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221
results.
Updated on
07/08/2025 [07:14:00]
Results 100 to 125 of 221
Absstract of: AU2024223881A1
The present invention relates to methods for the purification of lipid nanoparticles (LNPs) encapsulating a nucleic acid, comprising the steps of subjecting a solution containing said LNPs to a chromatographic medium with convective flow properties in the presence of at least one kosmotropic agent; and eluting LNPs from said chromatographic medium. The present invention further relates to respective uses of a chromatographic medium with convective flow properties for the purification of lipid nanoparticles (LNPs) encapsulating a nucleic acid.
Absstract of: AU2024206838A1
The disclosure provides compositions, methods of treatment, and methods of making and using compositions to deliver a nucleic acid to a subject that, optionally, have reduced reactogenicity and promotes a local innate immune response in the subject while promoting an adaptive immune response. Compositions described herein include nanoparticles, optionally including an inorganic particle, capable of admixing with nucleic acids encoding proteins, antibodies, or immunomodulators. Methods of using the compositions as a therapeutic vaccine for the treatment of an infection or cancer are also provided.
Absstract of: AU2024209247A1
41 P6105182WOThe invention relates to formulations comprising miRNA that have improved stability for the treatment of diseases including neurodegenerative diseases such as spinocerebellar ataxias type 3. 5
Absstract of: AU2023394992A1
The present invention relates to a messenger RNA (mRNA)-based immunogenic composition that is capable of inducing a mammalian cell to produce an influenza virus-like particle (VLP). The immunogenic composition comprises one or more mRNAs encoding an influenza virus matrix 1 (M1) protein and one or more influenza virus hemagglutinin (HA) proteins and/or one or more influenza virus neuraminidase (NA) proteins.
Absstract of: AU2025205348A1
Abstract The present disclosure relates to a copolymer and a polymersome for targeted delivery of biomolecules to a living organism. Hie exemplary copolymer comprises an initiator block, a propagator block, and a linkage connecting the initiator block and the propagator block. The initiator block comprises a glycan head configured to provide a targeted delivery, and the propagator block comprises a functional moiety configured to provide desired properties for the polymersome. Abstract The present disclosure relates to a copolymer and a polymersome for targeted delivery of biomolecules to a living organism. Hie exemplary copolymer comprises an initiator block, a propagator block, and a linkage connecting the initiator block and the propagator block. The initiator block comprises a glycan head configured to provide a targeted delivery, and the propagator block comprises a functional moiety configured to provide desired properties for the polymersome.
Absstract of: WO2025160552A1
A combinatorial cancer vaccine for cancer therapy includes a lipid nanoparticle that includes a plurality of pH sensitive protonatable or ionizable lipids, at least one of a TLR7 agonist, TLR 8 agonist or a TLR9 agonist complexed with and/or encapsulated by the pH sensitive protonatable or ionizable lipids, optionally a nucleic acid encoding a cancer antigen or neoantigen complexed or conjugated with and/or encapsulated by the pH sensitive protonatable or ionizable lipids, and optionally a stabilizing amount of at least one stabilizing polymer, polyethylene glycol or polysaccharide, or structural lipid that is conjugated to and/or complexed with the pH sensitive protonatable or ionizable lipids.
Absstract of: WO2025160462A1
Methods for treatment of hepatocellular carcinoma using formulations of tegavivint, alone or in combination with an immune checkpoint inhibitor.
Absstract of: WO2025160118A1
The present disclosure relates, in part, to hematopoietic stem cell (HSC) targeted lipid nanoparticle (LNP) compositions, and methods of use thereof for delivery of nucleic acids and/or therapeutic agents to HSCs. In certain embodiments, the LNPs of the disclosure are suitable for in utero administration. In another aspect, the disclosure provides methods for treating, preventing, and/or ameliorating HSC-related disease and/or disorders, including but not limited to hemoglobinopathies and immunodeficiencies.
