Abstract: This disclosure provides cell surface anchoring antigen conjugates, formulations comprising cell surface anchoring antigen conjugates, and methods of using the same for treating cancer.

Abstract: In some aspects, the present invention provides certain combination therapies comprising compstatin analogs.

Abstract: The specification describes an improved serum-free animal cell culture medium, which can used for the production of a protein of interest. Ornithine, or a combination of ornithine and putrescine can be added to serum-free media or chemically defined media to improve viable cell density, to reduce cell doubling time, and to increase the production of a protein of interest.

Abstract: Problem: A malaria transmission blocker or a malaria parasite growth inhibitor is provided as a result of finding a druq which inhibits malaria parasite growth in the body of a vector mosquito. Solution: A malaria parasite growth inhibitor or transmission blocker containing a rare sugar such as D-allose or D-psicose as an active ingredient.

Abstract: The invention relates to unmasking endotoxins in compositions so that previously present, but undetectable endotoxins are rendered detectable.

Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

Abstract: A graft copolymer having drug-containing side chains is described. The graft copolymer can be prepared, for example, by directly polymerizing a drug-containing monomer on multiple sites of a linear copolymer. When exposed to water, the graft copolymer can form uniform nanocarriers, e.g., nanomicelles, optionally encapsulating additional drugs, e.g., non-covalently in the interior of the nanocarriers, for coordinated drug delivery of a plurality of drugs. Also described herein is the use of the nanocarriers for delivery of therapeutic agents, particularly the dual delivery of chemotherapeutic agents to treat tumors.

Abstract: The invention relates to unmasking endotoxins in compositions so that previously present, but undetectable endotoxins are rendered detectable.

Abstract: This document relates to materials and methods for treating cancer (e.g., prostate cancer). For example, methods for using one or more androgen receptor variant (AR-V) inhibitors (e.g., morpholinos) to treat a mammal having prostate cancer (e.g., castration-resistant prostate cancer) are provided.

Abstract: The present invention relates to the uses of the protein PRG4 and therapeutic modulation thereof. In particular, the present invention relates compositions and methods utilizing PRG4 and therapeutic modulation thereof, including, use as a surgical lubricant, use in a treatment for prevention or reduction of post-surgical adhesions, use in a treatment for oral ulcerations, use as an athletic lubricating patch, use as a dermal filler, use in a treatment for dry mouth, use in a drug delivery method or composition, and use in nursing lubrication.

Abstract: Hydrogels comprising a macromolecular matrix and water may be used to augment soft tissue of a human being, promote or support cell or tissue viability or proliferation, create space in tissue, and for other purposes. A macromolecular matrix may comprise a hyaluronic acid component crosslinked to a silk fibroin component.

Abstract: This invention is related to the field of hypercholesterolemia. In particular, the invention provides compositions and methods to modulate circulating levels of low density lipoproteins by altering the conformation of the protein PCSK9 using synthetic ligands and/or synthetic ligand derivative sequences of 3-8 amino acids ranging between 350-2,000 Da. Altering the conformation of PCSK9 affects the interaction between PCSK9 and an endogenous low density lipoprotein receptor, and can lead to reduced or increased levels of circulating LDL-cholesterol. High LDL-cholesterol levels are associated with increased risk for heart disease. Low LDL-cholesterol levels may be problematic in other conditions, such as liver dysfunction; thus, there is also utility for ligands which can raise LDL levels.

Abstract: The invention provides compositions comprising Eno1 for delivery to a muscle. Further, the invention provides a method for normalizing blood glucose in a subject with elevated blood glucose, comprising administering to the subject enolase 1 (Eno1), thereby normalizing blood glucose in the subject. The invention also provides methods of treating one or more conditions including impaired glucose tolerance, insulin resistance, pre-diabetes, and diabetes, especially type 2 diabetes in a subject, comprising administering to the subject enolase 1 (Eno1), thereby treating the condition in the subject. In certain methods of the invention, the Eno1 is delivered to muscle.

Abstract: The invention provides formulations and methods for ameliorating symptoms associated with metabolic disorders, such as hypoglycemia, obesity, diabetes, and the like by targeted administration to the oesphagus of a subject of Zn-?2-glycoproteins or a functional fragment thereof, alone or in combination with additional agents, such as ? adrenergin receptor agonists, ? adrenergin receptor antagonists, and/or glycemic control agents.

Abstract: Methods of modulating the properties of a cell culture expressing a protein of interest are provided. In various embodiments the methods relate to the overexpression of proteins involved in the N-glycosylation pathway.

