Abstract: Engineered antibodies that are stable and can be conjugated to a ligand or drug at a ligand/drug to antibody ratio of above 3, making these conjugates suitable for treatment of a variety of indications, are provided. Methods of producing these engineered antibodies are also provided.

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: The present disclosure relates to methods and systems for the continuous production of recombinant proteins. In particular embodiments, the disclosure relates to methods and systems using capture chromatography, post-capture chromatography, virus filtration, and ultrafiltration/diafiltration for the continuous production of recombinant proteins.

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: Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.

Abstract: A medicament delivery device is described.

Abstract: This disclosure to methods for treating or preventing particular symptoms and disorders which are associated with lysosomal storage diseases using quinuclidine compounds of formula (I), optionally in combination with enzyme replacement therapy. This includes pain, such as abdominal pain, and dermatological disorders, such as angiokeratoma, in a patient having a disease such as Fabry disease. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods.

Abstract: The present disclosure provides anti-complement factor Bb antibodies, and compositions comprising the antibodies. The anti-Bb antibodies are useful for treating complement-me-diated disorders. The present disclosure provides methods of treating complement-mediated disorders.

Abstract: A medicament delivery device is described. The device includes a body having proximal and distal ends defining a longitudinal direction; a needle for injecting medicament into a user and configured to be arranged in an injecting position in which the needle protrudes from the distal end of the body; a cap moveable along the longitudinal direction to be arrangeable in a capped position in which the cap conceals the distal end of the body, and an uncapped position in which at least a portion of the cap is spaced apart from the distal end; and a blocking element. When the cap is in the uncapped position, the blocking element is arrangeable in a first state in which movement of the cap from the uncapped position into the capped position is permitted, and a second state in which movement of the cap from the uncapped position into the capped position is prevented.

Abstract: Provided are methods for the creation of ultra-high density cryopreserved cell banks. In certain embodiments, these methods employ altered perfusion culture techniques that allow for production of ultra-high density cell cultures that can be cryopreserved at unexpectedly high cell densities without the need for any cell concentration steps, while retaining excellent cell viability and quality.

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: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.