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35 results about "Hemoglobinopathy" patented technology
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Hemoglobinopathy or Hemoglobinopathies is the medical term for a group of blood disorders and diseases that affect red blood cells. It can be a kind of genetic defect that results in abnormal structure of one of the globin chains of the hemoglobin molecule. Hemoglobinopathies are inherited single-gene disorders; in most cases, they are inherited as autosomal co-dominant traits. Common hemoglobinopathies include sickle-cell disease. It is estimated that 7% of world's population (420 million) are carriers, with 60% of total and 70% pathological being in Africa. Hemoglobinopathies are most common in populations from Africa, the Mediterranean basin and Southeast Asia.
Methods and compositions for the treatment and management of hemoglobinopathy and anemia
InactiveUS20050143420A1Good effectRelieve symptomsBiocidePeptide/protein ingredientsRed blood cellThalassemia
The present invention is directed to the use of immunomodulatory compounds, particularly members of the class of compounds known as IMiDs™, and more specifically the compounds 4-(Amino)-2-(2,6-dioxo(3-piperidyl))-isoindoline-1,3-dione and 3-(4-amino-1-oxo-1,3-dihydroisoindol-2-yl)-piperidine-2,6-dione, to induce the expression of fetal hemoglobin genes, genes essential for erythropoiesis, and genes encoding alpha hemoglobin stabilizing protein, within a population of CD34+ cells. These compounds are used to treat hemoglobinopathies such as sickle cell anemia or β-thalassemia, or anemias caused by disease, surgery, accident, or the introduction or ingestion of toxins, poisons or drugs.
Owner:SIGNAL PHARMA LLC
Modulation of bcl11a for treatment of hemoglobinopathies
ActiveUS20110182867A1Increasing fetal hemoglobin levelHigh expressionOrganic active ingredientsVirusesHemoglobinopathyFetal hemoglobin
The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
Owner:PRESIDENT & FELLOWS OF HARVARD COLLEGE
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
Modulation of BCL11A for treatment of hemoglobinopathies
The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
Owner:PRESIDENT & FELLOWS OF HARVARD COLLEGE
Methods for therapy of cancer
InactiveUS6037376AInhibition formationAvoid seizuresBiocidePeptide/protein ingredientsHemoglobinopathyMedicine
Compositions and methods of treating anemia, cancer, AIDS, or severs beta -chain hemoglobinopathies by administering a therapeutically effective amount of phenylacetate or pharmaceutically acceptable derivatives thereof or derivatives thereof alone or in combination or in conjunction with other therapeutic agents. Pharmacologically-acceptable salts alone or in combinations and methods of preventing AIDS and malignant conditions, and inducing cell differentiation are also aspects of this invention.
Owner:UNITED STATES OF AMERICA
Therapeutic retroviral vectors for gene therapy
ActiveUS20060057725A1Maintaining therapeutic levelImprove the level ofPeptide/protein ingredientsGenetic material ingredientsHemoglobinopathyRed blood cell
Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.
Owner:MASSACHUSETTS INST OF TECH
Methods and compositions for the treatment and management of hemoglobinopathy and anemia
InactiveCN1913896AOrganic active ingredientsPeptide/protein ingredientsBeta thalassemiaSickle cell anemia
Owner:CELGENE CORP
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
2-(hetero)aryl-benzimidazole and imidazopyridine derivatives as inhibitors of asparagime emethyl transferase
ActiveUS20160222005A1Inhibitory activityModulate activityOrganic chemistryAntineoplastic agentsArylHemoglobinopathy
Substituted benzimidazole and 3H-imidazo[4,5-b]pyridines or formula I: where X and Y respectively are selected from: (i) N and N; and (ii) N and CR4; A2 is selected from: a C5 heteroarylene group, containing 2 or 3 ring heteroatoms, where the bonds to L1 and the core are β to one another; L1 is selected from: (i) A1—O—CH2—A2; (ii) A1—CH2—O—A2; (iii) A1—C(═O)—NH—A2; (iv) A1—CH(OH)—A2; (v) A1—CH2—NH—C(═O)—A2; (vi) A1—S—CH2—A2; (vii) A1—CH2—S—A2; (viii) A1—CH2—A2; and (ix) A1—CH(CH3)—O-A2; A1 is phenyl, optionally substituted by F or CF3; their use as pharmaceuticals, and in particular, in treating cancer and hemoglobinopathies.