Abstract: The object of the present invention is to provide a polypeptide having interferon ? activity glycosylated with highly uniform sialylated sugar chains. The present invention is a glycosylated polypeptide, wherein the polypeptide is any polypeptide selected from the group consisting of the following (1) to (4); (1) a polypeptide consisting of the amino acid sequence represented by SEQ ID NO. 1, (2) a polypeptide having one or a few amino acids deleted, substituted, or added in the polypeptide consisting of the amino acid sequence represented by SEQ ID NO. 1, (3) a polypeptide that is an analog of interferon ?, and (4) a polypeptide having 80% or more homology to the polypeptide consisting of the amino acid sequence represented by SEQ ID NO. 1, in which amino acids at 4 to 6 locations are substituted with glycosylated amino acids, and wherein all of the non-reducing terminals of said sugar chain are sialylated.

Abstract: The present invention relates to a process for the enzymatic modification of a glycoprotein. The process comprises the step of contacting a glycoprotein comprising a glycan comprising a terminal GlcNAc-moiety, in the presence of glycosyltransferase that is, or is derived from, a ?-(1,4)-N-acetylgalactosaminyltransferase, with a non-natural sugar-derivative nucleotide. The non-natural sugar-derivative nucleotide is according to formula (3): wherein A is selected from the group consisting of —N3, —C(O)R3, —(CH2)iC?C—R4, —SH, —SC(O)R8, —SC(O)OR8, —SC(S)OR8, —F, —CI, —Br —I, —OS(O)2R5, terminal C2-C24 alkenyl groups, C3-C5 cycloalkenyl groups, C4-C8 alkadienyl groups, terminal C3-C24 allenyl groups and amino groups.

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.

Abstract: The present invention relates to novel chimeric molecules of ficolin-associated polypeptides, such as fusion polypeptides for the use in the treatment of conditions associated with inflammation, apoptosis, autoimmunity, coagulation, thrombotic or coagulopathic related diseases. The present invention further relates to nucleic acid molecules encoding such fusion polypeptides, vectors and host cells used in the production of the fusion polypeptides.

Abstract: Disclosed are isolated mutant erythropoietin (EPO) polypeptides, functional fragment thereof, nucleic acid encoding such peptides, vectors including such nucleic acids and compositions including such peptides and nucleic acids. The mutant EPO peptides are unique in that they include a substitution at amino acid position number 76, such as a glutamic acid for arginine substitution at position 76. This substitution inhibits erythropoietic activity while retaining their neuroprotection. Also disclosed are methods of treating or inhibiting neuronal degeneration, reducing or inhibiting one or more symptoms associated with neuronal degeneration and/or glaucoma in a subject. The methods include administering a therapeutically effective amount of a isolated mutant erythropoietin EPO polypeptide, an expression vector encoding such a mutant erythropoietin EPO polypeptide, a viral particle including an expression vector, or a composition, thereby treating or inhibiting neuronal degeneration in the subject.

Abstract: Nutritive proteins are provided. In some embodiments the nutritive proteins comprise a first polypeptide sequence comprising a fragment of a naturally-occurring nutritive protein. In some embodiments the fragment comprises at least one of a) an enhanced ratio of branch chain amino acid residues to total amino acid residues present in the nutritive protein; b) an enhanced ratio of leucine residues to total amino acid residues present in the nutritive protein; and c) an enhanced ratio of essential amino acid residues to total amino acid residues present in the nutritive protein.

Abstract: Methods of preparing alpha-1-antiproteinase inhibitor and controlling the amount of des-lys alpha-1-antiproteinase inhibitor in the preparation, and compositions comprising the same, as well as methods of treatment using the same are provided.

Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.

Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.

Abstract: The present technology provides for enzyme compositions with enhanced enzyme activity, thermophilic and psychrophilic stability.

Abstract: The invention describes the use of an antibody specific for serum amyloid P component, for the treatment or prophylaxis of amyloidosis, and the use of a compound which depletes serum amyloid P component from the circulation in combination with an antibody specific for serum amyloid P component.

Abstract: The invention relates to the use of fibromodulin and lumican, particularly active fragments thereof, to increase muscle mass, especially in the treatment of muscular dystrophies.

Abstract: The present invention relates to inhibitors of the activity of Complex (III) of the mitochondrial electron transport chain and use thereof in treatment and/or prevention of cancers presenting tumour-initiating cells. The present invention further relates to pharmaceutical compositions containing said inhibitors alone or in combination with other pharmaceutically active agents, and their use as medicaments or as agrochemicals where their properties as inhibitors of the mitochondrial respiration is of benefit.

