Abstract: The present technology relates to a novel cleaning method for chromatography media and/or supporting equipment comprising treatment with a novel cleaning solution comprising sodium hydroxide and hexylene glycol.

Abstract: The current disclosure provides binding polypeptides (e.g., antibodies), and targeting moiety conjugates thereof, comprising a site-specifically engineered glycan linkage within native or engineered glycans of the binding polypeptide. The current disclosure also provides nucleic acids encoding the antigen-binding polypeptides, recombinant expression vectors and host cells for making such antigen-binding polypeptides. Methods of using the antigen-binding polypeptides disclosed herein to treat disease are also provided.

Abstract: A vial adapter comprises an adapter body comprising a top wall and a sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a spike fixed to the base part. The spike extends through the top wall in a distal direction into the receptacle. The movable part is movable relative to the adapter body from an undeployed position into a deployed position. An interlock is operable to lock the movable part in the undeployed position, and an actuating member extending into the receptacle is coupled to the interlock and operable to unlock the interlock when actuated.

Abstract: A medical device for mixing a medicament contained within a medicament vial, wherein the medical device comprises a track configured to support the medicament vial, and wherein the track comprises an inclined first track section arranged such that, in use, the medicament vial can roll along the track to mix the medicament contained within the medicament vial. A system comprising the medical device and a medicament vial containing the medicament. A method for using the medical device.

Abstract: The disclosure provides methods of purifying adeno-associated virus (AAV) particles by introducing a flocculant step into a large scale a manufacturing process, thereby improving AAV particle purity and yield.

Abstract: Compositions and methods for treating macular degeneration are disclosed. The methods utilize gene delivery to human eyes of soluble Flt-1 receptors, as well fusion proteins including a soluble Flt-1 receptor.

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.