Owner:CANCER THERAPEUTICS CRC
Globin gene therapy for treating hemoglobinopathies
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
Owner:MEMORIAL SLOAN KETTERING CANCER CENT +1
Globin gene therapy for treating hemoglobinopathies
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
Owner:MEMORIAL SLOAN KETTERING CANCER CENT +1
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
2-(hetero)aryl-benzimidazole and imidazopyridine derivatives as inhibitors of asparagime emethyl transferase
ActiveUS9856252B2Modulate activityDecreased arginine-methylated p53Organic chemistryAntineoplastic agentsArylHemoglobinopathy
Substituted benzimidazole and 3H-imidazo[4,5-b]pyridines or formula I: where X and Y respectively are selected from: (i) N and N; and (ii) N and CR4; A2 is selected from: a C5 heteroarylene group, containing 2 or 3 ring heteroatoms, where the bonds to L1 and the core are β to one another; L1 is selected from: (i) A1—O—CH2—A2; (ii) A1—CH2—O—A2; (iii) A1—C(═O)—NH—A2; (iv) A1—CH(OH)—A2; (v) A1—CH2—NH—C(═O)—A2; (vi) A1—S—CH2—A2; (vii) A1—CH2—S—A2; (viii) A1—CH2—A2; and (ix) A1—CH(CH3)—O—A2; A1 is phenyl, optionally substituted by F or CF3; their use as pharmaceuticals, and in particular, in treating cancer and hemoglobinopathies.
Owner:CANCER THERAPEUTICS CRC
Compositions and methods for the treatment of hemoglobinopathies
ActiveUS20170298329A1Improve the level ofAvoid the insertion of vector sequencesFusion with DNA-binding domainPeptide/protein ingredientsThalassemiaGlobin 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) and / 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 HbF silencing DNA regulatory element or pathway, such as a Bcl11a-regulated HbF 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:FRED HUTCHINSON CANCER RES CENT
Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
InactiveUS20090274671A1Increase percentageBiocideOrganic active ingredientsTransformation cellGlobin genes
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 untransformed cells allows selection by treatment of the cells with the corresponding drug.
Owner:MEMORIAL SLOAN KETTERING CANCER CENT
Bcl11a homing endonuclease variants, compositions, and methods of use
InactiveUS20190184035A1Decrease blood transfusionHydrolasesGenetic therapy composition manufactureHemoglobinopathyGenome editing
The present disclosure provides improved genome editing compositions and methods for editing a BCL11A gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.
Owner:BLUEBIRD BIO INC
Hemoglobin assay for neonatal screening
InactiveUS7544513B2Avoid pollutionChemiluminescene/bioluminescenceImmunoglobulins against animals/humansHemoglobinopathyPediatrics
The present invention provides an assay, suitable for neonatal screening of hemoglobinopathies by quantitatively determining the presence of at least one pair of hemoglobin (Hb) variants, determining the ratio between the signals obtained from each variant; and, based on that ratio, determining whether the tested subject is afflicted or not.
Owner:WALLAC
Methods for identifying and treating hemoglobinopathies
InactiveUS20190111114A1Efficient processingSuitable for automationCompound screeningFusion with DNA-binding domainHemoglobinopathyPathology
The invention relates to method of treating or inhibiting progression of hemoglobinopathy in a subject in need thereof comprising inhibiting interaction between LRF-BTB and CHD protein-LBD.
Owner:MAEDA TAKAHIRO +1
Donor repair templates multiplex genome editing
The present disclosure provides improved multiplex genome editing compositions and methods. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.
Owner:2SEVENTY BIO INC
Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
InactiveUS8058061B2Increase percentageOrganic active ingredientsGenetic material ingredientsGlobin genesDihydrofolate reductase
Owner:MEMORIAL SLOAN KETTERING CANCER CENT
Compounds for use in the treatment of disorders that are ameliorated by inhibition of HDAC
InactiveUS9371295B2Increase percentageInduce expressionOrganic active ingredientsOrganic chemistryHemoglobinopathyHistone deacetylase
Owner:IRBM SCI PARK +1
Methods and compositions for treating .beta.-thalassemia and sickle cell disease
The present invention provides compounds (such as compounds of formula I-a) that induce γ globin expression and pharmaceutical compositions thereof. Such compounds may have beneficial therapeutic effects. Compounds and compositions described herein may be used to treat hemoglobinopathies such as β-thalassemia and sickle cell anemia.
Owner:ACAD SINIC
Erythroid-specific promoter and method of use thereof
A DNA construct containing an erythroid lineage-specific promoter operably linked to a nucleotide coding sequence of interest and a method of using the same in the prevention or treatment of a hematopoietic disorder such as a hemoglobinopathy are described. Further disclosed are erythroid-specific promoters and erythroid-specific enhancers that can be used in the DNA construct.
