Abstract: Methods for preventing, delaying the onset of, or treating rejection of an allograft using a DNA vaccine, where the vaccine can comprise a polynucleotide encoding a pro-apoptotic protein, like BAX and/or a polynucleotide encoding an autoantigen or donor antigen, like secreted glutamic acid decarboxylase 55. Administering one of the DNA vaccines to a transplant recipient, as described herein, can induce a donor specific tolerogenic response.

Abstract: The present invention provides compositions comprising peptide-coupled biodegradable poly(lactide-co-glycolide) (PLG) particles. In particular, PLG particles are surface-functionalized to allow for coupling of peptide molecules to the surface of the particles (e.g., for use in eliciting induction of immunological tolerance).

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 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: A bioconjugate of an E. coli glucosylated O4 antigen polysaccharide covalently linked to a carrier protein and compositions thereof are provided. Also provided are recombinant host cells for producing the bioconjugate, and methods of producing the bioconjugate using the recombinant host cells. The recombinant host cells contain a nucleic acid encoding a glucosyl transferase capable of modifying the E. coli O4 antigen with glucose branching to produce the glucosylated O4 antigen polysaccharide. Bioconjugates of an E. coli glucosylated O4 antigen polysaccharide described herein can be used alone or in combination with one or more additional E. coli O-antigen polysaccharides to induce antibodies against an E. coli glucosylated antigen, and to vaccinate a subject against extra-intestinal pathogenic E. coli (ExPEC).

Abstract: A method of immunizing a human against infection by parasitic worms, comprising orally administering a live attenuated recombinant bacterium, expressing at least one antigen corresponding to a parasitic worm antigen; and a sterile injectable vaccine comprising the at least one antigen corresponding to a parasitic worm antigen. The method is effective against worms, including schistosomes.

Abstract: Provided herein are subunit vaccine compositions comprising a nanocarrier and a lipid antigen, a peptide antigen or combinations thereof that elicit bother a CD1-restricted and an MHC-restricted T cell response in a subject. Methods for making and using the subunit vaccine compositions are also provided.

Abstract: The present invention relates to chimeric yellow fever—Zika strains and attenuated versions thereof, wherein the nucleotide sequence encoding the signal sequence and prME protein of said yellow virus is replaced by a nucleotide sequence encoding the signal sequence and the prME protein of a Zika virus.

Abstract: Provided herein are monovalent pharmaceutical compositions (vaccine compositions) and methods for inducing a multi-arm (mucosal, humoral and cell-mediated) immune response and extended seroprotection of at least 12 months post vaccination against influenza virus.

Abstract: The present invention relates to vaccines comprising at least one mRNA encoding at least one antigen for use in the treatment of a disease in newborns and/or infants, preferably exhibiting an age of not more than 2 years, preferably of not more than 1 year, more preferably of not more than 9 months or even 6 months, wherein the treatment comprises vaccination of the newborn or infant and eliciting an immune response in said newborn or infant. The present invention is furthermore directed to kits and kits of parts comprising such a vaccine and/or its components and to methods applying such a vaccine or kit.

Abstract: Embodiments relate to a method comprising (a) expressing a vector comprising a PSGL-1 (P-selectin glycoprotein ligand-1) or a mutant thereof in a VPC (virus producing cell); and blocking a virus infection by inactivating an infectivity of a released virions from the VPC; or (b) expressing a glycoprotein or a mutant thereof in the VPC; blocking the virus infection by preventing binding of the released virions to a target cell; inactivating infectivity of the released virions; and targeting a viral infection. Other embodiments relate to (1) a broad-spectrum anti-viral product comprising: a vector expressing a glycoprotein or a mutant thereof in a VPC; and blocking a virus infection by inactivating infectivity of a released virion from the VPC; and (2) a vaccine comprising a viral particle is configured to a live attenuated or an inactivated or a non-infectious, wherein the viral particle are produced in a VPC.

Abstract: Methods and compositions are provided that can be used to vaccinate against and treat HIV. Specifically contemplated are vaccine compositions and methods of using these compositions treat HIV in patients. Aspects of the disclosure relate to an anti-CD40 antibody-HIV antigen fusion protein comprising (i) an anti-CD40 heavy chain (HCD40)-HIV antigen (Ag) fusion protein comprising the formula: HCD40-Ag, wherein Ag is a polypeptide with at least 80% sequence identity to SEQ ID NO:1; and (ii) an anti-CD40 light chain (LCD40).

