Abstract: The invention relates to pharmaceutical aerosols comprising the active compound cyclo(-Thr-Trp-Ile-Dab-Orn-DDab-Dab-Trp-Dab-Dab-Ala-Ser-DPro-Pro), or any pharmaceutically acceptable salt thereof. It further relates to liquid compositions comprising the active compound cyclo(-Thr-Trp-Il-Dab-Orn-DDab-Dab-Trp-Dab-Dab-Ala-Ser-DPro-Pro), dilutions thereof, kits comprising the liquid compositions, pharmaceutical compositions and kits for preparing and administering such aerosols. The invention can be used for the prevention, management or treatment of diseases or conditions of the lungs such as cystic fibrosis (CF), non-cystic fibrosis bronchiectasis (NCFB), asthma, chronic obstructive pulmonary disease (COPD), ventilator-associated pneumonia (VAP), ventilator-associated bacterial pneumonia (VABP), hospital-acquired pneumonia (HAP), hospital-acquired bacterial pneumonia (HABP), or healthcare-associated pneumonia (HCAP).

Abstract: The present invention relates to a formulation comprising a pharmaceutically active ingredient and a coating. The invention also relates to the use of the formulation in the treatment and prevention of disorders of the gastrointestinal tract. Also disclosed are methods for preparing the formulations.

Abstract: The present invention relates to novel fragments of AIMP1 protein and a composition for improving alopecia and promoting hair growth comprising the same, more specifically it relates to a polypeptide consisting of 4 to 21 consecutive amino acids from the amino acid sequence of SEQ ID NO: 1, wherein the polypeptide comprises the 28th to 31st amino acid residue (KEKA) of amino acid sequence of SEQ ID NO: 1, or a polypeptide consisting of an amino acid sequence having 70% or more sequence homology with the polypeptide; a polynucleotide encoding the polypeptide; a pharmaceutical, cosmetic and food composition for improving alopecia and promoting hair growth comprising the polypeptide.

Abstract: Provided herein are methods of treating corneal opacities in patients, such as neonates, including administering to the patient's eye or cornea a ZBTB7B and/or granulin polypeptide, and/or knocking down expression of HAS2 and/or HYAL1 in the patient's eye or cornea.

Abstract: The present invention relates to a synergistic combination of an FXR agonist and interferon for the treatment of hepatitis B.

Abstract: Provided is a method for administering to a subject, via an implantable delivery device, a continuous subcutaneous dose of glucagon-like peptide-1 (GLP-1) analog, where the subject is orally co-administered a drug after implantation of the implantable delivery device and during continuous subcutaneous dosing of the GLP-1 analog.

Abstract: An embodiment of the invention relates to method for increasing HDL-C in a patient in need thereof. An embodiment of the invention relates to a method for decreasing high blood pressure. An embodiment relates to a treatment method for a patient with refractory type 2 diabetes to provide normal HbA1C glycemia.

Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor IX variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia B. In some embodiments, the present disclosure provides methods for dosing a hemophilia B patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor IX polypeptide.

Abstract: Fibronectin type III domains (FN3) that specifically bind to serum albumin, related polynucleotides capable of encoding serum albumin-specific FN3 domains, cells expressing the FN3 domains, as well as associated vectors, detectably labeled FN3 domains and FN3 domains fused to a heterologous moiety are useful in extending the half-life of molecules in diagnostic and therapeutic applications.

Abstract: A water-soluble composition for bacteriostasis or for absorption through mucosal tissue which improves a cellular environment and a production method thereof are provided. The water-soluble composition contains magnesium, sodium, and protein. A content ratio between the magnesium and the sodium is magnesium:sodium=16:1 to 73:1. The protein is lactoferrin. A percentage content of the magnesium is 8.0 w/v % to 12.0 w/v %. A percentage content of the lactoferrin is 3.0 w/v % to 10.0 w/v %.

