Abstract: The present disclosure relates to the use of alkaline phosphatases, and in particular improved alkaline phosphatases such as RecAP, for the prevention, treatment, cure, or amelioration of the symptoms of acute respiratory distress syndrome (ARDS). The application relates to methods of preserving lung function, shortening the duration of mechanical ventilation therapy, increasing a the P/F ratio.

Abstract: The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.

Abstract: The present disclosure provides a method of treating peri-orbital fullness in a subject upon determining if the subject exhibits pseudoherniation of one or more upper eyelid fat pads and/or one or more lower eyelid fat pads, determining if the subject exhibits one or more conditions selected from the group consisting of: edema of the fat pad, edema along the soft tissue of the upper and/or lower eyelids, malar mounds due to edema, malar edema, and mild festoons aggravated by edema, and scoring the peri-orbital fullness from both determinations.

Abstract: Compositions and methods for the treatment of cancer are described, and, more preferably, to the treatment of cancers that do not express, or are otherwise deficient in, argininosuccinate synthetase, with enzymes that deplete L-Arginine in serum. In one embodiment, the present invention contemplates an arginase protein, such as a human Arginase I protein, comprising at least one amino acid substitution and a metal cofactor, said protein comprising an increased catalytic activity when compared with a native human Arginase I.

Abstract: A method and composition to treat a subject with arginase 1 (ARG1) deficiency (ARG1-D) and to rapidly reduce the levels of at least one of arginine and/or a guanidino compound in the subject.

Abstract: Disclosed herein are multifunctional polypeptides comprising a first domain comprising an antigen binding domain (e.g., anti-synuclein, anti-tau, anti-huntingtin) and a second domain comprising a programmable proteasome-targeting PEST motif, and methods for using these polypeptides in treatment of protein aggregation diseases, e.g., neurodegenerative diseases.

Abstract: A vaccine comprising tumor-homing bacteria which are genetically modified to express at least one cancer-associated antigen and a pharmaceutically acceptable carrier is disclosed. Uses thereof are also disclosed.

Abstract: A vaccine comprising a pharmaceutically acceptable carrier and bacteria which presents at least one cancer-associated antigen is disclosed. The bacteria are not genetically modified to express the at least one cancer-associated antigen. Uses thereof are also disclosed.

Abstract: Chimeric antigen receptors for use in treating malignant melanoma and other cancers expressing CS1 are described.

Abstract: A therapeutic vaccine for treating individuals who are carriers of a disease or pathogen that does not affect the brain, such as leishmaniasis, cancer or any pathogenic infection, comprises at least one cationic nanoparticle comprising a cationic polysaccharide core and a specific antigen, wherein the antigen is specific to the pathogen or disease.

Abstract: The disclosure provides immunogen polypeptides comprising fragments of VAR2CSA protein expressed by P. falciparum. Aspects of the disclosed immunogen polypeptides comprise all or portions of the CSA binding regions of VAR2CSA as identified by a structural study of VAR2CSA conducted by the inventors. Also provided are compositions comprising such immunogen polypeptides, and methods of using the immunogen polypeptides for vaccination and treatment of disease.

Abstract: The present invention is related to compositions and methods that reduce methane production in ruminant populations.

Abstract: The present disclosure relates to a BCG based therapeutic agent using a BCG strain that overexpresses the STING agonist, c-di-AMP. This BCG strain, called BCG-disA-OE, enhances the elevated trained immunity of macrophages and promotes early anti-viral Type I interferon responses in a subject, providing protection against viral infections such as primary respiratory infections and SARS-CoV-2 infection.

Abstract: The invention provides methods and compositions for treating fibromyalgia (FM) and chronic fatigue syndrome (CFS) in an individual. The methods provided herein entail administering a composition comprising an isolated Mycobacterium or antigenic fragments derived therefrom. Also provided herein are methods for assessing alleviation of symptoms and/or alteration of immune system functioning following administration of a composition comprising an isolated Mycobacterium or antigenic fragments derived therefrom.

