Abstract: A method of treating a neurodegenerative disorder or MD comprises administering to a subject a composition comprising an AGE antibody.

Abstract: In alternative embodiments, the invention provides a “triple combination” therapy for treating, ameliorating and preventing Crohn's Disease (or Crohn syndrome, terminal or distal ileitis or regional enteritis) or related disorders and conditions in mammals, such as paratuberculosis in mammals, or Johne's disease, including genetically-predisposed and chronic disorders, where the microbial or bacterial flora of the bowel is at least one causative or symptom-producing factor; and compositions for practicing same. In alternative embodiments, methods and compositions of the invention comprise or comprise use of therapies, medications, formulations and pharmaceuticals comprising active agents that can suppress or eradicate the microbiota super-infection that causes Crohn's Disease or paratuberculosis infection in mammals.

Abstract: Compounds useful as biologically active compounds are disclosed. The compounds have the following structure (I): (I) or a stereoisomer, tautomer or salt thereof, wherein R1, R2, R3, L, L1, L2, L3, L4, M, m and n are as defined herein. Methods associated with preparation and use of such compounds is also provided.

Abstract: Provided herein are thiazolidinedione analogues that are useful for treating non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), diabetes, and other metabolic inflammation-mediated disease and disorders.

Abstract: A conjugate and preparation method therefor, comprising a medication composition comprising the conjugate and a use of the medication composition in preparing medications for the treatment or prevention of diseases.

Abstract: The present invention provides a conjugate and preparation method thereof, a pharmaceutical composition comprising the conjugate and use of the pharmaceutical composition in the manufacture of a medicament for the treatment or prevention of a disease.

Abstract: The invention provides methods of using a human acid sphingomyelinase (e.g., olipudase alfa) in treating an abnormal bone condition in acid sphingomyelinase deficiency patients such as low bone density, high bone marrow burden, and other skeletal abnormalities presented in acid sphingomyelinase deficiency patients.

Abstract: The disclosure provides for improved pharmaceutical compositions containing deferasirox (DFX) and methods of manufacturing the same. In particular, the compositions are prepared using thermokinetic compounding and provide improved properties as well as more efficient methods of manufacture.

Abstract: Described herein are engineered ligand that binds a cell surface receptor (e.g., GPCR), with improved affinity, potency, and specificity. By conjugating a sub-optimal ligand for a cell surface receptor (e.g., GPCR) to a targeting molecule that binds an epitope (natural or exogenous epitope) in the extracellular portion of the cell surface receptor (e.g., GPCR), the affinity, potency, and/or specificity of the sub-optimal ligand is enhanced.

Abstract: The present technology provides compounds of Formula I (or pharmaceutically acceptable salts and/or solvates thereof) that are useful in treating a CNS-related disorder such as schizophrenia, schizoaffective disorder, migraine, depression, pain, drug addiction, drug use, and/or drug seeking behavior. Also provided are compositions, medicaments, and methods including such compounds.

Abstract: The invention relates to compositions containing water-soluble poly(oxazoline) and organic polymer particles chosen from the group of polyolefins, polyvinyl aromatics, polyvinyl esters, polyesters, polyamides, polyimides, polycarboxylic acids, polycarboxilic acid esters, polycarboxylic acid amides, polynitriles, polysulfonic acids, polyketones, polysulfones, polymeric polyols, polyurethanes, proteins, polymeric carbohydrates, nucleic acids or from a mixture of two or more of these polymers. The water-soluble poly(oxazolin) acts as a stabilizer for the polymer particles and can particularly advantageously be used as a stabilizer in the freeze-drying of aqueous polymer dispersions.

Abstract: The present invention includes a method of treating a drug-induced atrioventricular (AV) block comprising: providing a subject in need of therapy a drug that is contraindicated to treat a disease or condition in the subject, wherein the drug causes an AV block, with an amount of a lipid sufficient to reduce or eliminate the AV block caused by the drug.