Abstract: This invention relates to a method of treating a dog for canine diseases comprising administering to the dog therapeutically effective amounts of a vaccine, wherein the vaccine comprises viral antigens, a bacterin, or both, and wherein the vaccine is administered subcutaneously or orally according to the schedules provided herein.

Abstract: A vaccine comprising an immunologically effective amount of recombinant modified vaccinia Ankara (rMVA) virus which is genetically stable after serial passage and produced by a) constructing a transfer plasmid vector comprising a modified H5 (mH5) promoter operably linked to a DNA sequence encoding a heterologous foreign protein antigen, wherein the expression of said DNA sequence is under the control of the mH5 promoter; b) generating rMVA virus by transfecting one or more plasmid vectors obtained from step a) into wild type MVA virus; c) identifying rMVA virus expressing one or more heterologous foreign protein antigens using one or more selection methods for serial passage; d) conducting serial passage; e) expanding an rMVA virus strain identified by step d); and f) purifying the rMVA viruses from step e) to form the vaccine.

Abstract: The invention relates to compositions including a substance useful as an adjuvant for potentiating an immune response, and methods of using the composition in individuals with infections of tissue within or adjacent to a transformation zone, such as the transformation zone of the cervix or anal canal.

Abstract: The present invention relates to peptides that bind with high specificity and which functionally interact with the transferrin receptor (“TfR”) and which may be used in making molecular vehicles that carry biomolecules across membranes, including, e.g., across the blood brain barrier or the gastrointestinal tract. TfR specific binding moieties may also be used alone or as components in specific molecules that target the transferrin/transferrin receptor transport system. The invention relates more specifically to VNAR single chain antibodies derived from nurse shark that bind to TfR, compounds and compositions comprising a TfR specific VNAR binding moiety, methods for preparing them, diagnostic and therapeutic methods of use in vitro or in vivo, e.g., to diagnose, treat and/or prevent a pathological condition, disorder or disease in which it is beneficial to deliver a heterologous biomolecule across the blood brain barrier by association with a TfR specific VNAR binding moiety.

Abstract: The invention encompasses injectable therapeutic formulations for injection into patients in need thereof, wherein the injectable formulations are injected using a medical device having a hydrophobic surface; the injectable therapeutic formulation comprises a therapeutic protein and a stabilizing excipient that protects the therapeutic protein from forming particulates or aggregates in the presence of the hydrophobic surface. Also included are methods for reducing particulates or aggregates by using such injectable therapeutic formulations in medical devices having hydrophobic surfaces.

Abstract: The present invention relates to clinically proven subcutaneous pharmaceutical compositions comprising anti-CD38 antibodies and methods of their uses in combination with bortezomib and dexamethasone.

Abstract: The present invention relates generally to the fields of oncology, virology and immunotherapy. More particularly, it concerns the use of a recombinant modified vaccinia Ankara (MVA) virus comprising an MVA harboring a human Fms-like tyrosine kinase 3 ligand (hFlt3L) (MVA-hFtl3L). The foregoing vaccinia Ankara (MVA) virus can be delivered to tumor cells of a subject afflicted with a malignant solid tumor, to treat the tumor. In a related aspect, the present disclosure concerns a recombinant modified vaccinia Ankara virus with deletion of vaccinia virulence factor E3 (MV At.E3L) modified to express human Fins-like 5 tyrosine kinase 3 ligand (hFlt3L) isolated, suitable for use as an immunotherapeutic agent against a malignant solid tumor.

Abstract: The present invention broadly provides different compositions, kits, vectors, and methods including monoclonal antibodies directed to epitopes found within lipoarabinomannan (LAM) and phosphatidyl-myo-inositol mannoside 6 (PIM6) for the diagnosis and treatment of Mycobacterium tuberculosis infections.

Abstract: Devices, materials, compounds, systems, and processes for Cherenkov-Activated Nuclear-Targeted Photodynamic Therapy that involves generating Cherenkov light within the tissue of a target volume and using this light to activate photosensitizing material that is located in the nucleus of cells of the target volume.

Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.

Abstract: This application discloses methods for prevention and treatment of cancers. Due to the larger size of human white blood cells compared to red blood cells, a decrease in the diameter of a part of a blood vessel can block the flow of white blood cells while allowing the flow of red blood cells. As a result, in tissues with such reduction in the diameter of blood vessels, there are areas where red blood cells are present, while there is a lack of white blood cells to fight the parasites and cancer cells. Therefore, cancer cells and other parasites can reside in such areas, replicate, and develop into diseases. The disclosed methods provide for widening of the blood vessels in which a reduction in the diameter of a part of the vessel blocks the flow of white blood cells, while allowing the flow of red blood cells. As a result of this widening, white blood cells may pass through the vessel, reach to the parasites or cancer cells, and confront them.

