Abstract: The present invention provides a technique that enables introduction of active ingredients, in particular, nucleic acids, into cells with superior efficiency; and cationic lipids, etc., for use in the technique. The compound or a salt thereof according to the present invention is a compound represented by formula (I) or a salt thereof. In formula (I), n represents an integer of 2 to 5, R represents a linear C1-5 alkyl group, a linear C7-11 alkenyl group, or a linear C11 alkadienyl group, and wavy lines each independently represent a cis-bond or a trans-bond.

Abstract: The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity. A compound represented by the formula (I): wherein each symbol is as described in the specification, or a salt thereof, is useful as an agent for the prophylaxis or treatment of narcolepsy.

Abstract: Characterization of drug-drug interaction properties and pharmacological properties of maribavir is useful to inform potential drug-drug interactions and dosing strategies when administering with co-medications.

Abstract: The present invention provides the following method as an approach for enriching desired cells, particularly, yolk sac mesoderm cells or amniotic ectoderm cells, contained in an embryogenesis region-specific micropattern structure of a cell cluster formed by differentiation-inducing factors from a colony of pluripotent stem cells such as iPS cells: a method for producing a cell cluster enriched in desired cells from pluripotent stem cells, comprising the step of two-dimensionally culturing the pluripotent stem cells under Wnt signal regulation.

Abstract: The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.

Abstract: Provided herein are methods of producing an adeno-associated virus (AAV) product and methods of purifying adeno-associated virus. AAV is loaded onto an affinity resin, wash steps are undertaken, and AAV is eluted from the affinity resin. Various buffers are disclosed for use in the wash steps and elution.

Abstract: The present disclosure relates to Zika virus vaccines and immunogenic compositions having one or more antigens from a Zika virus (e.g., a Zika virus clonal isolate, a non-human cell adapted Zika virus, etc.), and methods of treatments and uses thereof.

Abstract: The invention relates to COnditional Bispecific Redirected Activation constructs, or COBRAs, that are administered in an active pro-drug format. Upon exposure to tumor proteases, the constructs are cleaved and activated, such that they can bind both tumor target antigens (TTAs) as well as CD3, thus recruiting T cells expressing CD3 to the tumor, resulting in treatment.

Abstract: Provided herein are methods and kits for analyzing a biological sample obtained from a subject having, suspected of having, or being at risk for a disease associated with the contact activation system.

Abstract: The present invention aims to provide a compound that may be useful for the prophylaxis or treatment of constipation and the like. The present invention provides a compound represented by the formula (I): wherein R1 is a hydrogen atom, a halogen atom, an optionally substituted C1-6 alkyl group, or an optionally substituted C1-6 alkoxy group; and ring A is an optionally further substituted ring, or a salt thereof.

Abstract: An antibody-resin coupling apparatus quickly and efficiently activates resin beads and couples them to antibodies, while preventing breakdown and crosslinking of the beads, thereby improving downstream column purification processes, extending the usable life of the resin beads, and increasing molecule capture efficiency of the resultant resin-antibody complexes, to allow improved isolation and purification of factor VIII molecules or other drug compounds.

Abstract: The present disclosure relates to methods for inactivating a Zika virus which can be used in vaccines and immunogenic compositions. The present disclosure also relates to a method for determining the completeness of inactivation of an arbovirus preparation.

Abstract: The present invention provides drug toxicity evaluation platforms (such as an evaluation method and a kit therefor) that enable detailed analysis of the possibility and the like of developing drug-induced damage (such as DILI) to the liver and other organs. The method for evaluating drug toxicity of the present invention includes: a step of adding a drug to a co-culture system of an organoid and blood cells; and a step of evaluating the toxicity of the drug to the organoid.

Abstract: The disclosure relates to antibodies that bind FcRn and methods of using these antibodies.

Abstract: Compounds and pharmaceutical compositions that modulate kinase activity, including mutant EGFR and mutant HER2 kinase activity, and compounds, pharmaceutical compositions, and methods of treatment of diseases and conditions associated with kinase activity, including mutant EGFR and mutant HER2 activity, are described herein.

Abstract: Methods for treating uterine fibroids, endometriosis, adenomyosis, or heavy menstrual bleeding in a subject, which include administering to the subject from 10 mg to 60 mg per day of N-(4-(1-(2,6-difluorobenzyl)-5-((dimethylamino)methyl)-3-(6-methoxy-3-pyridazinyl)-2,4-dioxo-1,2,3,4-tetrahydrothieno[2,3-d]pyrimidin-6-yl)phenyl)-N?-methoxyurea, and from 0.01 mg to 5 mg per day of a hormone replacement medicament. The present disclosure has methods for reducing menstrual bleeding in a subject, reducing bone mineral density loss in a subject caused by administering a GnRH antagonist to the subject, suppressing sex hormones in a subject, reducing vasomotor symptoms or hot flashes in a subject, and reducing symptoms of decreased libido in a subject having uterine fibroids, endometriosis, or adenomyosis.

Abstract: The present invention provides a heterocyclic compound having a HDAC6 inhibitory action, which is useful for the treatment of central nervous system diseases including neurodegenerative diseases, and the like, and a medicament comprising the compound. The present invention relates to a compound represented by the formula (I): wherein each symbol is as defined in the description, or a salt thereof.

Abstract: As a technique for proliferating neural crest cells without reducing differentiation capacity, provided is a method for producing neural crest cells, comprising the steps of: (1) obtaining neural crest cells; and (2) suspension-culturing the neural crest cells in a medium comprising a GSK3? inhibitor and a basic fibroblast growth factor (bFGF), wherein the medium comprises the GSK3? inhibitor with a concentration that exhibits an effect equivalent to that exhibited by CHIR99021 with a concentration of higher than 1 ?M and lower than 5 ?M.

Abstract: Provided are combinations, compositions and kits containing a immune globulin (IG) composition and a soluble hyaluronidase composition formulated for subcutaneous administration. Such products can be used in methods of treating IG-treatable diseases or conditions. Also provided are methods for subcutaneous administration of immune globulin whereby the dosing regimen is substantially the same as for intravenous administration of the same dosage for treatment of the same IG-treatable disease or condition.