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196 results about "Sickle Cell Diseases" patented technology
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Methods and low dose regimens for treating red blood cell disorders
ActiveUS20110251149A1Increase volumeIncrease the number ofBiocideCarbohydrate active ingredientsBeta thalassemiaRegimen
Disclosed herein are methods and low dose regimens for increasing fetal hemoglobin levels in patients with red blood cell disorders, such as beta thalassemia, sickle cell disease, other anemias, or blood loss. Fetal and total hemoglobin levels and red blood cell counts are increased by administering 2,2-dimethylbutyrate (DMB) alone or in combination with hydroxyurea, decitabine or an HDAC inhibitor. Treatment can be continued for at least two weeks.
Owner:HEMAQUEST PHARMA INC +1
Method for assessing disease states by profile analysis of isolated circulating endothelial cells
ActiveUS7901950B2Confident diagnosticConfident prognosticBioreactor/fermenter combinationsBiological substance pretreatmentsAntigenCirculating endothelial cell
Owner:MENARINI SILICON BIOSYSTEMS SPA
Anti-sickling agents
The provides compounds for the treatment of sickle-cell disease. In particular, the invention provides 5-membered heterocyclic anti-sickling agents that are highly effective and non-toxic, and methods for their use. The compounds include analogues and derivatives of naturally occurring occurring 5-hydroxymethyl-2-furfuraldehyde, 5-Ethyl-2-furfuraldehyde, 5-Methyl-2-furfuraldehyde, and 2-furfuraldehyde, and prodrug forms of the compound.
Owner:VIRGINIA COMMONWEALTH UNIV
Compositions and methods for the treatment of hemoglobinopathies
InactiveUS20150166969A1Improve the level ofAvoid the insertion of vector sequencesFusion with DNA-binding domainSugar derivativesThalassemiaGlobin genes
Provided are compositions and methods for the treatment of hemoglobinopathies such as thalassemias and sickle cell disease. Compositions and methods include one or more endonuclease(s) or endonuclease fusion protein(s), including one or more homing endonuclease(s) and / or homing endonuclease fusion protein(s) and / or CRISPR endonuclease(s) ad / or CRISPR endonuclease fusion protein(s): (a) to disrupt a Bcl11a coding region; (b) to disrupt a Bcl11a gene regulatory region; (c) to modify an adult human β-globin locus; (d) to disrupt a HbP silencing DNA regulatory element or pathway, such as a Bcl11a-regulated HbP silencing region; (e) to mutate one or more γ-globin gene promoter(s) to achieve increased expression of a γ-globin gene; (f) to mutate one or more δ-globin gene promoter(s) to achieve increased expression of a δ-globin gene; and / or (g) to correct one or more β-globin gene mutation(s).
Owner:NAT INST OF HEALTH DIRECTOR DEITR
Method of treating erythropoietin hyporesponsive anemias
InactiveUS20100266591A1Peptide/protein ingredientsAntibody ingredientsHemoglobinopathyRed blood cell
The invention relates to methods of using compositions comprising EPO-mimetic peptides to treat anemia. The invention relates to methods of treating disorders characterized by the insufficient amounts of erythrocytes and hemoglobulin in the blood due to myelodysplastic syndrome (MDS) or by hemoglobinopathies, such as alpha- or beta-thalessemia or sickle cell disease.
Owner:CENTOCOR ORTHO BIOTECH
Triaryl methane compounds and analogues thereof useful for the treatment or prevention of sickle cell disease or diseases characterized by abnormal cell proliferation
InactiveUS6028103AReducing sickle erythrocyte dehydrationDelaying occurrenceHalogenated hydrocarbon active ingredientsBiocideAbnormal cellSickled erythrocytes
The present invention provides a class of chemical compounds useful as efficacious drugs in the treatment of sickle cell disease and diseases characterized by unwanted or abnormal cell proliferation. The active compounds are substituted triaryl methane compounds or analogues thereof where one or more of the aryl groups is replaced with a heteroaryl, cycloalkyl or heterocycloalkyl group and / or the tertiary carbon atom is replaced with a different atom such as Si, Ge, N or P. The compounds inhibit mammalian cell proliferation, inhibit the Gardos channel of erythrocytes, reduce sickle erythrocyte dehydration and / or delay the occurrence of erythrocyte sickling or deformation.
Owner:HARVARD COLLEGE PRESIDENT & FELLOWS OF +2
Method to treat sickle cell disease
ActiveUS20080009460A1Inhibit inflammationReduced responseBiocideSugar derivativesAgonistSickle Cell Diseases
The present invention provides a therapeutic method for treating an inflammatory response caused by a sickle cell crisis, comprising administration of an effective amount amount of an A2A adenosine receptor agonist. Optionally, the method includes administration of a type IV PDE inhibitor (e.g., rolipram).
