Abstract: A device houses a lyophilized drug that is to be reconstituted and administered to a patient that has a disease. The device has a transparent cylinder that has a volume of at least 250 mL and that is configured to house the lyophilized drug. The cylinder has a first end that has a first spike, and the first end is opposite a second end that has a port. The first spike is configured to pierce an IV fluid bag and to form a fluid connection between the cylinder and the IV fluid bag. The port is configured to be pierced by a second spike that is part of a tubing, thereby forming a fluid connection between the cylinder and the tubing. At least a portion of the volume of the cylinder is occupied by at least 5 grams of the lyophilized drug.

Abstract: Disclosed are pharmaceutical compositions comprising a drug with a laccase-reactive functional group and a laccase (EC.10.3.2) and methods for manufacturing such compositions.

Abstract: The present disclosure provides methods and compositions useful for the prophylactic and therapeutic amelioration and treatment of infections caused by Gram negative bacteria, including Pseudomonas aeruginosa. The disclosure further provides compositions and methods of incorporating and utilizing lysin polypeptides of the present disclosure for augmenting the efficacy of antibiotics generally suitable for the treatment of Gram-negative bacterial infection.

Abstract: Provided are spray-dried thrombin powders comprising microcapsules, methods of preparation and uses thereof.

Abstract: The invention relates to a new, more potent, coagulation factor IX (FIX) expression cassette for gene therapy of haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.

Abstract: Devices and methods for treating defects in connective tissue are provided along with methods for making such devices. The devices can include enzyme-activated acellular tissue matrices that facilitate regrowth of the damaged tissue.

Abstract: A method for treating an abnormality of the first metatarsophalangeal joint of the foot of a mammal includes electrically stimulating a site on each of the extensor hallucis brevis and adductor hallucis muscles of a foot of the mammal affected by the abnormality, and visually confirming that the stimulated muscles responds to the stimulation by contracting. An amount of botulinum toxin effective to treat the abnormality is then injected to the confirmed sites on the extensor hallucis brevis and the adductor hallucis muscles of the affected foot.

Abstract: Variant Erwinia chrysanthemi L-asparaginases with reduced L-glutaminase activity and enhanced in vivo circulation are described as are fusion proteins containing an L-asparaginase and three tandem soluble domains of TRAIL for use in the treatment of cancers such as acute lymphoblastic leukemia and acute myeloid leukemia.

Abstract: Provided herein are, inter alia, compositions including a fusion protein containing an antigenic cancer peptide and a mature MHC class II peptide, a nucleic acid encoding the protein, a pharmaceutical formulation thereof, an antigen-presenting cell expressing the protein. Also provided herein are methods for treating cancer utilizing these compositions.

Abstract: Modified T-cells have paratopes against human TK1 epitopes, are made by producing monoclonal antibodies that are specific to TK1, creating chimeric antigen receptors (CARs) by fusion of the single-chain variable fragments (scFv) of the monoclonal antibodies to T-cell signalling domains, and transducing the CARs to the T-cells.

Abstract: Modified T-cells have paratopes against human TK1 epitopes, are made by producing monoclonal antibodies that are specific to TK1, creating chimeric antigen receptors (CARs) by fusion of the single-chain variable fragments (scFv) of the monoclonal antibodies to T-cell signalling domains, and transducing the CARs to the T-cells.

Abstract: Described herein is a reliable method for preparing a potent vaccine useful for immunotherapy comprising the step of cryopreserving a population of cells undergoing immunogenic cell death, and using such cells to activate dendritic cells for use in immunotherapy. In a specific embodiment, the method comprises cryopreserving cancer cells undergoing cell death, which can be used to prepare a pharmaceutical composition for immunotherapy against cancer.

Abstract: The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.

Abstract: The present invention pertains to a combination for simultaneous, separate or sequential use which comprises (a) an oncolytic virus and (b) a checkpoint inhibitor and to its use for the treatment of cancer.

Abstract: Provided herein are isolated proteins isolatable from a Klebsiella spp. Also provided are compositions that include one or more of the proteins, and methods for making and methods for using the proteins.