Abstract: The nucleic acid sequences provided by the present invention comprises sequence HPV16-AVLS1 and sequence HPV16-AVLC1 in a 1:1 ratio; and sequence HPV18-AVLS1 and sequence HPV18-AVLC1 in a 1:1 ratio; the sequence AVLS1 and the sequence AVLC1 comprise two concatenated E6 proteins, two LI short peptides, two L2 short peptides, two concatenated E7 proteins, one PADRE sequence, and one adjuvant sequence respectively; the N-terminal of sequence AVLS1 carries a mouse IgK secretion peptide sequence; the N-terminal of sequence AVLC1 carries a ubiquitin sequence. The nucleic acid sequences provided by the present invention can not only induce high titer antibodies against E6/E7, but also elicit a high level of functional cellular immune, demonstrating excellent preventive and therapeutic effects against tumors related to HPV.

Abstract: Disclosed are methods for non-cryogenic vitrification of particles, lipid particles, lipid particle compositions and mRNA vaccine compositions that include a lipid particle, the processes including the steps of providing a lipid particle within a vitrification medium on a capillary network within a desiccation chamber and providing both a heat energy and a lowered atmospheric pressure to provide for rapid vitrification without the vitrification medium or lipid particles experiencing cryogenic temperature or boiling as a result of lowered atmospheric pressure. The lipid particle can be later reconstituted after long term storage at ambient or higher temperature and still retain structural integrity and activity.

Abstract: Self-amplifying RNA (saRNA) molecules encoding an influenza virus antigen and methods of use thereof are disclosed herein.

Abstract: Recombinant vaccine compositions combined with a specified adjuvant to deliver a robust immune response against coronaviruses for inducing protective immunity to a coronavirus comprising providing a stable immunogenic composition capable of eliciting a robust and durable immune response to the coronavirus, wherein the composition comprises a protein subunit comprising a recombinant protein specific to the coronavirus and at least one adjuvant.

Abstract: This disclosure provides compositions, pharmaceutical preparations, and uses of polyribonucleotides encoding one or more immunogenic polypeptides. In particular, this disclosure features circular polyribonucleotide encoding one or more immunogenic polypeptides.

Abstract: The present invention provides compositions comprising polypeptides comprising a plurality of epitopes from the spike glycoprotein of SARS-CoV-2, systems, and methods of using thereof for the treatment of viral infections (e.g., coronavirus disease 2019 (COVID-19)).

Abstract: Disclosed herein are nanoparticles comprising one or more SARS coronavirus 2 (SARS-CoV-2) Spike receptor binding domain (RBD) antigen and nucleic acid molecules encoding the same. Also disclosed herein is a method of treating a SARS-COV-2 infection or treating or preventing a disease or disorder associated therewith in a subject in need thereof, by administering the nanoparticles, or encoding nucleic acid molecules, to the subject.

Abstract: The invention relates to an immunogenic composition for use as a medicament to prevent and/or treat a medical condition associated with a SARS-CoV infection, said composition comprising one or more bacteria of the human microbiota and/or immunogenic parts thereof, wherein said composition is capable of inducing acquired immunity to said infection. The invention further relates to isolated bacteria of the human microbiota that bind to a neutralising antibody that recognises the spike protein of SARS-CoV, preferably the spike protein of SARS-CoV2. In another aspect, the invention relates to an in vitro method for the diagnosis, prognosis, risk stratification and/or therapy management of a human subject with an increased risk of an adverse event associated with a medical condition associated with a SARS-CoV infection (preferably with SARS-CoV-2).

Abstract: The HERPES 2 ANTIBODY SYSTEM prevents people from getting HERPES 2. It prevents people from getting HERPES SIMPLEX 2 VIRUS (HSV-2) (HERPES 2) on their genitals, mouth, anus, hands, eyes and face. And it prevents a baby from getting infected with HERPES 2 during birth.

Abstract: The present invention pertains to a vaccine comprising in combination non-replicating immunogen of porcine circo virus type 2 (PCV2), non-replicating immunogen of Mycoplasma hyopneumoniae and conjugated deoxynivalenol (DON) for protecting swine against an infection with porcine circo virus type 2, an infection with Mycoplasma hyopneumoniae and DON induced mycotoxicosis.

Abstract: Provided is use of an NGF antibody in CIPN pain. In particular, the NGF antibody blocks the interaction of nerve growth factors (NGF) with receptors thereof, and thus can treat and/or prevent chemotherapy-induced peripheral neuropathy (CIPN) pain.

Abstract: Disclosed are methods of treating various disorders with anti-IL-4R? antibodies and fragments thereof.

