20 results about "Hematological disorders" patented technology

Novel compounds that act as antagonists to human GPR39 protein are disclosed. Pharmaceutical compositions and methods of use for antagonists to human GPR39 protein are disclosed. In particular, methods of using the antagonists in the treatment of diseases or conditions including cardiovascular conditions, endocrine system and hormone disorders, cancer disorders, metabolic diseases, gastrointestinal and liver diseases, hematological disorders, neurological disorders and respiratory diseases are disclosed herein.

The present invention pertains to periostin compounds for use in the prevention and treatment of haematological complications, such as adverse events from therapy or haematological diseases. In context of the present invention a therapeutic was developed for enhancing haematopoiesis in patients and to support haematopoietic stem cell (HSC) transplantation (HSCT) by administration of periostin compounds to patients or stem cell donors, or by contacting HSC directly with periostin compounds, for example ex vivo, to improve a transplant HSC preparation. The present invention provides periostin derived compounds such as polypeptides, peptides, nucleic acids, and other periostin-derived agents, that are used both in therapeutic applications and for improving haematopoiesis, for example in stem cell donor subjects or to treat HSC in vitro.

This invention discloses the application and method of a STAT1-targeting inhibitor in the treatment of immune thrombocytopenic purpura (ITP). It employs ivermectin, a STAT1 nuclear translocation inhibitor, and fludarabine, a STAT1 activation inhibitor. By injecting either the activation inhibitor or the nuclear translocation inhibitor, the platelet count in a mouse model of ITP is increased. Fludarabine, a fluorinated nucleotide analog of vidarabine, is non-radioactive and a small-molecule phosphorylation inhibitor. Ivermectin is a small-molecule inhibitor of nuclear translocation mediated by α / β1 introgression protein. Both have high bioavailability and are widely used to treat various hematological diseases with good safety profiles. Their application in treating ITP is safe, effective, and shows high compliance.

The present invention relates to products and compositions and their uses. In particular, the invention relates to nucleic acid products that interfere with the expression of the TMPRSS6 gene or inhibit its expression and to therapeutic uses such as for the treatment of hemochromatosis, porphyria and hematological disorders such as β-thalassemia, sickle cell disease and iron overload from transfusions or myelodysplastic syndrome.

To provide lentiviral vector (LV) formulations with improved stability and suitable for systemic administration, and pharmaceutical compositions comprising such LV formulations, and also to provide methods for treating disorders, especially blood disorders, using systemic administration of LV formulations.SOLUTION: Provided is a recombinant lentiviral vector preparation comprising: (a) a therapeutically effective amount of a recombinant lentiviral vector; (b) a TRIS-free buffer system; (c) a salt; (d) a surfactant; and (e) a carbohydrate, wherein the pharmaceutical composition is suitable for systemic administration to a human patient.SELECTED DRAWING: None

The present invention relates to compounds and compositions for the treatment of hematological disorders. The present invention provides methods for the treatment of hematological disorders, such as acute myeloid leukemia, using substituted heterocyclic compounds and pharmaceutically acceptable salts thereof. The compounds inhibit IRAK4 and FLT-3 kinases.

The invention provides a chemical entity of Formula (I):wherein R1, R2, X, Y and Z have any of the values described herein, and compositions comprising such chemical entities; methods of making them; and their use in a wide range of methods as disclosed herein, including metabolic and reaction kinetic studies; detection and imaging techniques; radioactive treatments; modulating and treating disorders mediated by PDE2 activity; treating neurological disorders, CNS disorders, dementia, neurodegenerative diseases, and trauma-dependent losses of function; treating stroke, including cognitive and motor deficits during stroke rehabilitation; facilitating neuroprotection and neurorecovery; enhancing the efficiency of cognitive and motor training, including animal skill training protocols; and treating peripheral disorders, including hematological, cardiovascular, gastroenterological, and dermatological disorders.

The present disclosure provides improved compositions and methods for detecting, diagnosing, prognosing, monitoring, and treating hematological disorders including multiple myeloma in a subject. In particular, the disclosure provides methods for detecting IL4I1 in subjects to reliably diagnose, predict survival, or monitor multiple myeloma in the subject and methods for inhibiting IL4I1 to treat multiple myeloma in the subject.

The present invention relates to a controlled-release pharmaceutical composition comprising at least a controlled-release pharmaceutical dosage form containing EPAG, the controlled-release pharmaceutical composition being suitable for local injection into an affected area. The present invention also relates to a controlled-release pharmaceutical composition in powder form for preparing the pharmaceutical composition suitable for local injection, and a kit for preparing the pharmaceutical composition comprising an aqueous injection vehicle and a controlled-release dosage form or a mixture of controlled and immediate-release dosage forms containing EPAG, the pharmaceutical composition optionally including an excipient. The composition is useful for local injection in patients to improve stem cell homing and / or treat non-malignant hematological disorders, malignant hematological diseases, primary immunodeficiencies, autoimmune diseases, inborn errors of metabolism, and / or degenerative diseases / injuries.

