Abstract: The present invention provides a method for inducing differentiation of motor neurons with less burden on a donor. The method for producing motor neurons from urine-derived cells according to the present invention includes a step of introducing into the urine-derived cell a transcription factor for inducing differentiation into a motor neuron. The transcription factor is introduced into the urine-derived cells by an adenoviral vector. The method of the present invention can further include a step of culturing the urine-derived cell into which the transcription factor has been introduced to obtain a cell positive for (I) at least one selected from the group consisting of Tuj1 and ISL1, and positive for (II) at least one selected from the group consisting of HB9, ChAT, and SMI32, preferably a cell positive for at least SMI 32 for (II).

Abstract: This present invention relates to BCMA binders (e.g. antibodies) and chimeric antigen receptor (CAR) constructs comprising a BCMA antigen binding molecule. The BCMA binders specifically bind to BCMA. The present BCMA CARs further comprise a hinge region (e.g., CD28 hinge), a transmembrane domain, and one or more intracellular NK cell signalling domains. NK cells expressing a BCMA CAR has increased efficacy in killing cancer cells. Provided herein also include therapeutic uses of the BCMA binders and BCMA CARs.

Abstract: The invention relates to parathyroid hormone (PTH) variants and pharmaceutical compositions comprising same. The invention further relates to PTH compositions with improved serum half-life and peak-trough ratios, and methods of controlling serum calcium levels with the PTH variants and compositions of the invention. The invention further relates to methods of treating hypoparathyroidism and/or hypocalcemia due to hypoparathyroidism with the PTH variants and compositions of the invention.

Abstract: The present invention relates generally to the field of pharmaceuticals, and specifically relates to isofagomine (IFG), novel salts thereof and preparation methods and uses of these, for example, in formulating pharmaceutical compositions for the treatment of Gaucher disease. Also provided are novel crystalline forms of isofagomine salts, methods for preparing the crystalline forms, and their use in formulating pharmaceutical compositions.

Abstract: Antibody formulations are described comprising a mixture of an anti-?4?7 antibody, an antioxidant or chelator, and at least one free amino acid. The disclosed formulations may have improved stability, reduced aggregate formation, or both. The present invention further provides a safe dosing regimen of these antibody formulations that is easy to follow, and which results in a therapeutically effective amount of the anti-?4?7 antibody in vivo.

Abstract: Disclosed herein are antibodies capable of binding to plasma kallikrein and inhibit its activity. Such antibodies interact with one or more critical residues in the catalytic domain of the plasma kallikrein. The antibodies may also contain specific heavy chain complementarity determining region 3 (CDRs) motifs and optionally specific residues at certain positions within both the heavy chain variable region and the light chain variable region.

Abstract: Provided is a substituted piperidine compound having an orexin type 2 receptor agonist activity. A compound represented by the formula (I): wherein each symbol is as described in the DESCRIPTION, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as a prophylactic or therapeutic agent for narcolepsy.

Abstract: The present invention relates to formulations of ADAMTS13 with enhanced or desirable properties. As such, the invention provides liquid and lyophilized formulations of ADAMTS13 that are suitable for pharmaceutical administration. Among other aspects, the present invention also provides methods of treating various diseases and conditions related to VWF and/or ADAMTS13 dysfunction in a subject. Also provided herein are kits comprising ADAMTS13 formulations useful for the treatment of various diseases and conditions.

Abstract: The present invention relates to methods for inactivating a lipid-enveloped virus using environmentally compatible detergents, and to methods for preparing a biopharmaceutical drug using environmentally compatible detergents. The invention also provides environmentally compatible detergents.

Abstract: Adeno-associated liquid and lyophilized pharmaceutical compositions are provided herein. In exemplary aspects, the pharmaceutical compositions comprise about 5 mM to about 25 mM L-histidine, about 0 mM to about 150 mM sodium chloride, about 0.001% (w/v) to about 0.01% (w/v) polysorbate 80 (PS80), and about 1% to about 10% (w/v) sucrose, trehalose, or combination thereof to AAV. In exemplary aspects, the pharmaceutical compositions further comprise glycine or mannitol. Methods of preparing a pharmaceutical composition comprising AAV, methods of treating a bleeding disorder in a subject, and methods of storing AAV compositions are also provided.

Abstract: The invention features new a method for treating inflammatory bowel disease, including Crohn's Disease (CD) or ulcerative colitis in a human patient, comprising administering to a patient a combination therapy comprising an anti-?4?7 antibody, a TNF? antagonist, and an immunomodulator.

Abstract: Provided herein are methods of producing an adeno-associated virus (AAV) product, methods of purifying adeno-associated virus, and methods of purifying full AAV capsids from a concentrated AAV fraction comprising empty AAV capsids and full AAV capsids.

Abstract: The present disclosure is directed to a device for screening pre-filled syringes configured to determine whether a stopper position within each syringe falls within an acceptable tolerance prior to final assembly of a syringe into a corresponding autoinjector device, thereby ensuring proper fit of the syringe within the autoinjector device and further ensuring accurate delivery of a desired dose of fluid from the syringe during operation of the autoinjector.

Abstract: Described herein are bispecific antibodies that bind to plasma kallikrein (pKal) and Factor XII and methods of producing and using such bi-specific antibodies for treating diseases or disorders associated with the contact system, e.g., hereditary angioedema or thrombosis.

Abstract: The present disclosure is directed to a vial adapter for interconnecting a vial and a fluid delivery device, and, more particularly, to a vial adapter having a modular design consisting of separately constructed components cooperatively arranged and coupled to one another. The modular construction allows for rapid manufacturing reconfigurations of one or more components with minimal costs to create new vial adapter configurations that meet specific needs.

Abstract: Disclosed is a method for predicting the gene transfer efficiency of animal cells, comprising (1) measuring the size of transgenic animal cells, and (2) predicting the gene transfer efficiency based on values measured in step (1).

Abstract: Disclosed are compounds of Formula 1, stereoisomers thereof, and pharmaceutically acceptable salts thereof, wherein L, r, s, R5, R6, R7, R9, R10, R11, R12, X1, X2, X3, X4, X13, and X14 are defined in the specification. This disclosure also relates to materials and methods for preparing compounds of Formula 1, to pharmaceutical compositions which contain them, and to their use for treating diseases, disorders, and conditions associated with SSTR4.

Abstract: The present disclosure provides antibody drug conjugates comprising STING modulators. Also provided are compositions comprising the antibody drug conjugates. The compounds and compositions are useful for stimulating an immune response in a subject in need thereof.

Abstract: The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.