10 results about "Hematological disorders" patented technology

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 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 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 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.

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.