Abstract: The present invention provides glycan-interacting antibodies and methods for producing glycan-interacting antibodies useful in the treatment and prevention of human disease, including cancer. Such glycan-interacting antibodies include monoclonal antibodies, derivatives, and fragments thereof as well as compositions and kits comprising them. In some embodiments, the present invention provides an antibody having a heavy chain with an amino acid sequence comprising at least 95% sequence identity to SEQ ID NO: 63 and having a light chain with an amino acid sequence comprising at least 95% sequence identity to SEQ ID NO: 64.

Abstract: Disclosed herein is a method of treating cancer, such as multiple myeloma, involving the combination of an anti-BCMA antigen binding protein (e.g., an anti-BCMA antibody) and an immunomodulatory drug (e.g. pomalidomide or lenalidomide). The combinations can also include an anti-inflammatory compound (e.g. dexamethasone).

Abstract: Provided is a chimeric antigen receptor, comprising a ligand-binding domain, a transmembrane domain, a co-stimulatory domain, and an intracellular signaling domain. The co-stimulatory domain comprises an intracellular region of an NK-activated receptor or a ligand thereof. Also provided are an engineered immune cell comprising the chimeric antigen receptor and a use thereof in treatment of diseases, such as cancers, autoimmune diseases, and infections.

Abstract: The present application relates to functionally improved third generation BCMA-CARs comprising modified intracellular co-stimulatory domains, which can be used in adoptive cell therapy, e.g., in treatment of diseases and disorders such as cancer.

Abstract: A recombinant nucleic acid expression construct including a first nucleic acid sequence region encoding a chimeric antigen receptor (CAR), a second nucleic acid sequence region encoding a checkpoint inhibitory molecule, and a third nucleic acid sequence region encoding an immune stimulatory cytokine. A recombinant nucleic acid expression construct encoding the CAR specifically recognizes CD44v6, and includes a PD1 checkpoint inhibitory molecule, and an immune stimulating cytokine. Further aspects relate to genetically modified cells, including a recombinant nucleic acid expression construct encoding the CAR, wherein the cells are preferably immune cells, more preferably NK cells or cytotoxic T lymphocytes or T helper cells. Medical use of the cells may be in the treatment of a medical disorder associated with the presence of pathogenic cells expressing CD44v6, preferably cancer cells, more preferably cancer stem cells of solid or liquid malignancies.

Abstract: Provided is an engineered immune cell. The engineered immune cell expresses (i) a chimeric receptor, and (ii) exogenous CCL3, CCL4 and/or CCL5, has improved tumor killing activity, and can be used to treat cancer, infection or autoimmune diseases.

Abstract: Embodiments of the invention relate generally to electrospun fibers and, more particularly, to the controlled release of an active pharmaceutical ingredient (API) from electrospun fiber scaffolds (EFSs).

Abstract: The present document describes a process for the preparation of a low functionalization polysaccharide having carboxyl groups, comprising a) swelling of a polysaccharide granule in boiling water or a water/polyol mixture, to obtain a swollen polysaccharide; b) partial gelatinization of said swollen polysaccharide in an alkaline solvent mixture of water and alcohol and/or polyol, to obtain a partially gelatinized polysaccharide; and c) partial functionalization of said partially gelatinized polysaccharide with a functionalizing agent, to obtain the low functionalization polysaccharide.

Abstract: The present invention relates to compounds that target monocytes, macrophages and other cells (such as dendritic cells) that express CD-206, particularly those cells at are assembled at a site of disease, using a target moiety coupled to a glucan backbone. The compounds disclosed here preferably comprise a glucan backbone, a targeting moiety, a targeting moiety linker, a payload and optionally a payload linker. The present invention also provides methods of making such compounds and compositions. The present invention also provides diagnostic methods and methods of treatment using compounds comprising a target moiety coupled to a glucan backbone.

Abstract: Particles having an ACE2 targeting moiety attached to an outer surface thereof are disclosed herein. The ACE2 targeting moiety comprises a polypeptide comprising an amino acid sequence of SARS CoV-2 receptor-binding domain (RBD), wherein said amino acid sequence comprises a modification at position 358 and at least two additional modifications at two positions selected from the group consisting of 484, 498 and 501. Uses of the particles for treatment and diagnosis of diseases are also disclosed.

