Abstract: Disclosed herein are methods and compositions related to coagulation factor complexes comprising a coagulation factor; a fusion protein; and a modifying molecule, wherein the modifying molecule is coupled to the coagulation factor in such a way as to allow binding by the fusion protein, thereby creating a modified coagulation factor; wherein the modified coagulation factor and the fusion protein interact in at least two independent sites.

Abstract: Disclosed are applications of a mussel adhesive protein or preparations thereof in suppression of skin inflammations. Specifically disclosed are applications of a mussel adhesive protein, preparations thereof and applications thereof in dermatitis, eczema, skin ulcer, technologies related to burns (comprising skin grafting), perniones, surgical incisions, herpes, abrasions, scars, psoriasis, erythema multiforme, skin damage after radiotherapy, skin cancers, folliculitis, urticaria and drug eruption, and applications in sunburn, polymorphous light eruption, pathological alopecia (comprising hair transplant), acne vulgaris, rosacea (that is, acne rosacea), and the like, and applications in the treatment of otitis externa.

Abstract: A pharmaceutical composition for use in inactivating an HIV-1 proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different multiplex guide RNAs (gRNAs), wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) of the HIV-1 proviral DNA, whereby treating the host cell with the composition cleaves a double strand of the HIV-1 proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease and cleaves a double strand of the HIV-1 proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease and thereby excises an entire HIV-1 proviral genome and eradicates the HIV-1 proviral DNA from the host cell, and a pharmaceutically acceptable carrier.

Abstract: A personalized method of inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus, by determining a nucleic acid sequence of the HIV-1 proviral DNA harbored by a subject, designing two or more different multiplex guide RNAs (gRNAs) complementary to the HIV-1 proviral DNA sequences in the subject, administering to the subject a therapeutically effective amount of a composition comprising a CRISPR-associated endonuclease, and the two or more different multiplex gRNAs, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, and eradicating the HIV-1 proviral DNA from the host cell.

Abstract: A method of preventing transmission of a retrovirus from a mother to her offspring, by administering to the mother a therapeutically effective amount of a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and the two or more different multiplex gRNAs, wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) of proviral DNA of the virus that is unique from the genome of the host cell, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, eradicating the HIV-1 proviral DNA from the host cell, and preventing transmission of the proviral DNA to the offspring.

Abstract: A method for treating Pompe disease including administration of recombinant human acid ?-glucosidase having optimal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue uptake of recombinant human acid ?-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid ?-glucosidase is provided.

Abstract: The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.

Abstract: The present invention provides methods of administering Factor IX; methods of administering chimeric and hybrid polypeptides comprising Factor IX; polynucleotides encoding such chimeric and hybrid polypeptides; cells comprising such polynucleotides; and methods of producing such chimeric and hybrid polypeptides using such cells.

Abstract: Methods for generating and identifying antibodies specifically binding target molecules expressed by cells embedded in a three-dimensional extracellular matrix resembling the in vivo environment and form of the target are provided. Also provided are methods of producing immunogens that yield targets in such forms. Further provided are methods for identifying anti-cancer therapeutics, such as antibody products. Hydrogels are also provided, and those hydrogels may comprise a cross-linked protein are also provided. Diagnostics, prophylactics and therapeutics identified using the methods disclosed herein are also provided.

Abstract: The success of anti-tumor immune responses requires effector T cells to infiltrate solid tumors, a process guided by chemokines. Herein, we demonstrate that in vivo post-translational processing of chemokines by dipeptidylpeptidase 4 (DPP4, also known as CD26) limits lymphocyte migration to sites of inflammation and tumors. Inhibition of DPP4 enzymatic activity enhanced tumor rejection by preserving biologically active CXCL10, and increasing trafficking into the tumor by lymphocytes expressing the counter-receptor CXCR3. Furthermore, DPP4 inhibition improved adjuvant-based immunotherapy, adoptive T cell transfer and checkpoint blockade. These findings provide the first direct in vivo evidence for controlling lymphocyte trafficking through CXCL10 cleavage and support the use of DPP4 inhibitors for stabilizing the biologically active form of chemokines as a strategy to enhance tumor immunotherapy.

