Resumen de: WO2025117815A1
Provided are lipid nanoparticles compositions comprising a payload, a helper lipid, a sterol, a PEG-lipid, an ionizable lipid, a permanently cationic lipids. Also provided herein are a method of delivering a payload to a cell in a lung of a subject by intravenous administration and a method for treating and/or preventing a lung disease in a subject in need thereof by intravenous administration. Also provided are a method for quantifying an amount of capped messenger RNA (mRNA) in an mRNA sample comprising contacting the mRNA with two or more of a nuclease, an alkaline phosphatase, and a polynucleotide kinase and separating the capped mRNA and the uncapped mRNA occurs using chromatography.
Resumen de: WO2025116441A1
The present disclosure relates to a pharmaceutical composition for preventing or treating osteoarthritis. The pharmaceutical composition for preventing or treating osteoarthritis according to the present disclosure is not only highly stable, thereby exhibiting excellent therapeutic effects, but is also expected to be a disease-modifying osteoarthritis drug (DMOAD)-class therapeutic agent that can prevent and preemptively treat osteoarthritis by overcoming the limitations of cell therapeutic agents through the use of mRNA.
Resumen de: WO2025117817A1
Provided are aerosolized pharmaceutical compositions including comprising aerosol particles, the aerosol particles comprising lipid nanoparticles (LNPs), the LNP comprising at least two selective organ targeting (SORT) lipids and/or at least six lipids. Also provided herein are methods of administering the aerosolized pharmaceutical compositions described herein.
Resumen de: WO2025117822A1
Described herein are self-assembled peptides and hydrogels comprising self-assembled peptides and glucose. Exemplary hydrogels comprising self-assembled peptides and glucose may be used to encapsulate glucagon and/or a glucagon analogue. Hydrogels with glucagon and/or a glucagon analogue encapsulated therein may be used to treat hypoglycemia and related disorders in subjects in need thereof.
Resumen de: WO2025117816A1
Disclosed herein are lipid nanoparticles (LNPS) and pharmaceutical compositions and vaccines comprising same. Also disclosed herein are methods of administering an LNP composition, pharmaceutical compositions, and vaccines described herein for preventing an infectious disease and/or for immunizing a subject or for delivering a payload to a cell in the central nervous system (CNS) of a subject. Also provided are methods for treating and/or preventing a CNS disease.
Resumen de: WO2025117732A1
The present disclosure provides for improved compositions of ionizable lipid nanoparticles for the delivery of therapeutic nucleic acids to cells. Anionic phospholipids, including phosphatidylserine and phosphatidylglycerol are included in the lipid nanoparticles to increase the transfection efficiency in human dendritic cells. The further incorporation of mono-unsaturated alkyl chain analogs in dimethylaminopropyl-dioxolane or heterocyclic ketal ionizable lipids in the formulation demonstrated high levels of transfection in human dendritic cells, compared to other ionizable lipids in the same family, and demonstrated good stability to oxidative damage. Finally, the use of an ammonium salt of phosphatidylserine allows for the efficient production of PS-targeted LNPs.
Resumen de: WO2025114612A1
Hybrid particles comprising a cross-linked polysaccharide matrix and metal oxide nanoparticles The invention relates to a method of manufacturing hybrid particles comprising a matrix of cross-linked polysaccharide and metal oxide nanoparticles embedded therein. It has been found that hybrid particles with low polydispersity and a high load of metal oxide nanoparticles could be obtained when non-coated metal oxide nanoparticles are used. Aggregation of the non-coated metal oxide nanoparticles could be controlled to achieve said polydispersity and load. The hybrid particles of the invention, when the metal oxide is a magnetic and photothermal metal oxide, could act on the thrombosis via a combination of thermal and mechanical action, through light and magnetic stimulation, and would be targeted to the occluded vessel by an external permanent magnet.
Resumen de: WO2025114613A1
Crosslinked polysaccharide nanoparticles The present invention relates to cross-linked polysaccharide nanoparticles, which can be used as drug delivery systems. The herein disclosed cross-linked polysaccharide nanoparticles are obtained via an emulsion cross-linking process. The careful choice of the surfactants used for forming the emulsion allows to reliably obtain small particles with a low polydispersity. Moreover, these particles are stable in saline solutions. The cross-linked polysaccharide nanoparticles can be loaded with proteinaceous fibrinolytic or thrombolytic agents and could be used as drug delivery systems for the treatment of thrombosis.
