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NCATS Inventions Available for Licensing

Word cloud of intellectual property termsInventions made by NCATS employees are owned by the federal government. Patenting and licensing are used to help ensure the technology is fully developed, commercialized and advancing public health. A list of NCATS patent applications available for licensing appears below.

Abstract details are provided by the NIH Office of Technology Transfer.



Alpha-Glucosidase Chaperones and Inhibitors for Treatment of Pompe Disease and Type 2 Diabetes

Inventors: Juan Marugan (NCATS), Ehud Goldin (NHGRI), Noel Southall (NCATS), Wei Zheng (NCATS), Jingbo Xiao (NCATS), Ellen Sidransky (NHGRI), Omid Motabar (NHGRI)

Abstract: Scientists at the NIH have discovered small molecules that can act as chaperones and correct the misfolding of mutated alpha-glucosidase enzyme. Pompe disease is caused by deficiency or dysfunction of alpha-glucosidase. The only FDA-approved treatment of Pompe disease is enzyme replacement, which in this case costs approximately $300,000 per year and elicits an immune reaction in most patients that limits clinical utility. Abstract Details

Arylthiazolyl Piperidines and Related Compounds as Modulators of Survival Motor Neuron (SMN) Protein Production

Inventors: Elliotn Adrophy (University of Massachusetts Medical School), Jonathan Cherry (University of Massachusetts Medical School), Juan Marugan (NCATS), Noel Southall (NCATS) Steven Titus (NCATS), Jingbo Xiao (NCATS), Wei Zheng (NCATS)

Abstract: This technology discloses compounds that modulate the amount of survival motor neuron protein (SMN). Low levels of SMN protein are associated with spinal muscular atrophy (SMA), which constitutes a group of inherited diseases that cause progressive muscle degeneration leading to death. Consequently, therapeutic inventions have focused on increasing SMN protein levels. This invention discloses novel arylthiazolyl piperidines, which are shown to be modulators of SMN production. This invention also discloses methods of treating SMA by administering SMN protein modulators. Abstract Details

Caspase 1 Inhibitor

Inventors: Matthew Boxer (NCATS), Craig Thomas (NCATS)

Abstract: Novel and potent caspase 1 inhibitors are available for licensing. In particular, this technology discloses potent and selective caspase 1 inhibitors that target the active site of the enzyme. Abstract Details

Delivery of a Therapeutic Protein, Transthyretin (TTR), Across the Blood-Brain Barrier (BBB) as a Treatment for Alzheimer's Disease

Inventors: Silvia Muro-Galindo (University of Maryland Biotechnology Institute), Juan Marugan (NCATS), Wei Zheng (NCATS)

Abstract: This invention describes products and methods of treating Alzheimer's disease, which is characterized by the formation of amyloid plaques and tangles in areas of the brain critical for learning and memory. Abstract Details

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

Inventors: Ehud Goldin (NHGRI), Juan Marugan (NCATS), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS)

Abstract: This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Abstract Details

Inhibitors of Human Apurinic/apyrimidinic Endonuclease 1 (APE1), an Anticancer Drug Target

Inventor: David Maloney (NCATS)

Abstract: APE1 is the primary mammalian enzyme responsible for the removal of abasic (AP sites) in DNA and functions as part of the base excision DNA repair pathway (BER). BER is instrumental in the repair of DNA damage caused by DNA alkylating agents (e.g. many cancer chemotherapeutics). APE1 has been shown to be overexpressed in cancer cells. It has been postulated that APE1 would be an attractive target in anti-cancer treatment paradigms; preclinical and clinical data confirm that APE1 is a valid anticancer drug target. Abstract Details

Non-toxic Compounds that Inhibit the Formation and Spreading of Tumors

Inventor: Samarjit Patnaik (NCATS)

Abstract: Available for licensing are novel pyrrolopyrimidine compounds that disrupt the assembly of the perinucleolar compartment (PNC), a sub-nuclear structure highly prevalent in metastatic tumors. These notable compounds act without overt cytotoxicity. Abstract Details

Novel Thio-Ether Analogues as a Treatment for Huntington's Disease

Inventors: Ehud Goldin (NHGRI), Juan Marugan (NCATS), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS)

Abstract: This technology is a collection of small molecules screened for their ability to prevent or reduce the cytotoxic effects of the protein, Huntingtin. Huntington's disease is a neurodegenerative disorder due to a dominantly acting expansion of a CAG trinucleotide repeat in exon 1 of the Huntington (HTT) gene resulting in production of the altered (mutant) protein Huntingtin, which has a long chain of polyglutamine (poly Q) attached to the exon 1 encoded protein sequence. Abstract Details

Pyruvate Kinase M2 Activators for the Treatment of Cancer

Inventors: Matthew Boxer (NCATS), Douglas Auld (NCATS), Craig Thomas (NCATS), Min Shen (NCATS)

Abstract: NIH investigators have discovered a series of small compounds with the potential to treat a variety of cancers as well as hemolytic anemia. Contrary to most cancer medications, these molecules can be non-toxic to normal cells because they target a protein specific to the metabolic pathways in tumors, thus representing a significant clinical advantage over less-specific chemotherapeutics. Abstract Details