Abstract: This invention provides novel carbonic anhydrase (CAIX) nucleic acid and peptide sequences, as well as related methods and compositions, including anti-cancer immunogenic agent(s) (e.g. vaccines and chimeric molecules) that elicit an immune response specifically directed against cancer cells expressing a CAIX antigenic marker. The novel CAIX variant and related compositions are useful in a wide variety of treatment modalities including, but not limited to protein vaccination, DNA vaccination, and adoptive immunotherapy.

Abstract: Antigenic molecules and compositions described herein protect against infection by typhoidal and non-typhoidal Salmonella serovars. Methods of immunization comprise the use of the antigenic molecules.

Abstract: An immunogenic composition containing leucocidin M/F antigen and capable of imparting a toxin-neutralizing activity to a ruminant animal as a subject animal. The immunogenic composition enables prevention of onset or reduction in symptoms of diseases caused by Staphylococcus aureus in ruminant animals. The leucocidin M/F antigen is a protein or peptide having at least a part of the amino acid sequence constituting the leucocidin M protein or a protein or peptide having at least a part of the amino acid sequence constituting the leucocidin F protein. The immunogenic composition may further contain a somatic antigen of Staphylococcus aureus.

Abstract: The present invention generally relates to compositions and methods for delivering a vaccine. The compositions and methods disclosed herein are particularly useful in making bacterial and viral vaccines.

Abstract: Disclosed herein are isolated flavivirus polypeptides comprising an amino acid sequence at least 75% identical to one of SEQ ID NOs: 1-11, or a nucleic acid encoding the polypeptide, or a viral like particle encoding the polypeptide, wherein the polypeptide does not comprise the amino acid sequence of a full-length flavivirus NS1 polypeptide. Methods of using these polypeptides are also disclosed, such as for preventing, treating, or determining the prognosis of a flavivirus infection.

Abstract: Embodiments of the present invention report compositions and methods for vaccinating a subject using trivalent dengue virus vaccine compositions. In some embodiments, more than one vaccine composition may be administered to a subject in different anatomical locations in order to induce a rapid response to at least three of four dengue virus serotypes. In certain embodiments, administration of a trivalent dengue virus vaccine composition can be combined with administration of a monovalent dengue virus vaccine composition.

Abstract: The invention relates to an active ingredient which is a live attenuated recombinant measles virus expressing influenza A virus antigen(s) and to its use in the elicitation of immunity, in particular protective immunity and advantageously broad-spectrum protective immunity against influenza A virus. In particular, the influenza A virus is selected among epidemic seasonal viruses and/or endemic viruses circulating in the human population and advantageously encompasses a pandemic virus such as H1N1v.

Abstract: The invention is directed to an oral vaccine composed of a micronized freeze-dried rotavirus particle emulsion with buffering excipients in a non-aqueous liquid. This IVT-06 formulation has imparted heat stability by protecting the virus at temperatures of 30° C. and 40° C. for at least twelve months. Extrapolations from the 12-month stability data indicate a shelf life of more than two years at 30° C., and six months at 50° C. In addition, for ease of administration, the formulated dose has a volume of 0.5 mL.

Abstract: The subject invention concerns methods and materials for inducing an immune response in an animal or person against an immunodeficiency virus, such as HIV, SIV, or FIV. In one embodiment, a method of the invention comprises administering one or more antigens and/or immunogens to the person or animal wherein the antigen and/or immunogen comprises one or more evolutionarily conserved epitopes of immunodeficiency viruses. In one embodiment, the epitope is one that is conserved between HIV and SIV, or between HIV and FIV. In another embodiment, the epitope is one that is conserved between HIV, SIV, and FIV.

Abstract: This invention describes novel adjuvant compositions and formulations with excellent stability at refrigerated and room temperatures and up to and about 37° C. that can be produced at remarkably low costs. This invention describes novel vaccine compositions and formulations to treat and prevent urinary tract infections caused by gram-negative bacteria including Escherichia coli and multi-drug resistant E. coli. This invention also describes methods of administration of said novel vaccine compositions and formulations and methods of treatment to prevent and treat urinary tract infections caused by gram-negative bacteria including E. coli and multi-drug resistant E. coli.