The present disclosure generally relates to blood disorders (e.g., cold agglutinin hemolytic anemia (cold agglutinin disease), cold antibody hemolytic anemia, ABO incompatibility acute hemolytic reaction, warm agglutinin hemolytic anemia, warm antibody hemolytic anemia, warm antibody autoimmune hemolytic anemia (WAIHA)), autoimmune hemolytic anemia (AIHA), autoimmune thrombocytopenia, antiphospholipid syndrome, Evans syndrome, red blood cell alloimmunization, Felty syndrome, neonatal alloimmune thrombocytopenia, heparin-induced thrombocytopenia (HIT), ... The present invention relates to a method for preventing, reducing the risk of developing, or treating a complement pathway-induced thrombocytopenia and thrombosis (HITT), thrombotic thrombocytopenic purpura (TTP), immune thrombocytopenic purpura (ITP), thrombocytopenia, thrombosis, vasculitis, lupus nephritis, systemic lupus erythematosus (SLE), glomerulonephritis, antiphospholipid syndrome (APS), infectious disease, or drug-induced hematological disorder, comprising administering to a subject an inhibitor of the complement pathway.

The present invention provides methods of treating hematological disorders such as acute myeloid leukemia, using substituted heterocyclic compounds and pharmaceutically acceptable salts thereof. The compounds inhibit the kinases IRAK4 and FLT-3.

The invention relates to compositions and methods for treating patients with hematological proliferative disorders who are ineligible for treatment with standard intensive chemotherapy, using low intensity treatment with CPX-351, a liposomal composition of daunorubicin and cytarabine.

The present disclosure provides pyrimidine nucleoside compounds, including torcitabine, thymidine, deoxyuridine, deoxycytidine and uridine, and methods to treat telomere biology disorders (TBDs) and aging-related diseases, including hematological disorder, liver disease, or hepatic fibrosis, comprising administering to a subject diagnosed with said telomere biology disorder a therapeutically effective amount of said compounds.

The present disclosure features chemical entities (e.g., compounds or pharmaceutically acceptable salts thereof) that degrade and / or otherwise modulate (e.g., inhibit) NIMA-related kinase 7 (NEK7). The chemical entities are useful, for example, in treating subjects having one or more conditions or diseases associated with NLRP3 inflammasome activation (e.g., human subjects). The conditions or diseases include, but are not limited to, autoinflammatory and autoimmune conditions (e.g., gout, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease), cardiovascular and metabolic conditions (e.g., pericarditis, atherosclerosis, type 2 diabetes mellitus, diabetes mellitus, diabetes mellitus, diabetes mellitus, diabetes mellitus, diabetes mellitus, diabetes mellitus, diabetes mellitus, and the like). ), fibrotic disorders (e.g., interstitial lung disease, chronic kidney disease), hematological disorders (e.g., inflammatory anemia), and ocular disorders (e.g., macular degeneration). In embodiments, although not desired to be constrained theoretically, the chemical entities described herein are believed to directly target (e.g., bind directly) NEK7, thereby altering (e.g., attenuating) the inflammatory response modulated by the NLRP3 inflammasome. The disclosure also features compositions containing the entities as well as methods of using and making the entities.

The present invention comprises novel aromatic molecules, which can be used in the treatment of pathological conditions, such as cancer, skin diseases, muscle disorders, and immune system-related disorders such as disorders of the hematopoietic system including the hematologic system in human and veterinary medicine.

Provided herein are methods and compositions for the inhibition complement component (C5). The methods and compositions involve the use of aptamer-containing molecules, comprising one or more moieties that bind to and inhibit C5. The application further provides aptamer-containing molecules for the treatment of retinal diseases and / or hematological diseases.

A chemical entity of Formula (I):wherein V, W, Y, and Z, have any of the values described herein, and compositions comprising such chemical entities; methods of making them; and their use in a wide range of methods, including metabolic and reaction kinetic studies; detection and imaging techniques; radioactive treatments; modulating and treating disorders mediated by PDE2 activity; treating neurological disorders, CNS disorders, dementia, cognitive disorders, neurodegenerative diseases, and trauma-dependent losses of function; enhancing the efficiency of cognitive and motor training, including in stroke or TBI rehabilitation; and treating peripheral disorders, including hematological, cardiovascular, gastroenterological, dermatological, inflammatory, and pain disorders.

The invention relates to compositions and methods for treating patients with hematological proliferative disorders with CPX-351, a liposomal composition of daunorubicin and cytarabine, and venetoclax.

Compositions and methods useful for the treatment of hemoglobinopathies and hematological diseases are disclosed herein. The compositions include an actinium-225 labeled anti-CD45 antibody (BC8) formulated as a single patient dose that is wholly deliverable to a patient in a single dose. The actinium-225 labeled anti-CD45 may be administered alone or in combination with additional therapeutic agents, such as other immunotherapeutics or a radiosensitizing agent, or additional therapeutic interventions, such as bone marrow transplant or adoptive cell therapies.