Abstract: The present disclosure provides delivery constructs comprising a carrier coupled to a heterologous payload, wherein coupling of the carrier to the payload can result in transportation of the payload (e.g., a therapeutic payload) into and/or across intact polarized epithelial cells (e.g., epithelial cells of the gut of a mammal). The delivery construct can be part of a pharmaceutical composition that can be orally administered to a subject to provide for improved, effective therapies for treatment of, e.g., inflammatory diseases or autoimmune diseases.

Abstract: The present invention relates to a long-acting conjugate of an interleukin-2 analog with altered binding affinity for interleukin-2 receptors.

Abstract: The present invention provides isolated antibodies that bind to the human EGFR protein, and ADCs of the antibodies. Pharmaceutical compositions including the antibodies and ADCs, and methods of treating cancer are also provided.

Abstract: The present invention provides engineered polypeptide conjugates (e.g., antibody-drug-conjugates) comprising specific acyl donor glutamine-containing tags and amine donor agents. The invention also provides methods of making such engineered polypeptide conjugates using transglutaminase and methods of using thereof.

Abstract: The present disclosure is directed to an organogel drug depot for use in a non-sterile environment and application to a non-sterile open wound site. The present disclosure is also directed to an organogel drug depot for use in delivering an active agent to a surgical site, such as an implant site, for instance an orthopedic implant site. In a further embodiment, there is disclosed a system for preparing an organogel drug depot including an organogel matrix comprising an organogelator and a biocompatible organic solvent, an active agent comprising solid particles, a container including at least one wall having an outer surface and defining a volume capable of containing the organogel matrix and active agent solid particles, and a heating component configured to contact the outer surface and supply an amount of heat to the container.

Abstract: Described herein are compositions and methods for treating cancer. The compositions comprise sphingomyelin-conjugated cancer drugs which can be formed into nanovesicles. These nanovesicles can be loaded with additional doxorubicin-conjugated drugs to provide combination therapeutics. These compositions are efficacious for cancer treatments.

Abstract: The present invention relates to methods of preparing a therapeutic exosome using a protein newly-identified to be enriched on the surface of exosomes. Specifically, the present invention provides methods of using the proteins for affinity purification of exosomes. It also provides methods of localizing a therapeutic peptide on exosomes, and targeting exosomes to a specific organ, tissue or cell by using the proteins. The methods involve generation of surface-engineered exosomes that include one or more of the exosome proteins at higher density, or a variant or a fragment of the exosome protein.

Abstract: The present application provides methods of treating a colon cancer (such as advanced and/or metastatic colon cancer) in an individual, comprising administering to the individual: a) an effective amount of a composition comprising nanoparticles comprising an mTOR inhibitor (such as a limus drug, such as sirolimus or a derivative thereof) and an albumin, b) an effective amount of anti-VEGF antibody (such as bevacizumab), and c) a therapeutically effective FOLFOX regimen (such as FOLFOX4 or a modified FOLFOX6).

Abstract: A method for treating a patient suffering from a neuronal hypo-kinetic disease or a neuronal hyper-kinetic disease by modulating neuronal activity in the: internal globus pallidus (GPi), in the anterior motor thalamus and/or in the external globus pallidum (GPe) and/or in the subthalamic nucleus (STN) by utilizing suppressor and/or enhancer DREADDs is provided.

Abstract: The present disclosure is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof, and to administering gene therapy to the subject by introducing normal human MCT8 into the subject's cells in order to increase T3 in the subject's brain.

Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.

Abstract: Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5? inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3? ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.

Abstract: The present disclosure relates to an upconversion nanoparticle, which is represented by the following Chemical Formula 1 and comprises a nanoparticle doped with lanthanide ion: Li3ZrF7:Ln3+[??Chemical Formula 1] where Ln is a lanthanide element selected from the group consisting of La, Ce, Pr, Nd, Pm, Sm, Eu, Gd, Tb, Dy, Ho, Er, Tm, Yb, Lu, and combinations thereof.

Abstract: The present invention to freeze-dried monodisperse microbubbles. The invention also relates to a process for preparing said microbubbles using microfluidic techniques. The microbubbles according to the present invention show interesting properties, making them particularly useful in applications that require monodispersity of the microbubbles.