Abstract: The present invention provides compositions and methods for treating KMA-expressing malignancies including chimeric antigen receptors (CARs) and T cells containing CARs (CAR T-cells). The invention also provides methods and compositions comprising CAR T-cells co-expressing other anti-tumoral agents including cytokines and antibodies.

Abstract: The present disclosure describes a pharmaceutical combination of an anti-CD19 antibody and a phosphoinositide 3-kinase inhibitor for the treatment of non-Hodgkin's lymphoma, chronic lymphocytic leukemia and/or acute lymphoblastic leukemia.

Abstract: The inventions describes a method for increasing effector T cell accumulation in cancer cell-containing sites of a tumor, comprising administering to a subject in need thereof a pharmaceutically effective amount of an inhibitor of CXCR4 signaling

Abstract: The present invention relates to a highly concentrated, stable pharmaceutical formulation of a pharmaceutically active anti-HER2 antibody, such as e.g. Trastuzumab (HERCEPTIN™), Pertuzumab or T-DM1, or a mixture of such antibody molecules for subcutaneous injection. In particular, the present invention relates to formulations comprising, in addition to a suitable amount of the anti-HER2 antibody, an effective amount of at least one hyaluronidase enzyme as a combined formulation or for use in form of a co-formulation. The formulations comprise additionally at least one buffering agent, such as e.g. a histidine buffer, a stabilizer or a mixture of two or more stabilizers (e.g. a saccharide, such as e.g. ?,?-trehalose dihydrate or sucrose, and optionally methionine as a second stabilizer), a nonionic surfactant and an effective amount of at least one hyaluronidase enzyme. Methods for preparing such formulations and their uses thereof are also provided.

Abstract: A patch for treating vascular ulcers caused by excessive enzymatic activity may include a substrate configured to span a vascular ulcer as well as a linking material that is disposed relative to the substrate and has an affinity for an enzyme involved in causing the vascular ulcer. A magnetic material may be coupled to the linking material. In some cases, the enzymes involved in causing the vascular ulcer may become coupled to the linking material and thus become coupled to the magnetic material so that that the enzymes can be removed by applying a magnetic field in the proximity of the vascular ulcer. The enzymes may include matrix metalloproteinases.

Abstract: Compositions for vapor phase delivery of one or more active compounds. The compositions comprise one or more polyol and one or more active compound. Optionally, the compounds further comprise one or more disruptive compound. The compositions can be used in methods of vapor phase delivery of one or more active compound to an individual. Also provided are kits comprising one or more of the compositions and instructions for use of the composition(s).

Abstract: This invention provides for biocompatible and biodegradable syringeable liquid, implantable solid, and injectable gel pharmaceutical formulations useful for the treatment of systemic and local disease states.

Abstract: The present disclosure provides inter alia compositions that comprise therapeutic agents (e.g., live attenuated viral antigens, therapeutic proteins, etc.) and a lipid component. The lipid component may comprise or consist of different types of lipid or lipids as described herein. In some embodiments the therapeutic agents are thermolabile. The present disclosure also provides methods for preparing compositions, including the aforementioned compositions (e.g., melt methods and spray injection methods among others).

Abstract: The present invention pertains to a composition containing an immunogenic cell preparation of killed Leptospira bacteria in an ethylenediaminetetraacetic acid solution. The invention also pertains to a vaccine to protect an animal against an infection with leptospira bacteria, wherein the vaccine comprises this composition. Also, the invention pertains to the use of ethylenediaminetetraacetic acid to stabilise an immunogenic preparation of killed Leptospira bacteria in a liquid carrier, by dissolving the ethylenediaminetetraacetic acid in the carrier.

Abstract: The present invention provides a stable and clear aqueous liquid preparation containing (3-{2-[4-isopropyl-2-(4-trifluoromethyl)phenyl-5-thiazolyl]ethyl}-5-methyl-1,2-benzisoxazol-6-yl) oxyacetic acid or a pharmaceutically acceptable salt thereof as an active ingredient, and bensalkonium chloride represented by the formula: [C6H5CH2N(CH3)2R]Cl wherein R is an alkyl group having 8-18 carbon atoms.