Owner:VIRGINIA PATENT FOUND UNIV OF +1
Methods of treatment with Syk inhibitors
ActiveUS20050232969A1Inhibit inflammationBiocideOrganic chemistryPercent Diameter StenosisSickle Cell Diseases
The present invention provides novel compositions and methods for inhibiting restenosis, thrombosis, and / or inflammation in a patient undergoing a vascular intervention. More particularly, the present invention provides intravascular devices coated with one or more spleen tyrosine kinase (Syk) inhibitors. Methods for inhibiting restenosis, thrombosis, and / or inflammation in a patient by treatment with such intravascular devices are also provided. In addition, the present invention provides methods for treating sickle cell disease using Syk inhibitors.
Owner:ALEXION PHARM INC
Methods of treating sickle cell disease
InactiveUS20050112124A1Decrease SS-RBC/leukocyte adherenceReduce adhesionImmunoglobulins against cytokines/lymphokines/interferonsImmunoglobulins against cell receptors/antigens/surface-determinantsWhite blood cellSickle Cell Diseases
The present invention relates to methods of treating sickle cell disease comprising reducing, in a subject in need of such treatment, the adherence between sickle RBCs and leukocytes. It is based, at least in part, on the discovery that leukocytes play a direct role in the initiation of venular occlusion. The present invention further provides for methods for identifying agents which decrease SS-RBC / leukocyte adherence and for animal models which may be used to further elucidate the mechanism of vaso-occlusion in sickle cell crises.
Owner:MT SINAI SCHOOL OF MEDICINE
Method of treating cardio pulmonary diseases with no group compounds
Treatment of pulmonary disorders associated with hypoxemia and / or smooth muscle constriction and / or inflammation comprises administering into the lungs as a gas compound with an NO group which does not form NO2 / NOx in the presence of oxygen or reactive oxygen species at body temperature. Treatment of cardiac and blood disorders, e.g., angina, myocardial infarction, heart failure, hypertension, sickle cell disease and clotting disorders, comprises administering into the lungs as a gas, a compound which reacts with cysteine in hemoglobin and / or dissolves in blood and has an NO group which is bound in said compound so that it does not form NO2 / NOx in the presence of oxygen or reactive oxygen species at body temperature. Exemplary of the compound administered in each case is ethyl nitrite.
Owner:DUKE UNIV
Red blood cells covalently bound with two different polyethylene glycol derivatives
InactiveUS6312685B1Reduce decreaseSignificant comprehensive benefitsBiocideDead animal preservationDiseaseBlood plasma
Living cells are modified at their surface with specially selected polymers. Covalently attaching specially selected polyethylene glycol (PEG) derivatives to the surface of red blood cells (RBC) in aqueous media under mild conditions is a preferred example. The selected PEG derivatives dramatically reduced aggregation and low shear viscosity of RBC resuspended in autologous plasma, and inhibited RBC agglutination by blood group-specific antibodies. The morphology and deformability of the PEG-treated cells were unaltered. PEG coating of the RBC surface is applicable to the treatment of a variety of diseases characterized by vaso-occlusion or impaired blood flow, e.g., myocardial infarction, shock, and sickle cell disease. An infusion solution is prepared containing red blood cells covalently bound to a PEG derivative having a molecular weight of between 2,000 and 5,000 Daltons and a PEG derivative having a molecular weight between 10,000 and 35,000 Daltons.
Owner:FISHER TIMOTHY C +1
Dosing regimens for 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde
ActiveUS20170157101A1High drug loadingGood physical propertiesOrganic active ingredientsCapsule deliveryRegimenMedicine
Provided herein are methods for treating sickle cell disease, comprising administering to a subject 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)-methoxy)benzaldehyde (Compound 1), or a polymorph thereof, in certain dosing regimens.
Owner:GLOBAL BLOOD THERAPEUTICS
Methods for increasing red blood cell levels and treating sickle-cell disease
InactiveUS20150361163A1Increase productionIncreased erythropoiesisSenses disorderPeptide/protein ingredientsPrimateSickle Cell Diseases
In certain aspects, the present disclosure provides compositions and methods for increasing red blood cell and / or hemoglobin levels in vertebrates, including rodents and primates, and particularly in humans. In some embodiments, the compositions of the disclosure may be used to treat or prevent sickle-cell disease or one or more complications associated with sickle-cell disease.
Owner:ACCELERON PHARMA INC
Compounds and methods for treatment of sickle cell disease or complications associated therewith
Owner:GLYCOMIMETICS
Inhibitors of 2-oxoglutarate dioxygenase as gamma globin inducers
InactiveUS20070042937A1Increase gene expressionInhibit hydroxylationBiocidePeptide/protein ingredientsDiseaseThalassemia
The present invention provides methods for increasing endogenous globin expression in a subject, specifically γ-globin expression. The invention also provides compounds and medicaments for use in the methods. The methods are particularly useful for increasing fetal hemoglobin production in a subject, and can be used to treat various disorders, e.g., β thalassemia and sickle cell disease.