Abstract: The disclosed molecules are inhibitors of Bcr-Abl and Src kinases. The molecules are cytotoxic to Gleevec resistant cells. Inhibitors of Bcr-Abl and Src kinases are used in the treatment of Chronic Myelogenous Leukemia among other diseases.

Abstract: The present invention relates to methods for diagnosing and treating focal segmental glomerulosclerosis. More particularly, the present invention relates to a method for determining whether a subject is at risk of having or developing a focal segmental glomerulosclerosis (FSGS) comprising the step consisting of determining the level of calcium/calmodulin-dependent serine protein kinase (CASK) in a blood sample obtained from the subject. The present invention also relates to an agent effective to inhibit the binding of CASK to hCD98 present on the surface of podocytes for use in the prevention or treatment of focal segmental glomerulosclerosis (FSGS) in a subject in need thereof.

Abstract: The present invention provides dendrimer conjugates. The present invention provides a composition comprising a dendrimer conjugate and a cell, such as a cell covered with dendrimer conjugates, in which dendrimer conjugates home the cell to a target tissue.

Abstract: A protein composition which comprises a mixture of glycine, phenylalanine and histidine and/or a cellulose ether derivative as an additive and has resistance to radiation sterilization.

Abstract: Aspects of the invention provide methods for selecting a candidate oligonucleotide for activating expression of a target gene. Further aspects of the invention provide methods of selecting a set of oligonucleotides that is enriched in oligonucleotides that activate expression of a target gene. Further aspects provide single stranded oligonucleotides that modulate gene expression and compositions and kits comprising the same. Methods for modulating gene expression using the single stranded oligonucleotides are also provided.

Abstract: The invention relates to methods of preparing and purifying conjugates between Granulocyte Colony Stimulating Factor and PEG moieties. The conjugates are linked via an intact glycosyl linking group that is interposed between and covalently attached to the peptide and the modifying group. The conjugates are purified using various chromatography methods.

Abstract: Compositions and methods for treating subjects with disorders characterized by hyper-proliferating cells such as tumors and cancers are provided. The compositions comprise agents that are combinations of saposin C (or prosaposin-related polypeptides) and phospholips (or inner leaflet components). This anti-tumor agent is administered in the methods of the invention according to a dosing regimen. Administering an agent of the invention results in a positive therapeutic response in a subject with a tumor.

Abstract: Methods and compositions are provided that load and encapsulate an agent, such as a protein, in a porous self-healing polymer. A delivery system includes a porous self-healing polymer, an ionic affinity trap within the pores of the self-healing polymer, and an agent associated with the ionic affinity trap. Methods of encapsulating an agent in a polymer include providing a porous self-healing polymer comprising an ionic affinity trap within the pores. The polymer is incubated with an agent having an affinity for the ionic affinity trap. At least a portion of the pores in the polymer are then healed. Active encapsulation of macromolecules at low concentrations may be achieved due to affinity of the agent for the ionic affinity trap within the pores.

Abstract: Conjugates comprising a N-oxime bond are disclosed. In one embodiment, a suitable conjugate is represented by the following Formula (I): wherein R? is derived from a compound comprising at least one reactive amide group, R? is derived from a compound comprising at least one reactive aminooxy group, and X is H, CnH(n+2) or other atoms. Additional methods are also provided.

Abstract: The disclosure provides a recipe for in-situ gel, formed by dissolving at least one polymer and at least one gel prevention agent in a polar solvent to form a solution and placing the solution in a condition for in-situ forming gels. The disclosure also provides an implant and a drug delivery system formed by the recipe.

Abstract: Nanoparticles having a core and a corona of ligands covalently linked to the core, wherein differing species of peptides are bound to the nanoparticles and incorporated into various dosage forms.

Abstract: It is an object of the present invention to provide: a polyion complex that sufficiently retains a photosensitizing substance in serum and is excellent in terms of structural stability; a nucleic acid polyplex as a constituent thereof; and a device and a kit for delivering a nucleic acid into a cell. The nucleic acid polyplex of the present invention comprises a cationic polymer represented by general formula (1) and a nucleic acid. The polyion complex of the present invention comprises the nucleic acid polyplex of the present invention and an anionic photosensitizing substance.

