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.

Abstract: The present invention provides a novel antisense oligonucleotide and a composition for preventing or treating glycogen storage disease type Ia. The present invention provides an antisense oligonucleotide which hybridizes with a pre-mRNA sequence derived from a region including at least one of a base at position 42911000, a base at position 42911004, and a base at position 42911005 in a base sequence of human chromosome 17 of GRCh38/hg38 and has activity to inhibit aberrant splicing of pre-mRNA of c.648G>T variant G6PC.

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 die 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.648C>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: The present invention provides a novel anti-DLL3 antibody-pyrrolodiazepine derivative and a novel anti-DLL3 anti-body-pyrrolodiazepine derivative conjugate using the same.

Abstract: It is an object of the present invention to provide an antibody-drug conjugate of an antibody binding to DLL3 and a drug having antitumor activity, a pharmaceutical composition comprising the antibody-drug conjugate and having therapeutic effects on a tumor, a method for treating a tumor using the antibody-drug conjugate or the pharmaceutical composition, and the like. The present invention provides an antibody-drug conjugate of an antibody binding to DLL3 and a drug having antitumor activity, a pharmaceutical composition comprising the antibody or the antibody-drug conjugate, and a method for treating a tumor.

Abstract: A novel antibody that can be used as an anti-tumor agent and an anti-tumor agent that comprises, as an active ingredient, a molecule containing such an antibody.

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: Provided is an oligonucleotide which may induce an editing activity of ADRC in cell and has excellent stability in a living body. The oligonucleotide includes a first oligonucleotide identifying a target RNA and a second oligonucleotide linked to the 5?-side of the first oligonucleotide. The first oligonucleotide consists of a target-corresponding nucleotide residue, an oligonucleotide of 10 to 24 residues at the 3?-side, and an oligonucleotide of 3 to 6 residues at the 5?-side. The second oligonucleotide has no nucleotide residue corresponding to a nucleotide residue of the target RNA or has a nucleotide residue which does not form a complementary pair at the 3?-end thereof and the number of residue is 3 to 6.

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.

Abstract: The present invention relates to a compound or a pharmacologically acceptable salt thereof having excellent tissue non-specific alkaline phosphatase inhibitory activity. The present invention provides a compound represented by the following formula (I): wherein X1 represents a nitrogen atom or CR9, R1 represents a hydrogen atom, a C1-C6 alkyl group, or a C1-C6 alkoxy group, R2 represents a halogen atom, R3 represents a hydrogen atom or a halogen atom, R4 represents a hydrogen atom or a halogen atom, and R5 represents a C1-C3 alkylsulfonyl group, a substituted C1-C6 alkyl group, a substituted C1-C6 haloalkyl group, a substituted C1-C6 alkoxy group, or a substituted C1-C6 alkylamino group, or a pharmacologically acceptable salt thereof.

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

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: The present invention provides a novel peptide that has an amino acid sequence represented by SEQ ID NO: 18, and binds to an active protease but does not bind to a pro-protease.

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: 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: To provide a novel pharmaceutical use of a peptide. A pharmaceutical composition for the treatment or prevention of retinitis pigmentosa, comprising a peptide which comprises the amino acid sequence shown in SEQ ID NO: 30 and inhibits the protease activity.

Abstract: The present invention provides a novel compound or a pharmaceutically acceptable salt thereof which has a TLR4 activating effect and can be used as an immunostimulant or an adjuvant in vaccines or allergen immunotherapy. The present invention provides a compound represented by the general formula (I) or a pharmaceutically acceptable salt thereof. In this context, X, Y, Z and n in the formula (I) are each as defined herein.

Abstract: A complex of ?(1?3) glucan and a nucleic acid, and having a controlled particle size is provided by the present invention. Furthermore, a complex of ?(1?3) glucan and a nucleic acid, and having a controlled particle size is provided by the present invention.

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: A pharmaceutical composition and a method of treatment having excellent antitumor effect and safety are provided. A pharmaceutical composition and a method of treatment wherein an antibody-drug conjugate, in which a drug-linker and an antibody are bound by a thioether bond represented by the following formula (A represents the binding position with an antibody), and a SIRP?-CD47 interaction inhibitor, such as an anti-SIRP? antibody, are administrated in combination. Alternatively, a pharmaceutical composition and a method of treatment wherein said antibody-drug conjugate and SIRP?-CD47 interaction inhibitor, such as an anti-SIRP? antibody, are administrated in combination and are used for the treatment of a disease improved by an action of activating antitumor immunity.