Abstract: The present disclosure provides methods of treating an antibody-mediated disorder (e.g., an autoimmune disease) using an FcRn antagonist, e.g., a binding polypeptide (e.g., an antibody and/or an immunoadhesin), comprising a modified Fc domain. The present disclosure also provides methods of enhancing diagnostic imaging using an FcRn antagonist disclosed herein. The present disclosure further provides methods of reducing exposure of normal tissue to a radiolabeled antibody during diagnostic imaging using an FcRn antagonist disclosed herein. Methods comprising administering to a subject a therapeutically effective amount of an FcRn antagonist disclosed herein are also provided.

Abstract: The present disclosure provides methods of producing in a single cell a mixed population of recombinant adeno-associated viruses for expressing two or more transgenes and methods of producing recombinant adeno-associated viruses containing self-complementary genomes. Also provided are methods of treating diseases using the rAAV compositions produced herein.

Abstract: The present disclosure includes, among other things, CFTR modulators, pharmaceutical compositions, and methods of making and using the same.

Abstract: A medicament delivery device has a needle for injecting medicament into a user, a body having a proximal end and a distal end and a needle cover. The needle cover is axially movable relative to the body between an initial position, in which the needle cover covers the distal end of the needle, and a holding position. In the holding position the needle protrudes from the distal end of the needle cover. The needle cover has a flexible arm. The device further has a needle cover guide comprising a track. The flexible arm engages the track. The track has a radially-extending ramp configured to flex the flexible arm when the needle cover moves from the initial position to the holding position. The flexible arm is flexed when the needle cover is in the holding position.

Abstract: The present disclosure relates to heterocyclic compounds, pharmaceutically acceptable salts thereof, and pharmaceutical preparations thereof. Also described herein are compositions and the use of such compounds in methods of treating diseases and conditions mediated by deficient CFTR activity, in particular cystic fibrosis.

Abstract: Methods for controlling a biological manufacturing system include directing a light beam to pass through a wall of a vessel containing a first fluid generated by the biological manufacturing system, measuring an angle of refraction of the light beam in the first fluid, the angle of refraction corresponding to an angle between a propagation direction of the light beam in the first fluid and a normal to an interface between the vessel wall and the first fluid, determining information about the first fluid based on the measured angle of refraction, and adjusting a parameter of the biological manufacturing system based on the information about the first fluid.

Abstract: The present disclosure relates to substituted aryl amide compounds, according to Formula (I): pharmaceutically acceptable salts thereof, and pharmaceutical preparations thereof. Also described herein are compositions and the use of such compounds in methods of treating diseases and conditions mediated by deficient CFTR activity, in particular cystic fibrosis.

Abstract: The present disclosure relates to heterocyclic compounds, pharmaceutically acceptable salts thereof, and pharmaceutical preparations thereof. Also described herein are compositions and the use of such compounds in methods of treating diseases and conditions mediated by deficient CFTR activity, in particular cystic fibrosis.

Abstract: The present invention relates to a method of producing a polypeptide having ?-hexosaminidase activity, comprising the steps of a) providing a yeast cell comprising a polynucleotide encoding a polypeptide having ?-hexosaminidase activity and having an amino acid sequence being at least 95% identical to the amino acid sequence shown in SEQ ID NO: 1, b) cultivating said yeast cell under conditions which allow for the production of the polypeptide, and c) obtaining the polypeptide produced in step b). The present invention further concerns a polynucleotide encoding a polypeptide having ?-hexosaminidase activity and having an amino acid sequence being at least 95% identical to the amino acid sequence shown in SEQ ID NO: 1, as well as polypeptide encoded by said polynucleotide. Moreover, the present invention concerns a yeast cell comprising the polynucleotide of the present invention.

Abstract: The present disclosure relates to a medicament delivery device. The medicament delivery device comprises a housing, a needle, and a needle cover. The needle cover is axially movable between an extended position and a retracted position. The medicament delivery device comprises a needle cover biasing member configured to bias the needle cover axially in the distal direction towards the extended position. The needle cover biasing member is configured to transition from a non-buckled state to a buckled state during proximal movement of the needle cover from the extended position to the retracted position. The present disclosure also relates to a method of preparing a medicament delivery device, and a method of locking a medicament delivery device.

Abstract: The present disclosure includes, among other things, CFTR modulators, pharmaceutical compositions, and methods of making and using the same.

Abstract: A medicament delivery device for injecting medicament is described. The device has a body having a proximal end and a distal end, a needle for injecting medicament, an actuation member which is movable relative to the body from a first position to a second position for dispensing medicament from the needle, a lock ring which is rotatable relative to the body from a pre-use position, in which movement of the actuation member from the first position to the second position is prevented, to a use position in which movement of the actuation member from the first position to the second position is permitted and a cap which is removably attached to the medicament delivery device. The cap has a portion which is configured to prevent rotation of the lock ring from the pre-use position to the use position when the cap is attached to the medicament delivery device.

Abstract: Systems for measuring a product quality attribute of an analyte of a biological sample include a first flow control device, a sample purification device, a second flow control device in fluid communication with first and second sample analyzers, where the first sample analyzer includes a first chromatography column, and a control unit configured so that during operation of the system, the control unit adjusts a configuration of the second flow control device to direct a portion of the biological sample to one of the first and second sample analyzers, and determines a product quality attribute of an analyte of the biological sample based on an analysis of the portion of the biological sample by the one of the first and second sample analyzers.

Abstract: The present invention relates to humanized monoclonal antibodies comprising the CDRs of murine antibody BMA031, which bind to the apTCR.CD3 complex and possess improved biological properties.

Abstract: The present invention relates to antibody molecules, in particular antibody molecules that bind Transforming Growth Factor beta (TGF?) and uses thereof. More particularly, the invention relates to antibody molecules that bind and preferably neutralise TGF?1, TGF?2 and TGF?3, so-called “pan-specific” antibody molecules, and uses of such antibody molecules. Preferred embodiments within the present invention are antibody molecules, whether whole antibody (e.g. IgG, such as IgG1 or IgG4) or antibody fragments (e.g. scFv, Fab, dAb).

Abstract: Provided herein are methods of selecting an optimal fixed orientation of a dissolved oxygen (DO) probe in a perfusion bioreactor and culturing a mammalian cell in a perfusion bioreactor having the DO probe positioned at the selected fixed orientation.

Abstract: The present disclosure provides methods of screening, diagnosing, monitoring and/or treating acid sphingomyelinase (ASM) disorders such as Niemann-Pick disease. In particular, the methods encompass techniques for improved diagnosis and/or treatment of an ASM disorder, for example using enzyme replacement therapy.

Abstract: Provided herein are methods and compositions for batch production of producer cells using fluorescence activated cell sorting (FACS). In some aspects, the disclosure provides a drug-selection-free method for batch production of producer cells using FACS. Such batch production methods and compositions can be further utilized to generate clonal populations of producer cells, e.g., for large-scale manufacturing of a polypeptide of interest.

Abstract: The invention relates to heteroaryl compounds, pharmaceutically acceptable salts thereof, and pharmaceutical preparations thereof. Also described herein are compositions and the use of such compounds in methods of treating diseases and conditions mediated by deficient CFTR activity, in particular cystic fibrosis.

Abstract: The present technology relates to a novel sanitization method for chromatography media and supporting equipment comprising treatment with a sanitization/sterilization solution comprising acetic acid and hexylene glycol.