Selective 12-Human Lipoxygenase Inhibitors for the Treatment of Diabetes and Clotting

Inventors: David Maloney (NCATS), Theodore Holman (University of California, Santa Cruz), Jerry Nadler (Eastern Virginia Medical School), Michael Holinstat (Thomas Jefferson University), Anton Simeonov (NCATS), Ajit Jadhav (NCATS)

Abstract: This invention discloses small molecule inhibitors of human 12-lipoxygenase (12-hLO). 12-lipoxygenase expression, activation, and lipid metabolites have been implicated in type 1 and type 2 diabetes, cardiovascular disease, hypertension, Alzheimer's, and Parkinson's disease. The development of 12-hLO inhibitors may be a potent intracellular approach to decreasing the ability of platelets to form large clots in response to vessel injury or activation of the coagulation pathway. Abstract Details

Small Molecule Activators of Human Pyruvate Kinase

Inventors: Douglas Auld (NCATS), Matthew Boxer (NCATS), James Inglese (NCATS), Jian-kang Jiang (NCATS), Amanda Skoumbourdis (NCATS), Craig Thomas (NCATS)

Abstract: NIH investigators have discovered a series of small compounds with the potential to treat a variety of cancers as well as hemolytic anemia. Contrary to most cancer medications, these molecules can be non-toxic to normal cells because they target a protein specific to the metabolic pathways in tumors, thus representing a significant clinical advantage over less-specific chemotherapeutics. Abstract Details

Small-Molecule Inhibitors of Human Galactokinase for the Treatment of Galactosemia and Cancers

Inventor: Matthew Boxer (NCATS)

Abstract: Lactose, found in dairy products and other foods, is comprised of two simple sugars, glucose and galactose. In galactosemia, where galactose is not properly metabolized, build-up of toxic compounds, such as galactose-1-phosphate, can lead to liver disease, renal failure, cataracts, brain damage, and even death if this disorder is left untreated. Currently, the only treatment for galactosemia is elimination of lactose and galactose from the diet, but in some cases this is not sufficient to avoid long-term complications from the disorder. Abstract Details

Small Molecule Kinase Inhibitors for the Potential Treatment of Down's Syndrome and Alzheimer's Disease and for Studies of Alternate Gene Splicing

Inventor: Craig Thomas (NCATS)

Abstract: NIH investigators have discovered a series of potent, selective small molecule inhibitors of cdc2-like kinases (Clk) and dual-specificity tyrosine-regulated kinase 1A (Dyrk1A) with potential as modulators of gene splicing and for the treatment of Down's syndrome and Alzheimer's disease. Abstract Details

Small-Molecule Modulators of Lipid Storage for Treatment of Obesity, Atherosclerosis, Metabolic Syndrome and Lipid Storage Diseases

Inventor: Matthew Boxer (NCATS)

Abstract: Lipid droplets are key organelles involved in lipid homeostasis. In normal physiology, these droplets are formed in response to elevated fatty acid levels, and are broken down when needed for energy production. Imbalances in lipid homeostasis trigger compensatory alterations in metabolism that can lead to diseases such as obesity, atherosclerosis, and metabolic syndrome. Abstract Details

Small Molecule Neuropeptide S (NPS) Antagonists for the Treatment of Addictive Disorders, Mood and Anxiety Disorders, and Sleep Disorders

Inventors: Markus Heilig (NIAAA), Ke Liu (NHGRI), Juan Marugan (NCATS), Samarjit Patnaik (NCATS), Noel Southall (NCATS), Wei Zheng (NCATS)

Abstract: The inventors have developed NPSR antagonists that hold the potential for being clinically useful treatments for alcohol and drug addiction. Abstract Details

Substituted 3-Oxo-3, 4-Dihydroquinoline-2H-Benzo [B] [1,4] Oxazine-7-Sulfonamides and 2-Oxo-1,2,3,4-Tetrahydroquinoline-6-Sulfonamides Small Molecule Activators of Human Pyruvate Kinase

Inventors: Douglas Auld (NCATS), Matthew Boxer (NCATS), Min Shen (NCATS), Craig Thomas (NCATS)

Abstract: NIH investigators have discovered a series of small compounds with the potential to treat a variety of cancers as well as hemolytic anemia. Contrary to most cancer medications, these molecules can be non-toxic to normal cells because they target a protein specific to the metabolic pathways in tumors, thus representing a significant clinical advantage over less-specific chemotherapeutics. Abstract Details

Substituted Oxadiazole 2-Oxides as a Treatment of Schistosomiasis

Inventors: Ganesha Bantukallu (NCATS), David Maloney (NCATS), Ahmed Sayed (Illinois State University), Anton Simeonov (NCATS), Craig Thomas (NCATS), David Williams (Illinois State University)

Abstract: Available for licensing and commercial development are pharmaceutical compositions and methods for the treatment of schistosomiasis in mammals. The various compositions are based on a number of compounds derived from 1,2,5-oxadiazole that are potent inhibitors of thioredoxin glutathione reductase (TGR), a critical parasite redox protein. Abstract Details

Inquiries

For technology and partnership inquiries, contact the NCATS Office of Strategic Alliances or call 301-217-4679.