Resumen de: US2024285768A1
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Resumen de: US2024285796A1
Provided herein are lipids having the Formula I or Formula Ia:and pharmaceutically acceptable salts thereof, wherein R′, R1, R2, R3, R4, R5, R6a, R6b, X1, X2, and n are as defined herein for Formula I and Formula Ia, respectively. Also provided herein are lipid nanoparticle (LNP) compositions comprising lipid having the Formula I or Ia and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).
Resumen de: US2024285740A1
This disclosure features human Lymphocyte-activation gene 3 (LAG3) and human Galectin-3 (GAL3) inhibitory agents, immunogenic peptides X-Box Binding Protein 1 (XBP1), CD2 Subset 1 (CS1), and CD138 peptides, and methods of using the inhibitory agents and peptides to treat blood cancers and pre-cancerous conditions.
Resumen de: US2024285798A1
Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.
Resumen de: US2024285781A1
Provided is a polymer comprising a hydrolysable polymer backbone, the polymer backbone comprising: (i) monomer units comprising a hydrophobic side chain; (ii) monomer units comprising a side chain comprising a polyamine group and a polyalkylene oxide group; and, optionally, (iii) monomer units comprising a side chain comprising a polyamine group without a polyalkylene oxide group, as well as a method of preparing said polymer, and a method of delivering a nucleic acid and/or polypeptide to a cell using the polymer.
Resumen de: US2024285767A1
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Resumen de: US2024285754A1
The disclosure provides mRNA vaccines which encode for coronavirus spike proteins exhibiting increased flexibility in the crown of the helix turn region. This increased flexibility is achieved by at least two glycine mutations within the crown of the helix turn region, located between the HR1 and CH regions.
Resumen de: US2024285792A1
Anticancer virus particles are described. Anticancer virus particles are filamentous or rod-shaped plant virus particle containing an anticancer agent within the interior of the virus particle. The anticancer agent can be attached either covalently or non-covalently within the interior of the virus particle. A therapeutically effective amount of an anticancer virus particle can be administered to a subject identified as having cancer to provide a method of cancer treatment.
Resumen de: US2024285652A1
The present invention relates to an amorphous nano-molecular association composed of an organic/inorganic material or a salt thereof, and more specifically, to an amorphous nano-molecular association, which is prepared by applying a shear stress to an organic/inorganic material or a salt thereof, and thus has excellent solubility and permeability to a lipid membrane.
Resumen de: WO2024177282A1
The present invention relates to a novel ionizable lipid comprising a sulfide. The ionizable lipid according to the present invention enables a drug to be encapsulated in lipid nanoparticles with high efficiency, by electrostatically interacting with the anionic drug during the production of the lipid nanoparticles, also enables the drug to be stably delivered within the body, and thus can be usefully employed in related technical fields such as lipid-nanoparticle-mediated gene therapy.
Resumen de: US2024285545A1
An inhalable nanocrystal-based suspension formulation of nintedanib (NTB) (NTB-NS) possessing specific physicochemical properties to enhance drug retention in the lung was developed for localized treatment of pulmonary fibrosis via inhalation. This NTB-NS formulation was prepared using a wet-milling procedure in the presence of a surfactant, PLURONIC® F127, to endow the formulation with non-adhesive surface coatings to minimize interactions with therapy-inactivating delivery barriers in the lung.
Resumen de: US2024285580A1
Extended-release pharmaceutical compositions comprising a biodegradable microparticle, nanoparticle or other polymeric formulation of Formula I, Formula IL, or Formula III or a pharmaceutically acceptable salt thereof are provided with advantageous properties for in vivo delivery to a patient in need thereof. The extended-release formulations overcome historic obstacles in cromakalim, including levcromakalim, delivery.
Resumen de: US2024285533A1
Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for cystic fibrosis (CF), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject, wherein the LNPs comprise mRNA encoding a Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein.
Resumen de: US2024285531A1
A submicron structure comprising a silica body defining a plurality of pores that are suitable to receive molecules therein, and having a surface, and a phospholipid bilayer coating the surface, wherein said submicron structure has a maximum dimension of less than one micron, and wherein the phospholipid bilayer stably seals the plurality of pores; and wherein the submicron structure is a member of a monodisperse population of submicron structures.