Abstract: Disclosed is a novel use of MPK38/MELK as a therapeutic agent for obesity or metabolic diseases specific to middle-aged men. Provided are a pharmaceutical composition and health functional food for preventing or treating obesity or a metabolic disease specific to middle-aged men, containing a murine protein serine-threonine kinase 38 (MPK38) protein or a gene encoding the same as an active ingredient. Obesity and metabolic disorders were found to occur in MPK38-knockout middle-aged male mice. As a result of inducing the expression of MPK38 in MPK38-knockout middle-aged male mice, MPK38 was found to have effects of decreasing the size of adipocytes, suppressing the expression of adipogenic genes, reducing glucose and insulin in the blood, enhancing insulin sensitivity, reducing triglycerides and total cholesterol in the blood, promoting ketone formation, and promoting the production of testosterone, a male hormone, in middle-aged male mice.

Abstract: Aspects of the disclosure relate to compositions and methods for positively or negatively regulating the expression of a gene therapeutic (e.g., a therapeutic protein expressed from an AAV vector). The disclosure is based, in part, on certain nucleic acids, for example antisense oligonucleotides (ASOs), configured to bind specific regions of an expression cassette (or an mRNA transcribed from such an expression cassette).

Abstract: The present disclosure is directed to recombinant mutant Granzyme A molecules that lack enzymatic activity and their use in inhibiting Mycobacterium tuberculosis and treating the related disease states.

Abstract: The present invention relates to chimeric and fusion proteins and their compositions, and the use of such proteins and compositions in the prevention and/or treatment diseases or conditions requiring plasminogen supplementation. In one aspect, the invention provides a chimeric or fusion protein comprising plasminogen and an Fc region of an antibody.

Abstract: The invention provides methods for treating primary disorders of mood and affect, including depressive disorders, anxiety and sleep disorders and CNS disorders comprising the administration of a neurotoxin.

Abstract: The present disclosure provides a method of regulating expression of a target polynucleotide in a cell. The method may comprise expressing a system in the cell, wherein the cell comprises a receptor having a ligand binding domain specific for a ligand. The method may comprise contacting the cell with the ligand that binds specifically the ligand binding domain. The system expressed in the cell may comprise a first chimeric polypeptide and a second chimeric polypeptide that are activatable upon the contacting. One of the first and second chimeric polypeptides may comprise a gene modulating polypeptide (GMP) comprising an actuator moiety linked to a cleavage recognition site. The actuator moiety may be capable of regulating the expression of the target polynucleotide in the cell. The other of the first and second chimeric polypeptides may comprise a cleavage moiety capable of cleaving the cleavage recognition site of the GMP.

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 invention features multi-specific fusion protein complexes with one domain comprising IL-15 or a functional variant and a binding domain specific to IL-12 or IL-18.

Abstract: Disclosed herein relates to immunotherapeutic compositions comprising immunotherapeutic peptides comprising neoepitopes, polynucleotides encoding the immunotherapeutic peptides, antigen presenting cells comprising the immunotherapeutic peptides or polynucleotides, or T cell receptors specific for the neoepitopes. Also disclosed herein is use of the immunotherapeutic compositions.

Abstract: The disclosure provides synthetic (e.g. recombinant) pneumococcal saccharide comprising one or more repeat unit(s) ?4)-?-D-Glcp-(1?3)-[[?-L-Rhap-(1?2)]-[Gro-(2?P?3)]-?-D-Galp-(1?4)]-?-L-Rhap-(1?. Also provided are conjugates comprising a ?4)-?-D-Glcp-(1?3)-[[?-L-Rhap-(1?2)]-[Gro-(2?P?3)]-?-D-Galp-(1?4)]-?-L-Rhap-(1?, immunogenic compositions, vaccines and their use in preventing or treating infection by Streptococcus pneumoniae.

Abstract: Immune-based approaches to treat and reduce the risk of cancer of mucosal tissues by boosting T cell immunity against commensal HPVs.

Abstract: The invention relates to a method for producing a ribonucleic acid (RNA) molecule composition comprising n different RNA molecule species, the method comprising a step of RNA in vitro transcription of a mixture of m different deoxyribonucleic acid (DNA) molecule species in a single reaction vessel in parallel, i.e. simultaneously, and a step of obtaining the RNA molecule composition. Also provided is the RNA composition provided by the inventive method and a pharmaceutical composition comprising the same as well as a pharmaceutical container. Moreover, the invention provides the RNA composition and the pharmaceutical composition for use as medicament.