Abstract: The present invention is intended to provide a nose drop having high absorption of peptide hormone oxytocin or an acid addition salt thereof, or a derivative thereof through nasal mucosa and little safety concern. The present invention is a pharmaceutical composition for administration to nasal mucosa containing oxytocin or an acid addition salt thereof, or a derivative thereof and a carboxyvinyl polymer, characterized by having an osmotic pressure ratio less than 1.

Abstract: The present invention provides compositions and methods for delivery of therapeutic agents across an barrier. The compositions include a therapeutic agent (e.g., antimicrobial agent, antibiotic, or anesthetic agent), a permeation enhancer which increases the flux of the therapeutic agent across the barrier, and a matrix forming agent. The matrix forming agent forms a gel at a suitable gelation temperature and rheological properties for use in drug delivery, and in some cases, the gelation temperature and rheological properties are not significantly changed from those of the composition without the permeation enhancer. The invention also provides a matrix forming agent and compositions thereof. Such compositions are particularly useful in the treatment of otitis media. Methods of treatment, methods of delivery, and kits for the compositions described herein are also provided.

Abstract: The present invention relates to new formulations, particularly useful in the topical treatment of skin and orormucosal wounds. Formulations can be either a solution or a gel form consisting of a Polyhexamethylene biguanide as an antimicrobial agent, purified water and a tri-blockcopolymer, particularly a triblockcopolymer of polyethylene oxide and polypropylene oxide, and more particularly a poloxamer.

Abstract: A method of inhibiting and/or reducing the activity, signaling, and/or function of leukocyte-common antigen related (LAR) family of phosphatases in a cell of a subject induced by proteoglycans includes administering to the cell a therapeutic agent that inhibits one or more of catalytic activity, signaling, and function of the LAR family phosphatases without inhibiting binding to or activation the LAR family phosphatases by the proteoglycans.

Abstract: A molecule comprising a saccharide bound via an O-glycosidic bond to a hydroxyl group of a toxic payload molecule is disclosed. An antibody-drug conjugate comprising an antibody covalently bound to a toxic payload molecule, optionally via a linker group, and a saccharide bound via an O-glycosidic bond to a hydroxyl group of the toxic payload molecule is further disclosed.

Abstract: The description relates to cereblon E3 ligase binding compounds, including bifunctional compounds comprising the same, which find utility as modulators of targeted ubiquitination, especially inhibitors of a variety of polypeptides and other proteins which are degraded and/or otherwise inhibited by bifunctional compounds according to the present disclosure. In particular, the description provides compounds, which contain on one end a ligand which binds to the cereblon E3 ubiquitin ligase and on the other end a moiety which binds a target protein such that the target protein is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of that protein. Compounds can be synthesized that exhibit a broad range of pharmacological activities consistent with the degradation/inhibition of targeted polypeptides of nearly any type.

Abstract: Provided herein are conjugates including a polypeptide and one or more drug molecules. The polypeptide includes one or more charged motifs, and may further include one or more uncharged motifs. The conjugates may be used to effectively deliver the drug molecule to a subject.

Abstract: The disclosure provided herein pertains to CCK2R-drug conjugates. In particular, the disclosure pertains to CCK2R-drug conjugates that target the delivery of drugs to a mammalian recipient. Also described are methods of making and using CCK2R-drug conjugates.

Abstract: The present invention relates to compounds, and pharmaceutically acceptable salts thereof, comprising a vascular disrupting agent (VDA) associated and a MMP proteolytic cleavage site. The compounds are useful in the treatment of cancer.

Abstract: The invention relates to 3-arms star-shaped polypeptides derivatives which are able to self-assemble to form bioresponsive nanometric globular structures with controllable size and shape. These multivalent constructs also present the ability of disassemble under specific physiological conditions and of linking to at least one active agent so that they can be used as carries in biomedical applications.

