Abstract: The present disclosure relates generally to compounds useful in treatment of conditions associated with mutant isocitrate dehydrogenase (mt-IDH), particularly mutant IDH1 enzymes. Specifically, the present invention discloses compound of formula (IA), which exhibits inhibitory activity against mutant IDH1 enzymes. Method of treating conditions associated with excessive activity of mutant IDH1 enzymes with such compound is disclosed. Uses thereof, pharmaceutical composition, and kits are also disclosed.

Abstract: The invention refers to the use of isoxazole derivatives to prepare medicament able to induce fetal hemoglobin (HbF) synthesis in ?-thalassemia and sickle cell disease (SCD) patients.

Abstract: The present invention relates to the (S) enantiomeric form of certain 6-chromanol derivatives for use as a medicament. Especially, the present invention relates to the (S) enantiomeric form of a 6-chromanol derivative for use as a medicament wherein said 6-chromanol derivative is chosen from the group consisting of (6-hydroxy-2,5,7,8-tetramethylchroman-2-yl)(piperazin-1-yl)methanone; N-(benzyl)-6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxamide; N-(phenyl)-6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxamide; methyl 4-(6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxamido)benzoate; (6-hydroxy-2,5,7,8-tetramethylchroman-2-yl)(morpholino)methanone, and pharmaceutically acceptable salts or bases thereof and pharmaceutically acceptable salts thereof.

Abstract: Therapeutic uses P2X7 inhibition and inhibition of IRAK1 and/or IRAK4, methods protecting a cell, and screening methods for identifying inhibitors are described herein.

Abstract: The invention is directed to compounds of formula (I): and salts thereof. The compounds of the invention are inhibitors of kinase activity, in particular PI3-kinase activity.

Abstract: A composition for the treatment of cellulitis is provided that includes an antibiotic. The composition may also include an analgesic, an anaesthetic, or a combination thereof. A method use is also provided by which the composition is applied topically or to affected skin. A method of use is also provided prior to a medical procedure involving an incision through the skin of a patient in a predetermined area, the method including the application, to the predetermined area and prior to the procedure of the composition.

Abstract: The current methods and compositions provide for therapeutic approaches to treating hepatocellular carcinoma (HCC) and other types of cancer including, for example, pancreatic and colon cancer. Accordingly, certain aspects of the disclosure relates to methods and compositions for treating HCC, pancreatic and colon cancer using one or more small molecule inhibitors disclosed herein. In certain embodiments, the small molecule inhibitor is a benzothiazine, a sulfonamide, a thiazolidinone or other chemical compound.

Abstract: A method of forming a dietary supplement can include steps of creating a composition of matter comprising tianeptine, sakae naa, and optionally, at least one of kava, CDP Choline, and Alpha GPC; and providing the composition of matter in a liquid or solid form. The ingredient of tianeptine can be tianeptine sodium, tianeptine free acid, or both. Providing the composition of matter can include filling a container with the composition of matter.

Abstract: The invention provides novel pharmaceutical compositions for the treatment of increased intraocular pressure based on semifluorinated alkanes which are useful as carriers for a broad range of active ingredients. Preferred active ingredients include poorly water-soluble prostaglandin analogues. The compositions can be administered topically into the eye. The invention further provides kits comprising such compositions.

Abstract: According to various embodiments of this disclosure, pharmaceutical compositions comprising solubilized estradiol are provided. In various embodiments, such compositions are encapsulated in soft capsules which may be vaginally inserted for the treatment of vulvovaginal atrophy.

Abstract: This invention provides for a method of h eating secondary adrenal insufficiency by co-administrating therapeutically effective amounts of a glucocorticoid and a glucocorticoid receptor antagonist to the patient in need thereof. In some embodiments, the method includes the proviso that the patient not be otherwise in need of treatment with a glucocorticoid and a glucocorticoid receptor antagonist. The treatment method can increase the patient's morning or basal cortisol level to at least about 12 ?g/dL or a standard control level, and in turn, expedite significantly the recovery of the HPA axis. The method provided herein can improve health outcomes and life-threatening complications associated with secondary adrenal insufficiency.

Abstract: Described herein are methods of promoting remyelination in a subject suffering from demyelination diseases by administering to the subject a combination of steroid hormones and Hedgehog signaling pathway modulators. Also described are methods of administering the combination of drugs, wherein the combination of drugs are in compositions adapted for nasal administration.

Abstract: A method of improving or maintaining muscle strength of aging muscle tissue.