Abstract: The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. The present invention also concerns methods to use these proteins. The present invention also relates to vectors, compositions and kits in which RVD domains and Transcription Activator-Like Effector (TALE) proteins of the present invention are used.

Abstract: A problem of this invention it to provide a method for differentiate a primordial germ cell into a functional GV stage oocyte by in vitro culture. This invention relates to a method for differentiating a primordial germ cell into a functional GV stage oocyte by in vitro culture, comprising: (a) a step of producing a secondary follicle by culturing the primordial germ cell and supporting cells adjacent to the primordial germ cells under conditions that eliminate the effects of estrogen or a factor having a similar function to estrogen; (b) a step of partially dissociating cells between a granulosa cell layer and a thecal cell layer, wherein an oocyte, the granulosa cell layer, and the thecal cell layer constitute the produced secondary follicle; and (c) a step of differentiating the oocyte into a functional GV stage oocyte by culturing the oocyte, the granulosa cell layer, and the thecal cell layer that constitute the secondary follicle in a medium containing a high-molecular-weight compound.

Abstract: The invention provides topical formulations and methods comprising charged bioactive agents complexed with amphipol polymers for dermal and transdermal delivery, optionally further including TJ-modulating peptides.

Abstract: The present invention relates to pharmaceutical compositions of the GLP-1 peptide semaglutide comprising no more than 0.01% (w/w) phenol, their preparation, kits comprising such compositions as well as uses thereof.

Abstract: [Problem to be Solved] The present invention provides a medicament for cytokine release syndrome, autoimmune-related adverse events, macrophage activation syndrome, hemophagocytic lymphohistiocytosis or Langerhans cell histiocytosis. [Means to Solve the Problem] The present invention provides a medicament for at least one selected from the group consisting of cytokine release syndrome, autoimmune-related adverse events, macrophage activation syndrome, hemophagocytic lymphohistiocytosis and Langerhans cell histiocytosis, wherein the medicament comprises a compound represented by the following formula I: or a pharmaceutically acceptable salt thereof.

Abstract: The present invention relates to the novel use of certain piperidinyl-indole derivatives in the treatment of patients suffering from renal diseases or disorders, and in particular for the treatment of patients suffering from C3G (C3 glomerulopathy) and IgAN (IgA nephropathy).

Abstract: The present invention relates to methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof. In particular, the present invention relates to a method for performing antisense oligonucleotide-mediated exon skipping in a retina cell of a subject comprising the step of injecting into the vitreous of the subject an amount of the antisense oligonucleotide.

Abstract: Disclosed herein are polypeptides, polynucleotides encoding, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.

Abstract: The present invention relates to Rucaparib and/or Talazoparib and/or Veliparib and/or Olaparib, and/or AZD 2461, or a pharmaceutically acceptable salt thereof, for use in the treatment of impaired skin wound healing in a subject, an in vitro method for identifying a subject suffering from impaired skin wound healing to be responsive to the treatment with Rucaparib and/or Talazoparib and/or Veliparib and/or Olaparib and/or AZD 2461, and kits and kits-of-part related thereto.

Abstract: The present invention relates to a process for spray drying of a feed solution comprising the GLP-1 peptide semaglutide wherein the ratio of atomising gas flow (kg/h) to feed solution flow (kg/h) is 1.0-1.7.

Abstract: The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.

Abstract: Compounds are provided for stabilizing protein transthyretin (TTR) and inhibiting amyloid fibril formation, for example, transthyretin-mediated amyloid fibril formation, and for treating, preventing, or ameliorating one or more symptoms of amyloid diseases, for example, transthyretin-related amyloidosis (ATTR).

Abstract: Methods for preparing particles and related compositions are provided. In some embodiments, the particles include at least one polynucleotide (e.g., mRNA), and in certain embodiments, the particles may include at least one ionizable molecule (e.g., a lipid). A method for preparing a suspension including the particles may comprise one or more filtration steps. In some such embodiments, prior to or during filtration, one or more properties of the particles (e.g., surface charge) and/or one or more properties of the suspension (e.g., pH) may be altered. In some embodiments, altering one or more properties of the particles and/or suspension may improve yield, improve a characteristic of the resulting composition, and/or prevent or reduce certain problems, such as fouling during the filtration process.

