Abstract: The present invention has an object of providing a pharmaceutical drug containing a compound having a good LCAT activation effect on a mutant LCAT protein and a therapeutic effect for LCAT deficiency, and also provides a therapeutic drug for LCAT deficiency containing a compound represented by Formula (I) wherein R1 is a hydrogen atom or a hydroxyl group, and R is a 2-(trifluoromethyl)pyrimidin-5-yl group or a 5-(trifluoromethyl)pyrazin-2-yl group, or a pharmacologically acceptable salt thereof.

Abstract: This application relates to a pharmaceutical composition for use in a method for treating and/or preventing a patient having ectopic ossification and/or brain tumor, wherein the patient has an active mutation in ALK2 protein which is responsible for ectopic ossification or brain tumor; an amino acid residue at position 330 of ALK2 is proline; and an active ingredient of this composition is an anti-ALK2 antibody or an antigen-binding fragment thereof comprising a property of binding to ALK2, a property of cross-linking ALK2, and a property of inhibiting BMP signal transduction.

Abstract: The present invention aims to provide a pharmaceutical drug that contains a compound having an excellent therapeutic effect on inflammatory bowel disease. A therapeutic agent for inflammatory bowel disease, containing as an active ingredient a compound represented by formula (I), wherein R1 represents a hydroxy C1-C6 alkyl group, a C2-C7 alkanoyl group, a C2-C7 alkanoyl C1-C6 alkyl group, a (C1-C6 alkoxy) carbonyl group, a (C1-C6 alkoxy) carbonyl C1-C6 alkyl group, a carboxy group or a carboxy C1-C6 alkyl group, or a pharmacologically acceptable salt thereof.

Abstract: The present invention provides a pharmaceutical composition or a diagnostic composition targeting human fibroblast growth factor receptor 2 (hFGFR2).

Abstract: The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to the cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82nd to the 92nd nucleotide from the 5? end of exon 5 of the G6PC gene with c.648G>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).

Abstract: It is an object of the present invention to provide an antibody binding to CDH6 and having internalization activity, an antibody-drug conjugate of the antibody and a drug having antitumor activity, a pharmaceutical product comprising the antibody-drug conjugate and having therapeutic effects on a tumor, a method for treating a tumor using the antibody, the antibody-drug conjugate or the pharmaceutical product, and the like. The present invention provides an anti-CDH6 antibody having internalization activity, an antibody-drug conjugate of the antibody and a drug having antitumor activity, a pharmaceutical product comprising the antibody or the antibody-drug conjugate, and a method for treating a tumor.

Abstract: Provided is a short-chain guide RNA that is able to induce site-specific editing even when only a small number of nucleotides is attached to the target recognition site. The guide RNA includes a first oligonucleotide that identifies the target RNA, and a second oligonucleotide that links to the 3? end of the first oligonucleotide. The first oligonucleotide contains: a target-corresponding nucleotide residue that corresponds to an adenosine residue in the target RNA; an oligonucleotide of 15 to 30 residues that links to the 5? end of the target-corresponding nucleotide residue and that has a base sequence complementary to the target RNA; and an oligonucleotide of 3 or 4 residues that links to the 3? end of the target-corresponding nucleotide residue and that has a base sequence complementary to the target RNA. The second oligonucleotide contains 2 to 24 nucleotide residues, and induces site-specific editing of the target RNA.

Abstract: A method for producing a compound represented by formula (C) wherein R1 represents an amino group protected with a protecting group, the method comprising a step of subjecting a compound represented by formula (B) wherein R1 represents the same meaning as above, to intramolecular cyclization to convert the compound into the compound represented by formula (C).

Abstract: Crystals of the compound represented by formula (1), a method for the production thereof, and a method for producing an antibody-drug conjugate using the crystals.

Abstract: As an antitumor drug which is excellent in terms of antitumor effect and safety and has an excellent therapeutic effect, there is provided an antibody-drug conjugate in which an antitumor compound represented by the following formula is conjugated to an anti-HER2 antibody via a linker having a structure represented by the following formula: -L1-L2-LP-NH—(CH2)n1-La-(CH2)n2-C(?O)— wherein the anti-HER2 antibody is connected to the terminal L1, and the antitumor compound is connected to the carbonyl group of the —(CH2)n2-C(?O)— moiety with the nitrogen atom of the amino group at position 1 as the connecting position.

Abstract: It is intended to provide a novel peptide. The present invention provides a peptide which comprises the amino acid sequence shown in SEQ ID NO: 30 and inhibits protease activity.