Abstract: The present invention relates to compositions for inhibiting inflammation. The composition can comprise one or more natural products in the form of a nutraceutical or functional food. The present invention further relates to the combinatorial use of nutritional supplements and nutraceuticals for the prevention and/or treatment of sterile inflammation and conditions associated with sterile inflammation.

Abstract: Methods of normalizing vitamin B12 levels in patients with low vitamin B12 and methods of normalizing intersubject variability in the treatment of such patients are described. Methods of reducing MMA and/or homocysteine levels, and pharmaceutical compositions useful to effect such changes are also described.

Abstract: The present invention relates to a novel combination drug in a solid oral dosage form comprising, as one of the three active ingredients, elsulfavirine sodium that may be suitable for medical use when treating viral infections including HIV and HBV.

Abstract: Injectable dosages and formulations of fosnetupitant and pharmaceutically acceptable salts thereof are provided that are efficacious, chemically stable and physiologically balanced for safety and efficacy.

Abstract: This invention relates to a method and composition for inducing allergen tolerance in a patient suffering from an atopic allergy or allergic asthma; hence reducing allergy symptoms in these patients.

Abstract: A method for preventing or mitigating an acute allergic response in a subject is disclosed. The method includes a step of administering a nutritional composition to the subject. The nutritional composition includes at least one of an acidic or a neutral HMO, but does not include an N-acetyl-lactosamine.

Abstract: D-ribose is administered to patients suffering an acute myocardial infarction during first response care, in order to prevent cardiac compromise. In those patients able to ingest fluids, two to five grams of D-ribose is administered orally. In a patients unable to ingest fluids, or in a patient with and intravenous line, pyrogen-free D-ribose is administered intravenously at a rate of 50-300 mg/kg/hour.

Abstract: Oral administration of a solid dosage form of the present invention comprising an effective amount of rifabutin, an effective amount of clarithromycin, an effective amount of clofazimine, and an effective amount of an absorption enhancer, is used to treat a subject suffering from, or susceptible to, Mycobacterium avium subspecies paratuberculosis infection. In an embodiment, the solid dosage form is sufficiently designed to result in a reduction in the increased metabolism of clarithromycin caused by rifabutin. In an embodiment, the solid dosage form is sufficiently designed to result in a reduction in the metabolism of rifabutin caused by clarithromycin. In an embodiment, the solid dosage form is sufficiently designed to result in a reduction in risk of a subject developing leucopenia or uveitis as a result of rifabutin.

Abstract: The disclosure relates generally to methods and reagents for reducing or shutting down lactation in a non-human mammalian subject. In particular, the disclosure relates to a method of reducing or shutting down lactation in a non-human mammalian subject by administering to the subject by intramammary infusion an agent which activate the OAS2 signalling pathway or induce expression of OAS2. In some examples, the methods and reagents of the disclosure may be useful for the prevention of mastistis in a non-human mammalian subject, such as a dairy cow.

Abstract: Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure relates to methods for treating a subject having a disease associated with RAN protein translation by administering the subject a modulator of eIF3 or an eIF3 subunit, or an antibody that bind to a RAN protein.

Abstract: The present invention provides compositions comprising a UBC antagonist and methods of use thereof for treating cancer in a patient. In some embodiments, the cancer patient may have a reduced expression level of a UBB gene product. Further provided are reagents and methods for detection of a UBB and/or UBC gene product.

Abstract: Provided is a homogeneous polysaccharide ESP-B4, method for producing the same and use thereof. The ESP-B4 is a polysaccharide monomer, which is isolated from the traditional Chinese medicine Ephedra sinica Stapf, Ephedra intermedia Schrenk et C. A. Mey, or Ephedra equisetina Bge. HPSEC-ELSD shows a single symmetrical peak. The chemical construction of ESP-B4 is an acidic heterosaccharide. It is composed of xylose, arabinose, glucose, rhamnose, mannose, galactose, glucuronic acid and galacturonic acid with a molar ratio of 1.0:4.5:1.0:2.0:5.5:1.5:50. The molecular weight of ESP-B4 is 2.37×107 Da, and the content of galacturonic acid was 75.2%. The application of ESP-B4 is significant activity against infectious acute lung injury, respiratory distress syndrome, asthma, pneumonia, trachea and bronchitis. Therefore, it can be used for severe infectious acute lung injury caused by SARS virus, influenza a virus, avian influenza virus.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.