Abstract: Nucleic acid molecules and compositions comprising one or more nucleic acid sequences that encode a consensus filovirus immunogen including a consensus Marburgvirus filovirus glycoprotein MARV GP immunogen, a consensus Ebolavirus Sudan filovirus glycoprotein SEBOV GP immunogen and a consensus Ebolavirus Zaire glycoprotein ZEBOV GP immunogen are disclosed. The coding sequences optionally include operable linked coding sequence that encode a signal peptide. Immunomodulatory methods and methods of inducing an immune response against filovirus, particularly Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire are disclosed. Method of preventing filovirus infection, particularly infection by Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire and methods of treating individuals infected with filovirus infection, particularly infection by Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire are disclosed. Consensus filovirus proteins are disclosed.

Abstract: Immunogenic compositions comprising viral vectors and surfactants are provided. Methods for administration and preparation of such compositions are also provided.

Abstract: Aspects of the disclosure relate to compositions of messenger RNA vaccines and methods of administration thereof. Compositions provided herein include one or more RNA polynucleotides having an open reading frame encoding a picornavirus capsid polyprotein and a protease. Compositions provided herein include one or more RNA polynucleotides having an open reading frame encoding an active product that modulates the expression, structure or function of at least one other RNA or product thereof.

Abstract: A composition, method of making, and method of using an active agent against a myriad of known as well as unknown pathogens comprising: binding a live influenza virus with a neuraminidase inhibitor in vitro; and administering the active agent with or without elimination of unbound neuraminidase inhibitors by intranasal administration or oral inhalation into patients. The active agent confers rapid and broad protection to a patient performing at least one of: i) elicit an innate immune response against known or unknown pathogens; ii) mitigate lymphopenia in general; iii) enable natural infection to activate adaptive immunity by allowing pathogens to harmlessly linger in an infected patient for a limited amount of time; and/or iv) eliciting protective immunity against influenza virus as a rapid-response influenza vaccine by making a clinically-isolated influenza virus immediately benign with a neuraminidase inhibitor in vitro without the time-consuming requirement to generate conventional influenza vaccines.

Abstract: The present disclosure relates to a chimeric influenza virus hemagglutinin (HA) polypeptide, comprising one or more stem domain sequence, each having at least 60% homology with a stem domain consensus sequence of H1 subtype HA (H1 HA) and/or H5 subtype HA (H5 HA), fused with one or more globular head domain sequence, each having at least 60% homology with a globular head domain consensus sequence of H1 subtype HA (H1 HA) or H5 subtype HA (H5 HA).

Abstract: Provided herein are non-naturally occurring, broadly reactive antigens derived from influenza viruses that are immunogenic and capable of eliciting a broadly reactive immune response, e.g., a broadly reactive neutralizing antibody response, directed against influenza virus antigens following introduction into a subject. Also provided are non-naturally, broadly reactive immunogens, vaccines, virus particles, virus-like particles (VLPs) and compositions comprising the immunogens and vaccines. Methods of generating an immune response in a human or non-human subject by administering the immunogens, vaccines, VLPs, or compositions thereof are provided. In particular, the immunogens comprise broadly reactive hemagglutinin (HA) protein antigens or soluble HA protein antigens of influenza virus strains, such as H1 or H3.

Abstract: Described herein is an anti-class II MHC antibody fused to a SARS-CoV-2 antigen. Also described is a vaccine comprising the antibody and methods for treating and/or preventing SARS-CoV-2, wherein the methods comprise administering the antibody to a subject in need thereof. In typical aspects, the vaccine is free of an adjuvant.

Abstract: Provided here are compositions for a bacterial artificial chromosome-based construct containing a replication-competent recombinant severe acute respiratory syndrome coronavirus-2 genome and methods of making such compositions and uses thereof.