Absstract of: WO2025160177A1
The present disclosure provides compositions which facilitate preferential targeting or delivery of a therapeutic agent to a particular organ, cell, or tissue. The presently disclosed compositions comprise lipid nanoparticles formed from a covalent-bond forming lipid, a cationic lipid, and, in some embodiments, a steroid or sterol, a phospholipid, and a PEG lipid.
Absstract of: WO2025160412A1
Particles, aqueous dispersions, and compositions having high lipophilic component (LC) concentrations and high LC to surfactant ratios are described. The particles, aqueous dispersions, and compositions can be used to efficiently deliver active compounds and/or active ingredients through a variety of formulation types.
Absstract of: WO2025160594A1
A method for immunizing a subject against a pathogen, comprising the steps of: (1) administering to the subject an effective amount of a prime vaccine, wherein the prime vaccine comprises one or more mRNA constructs encoding one or more immunogens from the pathogen; and (2) administering to the subject an effective amount of a boost vaccine, wherein the booster vaccine comprises a non-mRNA vaccine modality, such as a recombinant protein, against the pathogen, wherein the boost vaccine comprises the immunogen or immunogens encoded in one or more mRNA constructs in the prime vaccine, or wherein the boost vaccine comprises the immunogen or immunogens that are not the same as encoded in one or more mRNA constructs in the prime vaccine, wherein the boost vaccine is administered after the administration of the prime vaccine.
Absstract of: WO2025159411A1
An embodiment of the present invention relates to: a vaccine nanoparticle comprising an assembly of cell-penetrating peptides (CPPs) conjugated with deoxycholic acid (DOCA); a method for preparing same; and a vaccine composition for preventing or treating cancer, comprising same. The effects of migration, maturation, and antigen presentation through MHC I in dendritic cells, as well as antigen-specific T cell immune responses through the nanoparticle of the present invention were evaluated in vitro and in vivo. An improved effect was also confirmed through repolarization of tumor-associated macrophages. Additionally, the effect of combination therapy with an immune checkpoint inhibitor was confirmed in a tumor-bearing animal model. The nanoparticle of the present invention induces antigen-specific immunity and macrophage repolarization, thereby improving anti-tumor immunotherapy, and thus can be applied as an mRNA cancer vaccine.
Absstract of: WO2025159588A1
The present invention relates to a gene delivery platform for multi-organ sequential targeting oral administration, and to a gene delivery platform having a layer-by-layer chylomicron-mimicking self-assembly (lbl-CMSA), which is a lipid nanoparticle having a double-layer structure. A gene delivery platform for oral administration according to the present invention allows a second layer containing the outermost bile acid to exhibit high durability in the gastrointestinal tract even under various pH and enzyme actions, allows a gene drug encapsulated by an intestinal active target mechanism of bile acid to be primarily delivered to intestinal cells, and allows a first layer containing apolipoprotein to be delivered to lymphatic vessels through a chylomicron-mimicking pathway, thereby allowing the gene drug encapsulated therein to secondarily reach a target organ via a circulatory pathway in the body. Therefore, the gene delivery platform for oral administration of the present invention can be used as an effective oral administration formulation having increased bioavailability by loading, on the platform, a combination of gene therapeutic agents sequentially targeting the intestine and various organs.
Absstract of: WO2025159614A1
An embodiment of the present invention relates to: a cancer antigen peptide nanostructure comprising an assembly of cancer antigen peptides conjugated with deoxycholic acid; a method for preparing same; and use thereof. The inventors of the present invention confirmed that the nanostructure of the present invention effectively stimulates the maturation of dendritic cells, migrates to lymph nodes, and activates T cells. Also, in a cancer cell model, significant infiltration of cytotoxic T lymphocytes into primary tumors was observed, and an anti-metastatic effect was confirmed. Therefore, the nanostructure according to the present invention can be effectively used as a promising immunotherapy platform that can be applied to various intractable cancers.