Abstract: The invention relates to isolated immunogenic peptides comprising a MHC class II T cell epitope, and immediately adjacent or separated from said epitope a H-X(0,2)-C-X(2)-[CST] or [CST]-X(2)-C-X(0,2)-H redox motif.

Abstract: The invention provides compositions for oral delivery and methods of treatment using VSP carriers, such as Giardia sp. variable surface proteins (VSP), to deliver therapeutic agents. VSP drug carriers can be combined with bioactive peptides, e.g., glucagon, or hGH, and be administered orally or mucosally. VSP carriers are resistant to acidic pHs and to proteolytic degradation and protect therapeutic agents from degradation in the gastrointestinal tract.

Abstract: The present disclosure relates to pyrrolobenzodiazepine (PBD) prodrugs and conjugates thereof. The present disclosure also relates to pharmaceutical compositions of the conjugates described herein, methods of making and methods of using the same.

Abstract: The present invention relates to an antibody-drug-conjugate. From one aspect, the invention relates to an antibody-drug-conjugate comprising an antibody capable of binding to a Target, said antibody being conjugated to at least one drug selected from derivatives of dolastatin 10 and auristatins. The invention also comprises method of treatment and the use of said antibody-drug-conjugate for the treatment of cancer.

Abstract: Provided are Conjugate comprising PBDs conjugated to a targeting agent and methods of using such PBDs.

Abstract: A system is provided which includes nanoparticle conjugates configured to bind with a tumor cell, the nanoparticle conjugate comprising a nanoparticle, at least one targeting entity bound to the nanoparticle, and at least one shielding entity that shields at the at least one targeting entity, the nanoparticle, or both; a body-mountable device mounted on an external surface of a living body and configured to detect a tumor cell binding response signal transmitted through the external surface, wherein the tumor cell binding response signal is related to binding of the nanoparticle conjugates with one or more tumor cells; and a processor configured to non-invasively detect the one or more tumor cells based on the tumor cell response signal. Nanoparticle conjugates and methods for use for treating or imaging tumor cells are also provided.

Abstract: Silica-based biomolecule carriers, compositions comprising the same and preparation methods and uses thereof for delivering biomolecules into a cell are provided. The silica-based biomolecule carrier comprises a porous core; a first bioactive moiety; a second bioactive moiety functionally associated with the first bioactive moiety; and linkers for respectively conjugating the first bioactive moiety and the second bioactive moiety to the porous core.

Abstract: The present invention relates to a bicistronic expression vector for silencing a gene specifically in astrocytes and neurons, comprising two expression cassettes comprising a first and a second silencer sequence, respectively, wherein the expression of said first silencer sequence within astrocytes is regulated by an astrocyte-specific promoter and the expression of said second silencer sequence within neurons is regulated by a neuron-specific promoter. In a preferred embodiment, said first and second silencer sequences are SOD1 silencer sequences. Pharmaceutical composition comprising said bicistronic vector and the use of the same in the treatment of motoneuron diseases are further described.

Abstract: Provided is a tissue staining composition containing carbon particles having a mean particle diameter less than 0.3 ?m in diameter (optionally less than 0.2 ?m in diameter) together with one or more agents that maintain the carbon particles in suspension (for example, an anti-settling agent and/or surfactant). In certain circumstances, the anti-settling agent may also have mucoadhesive properties. The tissue staining composition is visually dark and does not disperse rapidly when introduced into regions of tissue of interest making it ideal for marking the regions that can be visualized clearly and over prolonged periods of time via, for example, direct visualization, endoscopic or laparoscopic inspection. The invention also provides methods of making and using the tissue staining composition for marking regions of tissue of interest, for example, gastrointestinal tissue, as well as other tissues.

Abstract: A reporter gene that can be administered to an individual such as HIV-BAL-eLuc in order to detect the presence of a virus. A method of detecting the presence of virus in an individual, by administering a reporter gene to the individual, associating the reporter gene with the virus, imaging the individual, and detecting the presence of virus in the individual. A method of determining the efficacy of a treatment for a virus, by administering a reporter gene to the individual receiving treatment for the virus, associating the reporter gene with the virus, imaging the individual, detecting the presence of virus in the individual, and determining if the treatment is effective.