Resumen de: US2024285544A1
The present disclosure provides a lipid nanoparticle for extrahepatic delivery of mRNA, the lipid nanoparticle comprising: (i) mRNA cargo; (ii) a phosphatidylcholine lipid content of from 30 mol % to 70 mol %; (iii) a ionizable, cationic lipid content of from 5 mol % to 50 mol %; (iv) a sterol selected from cholesterol or a derivative thereof; and (v) a hydrophilic polymer-lipid conjugate that is present at a lipid content of 0.5 mol % to 5 mol. Further provided is a lipid nanoparticle comprising encapsulated mRNA and 20 to 70 mol % of a phosphatidylcholine lipid, an ionizable lipid; and at least one of a sterol and a hydrophilic polymer-lipid conjugate, the lipid nanoparticle exhibiting at least a 10% increase in gene expression of the mRNA in vivo as measured in one or more extrahepatic organs or tissues. Further provided are methods of administration of such lipid nanoparticles.
Resumen de: WO2024178439A1
The present application discloses compositions comprising nanoparticles of vitamin K2, and their methods of use.
Resumen de: WO2024177973A1
The present application discloses compositions comprising nanoparticles of vitamin K2, and their methods of use.
Resumen de: US2024285575A1
Disclosed herein are compositions of nanoparticles with naringenin as a folate receptor (FR)-targeting ligand and methods of making and administering the nanoparticles with naringenin (NAR) ligands for drug delivery applications. The disclosed compositions include NAR-functional polyester-based nanoparticles encapsulating insulin (i.e. insulin-laden NAR-functional nanoparticles), which showed loading capacities of 10% and encapsulation efficiency of >70%. The insulin-laden NAR-functional nanoparticles are shown in examples to have a 3-fold higher bioavailability of insulin compared to unfunctionalized nanoparticles, suggesting the role of receptor-mediated transport across the intestinal barriers. Methods of making NAR-functional nanoparticles are also disclosed herein, and methods of administration for the peroral delivery of encapsulated drugs.
Resumen de: US2024285543A1
Described herein are methods of treating cancer by administering to a subject a composition comprising ultrasmall silica nanoparticles to enhance one or more of the following immunotherapies: chimeric antigen receptor (CAR) T-cell therapy, immune checkpoint blockade antibody therapy (ICB), immune inhibitor therapy (e.g., myeloid-targeting inhibitors). In some embodiments, the compositions are used in combination with external beam radiotherapy.
Resumen de: US2024285525A1
This document relates to methods and materials for treating cancer. For example, nanostructures (e.g., nanotubes) having one or more anti-cancer agents intercalated in the nanostructures are provided. In some cases, nanostructures (e.g., nanotubes) having one or more anti-cancer agents intercalated in the nanostructures can be administered to a mammal (e.g., a human) having cancer to treat the mammal.
Resumen de: US2024287522A1
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.
Resumen de: US2024287509A1
Nanocomplex for targeted repairing of neurovascular lesion. The nanocomplex comprises a lipid, an apolipoprotein and a targeted peptide, the targeted peptide formed by covalently linking, through a bridge structure, a nanocarrier linking end and a peptide chain targeting RAGE, which specifically binds to cerebrovascular lesion site. Based on AD and the high expression of RAGE in cerebrovascular and brain parenchyma in diabetic encephalopathy, the nanocomplex, as modified by the targeted peptide, can mainly bind, in a targeted manner, to cerebrovascular endothelium by linking the targeted peptide, to repair vascular endothelial cells, promote removal of AB plaque near blood vessels, and repair cerebrovascular and neurovascular unit components such as microglial cells, astrocytes and neurons. Ameliorating cognitive impairment is achieved by combined repairing of multiple components and restoring cerebrovascular functions and cerebral blood flow. The nanocomplex has a disease repair effect and can carry drugs, which are delivered to cerebrovascular lesion site.
Resumen de: WO2024176066A1
The present invention relates to a polymeric nanoparticle having a core@shell structure comprising poly(3-hexylthiophene) in the core and poly(3-hexylthio-phene)-S,S-dioxide in the shell with critical oxidation fraction for use in ameliorating and/or treating degenerative retinal diseases.
Nº publicación: WO2024175757A1 29/08/2024
Solicitante:
KATHOLIEKE UNIV LEUVEN [BE]
KATHOLIEKE UNIVERSITEIT LEUVEN
Resumen de: WO2024175757A1
The invention relates to a method for making unilamellar vesicles using Pickering emulsions of aqueous droplets comprising lipids in oil wherein the Pickering emulsions is stabilized by amphiphilic nanoparticles. The invention further relates to the use of the method of the invention to form synthetic cells.