Abstract: The invention relates to self-assembled organosilane- and small molecule drug-containing coatings for resorbable medical implant devices. The coatings can be prepared from precursor compositions containing an organosilane and a small molecule drug, and can be applied to substrates. Prior to applying the coatings, the surfaces of the substrates can be pretreated. The coatings can be functionalized with a binding compound that is coupled with an active component. The coatings can be applied using various techniques and apparatus, more particularly, by a deep-coating process conducted at ambient conditions.

Abstract: The present disclosure pertains to compositions comprising anti-VEGF proteins and methods for producing such compositions.

Abstract: The present invention relates to PCSK9 inhibitors and methods of use thereof. Specifically, the invention relates to PCSK9 cell-based assay, PCSK9 inhibiting polypeptides and derivatives thereof. The invention includes pharmaceutical compositions comprising a PCSK9 inhibitor polypeptide together with a pharmaceutically acceptable carrier and method for treating cardiovascular disorders, hyperlipidemia, inflammatory diseases or inflammatory response to infection.

Abstract: Polypeptides comprising a C3b binding region are disclosed, as well as nucleic acids and vectors encoding such polypeptides, and cells and compositions comprising such polypeptides. Also disclosed are uses and methods using the polypeptides for treating and preventing diseases and conditions.

Abstract: The present invention is directed to carrier systems comprising ether-lipids conjugated to one or more bioactive ligands and exposed on the surface of the carrier system for use in targeted delivery and/or antigen display systems. Optionally one or more further bioactive agents may be encapsulated or embedded within or attached to or adsorbed onto the carrier system. The present invention is further directed to methods of their preparation and their uses in medical applications, such as targeted delivery of bioactive agents to specific tissues or cells and antigen display systems for the study, diagnosis, and treatment of traits, diseases and conditions that respond to said bioactive agents.

Abstract: This invention provides compositions comprising at least one protein nanoparticle comprising a protein and a stealth polymer. In certain embodiments, the nanoparticle further comprises a therapeutic agent, such as but not limited to a miRNA and/or siRNA. In other embodiments, the nanoparticle further comprises a cell surface receptor ligand. Also included in the invention are methods of preparing the compositions of the present invention, and methods of treating, ameliorating or preventing a disease or disorder in a subject using the compositions of the present invention.

Abstract: The present invention provides an antisense oligonucleotide for inhibiting biosynthesis of chondroitin sulfate. The antisense oligonucleotide comprises at least one modified nucleotide, wherein the antisense oligonucleotide suppresses expression of one or both of the chondroitin sulfate N-acetylgalactosaminyltransferase-1 (CSGalNAcT1) gene and the chondroitin sulfate N-acetylgalactosaminyltransferase-2 (CSGalNAcT2) gene.

Abstract: This invention is directed to a zinc-charged insulin composition that is effective in treating diabetes and lowering and stabilizing blood glucose levels when administered orally. The zinc-charged insulin is acid and enzyme resistant, such that the zinc-charged insulin is capable of surviving the acidic conditions of the stomach. The zinc-charged insulin is capable of being absorbed through the gastrointestinal tract and stored in the liver, such that the zinc-charged insulin is long lasting and pharmacokinetically similar to the insulin normally generated by the body. The invention is further directed to a method of preparing the zinc-charged insulin composition, including: (1) removing any loosely bound surface ions present on an insulin molecule using a chelating agent; and (2) replacing all the loosely bound surface ions with zinc.

