Resumen de: WO2025076625A1
Provided is a glutamic or glutaric acid-based ionizable lipid compound of Formula (I) or a pharmaceutically acceptable salt thereof. The compound can be used to obtain lipid nanoparticles. In some embodiments, the lipid nanoparticle can comprise (a) from about 40 to about 100 mol % of the compound of Formula (I); (b) from 0 to about 20 mol % of a neutral lipid; (c) from 0 to about 50 mol % of a helper lipid; (d) from 0 to about 5 mol % of a polymer-conjugated lipid; and (e) from 0 to about 10 mol % of a hydrophobic component; wherein the mol % are based on the total lipids present in the nanoparticle. In some embodiments, the ionizable lipid compound is a glutamic acid-based ionizable lipid compound.
Resumen de: WO2025080206A1
This disclosure relates to methods of promoting infiltration of a nanoparticle-based anti-cancer drug into a solid tumor of a subject using mechanical stimulation. Devices for performing such a method are also disclosed. In one embodiment, the mechanical stimulation is a compressive force applied to the tumor or to a tissue surrounding the tumor, or to a tissue in the vicinity of the tumor. In another embodiment, the device comprises an actuator capable of providing mechanical stimulation to the solid tumor or to the tissue in the vicinity of the solid tumor when positioned at a proximate location from the tumor on an external skin surface.
Resumen de: US2025121098A1
The present invention provides nanoparticles or conjugates comprising at least one ligand that selectively targets major facilitator superfamily domain-containing protein-2a (MFSD2A). In various embodiments, the nanoparticles or conjugates of the invention target at least one cell comprising MFSD2A (e.g., endothelial cells of blood brain barrier). In some embodiments, the nanoparticles or conjugates of the invention cross the blood brain barrier and/or blood retinal barrier. In other aspects, the present invention relates to methods for in vivo delivery of diagnostic and/or therapeutic agents to a brain. In other aspects, the present invention relates to methods of preventing or treating a neurological or cognitive disease or disorder using the nanoparticles or conjugates of the invention.
Resumen de: US2025121043A1
The methods include selectively reducing or expanding T cells according to the antigenic specificity of the T cells. Therefore, the present invention can be used to reduce or eliminate pathogenic T cells that recognize autoantigens, such as beta cell specific T cells. As such, the present invention can be used to prevent, treat or ameliorate autoimmune diseases such as IDDM. Furthermore, the present invention can be used to expand desirable T cells, such as anti-pathogenic T cells to prevent, treat and/or ameliorate autoimmune diseases.
Resumen de: US2025120997A1
Disclosed herein is a composition including a nucleoside-modified mRNA encapsulated in a lipid nanoparticle wherein the nucleoside-modified mRNA encodes IL10, IL-6, MIP1a, GDNF (glial cell line-derived neurotrophic factor), or a combination thereof. A method of treated spinal cord injury is also described.
Resumen de: US2025120916A1
The instant disclosure teaches a highly efficient cellulose-based nanoadsorbent that can capture more than 6000 mg of doxorubicin (DOX), one of the most widely used chemotherapy drugs, per gram of the adsorbent at physiological conditions. Such drug capture capacity is more than 3200% higher than other nanoadsorbents, such as DNA-based platforms. The disclosure teaches how anionic hairy cellulose nanocrystals, also known as electrosterically stabilized nanocrystalline cellulose (ENCC), bind to positively charged drugs in human serum and capture DOX immediately without imposing any cytotoxicity and hemolytic effects. The disclosure further elucidates how ENCC provides a remarkable platform for biodetoxification at varying pH, ionic strength, ion type, and protein concentration. These discoveries pave the way for the next generation in vitro and in vivo drug capture additives and devices.
