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WO/2021/097326A1 |
Aminopyrimidine containing compounds that inhibit both Mer tyrosine kinase (MerTK) activity and Tyro3 kinase activity are disclosed herein. Additionally disclosed are methods of synthesis and use of the aminopyrimidine containing compoun...
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WO/2021/094210A1 |
The invention relates to substituted pyrazine carboxamide derivatives of formula (I) and to processes for their preparation, and also to their use for preparing medicaments for the treatment and/ or prophylaxis of diseases, in particular...
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WO/2021/096927A1 |
Disclosed herein are compositions and methods for treating pulmonary hypertension whereby a SCUBE1 polynucleotide or polypeptide is administered. Also disclosed herein are methods for diagnosing and/or prognosing pulmonary arterial hyper...
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WO/2021/092227A1 |
Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozyg...
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WO/2021/091846A1 |
The present disclosure relates to the use of Compound (I) or a pharmaceutically acceptable salt thereof, for the treatment of mast cell diseases and eosinophilic disorders.
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WO/2021/091532A1 |
The present disclosure relates to the use of 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[c]isoxazolo[4,5
-e]azepin-4-yl)acetamide, and pharmaceutically acceptable salts thereof, for treating myelofibrosis.
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WO/2021/090594A1 |
A purpose of the present invention is to produce neutrophil-like cells in numbers that can be used in granulocyte transfusion therapy. It became possible to vastly improve proliferation by further forcibly expressing at least one gene be...
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WO/2021/092485A1 |
The present invention features compositions and methods useful in inhibiting the expression of NFIX within a cell and thereby treating patients suffering from a hemoglobinopathy such as sickle cell disease or β-thalassemia. The present ...
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WO/2021/085504A1 |
Provided are a B-cell antibody receptor (BAR), a BAR-T cell and others which are effective for the treatment of diseases associated with antibodies produced in the bodies of patients. The B-cell antibody receptor (BAR) according to the p...
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WO/2021/081581A1 |
The present invention relates to antigen binding proteins comprising an antigen binding domain that binds to plasminogen, wherein the antigen binding protein reduces the activation of plasminogen to plasmin, pharmaceutical compositions c...
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WO/2021/086097A1 |
The present invention relates to a powder-type hemostatic composition and a preparation method therefor and, more specifically, to a powder aggregate obtained by binding biocompatible hemostatic material powders with a binder and having ...
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WO/2021/087177A1 |
Embodiments of the disclosure encompass methods and compositions related to overcoming or preventing cancer resistance to anti-apoptotic proteins, for example BH3 mimetics. The disclosure provides methods for modulating p73 to reduce can...
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WO/2021/083383A1 |
Disclosed are a nitrogen-containing fused cyclic compound represented by general formula I, or a pharmaceutically acceptable salt thereof, or an enantiomer, a diastereomer, a tautomer, a solvate, a polymorph, or a prodrug thereof, and a ...
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WO/2021/085575A1 |
[Problem] To provide a composition or the like that suppresses the physical destruction of red blood cells (hemolysis) due to exercise and behaviors associated with impact such as striking, for example, as when continually hitting the so...
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WO/2021/081582A1 |
The present invention provides an antigen binding protein comprising an antigen binding domain that binds to plasmin, wherein the antigen binding protein reduces the activity of plasmin. The invention also provides compositions comprisin...
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WO/2021/083073A1 |
The present invention provides a product for treating a hepatocyte Alb gene-based disease, comprising a reagent capable of inserting a target gene into intron 13 of Alb in hepatocytes and co-expressing the target gene and Alb. The diseas...
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WO/2021/087144A1 |
This invention provides methods for the treatment of anemia in patients with chronic kidney disease (CKD) using vadadustat (Compound 1), including methods suitable for conversion, correction, and maintenance therapy for patients. For exa...
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WO/2021/087050A1 |
The present invention relates to novel pharmaceutical formulations of an antibody against human P-selectin, especially SEG101, or an antibody having at most 3 amino acid difference from crizanlizumab, and processes for the preparation th...
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WO/2021/078889A1 |
The invention relates to compounds of the formula (I) and pharmaceutically acceptable salts thereof for the use in the treatment of sickle cell disease and for the use in preventing and treating vascular inflammation and vaso-occlusion.
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WO/2021/081277A1 |
The present invention provides dosing regimens of anti-C5 antibodies, such as pozelimab, for treating or preventing CS-associated diseases such as paroxysmal nocturnal hemoglobinuria or CHAPLE disease.
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WO/2021/080359A1 |
The present invention relates to a 6-6 bicyclic ring-containing compound derivative and use thereof. The compound according to the present invention acts as a PRMT5 inhibitor, and thus can be effectively used in the prevention or treatme...
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WO/2021/080262A1 |
The present invention relates to an "intravascular thrombus" lytic agent and, more specifically, to a polypeptide, a gene, and a pharmaceutical composition comprising same, wherein the polypeptide is composed of a thrombo-recognition dom...
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WO/2021/078076A1 |
A novel crystalline form of acetylated eltrombopag and a preparation method thereof belonging to the field of medicinal chemistry. The crystalline form has good stability and facilitates operations in storage, transfer, and production pr...
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WO/2021/079536A1 |
This composition for lysis of Staphylococcus aureus or this composition for prevention or therapy of diseases caused by Staphylococcus aureus contains as active ingredients: at least one bacteriophage selected from the group consisting o...
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WO/2021/079978A1 |
The present invention addresses the problem of providing a therapeutic or prophylactic agent that is for cachexia and that exhibits significant efficacy in therapy of cachexia with few side effects. The present invention provides a thera...