Abstract: This application discloses methods for prevention and treatment of infectious diseases. Due to the larger size of human white blood cells compared to red blood cells, a decrease in the diameter of a part of a blood vessel can block the flow of white blood cells while allowing the flow of red blood cells. As a result, in tissues with such reduction in the diameter of blood vessels, there are areas where red blood cells are present, while there is a lack of white blood cells to fight the parasites. Therefore, parasites can reside in such areas, replicate, and develop into infections. The disclosed methods provide for widening of the blood vessels in which a reduction in the diameter of a part of the vessel blocks the flow of white blood cells, while it allows the flow of red blood cells. As a result of this widening, white blood cells may pass through the vessel, reach to the parasites, and confront them.

Abstract: The present invention relates to pharmaceutical formulations comprising a peptide and propylene glycol, to methods of preparing such formulations, and to uses of such formulations in the treatment of diseases and conditions for which use of the peptide contained in such formulations is indicated. The present invention further relates to methods for reducing the clogging of injection devices by a peptide formulation and for reducing deposits on production equipment during production of a peptide formulation.

Abstract: The present specification discloses pharmaceutical compositions, methods of preparing such pharmaceutical compositions, and methods and uses of treating a severe pain in an individual using such pharmaceutical compositions.

Abstract: A concentrated salicylic acid gel comprising salicylic acid and a stabilizer compound, wherein the salicylic acid comprises at least 30 wt % of the total weight of the gel.

Abstract: Polymeric poly(bile acid) (pBA) nanoparticles have enhanced avidity and affinity to bile acid receptors and are effective anti-inflammatory agents. Oral delivery results in local accumulation and retention in the pancreas, liver, and colon as well as in systemic delivery of the nanoparticles. The nanoparticles are effective in alleviating inflammation and are useful as anti-inflammatory agents to treat inflammatory diseases of the organs. The nanoparticles provide a therapeutic and prophylactic benefit via the TGR5 pathway when used alone, or a more than additive benefit when used in combination with immunosuppressant(s). The nanoparticles induce immune tolerance in autoimmune diseases and are useful therapeutics for treating inflammatory and autoimmune diseases.

Abstract: Embodiments described herein generally relate to methods of processing of biopolymers and applications utilizing these biopolymers.

Abstract: A compound comprising at least one looped peptide structure attached via at least one alkylamino linkage to a scaffold. Preferably the looped peptide structure is a Bicycle structure comprising two peptide loops attached to the scaffold via two alkylamino linkages and one thioether linkage, one of the linkages being common to both loops.

Abstract: Provided herein are oligonucleotides, cell penetrating peptides, and peptide-oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein.

Abstract: Provided in the present disclosure are immunotherapy compounds, pharmaceutical compositions thereof and their use, wherein the immunotherapy compounds, upon local administration, form depots inducing cell mediated immune response while mitigating a systemic proinflammatory immune response.

Abstract: The present invention relates to prodrugs of vascular disrupting agents comprising a vascular disrupting agent (VDA) associated with a MMP proteolytic cleavage site and to the use of such prodrugs in the targeted treatment of cancer.

Abstract: The present invention is directed to a polymer platform comprising poly(L-lysine succinylated) which specifically targets scavenger receptor A1. This platform may be used to conjugate different types of drugs to the polymer for treatment of specific diseases or conditions in a patient. The resulting conjugates display moderate stability or controlled drug release of about 3-80 hours in plasma, and allow delivery and release of drugs and other therapeutic moieties to tissues/cells that express scavenger receptor A1 in a controlled manner.

Abstract: Disclosed herein are compounds, drug-conjugates thereof, methods of preparing drug-conjugates, and uses thereof. Also disclosed are pharmaceutical compositions and methods of treatment. The compounds and drug-conjugates disclosed herein can be used to treat a variety of conditions, diseases and ailments such as bladder cancer, breast cancer, colon cancer, rectal cancer, endometrial cancer, kidney cancer, lung cancer, melanoma, non-Hodgkin lymphoma, glioblastoma, pancreatic cancer, prostate cancer, and thyroid cancer.

Abstract: Provided are novel anti-SEZ6 antibodies and antibody drug conjugates, and methods of using such anti-SEZ6 antibodies and antibody dmg conjugates to treat cancer.