Abstract: One purpose of the present invention is to provide a high molecular weight glucan having both properties low digestion rate and high digestibility. A high molecular weight glucan that has property digested slowly and contains almost no indigestible ingredients is produced by enzymatic reactions of (1) a specific concentration of branching enzyme and (2) 4-?-glucanotransferase and/or an exo-type amylase to a branched glucan used as a substrate.

Abstract: Pharmaceutical compositions for and methods of treating an animal, including a human, and methods of preparing such compositions. The pharmaceutical compositions contain crosslinked amine polymers and may be used, for example, to treat diseases or other metabolic conditions in which removal of protons and/or chloride ions from the gastrointestinal tract would provide physiological benefits such as normalizing serum bicarbonate concentrations and the blood pH in an animal, including a human.

Abstract: Provided are compositions, methods, and solutions for generating aqueous glucomannan solutions with hydrogen compositions greater than 100 parts per billion. Said glucomannan solutions have application in nutritional, therapeutic, and energy fields.

Abstract: The invention relates to compositions containing selenite-containing compounds and pharmaceutically acceptable acids, selected from citric acid, acetic acid, malic acid, carbonic acid, sulphuric acid, nitric acid, hydrochloric acid, fruit acids or mixtures thereof, for use for treating cervical inflammations, dysplasia and/or carcinomas. The invention further relates to methods of using such compositions.

Abstract: The present invention in various aspects and embodiments provides methods and formulations for treating inflammatory conditions of the skin and/or conditions involving compromised skin barrier function. Such diseases include blistering diseases of the skin, hereditary defects in skin barrier function, hyperproliferative conditions involving the skin, conditions associated with aging or damaged skin, immunological disorders involving the skin, among others.

Abstract: Provided herein are topical formulations containing copper ions and methods of treating radiation dermatitis, damage caused by radiation therapy, or other radiation-induced damage conditions in various areas of the body using such formulations. Methods of treating radiation dermatitis using topical copper ion treatments are provided. A topical treatment in its basic form comprises a biocompatible copper ion solution or suspension obtained by leaching of the copper ions from copper metal. The copper ion solution or suspension is combined with various carriers to form the copper ion treatment including creams, gels, lotions, foams, pastes, tampons, solutions, suppositories, body wipes, wound dressings, skin patches, and suture material. Methods of making the copper ion solution or suspension from solid copper metal in a biocompatible solution are also provided.

Abstract: Provided herein are methods for delaying or inhibiting T cell maturation or differentiation in vitro for a T cell therapy, comprising contacting one or more T cells from a subject in need of a T cell therapy with an AKT inhibitor and at least one of exogenous Interleukin-7 (IL-7) and exogenous Interleukin-15 (IL-15), wherein the resulting T cells exhibit delayed maturation or differentiation. In some embodiments, the method further comprises administering the one or more T cells to a subject in need of a T cell therapy.

Abstract: The invention generally relates to the treatment of cancer with FLT3 targeting agents and kinase inhibitors. In particular, the invention relates to adoptive immunotherapy of Acute Myeloid Leukemia (AML) with chimeric antigen receptor (CAR)-modified T cells specific for FMS-like tyrosine kinase (FLT3) in combination with FLT3 inhibitors.

Abstract: Presented herein are systems and methods of producing “universal” and/or “off-the-shelf” CAR-T compositions suitable for cancer therapy to be administered to one or more individuals. A CAR-T composition is a composition comprising one or more types of chimeric antigen receptor T cells (CAR-T). The iPSCs and/or cell lines, and any iPSC-derived CAR-T compositions derived therefrom, are identified as compatible with one or more individuals using an identification of a cell type indicative of compatibility (e.g., HLA match and/or ABO blood match and/or RHD blood match). The compatible cells are then retrieved from a managed HLA-indexed (and/or otherwise indexed) repository or are derived from a biological sample of a donor. The retrieved compatible cells are then used to derive iPSC-derived CAR-T compositions, wherein the derived compositions are suitable for therapy of one or more individuals.

Abstract: The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.

Abstract: Multi-step methods for the in vitro production of enucleated red blood cells and the enucleated red blood cells thus prepared are provided. Such enucleated red blood cells may express fusion proteins comprising an antigen binding protein which allows the red blood cell to bind a toxin or an antigen of a pathogen. Also described herein are methods for neutralizing a toxin or pathogen in a subject by administering enucleated red blood cells that express any of the fusion proteins provided herein.