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WO/2021/079986A1 |
A composition for lysing Staphylococcus aureus or a composition for preventing or treating a disease caused by Staphylococcus aureus, said composition comprising, as active ingredients, at least one bacteriophage selected from the group ...
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WO/2021/079435A1 |
This composition is for lysing Staphylococcus aureus or treating or preventing diseases caused by Staphylococcus aureus, and contains, as active ingredients, at least one bacteriophage selected from the group consisting of the bacterioph...
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WO/2021/077994A1 |
Disclosed are a new crystal form of compound I and a preparation method therefor, a pharmaceutical composition comprising the crystal form, and the use of the crystal form in the preparation of hypoxia-inducible factor-prolyl hydroxylase...
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WO/2021/079984A1 |
The present invention provides a novel compound having an excellent β-lactamase inhibitory effect. The present invention provides: a compound which has an excellent β-lactamase inhibitory effect, and is represented by formula (1a), (1b...
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WO/2021/078077A1 |
Disclosed are a new crystal form of acetylated eltrombopag and a preparation method therefor, belonging to the field of pharmaceutical chemistry. The crystal form has better stability, which is conducive to storage, transfer, and operati...
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WO/2021/077034A1 |
Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of hemoglobinopathy, thalassemia, α-thalassemia, α-thalassemia minima, α-thalassemia minor, β-thalassemia, β-thalassemia minor, β...
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WO/2021/075568A1 |
The present invention provides a method for producing a megakaryocyte progenitor cell or a megakaryocytic cell, the method comprising a step of suppressing the expression of CDKN1A gene or the function of an expression product of the gen...
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WO/2021/076709A1 |
Embodiments of the present disclosure relate to doses of ornithine phenylacetate for treating or ameliorating hyperammonemia and the methods of administrating the same in a patient with a chronic liver disease, for example, cirrhosis. In...
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WO/2021/076991A1 |
The present disclosure relates generally to methods of preventing, reducing risk of developing, or treating a blood disorder ( e.g., cold agglutinin hemolytic anemia (cold agglutinin disease), cold antibody hemolytic anemia, ABO incompat...
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WO/2021/076514A1 |
Methods for treating immune thrombocytopenia comprising administering at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-
d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxeta
n-3-yl)pipe...
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WO/2021/076485A1 |
Disclosed herein are methods of reducing, preventing, or ameliorating tissue iron overload and disorders associated with depletion/reduction of plasm haptoglobin levels in the presence of increased plasma hemoglobin levels due to hemolys...
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WO/2021/073038A1 |
Disclosed in present invention are an o-hydroxybenzoic acid polyglycol ester compound, and a synthetic preparation method and pharmaceutical use thereof. The o-hydroxybenzoic acid polyglycol ester compound has a structure shown as formul...
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WO/2021/073610A1 |
Provided are methods and compositions for treating diseases or disorders related to hematopoietic dysfunctions and/or injuries, or for rejuvenating HSPCs.
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WO/2021/071751A1 |
The present embodiments provide systems and medical formulations suitable for delivering therapeutic powders to a target site. In one embodiment, the system comprises a delivery device, a first powder being formed of particles of a first...
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WO/2021/068239A1 |
Disclosed are a vaginal slow-release administration system for luteal support, a preparation method therefor and a use thereof. The system is a progesterone reservoir type vaginal ring, comprising a core layer, and a film layer double-la...
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WO/2021/072194A1 |
The present disclosure provides stabilized hemoglobin compositions, uses thereof, and devices for administration thereof. The stabilized hemoglobin compositions may be useful in the treatment of various anemic or traumatic conditions inv...
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WO/2021/070885A1 |
The present inventors have found that acquired hemophilia A can be prevented and/or treated effectively by administering a pharmaceutical composition containing emicizumab in accordance with a specific dosing regimen.
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WO/2021/067914A1 |
The present disclosure provides for methods and compositions for preventing and treating myelodysplastic syndromes, acute myeloid leukemia, and other diseases and disorders of the blood, using a combination therapy including all-trans re...
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WO/2021/066578A1 |
The present invention relates to a compound derivative bearing a 6-7 bicyclic ring and a use thereof. The compound according to the present invention acts as a protein arginine methyltransferases 5 (PRMT5) inhibitor and thus can be advan...
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WO/2021/064151A1 |
The present invention relates to the use of eicosapentaenoic acid ethyl ester and docosahexaenoic acid ethyl ester, optionally in combination with vitamin D3, in prevention of decline in lean muscle mass, falls with and without an injury...
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WO/2021/067389A1 |
Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorde...
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WO/2021/065768A1 |
Provided is a preparation which is co-administered with a protein preparation and can increase the administration volume of the protein preparation even when administered at a low concentration. This space-forming agent forms, under the ...
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WO/2021/066152A1 |
The purpose of the present invention is to provide a blood treatment material which can adsorb and remove blood components including activated leukocytes and inflammatory cytokines with high efficiency. The present invention provides a b...
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WO/2021/067668A1 |
The disclosure provides a method for treating and/or preventing graft rejection with A Disintegrin And Metalloproteinase with Thrombospondin type 1 motif, member-13 (ADAMTS13). The disclosure provides a method for increasing ADAMTS13-med...
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WO/2021/066649A1 |
The invention provides method of treatment comprising administering an AQGV peptide, or a functional analogue thereof, to a human subject, the human subject optionally having impaired kidney function, wherein the treatment of administeri...
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