Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: System for performing chemical spectroscopy on samples from the scale of nanometers to millimeters or more with a multifunctional platform combining analytical and imaging techniques including dual beam photo-thermal spectroscopy with confocal microscopy, Raman spectroscopy, fluorescence detection, various vacuum analytical techniques and/or mass spectrometry. In embodiments described herein, the light beams of a dual-beam system are used for heating and sensing.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: An oral product includes a body that is wholly receivable in an oral cavity. The body includes a mouth-stable polymer matrix, cellulosic fibers embedded in the mouth-stable polymer matrix, and an additive dispersed in the mouth-stable polymer matrix. The oral product is adapted to release the additive from the body when the body is received within the oral cavity and exposed to saliva.

Abstract: Multilayer beads for pharmaceutical use having a drug-in-polymer layer are disclosed. The disclosed multilayer beads for pharmaceutical use have (a) a core particle; (b) an optional barrier layer coated on the surface of the core particle; (c) a drug-in-polymer layer coated on the surface of the core or the barrier layer, (d) an optional sealant layer coated on the surface of the drug-in-polymer layer; and (e) optionally one or more outer layers external to the drug-in-polymer layer or the sealant layer. The drug-in-polymer layer consists essentially of (i) a drug selected from the group consisting of a 15-keto prostaglandin drug, a 13,14-dihydro prostaglandin drug, and a 13,14-dihydro-15-keto prostaglandin drug; and (ii) a polymer selected from the group consisting of polyvinylpyrrolidone, vinylpyrrolidone-vinylacetate copolymer or a mixture thereof. The drug-in-polymer layer may be solid dispersion of the drug in the polymer.

Abstract: The liquid composition includes a psychoactive alkaloid extract or synthetic psychoactive alkaloid. The alkaloids in the extract are predominantly dephosphorylated rather than phosphorylated. The injectable psychoactive alkaloid composition includes a carrier, an optional tonicity modifier, an optional antioxidant or preservative, an optional pH modifier, and optional further excipients. A process for obtaining an injectable psychoactive alkaloid composition includes dephosphorylating the alkaloid during extraction, purifying the extracted alkaloid, and standardizing it to a specific concentration by adding measured quantities of excipients.

Abstract: This disclosure provides a novel compositions and methods to deliver cyclosporine A using genetically engineered protein polymers.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: The present invention relates to a peptide compound of PDL2 selected from a peptide fragment, a functional homologue, and a functional analogue, as well as to a nucleic acid, such as DNA or RNA, encoding the peptide compound, a vector, such as a virus vector, and a host cell, such as mammalian cell, comprising the vector. The peptide compound, nucleic acid, vector and host cell of the present invention are in particular, useful for the treatment or prevention of a cancer characterized by expression of PDL2.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Site-specific modification of proteins with microbial transglutaminase (MTG) is a powerful and versatile strategy for a controlled modification of proteins under physiological conditions. Solid-phase microbead-immobilization is used to site-specifically and efficiently attach different functional molecules important for further downstream applications to proteins of therapeutic relevance including scFV, Fab-fragment and antibodies. MTG remained firmly immobilized with no detectable column bleeding and enzyme activity was sustained during continuous operation. Immobilized MTG shows enhanced selectivity towards a certain residue in the presence of several reactive residues which are all targeted when the conjugation was carried out in solution. The generation of dual site-specifically conjugated IgG1 with immobilized and MTG in solution is reported, i.e. site-specific conjugation to glutamine and lysine residues of IgG1 antibody.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: The present disclosure relates to methods of treating, ameliorating or preventing a disorder comprising administering a therapeutically effective amount of a composition comprising a protein which inhibits the ligand of Uniprot #P05112 and/or the ligand of Uniprot #P35225 from binding to their respective receptors to a subject in need thereof. In some embodiments, the disorder is preferably associated with an increase of the Th2 immune response. In some embodiments, administration is preferably locally to the lung in order to treat, ameliorate or prevent allergic asthma, rhinitis, conjunctivitis, lung fibrosis, cystic fibrosis, chronic obstructive pulmonary disease, pulmonary alveolar proteinosis or adult respiratory distress syndrome.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: The present invention refers to a secretion-competent mutein of the ?-subunit of human Interleukin 27 and to a human heterodimeric Interleukin 27. The present invention further refers to a nucleic acid molecule comprising a nucleotide sequence encoding a secretion-competent mutein of the ?-subunit of human Interleukin 27 or the human heterodimeric Interleukin 27, to a host cell containing a nucleic acid molecule comprising a nucleotide sequence encoding a secretion-competent mutein of the ?-subunit of human Interleukin 27 or of the human heterodimeric Interleukin 27. The invention also refers to an immune modulator comprising a secretion-competent mutein of the ?-subunit of human Interleukin 27 or of the human heterodimeric Interleukin 27, to the respective use thereof as well as to a method of producing said secretion-competent muteins and to a secretion-incompetent mutein of the ?-subunit of mouse Interleukin 27 and a secretion-competent mutein of the ?-subunit of mouse Interleukin 27.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