Abstract: The disclosure provides coronavirus mRNA vaccines, including vaccines directed against one or more variant strains of SARS-CoV-2, as well as methods of using the vaccines.

Abstract: Recombinant polynucleotides including a nucleic acid sequence encoding an engineered SARS-CoV-2 spike protein (S12) or an immunogenic fragment thereof, in which the engineered spike protein or immunogenic fragment includes one or more mutations in the S2 segment of the S12 ectodomain. Vaccines, pharmaceutical compositions, and methods of use of the polynucleotides, vaccines, and pharmaceutical compositions in treating, alleviating, or managing SARS-CoV-2 infection and/or one or more symptoms thereof.

Abstract: The present invention relates to polypeptides and cytomegalovirus (CMV) antigens that include at least two introduced amino acid mutations relative to the amino acid sequence of the wild-type HCMV glycoprotein B (gB). In some embodiments, the polypeptide is stabilized in a conformation alternative to the gB postfusion conformation. Also disclosed are compositions including the polypeptides and uses thereof.

Abstract: Disclosed herein are methods of producing glyco-modified viral antigens that provide a shift of the glycosylation profile of recombinant produced viral antigens (e.g. glycoproteins) towards the naturally occurring viral antigens (e.g. glycoproteins). Disclosed are methods of producing a modified viral antigen comprising expressing a viral antigen in a recombinant mammalian cell line having one or more of the endogenous genes Mgat2, Mgat4A, Mgat4B, Mgat5, St3Gal3, St3Gal4, B4galt1, B4galt2, B4galt3, B4galt4, B4galt5, B3gnt2, St3Gal6, SPPL3, and/or FUT8 inactivated and/or downregulated; and optionally a gene ST6Gall inserted. Disclosed are glyco-modified viral antigens produced by the method of using a recombinant mammalian cell line. Disclosed are methods of treating a subject in need thereof comprising administering a composition comprising a therapeutically effective amount of one or more of the glyco-modified viral antigens.

Abstract: This invention relates to alpha-4 binding antibodies, and fragments thereof.

Abstract: The invention provides methods or compositions for enhancing the potency of a targeted cancer immunotherapy in a subject by using a superantigen in combination with a PD-1 inhibitor.

Abstract: Disclosed herein are MSLN binding proteins with improved binding affinities and improved ability to mediate T cell dependent killing of cancer cells expressing mesothelin. Pharmaceutical compositions comprising the binding proteins disclosed herein and methods of using such formulations are further provided.

Abstract: The present invention discloses an optimal lyophilization method to prepare a room-temperature-stable freeze-dried formulation of an anti-?4?7 antibody wherein the lyophilized anti-?4?7 antibody formulation obtained from the said method exhibits stability at room temperature for at least three months and reconstituted anti-?4?7 antibody formulation exhibits stability at room temperature at least for 24 hours.

Abstract: The use of IL-7R??c agonist peptides in cell manufacturing is disclosed. The IL-7R??c agonist peptides can facilitate the selective growth of immune cell populations. Culture media comprising the IL-7R??c agonist peptides, methods of expanding a target immune cell population, and methods of manufacturing a target immune cell population are disclosed. The enriched immune cell populations can be used in immunotherapy.

Abstract: Disclosed are means, methods and compositions of matter useful for stimulation enhancement of T cell homing into tumors and overcoming tumor microenvironment. In one embodiment the invention provides an engineered T cell in which engagement of T cell receptor on said T cell results in upregulation of T cell attracting chemokines so as to induce an increased proportion of T cells to tumor cells. In another embodiment, T cell chemokine secreting cells are administrated intratumorally in order to augment T cell infiltration into said tumors. In other embodiments administration of lymphopoietic cytokines is performed intratumorally to enhance viability of T cells approaching and residing in the microenvironment. Combinations of agents which counteract tumor microenvironment induced immune suppression are also disclosed.

Abstract: The present invention relates to chimeric receptors capable of facilitating cross-presentation (XP) of antigens, and methods of doing the same.

