an inducible fluorescent cAMP reporter in targeted cells and tissues. To achieve this, transgenic mice carrying the newly generated and unique cAMP reporter are mated with mice carrying an appropriate, tissue-specific, antibiotic-sensitive triggering mechanism in their genome. Mice tailored to a specific tissue can be readily generated. By injecting a commonly available antibiotic, researchers can then induce the synthesis of the cAMP reporter in pre-specified cells. Using this mouse model, cAMP can be examined in intact organs, tissues and cells both in vitro and in vivo. The transgenic mouse will make it possible to monitor how intracellular cAMP levels change in response to metabolites, transmitters, hormones, and drugs in real time and with high spatial resolution (i.e., single cells and even subcellular regions).
Commercial Opportunity
This mouse is an important drug discovery tool. Pharmaceutical industry scientists as well as academic researches can use this mouse to monitor responses to existing drugs and develop new ones. Additionally, this mouse could be used to test the effects of treatments affecting the brain, spinal cord or autonomic nervous system.
Competitive Advantage
This is the only mouse model available that allows responses to drugs, metabolites, transmitters, hormones, pharmaceuticals, etc. to be monitored visually in vivo. Furthermore, the response can be seen in real time with high spatial resolution.
Heart Rate Variability (HRV) Index to Assess the Severity of Traumatic Brain Injury (TBI)
Scientific Relevance
The invention is a method of predicting a pathological medical condition using an algorithm based on HRV and several other routinely measured physiological variables.
Commercial Opportunity
According to the National Center for Injury Prevention and Control, of the 1.4 million who sustain a TBI each year in the United States: 50,000 die, 235,000 are hospitalized, and 1.1 million are treated and released from an emergency department.
Competitive Advantage
The method provides an algorithm that can be normalized to a simple scale for interpretation by a health care provider, such as an ambulance technician. The technology is an automated triage tool that aids decision making about the severity of trauma.
The invention involves an 11 amino acid peptide (IEP11) which corresponds to a unique region of the secreted form of human mucin protein (sec-mucin). The 11 amino acid peptide can be used as a vaccine adjuvant to enhance in vivo immune responses, to induce the production of cytokines, as a mitogen of one or more cell subpopulations, or to enhance cellular cytotoxicity.
Commercial Opportunity
Cancer is the second leading cause of death in the U.S. with over 500 thousand per year.
Competitive Advantage
This small protein drug has the potential to be a universal adjuvant for use with or without traditional chemotherapy. Immunomodulation to help one's body fight cancer can reduce the need for toxic chemotherapy and increase the survival rate and quality of life.
Early Detection of Head and Neck Squamous Cell Carcinoma (HNSCC)
Scientific Relevance
This invention is a saliva based test (ELISA) incorporating a panel of four biomarkers that can detect early stage HNSCC and very early pre-cancerous lesions. At this early stage the cancer or pre-cancer is treatable or reversible. At this point, the complete biomarker panel has 93% sensitivity and 75% specificity.
Commercial Opportunity
HNSCC accounted for $3 billion in expenditures in 2004 or 4.4% of all cancer expenditures. 85% of HNSCC occurs in drinkers and smokers. There are currently 56 million smokers and 15 million alcohol abusers in the U.S and significantly more worldwide.
Competitive Advantage
This early detection panel of biomarkers is simple and low cost, detecting cancer without visual examination of the patient by a primary care provider or dentist.
Inventors
Elizabeth Franzmann, Vinata Lokeshwar, Erika Reategui and Rakesh Sihgal
This invention demonstrates that hyaluronidase (HAase) inhibitors may be used to inhibit tumor cell proliferation and/or progression through the cell cycle. These inhibitors are being developed to treat cancer or a precancerous condition with special emphasis on solid tumors. In addition these compounds may be anti-angiogenic and reduce the risk of cancer cell metastases. These compounds are likely to be effective with little or no side effects.
Commercial Opportunity
Cancer is the second leading cause of death in the U.S. with over 500 thousand per year.
Competitive Advantage
This non-toxic anti-cancer therapeutic can be administered alone or as an adjunct to traditional chemotherapy or radiation therapy. This feature allows for reduced use of toxic chemotherapy and an improved quality of life.
Method of Treating Multiple Myeloma by Targeting CD28
Scientific Relevance
Targeting CD28 on myeloma cells can be used as a single agent for the treatment of multiple Myeloma. This approach can also be used in combination with other chemotherapy.
Commercial Opportunity
Multiple Myeloma is the second most common hematological malignancy behind non-Hodgkin' lymphoma, with 15,000 new cases diagnosed each year. It is currently incurable. Patients characteristically respond initially to chemotherapy, but then relapse with increasingly more chemoresistant disease. Thus novel therapies that can bypass the underlying resistance mechanisms are clearly needed.
Competitive Advantage
Targeting CD-28 on myeloma cells could be a single agent treatment as opposed to the existing highly toxic treatments available.
Antibody NKG2D Ligand Fusion Protein for Cancer Therapy
Scientific Relevance
Certain anticancer drugs (e.g. Herceptin for breast cancer) act by attaching to specific receptors (HER2/neu) and thereby destroy tumor cells that express the receptor. However, these drugs have reduced efficacy when the target antigen is inadequately expressed, or if immune function is impaired. One approach to overcome this is to take advantage of a protein NKG2D-Ligand (NKG2D-L) that is ubiquitously expressed during times of infection and stress. When NKG2D-L is bound to its specific receptor, there is increased cell killing potential. Linking anti-tumor antibodies to NKG2D-L proteins increases the ability of immune effector cells to recognize and eliminate tumor cells. This approach could be used for a variety of anti-oncogenic applications.
Commercial Opportunity
Adjuvant therapy for cancer is a multi billion dollar industry. In the near future, as the population ages, it is expected that expenditure for this type of drug will increase at a faster rate than any other category of medication.
Competitive Advantage
Targeting molecules for cancer therapy allow much lower concentration of the toxic chemotherapeutic agent to be used. The advantage of this approach over non-targeted molecules is its stability and the ability to use lower concentration.
Inventors
Joseph D. Rosenblatt, Seung-Uon Shin, and Khaled A. Tolba