Abstract: Disclosed are the methods of curing HBV infection and providing complete protection against HBV infection in a simplified HBV immunization schedule. The mechanistic basis for curing HBV infection is founded on the understanding that hepatitis B virus infection is established and prolonged by new rounds of infection with continuously produced viruses, which are not fully neutralized because of insufficient endogenous neutralizing antibodies. The methods of curing HBV infection including chronic HBV infection are aimed to block new rounds of HBV infection. The guidelines for establishing treatment regimens for curing HBV infection, include production or administration of sufficient level of HBV neutralizing antibodies in treated patients.

Abstract: The invention in suitable embodiments is directed to dynamic bio-nanoparticle elements and bio-nanoparticle platforms employing such bio-nanoparticle elements. In one aspect, one or more elements of one or more types, formed from isolated, synthetic and or recombinant amino acid residues comprising in whole or in part one or more types of Clathrin and or Coatomer I/II proteins of one or more isoforms, execute one or more functions and or effect one or more ends, in vivo and or in vitro.

Abstract: This disclosure provides a composition containing a conjugate with a modified insulin molecule. The conjugate has an insulin molecule, which can be insulin or an insulin analog, glucagon, GLP-1, GLP-2 or a GLP-1 agonist. The conjugate also contains one or more polymers. Each of the one or more polymers is covalently linked to the insulin molecule. Additionally, each of the one or more polymers is covalently linked to between 0 to 50 copies of a decoy ligand, and to between 0 to 50 copies of a glucose-binding agent, such that the combined total number of glucose-binding agents and decoy ligands covalently linked to each of the one or more polymers is at least 1. The conjugate can reversibly bind to soluble glucose and in which the extent of its glucose-binding controls the extent to which the modified insulin is able to bind to and activate the insulin receptor. Methods of making the conjugate, as well as use of the conjugate in treatment, are also provided.

Abstract: Provided are a fusion protein having a CCL3 variant with improved in vivo persistency, protein stability and pharmacological activity and a use thereof, more particularly, a fusion protein comprising a CCL3 variant and an immunoglobulin Fc region and a use thereof as a therapeutic agent for lymphopenia, cancer or infection, in which an N-terminal amino acid of a wild-type CCL3? or CCL3? is deleted and an amino acid at a specific position is substituted with a different amino acid at the same position of the wild-type CCL3? or CCL3? in the CCL3 variant.

Abstract: The present invention relates to extended recombinant polypeptide (XTEN) compositions, conjugate compositions comprising XTEN and XTEN linked to cross-linkers useful for conjugation to pharmacologically active payloads, methods of making highly purified XTEN, methods of making XTEN-linker and XTEN-payload conjugates, and methods of using the XTEN-cross-linker and XTEN-payload compositions.

Abstract: The invention provides peptides and analogs of INGAP and HIP peptides. The peptides and analogs can be used in methods for treating various diseases and conditions. Such diseases and conditions can include impaired pancreatic function, treating a metabolic disease, for example, diabetes, both type 1 and type 2 diabetes, islets induction, expansion and proliferation for transplantation, promoting neuroprotection or nerve regeneration, promoting liver regeneration or inhibiting inflammation.

Abstract: The present disclosure is directed to an engineered phospholipid or lysophospholipid (e.g., sphingosine 1-phosphate (S1P)) chaperone derived from an Apolipoprotein M (ApoM)-Fc fusion protein having an extended half life in vivo. The disclosed ApoM-Fc fusion protein provides a safe, efficient and effective means of delivering S1P to endothelial cells and to all tissues of the body.

Abstract: The present invention relates to binding fusion protein compositions comprising targeting moieties linked to extended recombinant polypeptide (XTEN), binding fusion protein-drug conjugate compositions, and XTEN-drug conjugate compositions, isolated nucleic acids encoding the compositions and vectors and host cells containing the same, and methods of using such compositions in treatment of diseases, disorders, and conditions.

Abstract: The disclosure provides a fusion polypeptide specific for both CD137 and HER2/neu, which fusion polypeptide can be useful for directing CD137 clustering and activation to HER2/neu-positive tumor cells. Such fusion polypeptide can be used in many pharmaceutical applications, for example, as anti-cancer agents and/or immune modulators for the treatment or prevention of human diseases such as a variety of tumors. The present disclosure also concerns methods of making the fusion polypeptide described herein as well as compositions comprising such fusion polypeptide. The present disclosure further relates to nucleic acid molecules encoding such fusion polypeptide and to methods for generation of such fusion polypeptide and nucleic acid molecules. In addition, the application discloses therapeutic and/or diagnostic uses of such fusion polypeptide as well as compositions comprising one or more of such fusion polypeptides.