Abstract: The present invention relates to new immunogenic compositions comprising conjugated Streptococcus pneumoniae capsular saccharide antigens (glycoconjugates), kits comprising said immunogenic compositions and uses thereof. Immunogenic compositions of the present invention will typically comprise at least one glycoconjugate from a S. pneumoniae serotype not found in PREVNAR®, SYNFLORIX® and/or PREVNAR 13®. The invention also relates to vaccination of human subjects, in particular infants and elderly, against pneumoccocal infections using said novel immunogenic compositions.

Abstract: The invention relates to isolated Streptococcus pneumoniae serotype 15B capsular polysaccharide and processes for their preparation. The invention also relates to immunogenic conjugates comprising Streptococcus pneumoniae serotype 15B capsular polysaccharide covalently linked to a carrier protein, processes for their preparation and immunogenic compositions comprising them.

Abstract: Disclosed herein are methods of making attenuated Arenaviruses, compositions comprising the attenuated Arenaviruses, and methods of using the attenuated Arenaviruses. As disclosed herein, the attenuated Arenaviruses have a deletion of at least a portion of the intergenic region (IGR) of one or both genome segments.

Abstract: The present invention provides a composition comprising hepatitis B virus (HBV) component(s), and which may be either nucleic acid- or polypeptide-based as well as nucleic acid molecules and vectors encoding such HBV component(s). It also relates to infectious viral particles and host cells comprising such nucleic acid molecules or vectors. It also provides composition and kits of parts comprising such nucleic acid molecules, vectors, infectious viral particles or host cells and the therapeutic use thereof for preventing or treating HBV infections.

Abstract: The present invention relates to improved methods for vaccination of a subject. Particularly, the present invention discloses the use of skin antigen application to amplify and improve a pre-existing immunity against a selected pathogen in a subject. The present invention discloses the use of skin application in combination with conventional vaccination or priming for improved immunization or vaccination of a subject against a selected pathogen.

Abstract: The present disclosure is directed to compositions comprising one or more components of a polypeptide, a reovirus-derived targeting protein, and a cleavable linker, and related methods and compositions for the generation of tolerance against the polypeptide. In some embodiments, the polypeptide is antigenic, such as comprising at least one epitope from a food allergen, an environmental allergen, an auto-antigen, and/or a biological therapeutic, and/or at least one epitope derived therefrom.

Abstract: The present invention relates generally to the field of allergies. More particularly, the present invention provides a method for treating an allergy in a subject by inducing tolerance to an allergen associated with the allergy. Medicinal kits useful in protocols to induce tolerance or reduce intolerance in a subject also form part of the present invention.

Abstract: A method and combination for treating a cancer patient by combining two distinct immuno-therapy solutions for administration to a patient within a common time period, comprising a checkpoint inhibitor antibody component such as a PD-1 or PD-L1 antibody administered by infusion, and a TAA/ecdCD40L vaccine component administered subcutaneously, wherein an initial antibody component administered is followed by at least several successive antibody boosts and an initial vaccine component administered is followed by at least several successive vaccine boosts, both the initial and boosts of each administered within at least said common time period, wherein the combined administration of said two distinct immuno-therapy solutions provides for an enhanced therapeutic effect, over that of the therapeutic effect of either of the two distinct immuno-therapy component solutions when administered alone as monotherapy.

Abstract: The present invention relates to uses of bispecific antibodies exhibiting cross-species specificity for evaluating the in vivo safety and/or activity and/or pharmacokinetic profile of the same in non-human species and humans. The present invention moreover relates to methods for evaluating the in vivo safety and/or activity and/or pharmacokinetic profile of said bispecific antibodies exhibiting cross-species specificity. The present invention also relates to methods of measuring the biological activity and/or efficacy of such bispecific antibodies exhibiting cross-species specificity. In addition, the present invention relates to pharmaceutical compositions comprising bispecific single chain antibodies exhibiting cross-species specificity and to methods for the preparation of pharmaceutical compositions comprising said bispecific single chain antibodies exhibiting cross-species specificity for the treatment of diseases.

Abstract: The invention provides a method for more effective treatment of patients susceptible to or diagnosed with tumors overexpressing EGFR, as determined by a gene amplification assay, with an EGFR antagonist. Such method comprises administering a cancer-treating dose of the EGFR antagonist, preferably in addition to chemotherapeutic agents, to a subject in whose tumor cells erbB1 gene has been found to be amplified e.g., by fluorescent in situ hybridization. EGFR antagonists described include an anti-EGFR antibody.