Abstract: An individual at risk for necrotizing enterocolitis and related disorders can be identified by measuring the level of at least one secretor antigen in a biological sample from the individual and comparing the measured level of the at least one secretor antigen to a predetermined value or a predetermined range of values. Among the secretor antigens which can be measured are: the H-1, H-2, Lewisb and Lewisy antigens and derivatives thereof (e.g., a sialylated form of Lewis a, Lewis x, Lewis b, Lewis y; H-1, H-2, Lewis a, Lewis x, Lewis b or Lewis y).

Abstract: Described are embolization devices which carry M1 macrophage promoting agents and/or M2 macrophage inhibiting agents, as well as methods for their manufacture and use. An illustrative embolization device of the disclosure comprises an embolic body and one or more M1 macrophage promoting agents and/or M2 macrophage inhibiting agents carried by a surface of the embolic body. In certain embodiments the embolic body of the present disclosure comprises an embolic coil or an embolic bead.

Abstract: A compound of the general formula (III): wherein X is O, S, NH or CH2; Y is O, S or NH; Z is O, S or CH2; R1 is C1-8 alkyl, especially C1-6 alkyl, preferably n-alkyl, e.g., n-pentyl or n-hexyl; at least one of R2 and R3 is H—[R4-R5]n—R6—, in which: H—[R4-R5]n— comprises an oligopeptide, R4 being an amino acid and R5 being an amino acid selected from proline, alanine, hydroxyproline, dihydroxyproline, thiazolidinecarboxylic acid (thioproline), dehydroproline, pipecolic acid (L-homoproline), azetidinecarboxylic acid, aziridinecarboxylic acid, glycine, serine, valine, leucine, isoleucine and threonine, R6 is a neutral, non-polar amino acid moiety that is bonded to R5 by a peptide bond, and n is 1, 2, 3, 4 or 5; and the other of R3 and R2 is H—[R4-R5]n—R6— or H; or a pharmaceutically acceptable salt thereof.

Abstract: The present invention relates to the management of vaginal health. In particular, the present invention relates to pharmaceutical compositions, and methods of use thereof, for treating diseases associated with compromised boundary lubrication at the vaginal epithelium.

Abstract: The present disclosure relates to milk-based nutritional compositions comprising lactoferrin and/or a prebiotic component, wherein, when combined, the lactoferrin and prebiotic component may exhibit additive or synergistic beneficial effects on the health and development of a pediatric subject. The disclosure further relates to methods comprising the administration of said milk-based nutritional compositions to pediatric subjects.

Abstract: Conjugates comprising a N-oxime bond are disclosed. In one embodiment, a suitable conjugate is represented by the following Formula (I): wherein R? is derived from a compound comprising at least one reactive amide group, R? is derived from a compound comprising at least one reactive aminooxy group, and X is H, CnH(n+2) or other atoms. Additional methods are also provided.

Abstract: Described herein are methods of syntheses and therapeutic uses of covalently modified peptides and/or proteins. The covalently modified peptides and/or proteins allow for improved pharmaceutical properties of peptide and protein-based therapeutics.

Abstract: This invention relates to collagen-binding synthetic peptidoglycans and engineered collagen matrices comprising a collagen matrix and a collagen-binding synthetic peptidoglycan where the collagen-binding synthetic peptidoglycan can be aberrant or can have amino acid homology with a portion of the amino acid sequence of a protein or a proteoglycan that regulates collagen fibrillogenesis. The invention also relates to kits, compounds, compositions, and engineered graft constructs comprising such collagen-binding synthetic peptidoglycans or engineered collagen matrices and methods for their preparation and use.

Abstract: The present invention relates to a myostatin inhibitor comprising extracellular water-soluble domains of delta-like 1 homolog (DLK1) as active ingredients. More particularly, the present invention relates to a composition for inhibiting myostatin activity, comprising, as active ingredients, extracellular water-soluble domains of DLK1 or a deletion mutant of extracellular water-soluble domains of DLK1. The myostatin inhibitor of the present invention is bonded to the myostatin or activin receptor type IIB so as to inhibit the action mechanism of the myostatin, to thereby promote myogenesis and prevent differentiation into fat cells. Therefore, the myostatin inhibitor of the present invention may be used in preventing and treating diseases such as muscular dysplasia that requires differentiation to muscular cells, or metabolic diseases.

Abstract: New liposomes are described, comprising: (i) phosphatidic acid and/or cardiolipin; (ii) apolipoprotein E (ApoE) or derivatives thereof. The so modified liposomes, administered systemically, obtain a dramatic in-vivo reduction of the amyloid plaque in the central nervous system, allowing an effective treatment of neurodegenerative diseases, in particular Alzheimer's disease.