Abstract: The present invention relates generally to compositions and methods comprising abiotic, synthetic polymers with affinity and specificity to proteins. The synthetic polymers are an improvement over biological agents by providing a simpler, less expensive, and customizable platform for binding to proteins. In one embodiment, the compositions and methods relate to synthetic polymers with affinity and specificity to vascular endothelial growth factor (VEGF). In one embodiment, the compositions are useful for treating diseases and disorders related to the overexpression of VEGF. In one embodiment, the compositions are useful for treating cancer. In one embodiment, the compositions are useful for detecting VEGF levels from biological samples. In one embodiment, the compositions are useful for detecting overexpression of VEGF from biological samples. In one embodiment, the compositions are used to diagnose cancer.

Abstract: The invention provides methods for selective targeting of live cells, which have undergone or are undergoing radiation or chemotherapy, at a site of interest with a cell-penetrating polypeptide. In one embodiment of the invention, the method comprises contacting the live cells with a cell-penetrating polypeptide comprising cell-penetrating determinants so that the cell-penetrating polypeptide binds extracellular DNA near or around the live cells so as to form a complex or association therewith such that the complex or associated polypeptide-DNA so bound bind the live cells and penetrates the live cells thereby selectively targeting live cells at a site of interest with a cell-penetrating polypeptide.

Abstract: The present invention provides regulatable biocircuit systems. Such systems provide modular and tunable protein expression systems in support of the discovery and development of therapeutic modalities.

Abstract: Novel peptide analogs of a ?-defensin have been developed that provide a biphasic effect in treating disseminated fungal disease and/or associated septic shock. These analogs are active at concentrations below those needed to provide a fungicidal effect, and function by initially mobilizing effector cells of the immune system to address the infective organism followed by regulation of the immune system to down regulate the inflammatory response. These novel ?-defensin analogs are protective at concentrations where naturally occurring ?-defensins have no apparent effect, and include a core set of structural and sequence features not found in native ?-defensins.

Abstract: History for data objects may be maintained to detect data events. An indication of an Extract, Transform, Load (ETL) process applied to one or more source data objects to generate one or more transformed data objects may be received. History for the source data objects may be updated to include the transformed data objects and the ETL process that generated the transformed data objects. An evaluation of the update may be performed to determine whether an event associated with the data lineage is triggered. If the event is triggered, a notification of the event may be sent to one or more subscribers for the event.

Abstract: The present disclosure provides methods of preventing or treating renal ischemia-reperfusion injury in a mammalian subject and methods for chronic treatment of ARVD, including administering an effective amount of an aromatic-cationic peptide to a subject in need thereof. The methods include administering aromatic-cationic peptides to prevent or treat renal injury during the treatment of renal artery stenosis. The methods include administering an effective amount of an aromatic-cationic peptide to subjects in need thereof.

Abstract: The invention provides ophthalmic compositions comprising about 0.1 % (w/v) cyclosporine dissolved in 1-(perfluorobutyl)pentane for use in the topical treatment of dry eye disease. The invention further provides kits comprising such compositions for the same use.

Abstract: Methods and compositions for treating and/or preventing aging-related conditions are described. The compositions used in the methods include blood plasma and blood plasma fractions derived from blood plasma with efficacy in treating and/or preventing aging-related conditions such as cognitive disorders. The methods relate to a regimen of pulsed dosing of blood plasma or blood plasma fractions.

Abstract: Methods, systems and computer-accessible medium for imaging a living cell or a living organism with bond-edited compounds using stimulated Raman scattering are disclosed. The method comprises the steps of introducing one or more bond-edited compounds into a live cell or a living organism, and detecting a vibrational tag in the cell or organism with stimulated Raman scattering. Also disclosed are methods for detecting a disease condition in a subject, methods for monitoring treatment for a disease condition, methods for screening an agent, methods for tracing a cellular process in a live cell using bond-edited compounds in combination with stimulated Raman scattering. Also disclosed are a composition for labeling a target cell with at least one bond-edited compound and devices for imaging bond-edited compounds by stimulated Raman scattering.