Abstract: The invention provides aqueous pharmaceutical adalimumab compositions suitable for long-term storage of adalimumab, methods of manufacture of these compositions, methods of administration, and kits containing same.

Abstract: The invention provides aqueous pharmaceutical adalimumab compositions suitable for long-term storage of adalimumab, methods of manufacture of these compositions, methods of administration, and kits containing same.

Abstract: The invention provides aqueous pharmaceutical adalimumab compositions suitable for long-term storage of adalimumab, methods of manufacture of these compositions, methods of administration, and kits containing same.

Abstract: This invention relates to photoswitchable inhibitors of histone deacetylases and methods of using the same.

Abstract: The present disclosure provides methods for treating diseased cells in a subject using sonodynamic therapy (SDT), comprising: administering to the subject a sonosensitizer composition comprising IRDye® 700DX, wherein the sonosensitizer composition associates with the diseased cell; and thereafter applying an ultrasonic wave to the diseased cell.

Abstract: Methods and compositions are provided here to manage tractional membranes, as well as other diseases and disorders of the eye. Compositions comprising microbubbles associated with enzymes or vitreolytic agents, are provided. The method of the present invention involves administration of the microbubble and an enzyme or vitreolytic agent to a subject in need thereof, followed by application of ultrasound.

Abstract: The present invention provides compositions for extended release of an active ingredient, comprising a lipid-saturated matrix formed from a biodegradable polymer. The present invention also provides methods of producing the matrix compositions and methods for using the matrix compositions to provide controlled release of an active ingredient in the body of a subject in need thereof.

Abstract: The present document describes a pharmaceutical excipient composition comprising a functionalized anionic polysaccharide having carboxyl groups complexed with an amino acid-divalent cation complex, monolithic solid dosage forms for dual rate release of an active pharmaceutical ingredient, comprising the pharmaceutical excipient composition and active pharmaceutical ingredients, as well as processes for preparing the pharmaceutical excipient composition.

Abstract: Zwitterionic polymer and mixed charge copolymer bioconjugates, methods for making and using the bioconjugates.

Abstract: The present disclosure provides POZ derivatives having a range of active functional groups allowing conjugation of POZ derivatives to a variety of target molecules under a wide range of reaction conditions to produce a hydrolytically stable target molecule-POZ conjugate. Furthermore, the present disclosure provides novel methods of synthesis for the disclosed POZ derivatives and hydrolytically stable target molecule-POZ conjugates created using the disclosed terminally activated monofunctional POZ derivatives. In one embodiment, the POZ derivative is a terminally activated monofunctional POZ derivative.

Abstract: Non-linear multiblock copolymer-drug conjugates for the treatment and prevention of diseases and disorders of the eye are provided. The polymer-drug conjugates can form nanoparticles, microparticles, and implants that are capable of effectively delivering therapeutic levels of one or more active agents for an extended period of time. Administration to the eye of an active agent in the form of a non-linear multiblock copolymer-drug conjugate produces decreased side effects when compared to administration of the active agent alone. Also provided are methods of treating intraocular neovascular diseases, such as wet age-related macular degeneration as well as diseases and disorders of the eye associated with inflammation, such as uveitis.

Abstract: SAE-CD compositions are provided, along with methods of making and using the same. The SAE-CD compositions comprise a sulfoalkyl ether cyclodextrin having an absorption of less than 0.5 A.U. due to a drug-degrading agent, as determined by UV/vis spectrophotometry at a wavelength of 245 nm to 270 nm for an aqueous solution containing 300 mg of the SAE-CD composition per mL of solution in a cell having a 1 cm path length.

Abstract: The disclosure provides for vectors, and methods of using the vectors to efficiently deliver mRNA and/or ssRNA into cells.

Abstract: Conjugates and compounds for making conjugates which are PBD molecules linked via the N10 position are disclosed, along with the use of the conjugates for treating proliferative diseases, including cancer.

Abstract: Provided herein are methods and compositions for site-specific conjugation of antibodies in the presence of a transglutaminase.

