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 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: 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 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: 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 invention provides compounds of Formula I?: compositions thereof, and methods of using the same for the inhibition of TYK2, and the treatment of TYK2-mediated disorders.

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.

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: Provided herein are expanded allogeneic adipose tissue-derived stromal stem cells for use in treating complex perianal fistulas in Crohn's Disease.

Abstract: A medicinal fluid pooling device may be used to pool multiple containers of medicinal fluid to facilitate administration of the medicinal fluid to a patient. A medicinal pooling device may include spikes covered by spike sheaths which are pierced when a container of medicinal fluid is inserted into the medicinal pooling device. The medicinal pooling device may also include a cover configured to cover the spikes. The medicinal pooling device may also include a fluidic interface which may be used to fluidly connect the medicinal pooling device to an infusion pump or syringe.

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: Provided is a compound that can have an effect of inhibiting MALT1 and is expected as useful as a prophylactic or therapeutic drug for cancer, etc. A compound represented by formula (I) [wherein each symbol is as defined in the description], a salt thereof, or a cocrystal, a hydrate or a solvate of the same.

Abstract: The present invention provides, among other things, methods and compositions for treating complement mediated disease. In some embodiments, recombinant human C1 esterase inhibitor proteins having similar or longer half-life than native plasma-derived human C1 esterase inhibitor, and methods of making the same are provided. In some embodiments, the invention provides a method for administering an effective amount of a recombinant human C1 esterase inhibitor protein to an individual who is suffering from or susceptible to a complement-mediated disease such that at least one symptom or feature of said complement-mediated disease is prevented and/or reduced in intensity, severity, or frequency.

Abstract: Isolated antibodies that bind to human CD38 and cynomolgus CD38 are disclosed. Also disclosed are pharmaceutical compositions comprising the disclosed antibodies, and therapeutic and diagnostic methods for using the disclosed antibodies.

Abstract: The present disclosure provides isolated, engineered, non-naturally occurring CB1 binding proteins, including anti-CB1 antibodies or antigen-binding fragment thereof. The CB1 binding proteins find utility in the treatment and diagnosis of CB1 mediated conditions, diseases and disorders.