Abstract: Provided herein are T-cell receptors (TCRs) that when expressed recombinantly on the surface of a T cell are able to recognize the MAGE-B2-derived peptide GVYDGEEHSV (SEQ ID NO: 1) when presented by HLA-A*02:01 sufficiently to activate the recombinant T cell. Certain TCRs provided herein also are able to recognize the MAGE-A4-derived peptide GVYDGREHTV (SEQ ID NO:2) sufficiently to activate the recombinant T cell. Importantly, exemplary TCRs provided herein were thoroughly screened for lack of cross-reactivity with similar peptides that may be presented by normal cells or tissue and for alloreactivity.

Abstract: The present application relates to activable inorganic nanoparticles which can be used in the health sector, in particular in human health, to disturb, alter or destroy target cancerous cells, tissues or organs. It more particularly relates to nanoparticles which can generate a surprisingly efficient therapeutic effect, when concentrated inside the tumor and exposed to ionizing radiations. The invention also relates to pharmaceutical compositions comprising a population of nanoparticles as defined previously, as well as to their uses.

Abstract: A phototherapy method includes delivering a photosensitizer to a patient to accumulate in target treatment tissue. The target treatment tissue is illuminated with light from an illumination source in a wirelessly powered illumination device thereby photoactivating the photosensitizing agent. The combination of oxygen in cells of the target treatment tissue and the photoactivated photosensitizer generates cytotoxic reactive oxygen species which destroys cells in the target treatment tissue.

Abstract: A method of preparing a specific solid self-nanoemulsifying drug delivery system involves applying an obtained self-nanoemulsifying drug delivery system on a mixture. The mixture contains (ia) 60 to 90 parts by weight of at least one dimethylaminoethyl methacrylate-butyl methacrylate-methyl methacrylate copolymer; (iia) 10 to 40 parts by weight of at least one methacrylic acid-ethyl acrylate copolymer; and (iiia) optionally, at least one additive; where the sum of (a) and (iia) is 100 parts by weight. The solid self-nanoemulsifying drug delivery system obtained by the method of the present invention is useful as a medicament.

Abstract: The present invention provides novel, serum-stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the present invention provides serum-stable nucleic acid-lipid particles (SNALP) comprising a nucleic acid (e.g., one or more interfering RNA molecules), methods of making the SNALP, and methods of delivering and/or administering the SNALP (e.g., for the treatment of cancer). In particular embodiments, the present invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the present invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.

Abstract: The present disclosure relates to a compound of Formula (I) or a pharmaceutically acceptable salt thereof, which can be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid.

Abstract: This disclosure demonstrated that cyanobacteria bioengineered with cyanobacterial lipid-biosynthetic-promoting genes could produce large quantities of SDA, as well as rarely observed ETA. Importantly, the biosynthesized omega-3 fatty acids, such as SDA and ETA, are found conjugated to more bioavailable glycolipids, including MGDG and DGDG. Novel compositions include MGDG, DGDG, SQDG, and PG molecular species that contain the following n-3 PUFAs and n-3 LC-PUFAs at both sn-1 and sn-2 positions: 18:3/18:4, 18:3/20:4, 18:3/20:5, 18:4/18:4, 18:4/20:4, 18:4/20:5. These compositions are not found in nature and result from the engineering of cyanobacteria; and can serve as highly bioavailable, cost-effective anti-inflammatory compounds. These compositions, therefore, have strong anti-inflammatory properties with the likely capacity to block activities of cyclooxygenases, lipoxygenases, and cytochrome P450s that metabolize PUFAs and LC-PUFAs to pro-inflammatory mediators.

Abstract: Provided herein are compositions, methods of making the same, and methods for targeted delivery of therapeutic agents for modifying expression and function of target genes, e.g. proteins involved in lipid and cholesterol metabolism such as PCSK9. Further provided herein are compositions and methods of treating conditions related to coronary disease.

Abstract: Provided are bivalent compounds (e.g., bi-functional small molecule compounds), compositions comprising one or more of the bivalent compounds, and methods of use the bivalent compounds for the treatment of certain disease in a subject in need thereof, and methods for identifying such bivalent compounds.