Resumen de: WO2025114520A1
The present invention relates to ionizable lipid nanoparticles (LNPs) including at least one lipid molecule comprising a thiourea moiety linked to a functional moiety. The ionizable lipid nanoparticles (LNPs) including at least one lipid molecule comprising a thiourea moiety linked to a functional moiety, disclosed herein, are useful in delivering an agent to a cell or in transfecting a cell, especially for gene therapy or for gene editing.
Resumen de: WO2025116961A1
The disclosed technology includes cannabinoid oral products, and methods for making same. In some embodiments, the oral products are pouches including cannabinoid infused wafers. In some embodiments, the method of making the cannabinoid oral products includes preparing an emulsion, preparing a spray dry solution, spray drying solution into cannabinoid and carbohydrate particles, preparing a paste, preparing a wafer in a heated vacuum oven, fragmenting the wafer into cannabinoid infused wafer fragments, mixing the cannabinoid infused wafer fragments with starch, and dispensing the cannabinoid infused wafer fragments into pouches. The methods may also include labeling the oral products, for example, with a THC warning symbol.
Resumen de: WO2025116530A1
The present invention relates to nanoparticles comprising sirolimus and albumin, a pharmaceutical composition for subcutaneous administration, comprising same, and a preparation method therefor. If the preparation method of the present invention is used, nanoparticles comprising sirolimus and albumin and having excellent improved particle size distribution maintenance stability can be prepared, and the prepared nanoparticles can be prepared in a pharmaceutical composition suitable for subcutaneous injection, and thus dosage is increased and convenience of administration is improved.
Resumen de: WO2025116522A1
The present invention relates to silica-based nanoparticles for sustained release of a hydrophilic drug and a manufacturing method thereof, the nanoparticles comprising: silica-based hollow nanoparticles comprising a hollow core layer and a silica matrix shell layer infiltrated with metal; and a hydrophilic drug supported by the hollow nanoparticles.
Resumen de: WO2025116518A1
The present invention relates to a pharmaceutical composition and an oral composition, which comprise nano-graphene oxide or a variant thereof for preventing or treating oral diseases, wherein the nano-graphene oxide or the variant thereof can prevent and treat various oral diseases including gingivitis through antibacterial, anti-inflammatory and osteoclast differentiation-promoting factor reduction effects in the oral cavity, and thus the present invention can be applied to various oral care products.
Resumen de: WO2025116176A1
The present invention relates to nanoparticles which comprise: a liposome core; a chitosan coating layer on the surface thereof; and bromelain bound to at least a part of chitosan of the chitosan coating layer, thereby facilitating mucus attachment and penetration and having excellent stability.
Resumen de: WO2025116177A1
The present invention relates to a pharmaceutical composition comprising mesenchymal stem cells overexpressing interleukin-10 (IL-10), poly-L-lactic acid particles, and hydrogel, wherein the composition has remarkably excellent adhesion and proliferation abilities of stem cells with a medicinal effect on degenerative arthritis, thereby exhibiting an excellent medicinal effect on degenerative arthritis.
Resumen de: WO2025113478A1
A tumor nano-vaccine and a preparation method therefor, belonging to the technical field of biopharmaceuticals. The tumor nano-vaccine comprises tumor cell lysate nanoparticles. The tumor cell lysate nanoparticles are formed by taking an acryloyl ester as a monomer, utilizing a biodegradable cross-linking agent, and synthesizing in situ a nano-thickness polymer shell on the surface of a protein or a tumor specific antigen in a tumor cell lysate by utilizing a free radical polymerization reaction. The tumor cell lysate nanoparticles can enhance the immunogenicity of a tumor antigen, thereby improving the immune response of an organism to a tumor. The tumor nano-vaccine enhances the stability of the tumor antigen, thereby ensuring the in vivo persistent circulation of the tumor antigen, and induces the organism to generate cell immunity and activate T cells, thereby achieving the effect of treating and/or preventing tumors.
Resumen de: WO2025113656A1
An ionizable cationic lipid compound having a structure represented by formula (I), which can be used to prepare lipid nanoparticles (LNP) for the delivery of a therapeutic and/or prophylactic agent. The LNP prepared using the ionizable cationic lipid compound has good stability and transfection efficiency, and can efficiently and stably deliver bioactive substances (including nucleic acids, such as mRNA) to target cells or organs, thereby eliciting a high-specificity antibody response in vivo.
Resumen de: WO2025113654A1
An ionizable cationic lipid compound having a structure as represented by formula (I), which can be used for preparing a lipid nanoparticle (LNP) for the delivery of a therapeutic agent and/or a prophylactic agent. The LNP prepared with the ionizable cationic lipid compound has a better stability and transfection efficiency, and can efficiently and stably deliver a bioactive substance (comprising a nucleic acid, e.g., mRNA) to a target cell or organ, thereby eliciting a highly specific antibody response in vivo.