Abstract: A linker-drug represented by formula (I) or a pharmaceutically acceptable salt or solvate thereof is provided. In formula (I), C is a conjugator, L is a linker unit, D is a toxin unit, and n is an integer ranging from 1 to 4. The structure of the conjugator is represented by formula (II). In formula (II), X is a leaving group, each of R1 and R2 is independently a single bond or —NH—, and Z is substituted aryl, heteroaryl, linear alkyl, cycloalkyl, heterocycloalkyl, or a combination thereof. The antibody is conjugated to the linker unit through a cysteine residue of the antibody. An antibody-drug conjugate (ADC) employing the above linker-drug is also provided.

Abstract: The present disclosure relates to methods of treating, ameliorating or preventing a disorder comprising administering a therapeutically effective amount of a composition comprising a protein which inhibits the ligand of Uniprot # P05112 and/or the ligand of Uniprot # P35225 from binding to their respective receptors to a subject in need thereof. In some embodiments, the disorder is preferably associated with an increase of the Th2 immune response. In some embodiments, administration is preferably locally to the lung in order to treat, ameliorate or prevent allergic asthma, rhinitis, conjunctivitis, lung fibrosis, cystic fibrosis, chronic obstructive pulmonary disease, pulmonary alveolar proteinosis or adult respiratory distress syndrome.

Abstract: The present invention provides a method for diagnosing in a subject Acute Kidney Injury or a related or analogous disease state. The method comprises the steps of a) providing a sample taken from the subject: b) analysing the sample from the subject to determine the level of a biomarker selected from certain atomic species; and c) comparing the level of the biomarker in the sample to a reference level of the biomarker that is characteristic of a healthy subject, eg a subject having normal renal function. An elevated level of the biomarker in the sample relative to the reference level is indicative of Acute Kidney Injury or a related or analogous disease state. The invention also relates to the use of an atomic species selected from those in Table 1 as a biomarker for Acute Kidney Injury or a related or analogous disease state, and test kits comprising an analytical element sensitive to those biomarkers.

Abstract: Disclosed are short DNA aptamers that selectively recognize CD200R1, a protein expressed on the surface of myeloid and lymphoid cells that delivers immune inhibitory signals to modulate inflammation when engaged with its ligand, CD200. Also disclosed is the use of said aptamers as therapeutic agents, for the purpose of decreasing inflammatory response; treatment of spinal cord injury; treatment of an immune related disease such as arthritis, asthma, allergy, infection; as a course of treatment during or after transplantation; or for treatment of an autoimmune disorder such as systemic lupus erythematosus, Parkinson's Disease, or multiple sclerosis.

Abstract: The monoclonal IgG-type antibodies were suggested comprising variable domains represented by a combination of VHH-derivative with a variable domain of the light chain VL. Said antibodies can comprise amino acid substitutions at positions 44 and 45 (Kabat numbering) or combinations thereof. Antibodies of the invention possess increased affinity and improved aggregation stability.

Abstract: Disclosed herein are Htt repressors and methods and compositions for use of these Htt repressors.

Abstract: The present disclosure relates to methods and computational tools based, at least in part, on computer simulations that identify hot-spot amino acid residues and binding-region amino acid residues of a protein.

Abstract: Bi-specific fusion proteins with therapeutic uses are provided, as well as pharmaceutical compositions comprising such fusion proteins, and methods for using such fusion proteins to repair or regenerate damaged or diseased tissue. The bi-specific fusion proteins generally comprise: (a) a targeting polypeptide domain that binds to a target molecule; and (b) an activator domain that detectably modulates tissue regeneration.

Abstract: The present invention relates generally to stable formulations comprising CTLA4Ig molecules, including lyophilized, and liquid formulations for administration via various routes including, for example, routes such as intravenous (IV) and subcutaneous (SC) for treating immune system diseases and tolerance induction.

Abstract: This disclosure provides an isolated polypeptide comprising no more than 35 amino acids, wherein the amino acid sequence comprises, or alternatively consists essentially of, or alternatively consisting of XXXRYSYXXSYX (SEQ ID NO: 1) and equivalents thereof, wherein X is a basic amino acid and Y is a hydrophobic amino acid. Polynucleotides encoding the polypeptides and antibodies that bind to the polypeptides are also provided. Therapeutic and diagnostic uses are further provided.

Abstract: The present invention relates to new extended release pharmaceutical compositions and methods of use thereof for the treatment of disorders. In certain embodiments, disclosed herein is an extended-release pharmaceutical formulation comprising, in a solid dosage form for oral delivery of between about 100 mg and about 1 g total weight: between about 2 and about 18% of an active ingredient; between about 70% and about 96% of one or more diluents; between about 1% and about 10% of a water-soluble binder and between about 0.5 and about 2% of a surfactant.