Absstract of: WO2025158300A1
Provided is a method of increasing bioavailability of at least one administered active pharmaceutical ingredient (API) in a subject in need thereof, the method comprising administering at least a first composition comprising the API and at least one nanoporous solid carrier, wherein the at least one API is contained in pores of the at least one nanoporous carrier.
Absstract of: WO2025157198A1
The present application belongs to the technical field of polypeptides and biomedicine, and specifically relates to a polypeptide monomer molecule MBP, a polypeptide co-assembled nanoparticle and the use thereof. In the polypeptide co-assembled nanoparticle of the present application, a lipopolysaccharide (LPS)-targeting unit in the polypeptide monomer molecule MBP is used to target a lipopolysaccharide on a bacterial outer membrane, the protective effect of the bacterial permeability barrier is overcome, and the nanoparticle translocates across the membrane and enters a cell. A thiol oxidase (DsbA)-targeting unit in a polypeptide monomer molecule EIP in the polypeptide co-assembled nanoparticle is used to target a bacterial intracellular DsbA enzyme, the bacterial DsbA enzyme is strongly bound, and the polypeptide co-assembled nanoparticle undergoes a transition from a nanoparticle into a nanofiber, so that the nano composition has a stronger capacity of competitively binding to the DsbA enzyme, and the activity of the DsbA enzyme is inhibited, which causes the synthesis of a downstream key resistance enzyme β-lactamase to be blocked, thereby exerting the effect of an antibiotic adjuvant and realizing accurate delivery of antibiotics.
Absstract of: WO2025157222A1
A manganese-doped mesoporous silica nanomaterial, and a preparation method therefor and the use thereof. The nanomaterial comprises a manganese-doped mesoporous silica nanosphere, an anti-angiogenic drug and a surface delivery system. The anti-angiogenic drug is adsorbed in the pores of the manganese-doped mesoporous silica nanosphere, and the surface delivery system is coated on the surface of the manganese-doped mesoporous silica nanosphere, wherein metal manganese ions are doped on the surface and in the framework of the mesoporous silica nanosphere. The anti-angiogenic drug is an HIF-2α inhibitor. After targeted degradation at a tumor site, the nanomaterial releases the manganese ions and the anti-angiogenic drug to realize the dual anti-tumor effects of activating local anti-tumor immunity and inhibiting angiogenesis within the tumor at a targeted tumor site; and the manganese ions and the anti-angiogenic drug have a synergistic effect. In addition, the nanomaterial has no potential toxic effect, has good biosafety, and provides a new choice for the treatment of tumors, especially pVHL-deleted tumors.
Absstract of: WO2025157180A1
Provided are an anti-albumin antibody or an antigen-binding fragment thereof and the use thereof, and an anti-albumin nanobody with improved affinity or an antigen-binding fragment thereof. The anti-albumin antibodies or antigen-binding fragments thereof can bind to albumins from different species with high affinity. In addition, the anti-albumin antibody can be linked to a bioactive effector molecule to form a fusion construct without affecting the activity of the bioactive effector molecule.
Absstract of: WO2025157144A1
An oil-in-water emulsion, comprising an oil phase and a water phase. The water phase comprises: 0.5-100 mg/mL of a PEG lipid or lipid-based substance, and 10-1000 μg/mL of an antigen or 10-10000 μg/mL of a natural protein or 100-10000 μg/mL of an antibody or any combination thereof. The oil phase comprises: 30-1100 μg/mL of a cationic polymer or an ionizable lipid or protein, the pKa of the ionizable lipid or protein being less than 6.8, and 0.3-25 v/v% of a metabolizable lipid based on the volume of the oil-in-water emulsion, wherein the oil-in-water emulsion has the Young's modulus of 20-800 MPa, and the mean droplet size of 50-800 nm. In addition, further provided are an oil-in-water emulsion adjuvant, and a use of a related emulsion and adjuvant.