Abstract: Provided herein are compositions, uses thereof, and methods for treating Autism or Autism Spectrum Disorder (ASD) in a subject in need thereof, wherein the compositions comprise a whey protein isolate and/or whey protein concentrate.

Abstract: Provided herein are methods and compositions for dynamically controlling and targeting multiple immunosuppressive mechanisms in cancer. Some aspects provide cells engineered to produce multiple effector molecules, each of which modulates a different immunosuppressive mechanisms of a tumor, as well as methods of using the cells to treat cancer, such as ovarian, breast, or colon cancer.

Abstract: A cell product for treatment of epidermolysis bullosa, comprising a SSEA-3-positive pluripotent stem cell (Muse cell) derived from a mesenchymal tissue in a living body or a cultured mesenchymal cell. Preferably, the epidermolysis bullosa is epidermolysis bullosa simplex, junctional epidermolysis bullosa, or dystrophic epidermolysis bullosa.

Abstract: A method of radiation-free hematopoietic stem cell (HSC) transplantation comprises administering to a mammalian subject one or two doses of 2 to 10 mg/kg body weight of a purine base analog, such as 6TG as a pre-conditioning step. The method further comprises engrafting into the subject hypoxanthine-guanine phosphoribosyltransferase (HPRT)-deficient donor HSCs within 48 to 72 hours of the pre-conditioning step; and administering to the subject about 1 to 5 mg/kg of the purine base analog every two to four days for two to eight weeks following the engrafting step. The method is performed in the absence of pre-conditioning via radiation. The subject is therefore not treated with myeloablative radiation in preparation for transplantation, and thus the subject is free of myeloablative radiation-induced toxicity.

Abstract: Provided herein are novel methods and compositions utilizing adipose tissue-derived stromal stem cells for treating fistulae.

Abstract: Compositions comprising collagen and insulin-producing cells are provided. Processes for making such a collagen and insulin-producing cell compositions are also provided. Methods for controlling, or lowering blood glucose levels and treating metabolic disorders in mammals, including type 1 diabetes, with such compositions are further provided. Methods to prolong insulin-producing cell viability and function in vitro or during transport are also provided.

Abstract: The invention provides compositions comprising bacterial strains for treating and preventing inflammatory and autoimmune diseases.

Abstract: A method for degrading a biofilm on a surface is provided. A method of preventing formation of a biofilm on a surface is provided. The method includes administering, e.g., applying, ammonia oxidizing microorganisms, e.g., a preparation comprising ammonia oxidizing bacteria, to the surface. Preparations comprising ammonia oxidizing microorganisms for biofilm treatment are also provided.

Abstract: A method of treating pulmonary hypertension in a subject is provided. The method comprises administering a preparation comprising ammonia oxidizing microorganisms to the subject, thereby treating the pulmonary hypertension. Related preparations, kits, and devices also provided.

Abstract: Ammonia oxidizing microorganism preparations for delivery to the urogenital system, kits including ammonia oxidizing preparations for delivery to the urogenital system, and devices for administering ammonia oxidizing preparations to the urogenital system are provided. Methods of introducing ammonia oxidizing microorganisms to the urogenital system are provided. Methods of treating disorders, including urogenital disorders and inflammatory disorders, with ammonia oxidizing microorganism preparations are provided.

Abstract: The invention provides compositions comprising bacterial strains for treating and preventing neurodegenerative disorders.

Abstract: The present invention relates to methods and compositions for diagnosing and treating degenerative mitral valve disease in a canine. In one embodiment, a method of diagnosing early stage degenerative mitral valve disease in a canine can comprise measuring a normalized relative abundance of a biomarker selected from the group consisting of Erysipelatoclostridium, Ruminococcaceae UCG014, Butyricicoccus, Faecalibacterium, and combinations thereof, and determining that the canine has early stage degenerative mitral valve disease if the Erysipelatoclostridium is from 0 to 0.5 normalized relative abundance, the Ruminococcaceae UCG014 is from 0 to 0.1 normalized relative abundance, the Butyricicoccus is from 0 to 0.1 normalized relative abundance, or the Faecalibacterium is from 0 to 0.1 normalized relative abundance.

Abstract: The present invention relates to an extracellular vesicle derived from Lactobacillus paracasei and a use thereof, and more particularly, to a composition for alleviating, preventing or treating an inflammation disease and a cancer, which comprises an extracellular vesicle derived from Lactobacillus paracasei, which may effectively inhibit various inflammation disease, as an active ingredient, and the like.