Abstract: Compositions and methods for conferring lepidoptericidal activity to bacteria, plants, plant cells, tissues and seeds are provided. Compositions comprising a coding sequence for the Axmi222z toxin polypeptide are provided. The Axmi222z coding sequences can be used in DNA constructs or expression cassettes for transformation and expression in plants and bacteria. Compositions also comprise transformed bacteria, plants, plant cells, tissues, and seeds. In particular, isolated Axmi222z toxin nucleic acid molecules are provided. Additionally, amino acid sequences corresponding to the Axmi222z polynucleotides are encompassed, and antibodies specifically binding to those amino acid sequences. In particular, the present invention provides for isolated nucleic acid molecules comprising nucleotide sequences encoding the amino acid sequence shown in SEQ ID NO:24-26, or the nucleotide sequence set forth in SEQ ID NO:2, 7, 12, and 17, as well as variants and fragments thereof.

Abstract: The present invention relates to compositions and methods for treating autoimmune diseases by administering to a subject a CTLA4 molecule that block endogenous B7 molecules from binding their ligands.

Abstract: The present disclosure provides compositions and methods for treating diseases or conditions associated with an abnormal level or activity of biglycan; disorders associated with an unstable cytoplasmic membrane, for example, due to an unstable dystrophin associated protein complex (DAPC); disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation. Examples of diseases include muscular dystrophies, such as Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, neuromuscular disorders and neurological disorders.

Abstract: Compositions and methods for conferring insecticidal activity to host cells are provided. Compositions comprising a coding sequence for a delta-endotoxin polypeptide are provided. The coding sequences can be used in DNA constructs or expression cassettes for transformation and expression in host cells. Compositions also comprise transformed host cells. In particular, isolated delta-endotoxin nucleic acid molecules are provided. Additionally, amino acid sequences corresponding to the polynucleotides are encompassed, and antibodies specifically binding to those amino acid sequences. In particular, the present invention provides for isolated nucleic acid molecules comprising nucleotide sequences encoding the amino acid sequence shown in SEQ ID NO:4, 5, 6, 13, or 14, or the nucleotide sequence set forth in SEQ ID NO:1, 2, 3, 11, or 12, as well as variants and fragments thereof.

Abstract: The present invention provides a method for inducing chronic elevation of intraocular pressure in the eyes of an animal by introducing into the eyes a cross-linking hydrogel, an animal produced by this method, as well as a screening method useful for identifying compounds capable of modulating intraocular pressure as well as for identifying compounds capable of modulating retinal ganglion cell survival and/or regeneration.

Abstract: Methods of treating peripheral vascular disease comprising administering a protein solution site of a defect at least two proteins from the group IL-1ra, sTNF-RI, sTNF-RII, IGF-I, EGF, HGF, PDGF-AB, PDGF-BB, VEGF, TGF-?1, and sIL-1RII. The solution may also comprise white blood cells, platelets, concentrated bone marrow aspirate, and combinations thereof.

Abstract: Variants of cytotoxic T-lymphocyte antigen 4 (CTLA-4) with high affinity, potency and stability. Formulations of CTLA-4 variants at high concentration for subcutaneous or intravenous administration, e.g. at monthly or less frequent dosage intervals. Use of CTLA-4 variants for treating rheumatoid arthritis and other inflammatory disorders. Fusion of CTLA-4 with IgG Fc having improved stability and longer in vivo half-life.

Abstract: A bioadhesive formulation, comprising gelatin, alginate and a coupling agent, capable of forming a bioadhesive matrix, which is characterized by rapid curing, optimal viscosity, high bonding strength, flexibility, biocompatibility and biodegradability, is disclosed. Further disclosed is such a bioadhesive formulation which further comprises a bioactive agent, and a drug-eluting bioadhesive matrix formed therefrom, the bioadhesive matrix being capable of delivering the bioactive agent to a bodily site. Methods utilizing the bioadhesive formulations and matrices in various biological and medical procedures are also disclosed.

Abstract: The present invention is based on the seminal discovery that targeted immunomodulatory antibodies and fusion proteins can counter act or reverse immune tolerance of cancer cells. Cancer cells are able to escape elimination by chemotherapeutic agents or tumor-targeted antibodies via specific immunosuppressive mechanisms in the tumor microenvironment and such ability of cancer cells is recognized as immune tolerance. Such immunosuppressive mechanisms include immunosuppressive cytokines (for example, Transforming growth factor beta (TGF-?)) and regulatory T cells and/or immunosuppressive myeloid dendritic cells (DCs). By counteracting tumor-induced immune tolerance, the present invention provides effective compositions and methods for cancer treatment, optional in combination with another existing cancer treatment.