Owner:ISIS INNOVATION LTD +1
Anti-P-selectin antibodies and methods of using the same to treat inflammatory diseases
Owner:NOVARTIS AG +1
Pan-selectin inhibitor with enhanced pharmacokinetic activity
Compounds, compositions and methods are provided for treatment of diseases or complications associated therewith, in which a selectin plays a role. More specifically, particular glycomimetics and uses thereof are described. For example, use of particular glycomimetics for treating sickle cell disease or a cancer involving a selectin, or complications associated with either, is described.
Owner:GLYCOMIMETICS
USE OF PHTHALIMIDE AND/OR SULPHONAMIDE DERIVATIVES IN THE TREATMENT OF DISEASES WHICH REQUIRE REDUCING THE TNF-alpha LEVELS AND AN EXOGENOUS SOURCE OF NITRIC OXIDE, PHTHALIMIDE DERIVATIVES, SULPHONAMIDE DERIVATIVES, AND A METHOD FOR OBTAINING A SULPHONAMIDE DERIVATIVE
InactiveUS20100324107A1Improve the quality of lifeBiocideOrganic chemistryPhthalocyanine derivativesNitric oxide
Owner:UNIV ESTADUAL DE CAMPINAS UNICAMP +2
Transdermal buprenorphine to treat pain in sickle cell crisis
ActiveUS7413748B2Effective analgesiaEffective pain reliefBiocideAnimal repellantsTransdermal patchDosing regimen
A specific dosage regimen of buprenorphine achieves pain relief from painful episodes due to sickle cell disease. The dosage regimen comprises administering to a patient in need of pain relief from sickle cell disease at least one BTDS transdermal patch. Alternatively, the dosing regimen comprises administering to the patient (1) a first buprenorphine-containing transdermal dosage form for a first dosing period; (2) administering to the patient a second buprenorphine-containing transdermal dosage form for a second dosing period, where the second dosage form comprises the same dosage of buprenorphine as, or a greater dosage of buprenorphine than, the first dosage form; and (3) administering to the patient a third buprenorphine-containing transdermal dosage form for a third dosing period, where the third dosage form comprises a greater dosage of buprenorphine than the second dosage form.
Owner:PURDUE PHARMA LP
Methods for the treatment or prevention of inflammatory diseases characterized by abnormal cell proliferation
InactiveUS20020128256A1Reducing sickle erythrocyte dehydrationDelaying occurrenceBiocideHydroxy compound active ingredientsPotassium channelPotassium
The present invention provides substituted 3,3-diphenyl indanone, indane and indole compounds, as well as analogues thereof which are specific, potent and safe inhibitors of the Ca.sup.2+-activated potassium channel (Gardos channel) of erythrocytes. The compounds can be used as efficacious drugs in the treatment of sickle cell disease and diseases characterized by unwanted or abnormal cell proliferation, and in particular inflammatory diseases associated with unwanted cellular proliferation.
Owner:BRUGNARA CARLO +12
Compositions and methods for the treatment of hemoglobinopathies and thalassemias
ActiveUS20190388376A1Function increaseIncrease synthesisOrganic active ingredientsDipeptide ingredientsHemoglobinopathyThalassemia
This disclosure provides compositions and methods for improving erythrocyte dysfunction or treating a hemoglobinopathy or a thalassemia (e.g., sickle cell disease or β-thalassemia).
Owner:AXCELLA HEALTH INC
Methods of Treating Sickle Cell Disease and Related Disorders Using Fumaric Acid Esters
InactiveUS20140171504A1Improve update processHigh expressionBiocideAmide active ingredientsBeta thalassemiaSickle-cell disorders
Methods of using one or more fumaric acid esters or pharmacologically active salts, derivatives, analogues, or prodrugs thereof to increase expression of fetal hemoglobin (HbF) are disclosed. The methods typically include administering to a subject an effective amount of one or more fumaric acid esters optionally in combination or alternation with hydroxyurea to induce HbF expression in the subject in an effective amount to reduce one or more symptoms of a sickle cell disorder, a hemoglobinopathy, or a beta-thalassemia, or to compensate for a genetic mutation is the human beta-globin gene (HBB) or an expression control sequence thereof. Pharmaceutical dosage units and dosage regimes for use in the disclosed methods are also provided.
Owner:AUGUSTA UNIV RES INST INC
Use of long chain polyunsaturated fatty acid derivatives to treat sickle cell disease
InactiveUS20140088047A1Improve the quality of lifeBiocideGroup 5/15 element organic compoundsDiseaseLong chain fatty acid
The present invention relates to the use of long chain polyunsaturated fatty acids to treat diseases associated with red blood cells and cell membranes, and in particular to the use of derivatives of long chain fatty acids to treat sickle cell disease.