Abstract: The invention discloses a fusion protein, preparation method thereof and use thereof and belongs to the field of biopharmaceutical technology. The fusion protein according to the present invention has anti-tumor, anti-autoimmune diseases and anti-inflammatory functions, and therapeutic effects on ophthalmic diseases. According to the long-acting, multifunctional fusion protein of the present invention, the EDSM-Y or EDSM-X polypeptide is fused to the antibody immunoglobulin Fc fragment by a flexible linker so as to obtain the fusion proteins I-V, which can improve the efficacy, prolong the half-life and enhance stability, have characteristics of strong effect, low toxicity and the like, and can be used for the prevention and treatment of solid tumors and various types of inflammation and neovascular ophthalmic diseases. The fusion protein is expressed in a prokaryotic cell or a eukaryotic cell by a genetic engineering method, and the expressed fusion protein is obtained by affinity chromatography.

Abstract: Polyethylene glycol (PEG)-b-poly(?-amino ester) (PBAE) co-polymers (PEG-PBAE) and blends of PEG-PBAEs and PBAEs and their use for delivering drugs, genes, and other pharmaceutical or therapeutic agents safely and effectively to different sites in the body and to different cells, such as cancer cells, are disclosed.

Abstract: The present specification discloses plasma processing systems that include a number of different fluid flow circuits that are defined by sources of fluid, fluid lines, fluid flow paths, waste containers, a mixer, a separator, valves, and a pump. The systems also include a connector tube and a solvent extraction device, wherein the connector tube and solvent extraction device are configured to be alternatively inserted in a same position along a fluid flow line. In addition, the systems include a controller that is configured to execute a plurality of programmatic instructions to open and close each of a first fluid flow line valve, a second fluid flow line valve, a third fluid flow line valve, and a fourth fluid flow line valve in a predetermined sequence to either enable or prevent a flow of fluid through various fluid flow lines.

Abstract: A bone cement paste containing a powder component comprising ?-tricalcium phosphate (?-TCP) particles having an average size greater than or equal to 9 ?m, and a liquid component comprising blood is disclosed. A method for preparation of the phosphocalcic cement composition is also disclosed.

Abstract: The disclosure provides a chimeric antigen receptor (CAR) comprising a modified hinge, transmembrane and/or intracellular domain disclosed herein. Some aspects of the disclosure relate to a polynucleotide encoding a chimeric antigen receptor (CAR) comprising the costimulatory domain disclosed herein. Other aspects of the disclosure relate to cells comprising the CAR and use in a T cell therapy.

Abstract: The present invention relates generally to methods of use and compositions useful for treating skin. The composition includes a combination of Argania spinosa kernel extract, dill extract, Myrciaria dubia fruit extract, and Croton lechleri extract and/or Morus alba fruit extract. This combination can be used to create topical skin compositions that reduce the appearance of loose, sagging, and/or flaccid skin, and/or improve facial contouring.

Abstract: The subject matter of the invention is a pharmaceutical formulation which, according to the invention, contains curcumin or a curcumin derivative dissolved in an alcohol, an acid and an solubiliser. The formulation contains at most 12 percent by weight water and makes possible the production of an aqueous infusion solution, by means of which curcumin can be administered intravenously in dissolved form.

Abstract: The present invention relates to nanoparticles. In particular, the present invention provides nanoparticles for clinical (e.g., targeted therapeutic), diagnostic (e.g., imaging), and research applications in the field of cardiology. For example, in some embodiments, the present invention provides a method of treating (e.g., ablating) cardiac tissue, comprising: a) contacting an animal with a nanoparticle comprising a matrix, a toxic (e.g., ablative) agent (e.g., sonosensitizer, chemotherapeutic agent (e.g., doxorubicin or cisplatin), or photosensitizer), and a cardiac targeting moiety; and b) administering an activator of the toxic agent (e.g., light, chemical (e.g., pharmaceutical agent) or ultrasound) to at least a portion of the cardiac tissue (e.g., heart) of the animal to activate the toxic agent.