Abstract: The present disclosure provides a method for efficiently producing and providing compounds having a spirooxindole skeleton, for example compounds having a spirooxindole skeleton and having antitumor activity that inhibit the interaction between Mdm2 protein and p53 protein, or intermediates thereof, using an asymmetric catalyst. Compounds having optically active tricyclic dispiroindole skeletons are obtained through catalytic asymmetric 1,3-dipolar cycloaddition reaction using ketimine as a reaction substrate and using a chiral ligand and a Lewis acid.

Abstract: It is intended to provide a therapeutic and/or prophylactic agent for transplant rejections, immunological diseases, allergic diseases, inflammatory diseases, thrombosis, cancers, etc., targeting human Orai1. The present invention provides, for example, a pharmaceutical composition comprising an antibody that specifically recognizes human Orai1 and has the activity of inhibiting human T cell activation.

Abstract: The present invention relates to a compound having RET kinase inhibiting action or a pharmaceutically acceptable salt thereof, useful in the treatment of diseases such as cancer. In particular a compound represented by the following general formula (I) as defined herein: (I) or a pharmaceutically acceptable salt thereof.

Abstract: It is an object of the present invention to provide an antibody specifically binding to GPR20-positive tumor cells such as GIST, a pharmaceutical product comprising the antibody and having therapeutic effects on a tumor, a method for treating a tumor using the aforementioned pharmaceutical product, and the like. It is another object of the present invention to provide an anti-GPR20 antibody having internalization activity, an antibody-drug conjugate containing the antibody, and the like.

Abstract: An object of the present invention is to provide a novel anti-CD147 antibody exhibiting potent antitumor efficacy and having excellent safety. Another object of the present invention is to provide a pharmaceutical product comprising such an antibody. Another object of the present invention is to provide a method for treating tumors using the antibody or the pharmaceutical product, for example. The present invention provides a CD147-specific antibody that activates CD147 and exhibits high antitumor efficacy. The present invention provides the anti-CD147 antibody that exhibits high antitumor efficacy independent of effector functions. The present invention provides a pharmaceutical composition comprising such an anti-CD147 antibody. The present invention provides a method for treating tumors using such an anti-CD147 antibody and/or pharmaceutical composition.

Abstract: An ophthalmic aqueous composition comprises levofloxacin, a salt thereof, or a solvate thereof; dexamethasone, an ester thereof, or a salt thereof; and one or at least two isotonic agents. The ophthalmic aqueous composition is substantially free of sodium chloride. This ophthalmic aqueous composition is excellent in drug stability and drug migration and has a clear appearance.

Abstract: An object of the present invention is to provide a compound having an anti-inflammatory activity or a pharmacologically acceptable salt thereof. The solution of the present invention is a compound of general formula (1) or a pharmacologically acceptable salt thereof. wherein the symbols in the formula are defined below: A: e.g., Benzene, E: e.g., —CH2—, G: e.g., a 5-membered aromatic heterocyclic ring, X: e.g., cyclohexane, J: e.g., a 5-membered aromatic heterocyclic ring, Y: e.g., a phenyl group, R1, R2, R3: e.g., a halogen atom, R4: e.g., a C1-C6 alkyl group, R5: e.g., a hydrogen atom, R6a, R6b, R6c, R6d: e.g., a hydrogen atom, R7: e.g., a hydrogen atom, R8: e.g., a hydrogen atom, n1, n2, n3: e.g., 1.

Abstract: Provided are novel crystals of [2-(1-methyl-1H-pyrazol-4-yl)-6-(morpholin-4-yl)-9H-purin-8-yl][4-(morpholin-4-yl)piperidin-1-yl]methanone and a pharmaceutically acceptable salt thereof having advantageous characteristics. [2-(1-Methyl-1H-pyrazol-4-yl)-6-(morpholin-4-yl)-9H-purin-8-yl][4-(morpholin-4-yl)piperidin-1-yl]methanone and a pharmaceutically acceptable salt thereof provided by the present invention are excellent in stability and have satisfactory physical properties as a drug substance of a pharmaceutical product.

Abstract: An object of the present invention is to provide a medicine that can simply treat and/or prevent a retinal degenerative disease associated with photoreceptor degeneration, including retinitis pigmentosa. The solution is to provide an agent for treating and/or preventing a retinal degenerative disease associated with photoreceptor degeneration, containing a compound having a retinoic acid receptor agonistic activity (for example, tamibarotene, tamibarotene methyl ester, tamibarotene ethyl ester, tazarotene, tazarotenic acid, adapalene, palovarotene, retinol, isotretinoin, alitretinoin, etretinate, acitretin or bexarotene) or a salt thereof.