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 treat a bleeding event in a subject having a hemophilia (e.g., with or without inhibitors).

Abstract: The present disclosure provides methods for treating and improving b1osteogenesis imperfecta (OI) in a subject by administering to the subject a therapeutically effective amount of an agent that binds and neutralizes transforming growth factor beta (TGF-?).

Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

Abstract: Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.

Abstract: Described herein are compositions of Thyroid Stimulating Hormone (TSH), wherein at least one polyalkylene glycol polymer is attached to a carbohydrate site of the TSH. Also described are compositions of mutated Thyroid Stimulating Hormone (TSH) and at least one polyalkylene glycol polymer, wherein the mutated TSH comprises a TSH in which one or more amino acid residues has been substituted with cysteine residue, and the polyalkylene glycol polymer is attached to the mutated TSH at the site of the substituted cysteine residue. Pharmaceutical compositions comprising these TSH compositions and method of treating a thyroid condition in a patient in need thereof, by administering to the patient an effective amount of the pharmaceutical compositions are also described.

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 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: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

Abstract: Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

Abstract: The present disclosure provides pharmaceutical compositions comprising anti-TGF-? antibodies and methods of use thereof.

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

Abstract: Disclosed herein are deuterated compounds of the formula which are useful as colony stimulating factor-1 receptor inhibitors (“CSF-1R inhibitors”).

Abstract: Compounds of the formulas which are useful as colony stimulating factor-1 receptor inhibitors (“CSF-1R inhibitors”).

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.

Abstract: Anti-FGFR3 antigen-binding proteins and antigen-binding binding fragments thereof are provided. Methods of inhibiting FGFR3 activity and methods of treating FGFR3-mediated diseases and disorders are also provided.

Abstract: Viral filters include a filter member featuring a first surface and a second surface and having a thickness extending between the first and second surfaces in a first direction, and a plurality of channels formed in the filter member, each of the channels having a channel axis, where during use, a solution carrying a viral load flows in a direction parallel to the first surface, and at least a portion of the viral load enters the membrane through the first surface and propagates in the first direction, and where for at least 50% of the channels in the filter member, the channel axis is oriented at an angle of between 5 degrees and 85 degrees relative to the first direction.

Abstract: An scFv-Fc dimer binds and neutralizes TGF?1 selectively and with high affinity and avidity. The scFv region may comprise the same VH and VL domains or CDR regions as metelimumab. The unique combination of their smaller size, high selectivity, potency against TGF?1, and long in vivo half-life makes the scFv-Fc dimers ideal candidates for therapeutic applications.

Abstract: A modified IgG antibody binds and neutralizes TGF?1 selectively and with high affinity and avidity. The modified IgG antibody comprises four polypeptide chains and may comprise modifications to the elbow regions of the polypeptide chains. The modified IgG antibody may comprise the same VH and VL domains or CDR regions as metelimumab. The modified IgG anti-body is useful in therapeutic and diagnostic applications.

Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.