Abstract: The present invention provides combination therapy methods of treating proliferative diseases (such as cancer) comprising a first therapy comprising administering to an individual an effective amount of a taxane in a nanoparticle composition, and a second therapy which may include, for example, radiation, surgery, administration of chemotherapeutic agents (such as an anti-VEGF antibody), or combinations thereof. Also provided are methods of administering to an individual a drug taxane in a nanoparticle composition based on a metronomic dosing regime.

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: Provided herein are cell surface conjugates containing a cell surface molecule and at least one agent, such as at least one affinity tag, and engineered cells expressing such cell surface conjugates. In some embodiments, the cell surface molecule does not contain an intracellular signaling domain or is not capable of mediating intracellular signaling. In some embodiments, the cells engineered to contain the cell surface conjugate, such as T cells, further contain a genetically engineered recombinant receptor that specifically binds to antigens, such as a chimeric antigen receptor (CAR). Also provided are methods of detecting, identifying, selecting or targeting cells expressing the cell surface conjugates, such as in connection with methods of manufacturing engineered cells or in connection with administration of such cells to subjects, including methods of adoptive cell therapy.

Abstract: Described herein is a method of induced cell death via ferroptosis by nanoparticle ingestion. Moreover, the present disclosure describes the administration of high concentrations of ultrasmall nanoparticles at multiple times over the course of treatment in combination with a nutrient-depleted environment, thereby modulating cellular metabolic pathways to induce cell death by the mechanism ferroptosis. Ferroptosis involves iron, reactive oxygen species, and a synchronous mode of cell death execution.

Abstract: Embodiments of the present disclosure provide for electroactive supramolecular polymeric assemblies, methods of making electroactive supramolecular polymeric assemblies, methods of using electroactive supramolecular polymeric assemblies, and the like.

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Abstract: This invention relates to polynucleotides comprising a nucleotide sequence encoding a PPT1 polypeptide or a fragment thereof, vectors (viral or non-viral vectors) comprising the same, and methods of using the same for delivery of the open reading frame to a cell or a subject and to treat infantile neuronal lipofuscinosis (infantile Batten disease). The polynucleotides comprise an optimized CLN1 open reading frame.

Abstract: Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Abstract: A method of treating a congenital birth defect includes detecting the presence of at least one mutated gene associated with a birth defect and injecting foreign genetic material containing at least one non-mutated version of the detected mutated gene into a patient, thereby promoting a desired therapeutic outcome in the patient. A kit for treating a congenital birth defect in a patient comprising normal genetic material tailored to treat the congenital birth defect and an injection device for providing the normal genetic material to the patient is also described.

Abstract: Targeted non-surgical administration of a nucleic acid formulation to the suprachoroidal space (SCS) of the eye of a human subject permits effective treatment of ocular disorders, including posterior ocular or choroidal maladies. In one embodiment, the method comprises inserting a hollow microneedle into the eye at an insertion site and infusing a nucleic acid formulation through the inserted microneedle and into the suprachoroidal space of the eye. The infused nucleic acid formulation flows within the suprachoroidal space away from the insertion site. In one embodiment, the fluid nucleic acid formulation comprises nucleic acid nanoparticles consisting of one molecule of nucleic acid.

Abstract: Several embodiments relate to engineered extracellular vesicles (EVs) using the membrane cloaking platform technology described herein, the cloaking imparting to the EVs enhanced delivery to tissues of interest, such as damaged or dysfunctional tissue. Several embodiments relate to engineering exosomes derived from cardiosphere-derived cells (CDCs) using the membrane cloaking platform technology described herein to confer enhanced tissue homing specificities, thereby leading to repair and regeneration at sites of injury. Uses of engineered EV compositions to treat diseases are also provided for in several embodiments.

Abstract: The present disclosure relates to methods of administering [18F]-FACBC. The present disclosure also relates to use of [18F]-FACBC in methods for imaging, diagnosing and monitoring metastasis or recurrence of cancer.

Abstract: The present disclosure relates to methods of administering [18F]-FACBC. The present disclosure also relates to use of [18F]-FACBC in methods for imaging, diagnosing and monitoring metastasis or recurrence of cancer.

Abstract: Provided herein are compounds and compositions useful for imaging, detecting, and/or diagnosing oxidative stress and/or a ROS modulated illness by detection of gamma radiation emitted by the compound, as well as intermediate compounds and methods useful to make the compounds and/or compositions, and methods of use thereof.