Owner:AKER BIOMARINE ANTARCTIC
Imines as ion channel modulators
The present invention provides a class of chemical compounds useful in the treatment of sickle cell disease, diseases characterized by unwanted or abnormal cell proliferation and for the treatment of ocular disorders such as glaucoma. The active compounds are tri-(aryl or heteroaryl) methane compounds or analogues thereof which further comprise an imine moiety and where the tertiary carbon atom can be replaced with a different atom such as Si, Ge, N or P. The compounds enhance resistance to degradation in a biological medium, inhibit potassium flux in a cell, reduce mammalian cell proliferation, reduce the Gardos channel of erythrocytes, reduce sickle erythrocyte dehydration and / or delay the occurrence of erythrocyte sickling or deformation.
Owner:ICAGEN INC
Feedback sensor for real-time management of sickle cell disease
InactiveUS20100198037A1Reduce the possibilityReduce expenditureDiagnostics using lightHealth-index calculationTissue damageSickle Cell Diseases
Described herein are devices, systems and methods for real-time (or near real-time) monitoring of red blood cell morphology, and in particular, monitoring of sickling to provide an indication of the risk of a the downstream consequences of sickling, such as the pain crises and associated hypoxic tissue damage which may occur as sickling progresses. The monitors describe herein may be continuous (e.g., sampling the subject continuously while worn or activated), or they may operate at a predetermine or selectable sampling rate. In some variations, the devices described herein are worn or applied to the patient non-invasively or minimally invasively.
Owner:HOPELAB FOUND
Treating sickle cell disease
InactiveUS20050101659A1Reduce morbidityIncrease hemoglobin F productionBiocideAnimal repellantsSickle Cell Diseases5-Lipoxygenase Inhibitor
Owner:SOUTH ALABAMA MEDICAL SCI FOUND
Topical mecamylamine formulations for ocular administration and uses thereof
Provided are methods, pharmaceutical formulations and kits thereof for the treatment and / or prevention of conditions mediated by neovascularization, abnormal angiogenesis, vascular permeability, or combinations thereof, of posterior and / or anterior tissues and fluids of the eye, including conditions associated with proliferative retinopathies, for example, diabetic retinopathy, age-related maculopathy, retinopathy of prematurity, retinopathy associated with macular edema, or retinopathy associated with sickle cell disease, using the topical administration of mecamylamine or a pharmaceutically acceptable salt thereof to the eye. Methods of preparing the pharmaceutical formulations are also provided.
Owner:COMENTIS
Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
ActiveUS7541179B2Increase percentageBiocideOrganic active ingredientsGlobin genesDihydrofolate reductase
Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than un-transformed cells allows selection by treatment of the cells with the corresponding drug.
Owner:SLOAN KETTERING INST FOR CANCER RES
Methods of treating cardio pulmonary diseases with NO group compounds
Treatment of pulmonary disorders associated with hypoxemia and / or smooth muscle constriction and / or inflammation comprises administering into the lungs as a gas a compound with an NO group which does not form NO2 / NOx in the presence of oxygen or reactive oxygen species at body temperature. Treatment of cardiac and blood disorders, e.g., angina, myocardial infarction, heart failure, hypertension, sickle cell disease and clotting disorders, comprises administering into the lungs as a gas, a compound which reacts with cysteine in hemoglobin and / or dissolves in blood and has an NO group which is bound in said compound so that it does not form NO2 / NOx in the presence of oxygen or reactive oxygen species at body temperature. Exemplary of the compound administered in each case is ethyl nitrite. Treatment of patient in need of improved oxygenation, blood flow of and / or thinning of blood comprises providing in the patient a therapeutic amount of red blood cells loaded with nitrosylated hemoglobin. A method is directed to screening drugs that increase level of nitrosoglutathione in airway lining fluid.
Owner:DUKE UNIV
Automated Cell Identification Using Shearing Interferometry
The present disclosure provides improved systems and methods for automated cell identification / classification. More particularly, the present disclosure provides advantageous systems and methods for automated cell identification / classification using shearing interferometry with a digital holographic microscope. The present disclosure provides for a compact, low-cost, and field-portable 3D printed system for automatic cell identification / classification using a common path shearing interferometry with digital holographic microscopy. This system has demonstrated good results for sickle cell disease identification with human blood cells. The present disclosure provides that a robust, low cost cell identification / classification system based on shearing interferometry can be used for accurate cell identification. For example, by combining both the static features of the cell along with information on the cell motility, classification can be performed to determine the type of cell present in addition to the state of the cell (e.g., diseased vs. healthy).
Owner:UNIV OF CONNECTICUT
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