Absstract of: WO2025156577A1
A preparation method for and use of a scopoletin-functionalized magnetic nanoprobe. The preparation method comprises steps such as preparation of amino-terminal modified nanoparticles Fe3O4@SiO2-NH2, preparation of a photoaffinity linker-scopoletin conjugate, preparation of the scopoletin-functionalized magnetic nanoprobe, etc. The method features simple and rapid operation, and eliminates the need for prior clarification of the structure-activity relationship of the active compounds, without compromising all-round contact between the active compounds and target proteins. The prepared scopoletin-functionalized magnetic nanoprobe is used for target fishing in cellular and animal models of rheumatoid arthritis. After the captured target proteins are isolated and subjected to mass spectrometry identification, a western blot experiment successfully verifies vimentin as the target protein of scopoletin on HFLS-RA cell membranes, thereby providing a novel therapeutic target for rheumatoid arthritis.
Absstract of: WO2025156581A1
Provided are a bionic drug-loaded nanoparticle with expressed CXCR4, a method for preparing same, and use thereof. The method comprises: mixing a lactic acid-glycolic acid copolymer and an immunosuppressant in a certain ratio, and preparing the mixture into a drug-loaded nanoparticle using a microfluidic technology; preparing an MSC cell stably expressing membrane protein CXCR4 and lysing same to extract the cell membrane, so as to give an engineered stem cell membrane; and fusing the engineered stem cell membrane with the drug-loaded nanoparticle to give a bionic drug-loaded nanoparticle with expressed CXCR4. The bionic drug-loaded nanoparticle with expressed CXCR4 is a bionic cell membrane drug-loaded nanoparticle with chemotaxis functionality. By means of genetic engineering, the engineered stem cell membrane is modified to encapsulate a therapeutic agent, and directionally migrates to the lesions in the body and targets specific cells, thereby improving the drug distribution in vivo and reducing adverse effects of immunosuppressants.
Absstract of: MX2025006656A
The invention relates to the field of diseases caused by high levels of LDL-C and/or fibrinogen, such as cardiovascular disease. The invention involves oligonucleotides for RNA editing technology in deaminating target adenosine nucleotides, such as the adenosine at position 1055, in transcripts of the human <i>B4GALT1 </i>gene.
Absstract of: MX2025003345A
Disclosed is a lipid nanoparticle (LNP) encapsulating a nucleic acid cargo preferably comprising messenger ribonucleic acid (mRNA). The LNP comprises at least a cationic lipid fraction, and a stabilizer fraction. The stabilizer fraction preferably comprises at least one polyethylenglycol (PEG) lipid. Furthermore, the LNP comprises at least one glycerol dialkyl glycerol tetraether (GDGT) lipid, as obtained e.g. from archaea of the genus Sulfolobus, optionally among other ether lipids. Also disclosed is a pharmaceutical composition comprising the LNP, such as an mRNA vaccine.
Absstract of: TW202449157A
The present invention relates to an mRNA construct containing 5'-UTR with improved translation efficiency and a vaccine composition comprising same, and more particularly to an mRNA construct containing 5'-UTR with improved translation efficiency that have been engineered to include specific motifs, a codon-optimized signal sequence and an antigen encoding sequence, and a vaccine composition comprising the same. The mRNA construct according to the present invention contains a 5'-UTR with improved translation efficiency, which can effectively induce the expression of an antigenic polypeptide and is useful for vaccine development as it can be expected to increase immunogenicity as a vaccine.
Nº publicación: AR132762A1 30/07/2025
Applicant:
COUR PHARMACEUTICALS DEV COMPANY INC [US]
COUR PHARMACEUTICALS DEVELOPMENT COMPANY INC
Absstract of: TW202508618A
The present disclosure relates to a process for the preparation of tolerizing immune modifying nanoparticles encapsulating antigens associated with primary biliary cholangitis (PBC), compositions comprising the particles and use thereof for the treatment of PBC.
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