Resumen de: WO2025113662A1
Provided are a lipid nanoparticle composition for delivering a nucleic acid drug, a preparation method therefor, and use thereof. The lipid nanoparticle composition comprises three lipid components: a steroid-cationic lipid compound, a neutral phospholipid, and a polyethylene glycol lipid. The composition prepared by mixing the described components has relatively good stability and transfection efficiency. The lipid nanoparticle is used for delivering nucleic acid, such as mRNA, can efficiently and stably deliver the nucleic acid drug to a target cell or organ, and can induce a relatively high specific antibody response in an experimental animal, and the antibody has a better safety profile.
Resumen de: WO2025113255A1
The present invention provides a composite molecule, a nucleic acid, a vector, a host cell, and a pharmaceutical composition, and uses of the composite molecule, the nucleic acid, the vector, the host cell, and the pharmaceutical composition in preparation of drugs for treating cancers. The composite molecule comprises an anti-tumor antibody domain, a linker, and pro-IL-15; the anti-tumor antibody domain is linked to the pro-IL-15 by means of the linker; the anti-tumor antibody domain is a complete antibody against an immune checkpoint molecule, a tumor antigen molecule or an immune activation molecule, or a nano antibody or an antigen binding fragment thereof; the linker is a polypeptide linker or a non-peptide linker; the pro-IL-15 is a fusion protein comprising IL-15, an IL-15Rα sushi domain, and a linker peptide, and optionally comprising an IL-15Rβ extracellular domain; and the IL-15Rβ extracellular domain, the IL-15, and the IL-15Rα sushi domain are linked by means of the linker peptide.
Resumen de: WO2025112086A1
Disclosed are an oxidation-responsive cationic water-soluble pillararene, and a preparation method and a use. The oxidation-responsive water-soluble cationic pillararene is a cyclic small-molecule nucleic acid carrier and is obtained by carrying out quaternization reaction on 4-methyl borate and a tertiary amine modified pillararene. A delivery carrier having a high positive charge density is obtained by means of small molecule synthesis, the delivery carrier can tightly bind with a negatively-charged nucleic acid substance to form a nanocomposite, and after entering cells, in an oxidative environment, positive charges of the carrier fall off and the nucleic acid substance is released to efficiently express nucleic acid information.
Resumen de: US2025179096A1
Disclosed are an oxidation-responsive water-soluble cationic pillararene, and a preparation method and application thereof, the oxidation-responsive water-soluble cationic pillararene is a cyclic small-molecule nucleic acid vector, and is obtained by quaternization reaction of 4-methylborate and tertiary amine-modified pillararene. According to the invention, a delivery vector with a high positive charge density is obtained through small molecule synthesis, the delivery vector is capable of being tightly complexed with negatively charged nucleic acid substances to form a nano-composite, and after the nano-composite enters cells, vector positive charges fall off to release the nucleic acid substances in an oxidation environment, and nucleic acid is efficiently translated and expressed. The delivery vector has the characteristics of simplicity and convenience in synthesis, high delivery efficiency and good biological safety, and has a good application prospect.
Resumen de: US2025177423A1
Provided herein is a photoresponsive prodrug comprising an active agent conjugated to a photoresponsive group, and a nanoparticle, wherein the photoresponsive prodrug is co-assembled with a polymer to form a nanoparticle. Also provided is a method of treating a subject, comprising administering photoresponsive prodrug or the nanoparticle to the subject, and irradiating at the target site with a light source.
Resumen de: US2025177360A1
Disclosed are compositions and methods that provide pharmacodynamic effects specific to therapeutic macromolecules. The effects may result from reduced doses of therapeutic macromolecules in combination with immunosuppressant doses. The effects may also be enhanced with such compositions.
Nº publicación: US2025177356A1 05/06/2025
Solicitante:
EAGLE PHARMACEUTICALS INC [US]
Eagle Pharmaceuticals, Inc
Resumen de: US2025177356A1
Long term storage stable bendamustine-containing compositions are disclosed. The compositions can include bendamustine or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable fluid which can include in some embodiments PEG, PG or mixtures thereof and an antioxidant or chloride ion source. The bendamustine-containing compositions have less than about 5% total impurities, on a normalized peak area response (“PAR”) basis as determined by high performance liquid chromatography (“HPLC”) at a wavelength of 223 nm, after at least about 15 months of storage at a temperature of from about 5° C. to about 25° C.