Owner:ST JUDE CHILDRENS RES HOSPITAL INC
Hemoglobin assay for neonatal screening
InactiveUS20050118654A1Simple methodAvoid pollutionHybrid immunoglobulinsChemiluminescene/bioluminescenceHemoglobinopathyPediatrics
The present invention provides an assay, suitable for neonatal screening of hemoglobinopathies by quantitatively determining the presence of at least one pair of hemoglobin (Hb) variants, determining the ratio between the signals obtained from each variant; and, based on that ratio, determining whether the tested subject is afflicted or not.
Owner:WALLAC
3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies
ActiveUS10519167B2Improve efficacyDecreased arginine-methylated p53Organic active ingredientsOrganic chemistryHemoglobinopathyMethyl group
A compound of formula Ia, Ib, Ic or Id: wherein: n is 1 or 2; RN is H or Me; R1 is optionally one or more halo or methyl groups; R2a and R2b are independently selected from the group consisting of: (i) F; (ii) H; (iii) Me; and (iv) CH2OH; R2c and R2d (if present) are independently selected from the group consisting of: (i) F; (ii) H; (iii) Me; and (iv) CH2OH; R3a and R3b are independently selected from H and Me; R4a is selected from OH, —NH2, —C(═O)NH2, and —CH2OH; R4b is either H or Me; R5 is either H or Me; m is 1 or 2; q is 0 or 1; R11a, R11b, R11c and R11d are independently selected from H, halo, C1-4 alkyl, C1-4 fluoroalkyl, C3-4 cycloalkyl, C1-4 alkyloxy, NH—C1-4 alkyl and cyano; R12a and R12b are independently selected from the group consisting of: (i) F; (ii) H; (iii) Me; and (iv) CH2OH; R12c and R12d are independently selected from the group consisting of: (i) F; (ii) H; (iii) Me; and (iv) CH2OH; R12e is H or Me; R13a and R13b are independently selected from H and Me; R14 is either H or Me; R16a and R16b are independently selected from H and Me; R6 is selected from H, OMe, and OEt.
![3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies](https://images-eureka.patsnap.com/patent_img/c4f86983-efd9-4b01-85ce-25dcc4af7197/US10519167-C00001.png "3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies")
![3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies](https://images-eureka.patsnap.com/patent_img/c4f86983-efd9-4b01-85ce-25dcc4af7197/US10519167-C00002.png "3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies")
![3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies](https://images-eureka.patsnap.com/patent_img/c4f86983-efd9-4b01-85ce-25dcc4af7197/US10519167-C00003.png "3-oxa-8-azabicyclo[3.2.1]octane derivatives and their use in the treatment of cancer and hemoglobinopathies")
Owner:CTXT PTY LTD
Methods of Treating Sickle Cell Disease and Related Disorders Using Fumaric Acid Esters
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
Methods and compositions for treating beta-thalassemia and sickle cell disease
ActiveUS20140088133A1Avoid developmentAvoid adjustmentBiocideOrganic chemistryHemoglobinopathyBeta thalassemia
The present invention provides compounds (such as compounds of formula I-a) that induce γ globin expression and pharmaceutical compositions thereof. Such compounds may have beneficial therapeutic effects. Compounds and compositions described herein may be used to treat hemoglobinopathies such as β-thalassemia and sickle cell anemia. (I-a)
Owner:ACAD SINIC
Oligonucleotide compositions and their use to induce differentiation of cells
InactiveUS20070010472A1Augment net therapeutic effectOrganic active ingredientsSugar derivativesBone Marrow-Derived CellLeukopenia
The present invention provides compositions comprising a 3′-OH, 5′-OH, chemically unmodified, synthetic phosphodiester nucleotide sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, and SEQ ID NO: 4, and a pharmaceutically acceptable carrier, wherein the compositions are useful to induce differentiation of cells or to stimulate differentiation or production of pluripotent cells. The present invention provides methods of using these compositions to induce differentiation of pluripotent cells, including bone marrow derived cells, and to treat disease associated with insufficient differentiation of cells in animals and humans, including but not limited to leukemia, lymphoma, non-malignant blood disorders such as hemoglobinopathies, sickle cell disease, myelodysplastic syndrome, pancytopenia, anemia, thrombocytopenia and leukopenia.
Owner:BIONICHE LIFE SCI
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