Abstract: A recombinant fusion protein comprising a human erythropoietin peptide portion linked to an immunoglobulin peptide portion is described. The fusion protein has a prolonged half-life in vivo in comparison to naturally occurring or recombinant native human erythropoietin. In one embodiment of the invention, the protein has a half-life in vivo at least three fold higher than native human erythropoietin. The fusion protein also exhibits enhanced erythropoietic bioactivity in comparison to native human erythropoietin. In one embodiment, the fusion protein comprises the complete peptide sequence of a human erythropoietin (EPO) molecule and the peptide sequence of an Fc fragment of human immunoglobulin IgG1. The Fc fragment in the fusion protein includes the hinge region, CH2 and CH3 domains of human immunoglobulin IgG1. The EPO molecule may be linked directly to the Fc fragment to avoid extraneous peptide linkers and lessen the risk of an immunogenic response when administered in vivo.

Abstract: The present invention relates to therapeutic immunoconjugates comprising SN-38 attached to an antibody or antigen-binding antibody fragment. The antibody may bind to Trop-2 or CEACAM5 and the immunoconjugate may be administered at a dosage of between 4 mg/kg and 16 mg/kg, preferably 4, 6, 8, 9, 10, 12, or 16 mg/kg. When administered at specified dosages and schedules, the immunoconjugate can reduce solid tumors in size, reduce or eliminate metastases and is effective to treat cancers resistant to standard therapies, such as radiation therapy, chemotherapy or immunotherapy. Surprisingly, the immunoconjugate is effective to treat cancers that are refractory to or relapsed from irinotecan.

Abstract: Aspects of the invention described herein include a hydrogel prodrug and methods of making a hydrogel prodrug for drug deliver}?. Also contemplated are methods of treating, inhibiting, ameliorating or inhibiting a disease or disorder. Without being limiting, the methods for treatment can be directed to a cancer, HIV, a virus, pain, a bacterial infection, a neurological disorder, hemorrhaging, multiple sclerosis, diabetes, high blood pressure, Alzheimer's, or inhibiting a fungal growth in a subject in need.

Abstract: Described herein are nanoparticle drug conjugates (NDCs), which, in certain embodiments, comprise a non-toxic, multi-modality, clinically proven silica-based nanoparticle platform with covalently attached drug molecules/moieties. The nanoparticle drug conjugates (NDCs) demonstrate imaging capability and targeting ligands which efficiently clear through the kidneys. Furthermore, the conjugates incorporate therapeutic agents for cancer detection, prevention, and/or treatment.

Abstract: A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a hIDS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.

Abstract: A polynucleotide comprising a nucleotide sequence encoding a thymidine kinase wherein at least one of the nucleotides corresponding to the splice donor site nucleotides is replaced by another nucleotide and wherein the nucleotides of the splice acceptor sites are not altered.

Abstract: Provided herein are nucleic acid delivery compositions, surfactants, kits comprising said materials, methods and uses thereof, and methods for the preparation thereof. In particular, nucleic acid delivery compositions described herein may include tri-modal nucleic acid delivery compositions which may comprise at least one peptide enhancer, at least one surfactant, and at least one helper lipid. By way of example, certain of the tri-modal nucleic acid delivery compositions described herein include a peptide enhancer; a surfactant which is a functionalized cationic gemini surfactant; and a helper lipid which is a neutral lipid such as DOPE.

Abstract: Methods, systems, and devices are disclosed for intracellular payload delivery by nanomotor structures. In some aspects, a nanomotor for intracellular payload delivery includes an asymmetric body having a concave cavity at one end of the nanowire body; a functionalization layer on an outer surface of the nanowire body; and a payload substance coupled to the nanomotor by the functionalization layer in a biologically active conformation, wherein the payload substance is attached to a portion of the functionalization layer or at least partially encapsulated within the functionalization layer, in which the nanomotor is operable to propel in a biological medium and into an intracellular region of a living cell to initiate an interaction of the biologically active payload substance with an intracellular constituent of the living cell.

Abstract: Embodiments described herein relate to suppressing the immune response locally within tissue transplants and certain conditions improperly affecting the immune system using optogenetically controlled cells. More specifically, embodiments described herein provide for localized immunosuppression surrounding tissue transplants and illness locations as an alternative to systemically suppressing a patient's entire immune system. Methods include implantation of optogenetically modified immunosuppressive cells that are configured to alter their biological activity to enhance their immunosuppressive activity in response to exposure of wavelengths of light in the red and near-infrared window spectral region (620-900 nm).

Abstract: The present invention provides novel drug discovery platforms and methods for treating diabetes.