Resumen de: US2025120913A1
A pharmaceutical composition of Quercetin nanosuspension comprising Quercetin dihydrate, about 0.1% w/w to 10% w/w of Poloxamer, about 0.1% w/w to 10% w/w of polyvinyl pyrrolidone, and about 0.1% w/w to 10% w/w of polyethylene glycol. A method for preparing Quercetin nanosuspension, comprising the steps of dissolving polyvinyl pyrrolidone, Poloxamer, and polyethylene glycol in purified water, adding Quercetin to the above solution, subjecting the resulting mixture to bead milling to form Quercetin nanocrystals or nanosuspension.
Resumen de: US2025121079A1
This disclosure relates to targeted protease compositions and uses related thereto. In certain embodiments, the disclosure relates to nanoparticles wherein a targeting molecule is linked to the nanoparticle and wherein a catalytic domain of a protease is linked to the nanoparticle. In certain embodiments, the targeting molecule and the catalytic domain are within a single polypeptide sequence. In certain embodiments, the targeting molecule binds a molecule more highly expressed on cancer cells then non-cancerous cells, and the nanoparticles disclosed herein are used for the treatment of cancer by further attaching an anti-cancer agent to the nanoparticle or incorporating an anticancer agent within the nanoparticle.
Resumen de: AU2023357320A1
The present disclosure provides RNA technologies for targeting Claudin-18.2 polypeptides. In some embodiments, such RNA technologies can be useful for treatment of diseases associated with positive expression of Claudin-18.2. For example, in some embodiments, such RNA technologies can be useful for treatment of Claudin-18.2 positive cancer, including, e.g., but not limited to biliary cancers, ovarian cancers, gastric cancers, gastro-esophageal cancers, pancreatic cancers. In some embodiments, such RNA technologies can be used in combination therapy (e.g., in combination with a chemotherapeutic agent). The present disclosure further provides RNA backbones containing specific sequences upstream and/or downstream from the coding sequence.
Resumen de: WO2025077898A1
An effective and safe composition comprises an anionic gold-polydopamine core-shell nanoworm as an alternative gene carrier for bypassing the bottleneck of endosomal entrapment. The nanoworm can be used in methods of delivering therapeutic oligonucleotides to a subject. A polydopamine shell supports the surface adsorption of nucleic acids. The anionic nucleic acid-encased nanoworm can then enter cells without transfection agents and activate the ClC3 H +/Cl - exchanger in late endosomes to mediate vesicular accumulation of H + and Cl -, which causes membrane rupture, and finally escape to cytosol without cell-penetrating peptides or mechanical stimuli. The nanoworm can be further used for programming cellular responses, i. e., primary macrophage polarization and stem cell differentiation, for the treatment of diseases, such as kidney fibrosis and acute liver injury.
Resumen de: WO2025081192A1
The disclosure concerns oral formulations that enable delivery of therapeutic agents that are sensitive to stomach pH and/or have reduced ability to be absorbed. In one aspect, the oral formulation comprises: (a) phenyl boric acid (PBA)-functionalized chitosan grafted with branched polyethyleneimine (PEI) and (b) a therapeutic agent, wherein the therapeutic agent is encapsulated by the PB A- functionalized chitosan grafted with a branched PEI. In other aspects, the oral formulation comprises: (a) chitosan grafted with branched polyethyleneimine (PEI), (b) a therapeutic agent, wherein the therapeutic agent is encapsulated by the chitosan grafted with a branched PEI, and optionally (c) a eukaryotic cell membrane fragment. The disclosure also concerns methods of making and administering such oral formulations.
Resumen de: WO2025081185A1
Disclosed herein are methods for delaying the onset of type I diabetes and preventing nosocomial infections by administering PEG-b-PPS nanocarriers loaded with rapamycin. This invention aims to reduce the frequency of visits to a transfusion clinic, reduce the costs of treatments, and reduce adverse side effects, without reducing the effects of the islet transplant. This is accomplished by the use of a nanocarrier which targets treatment to the desired location.
Resumen de: WO2025077933A1
The present invention relates to the technical field of microencapsulation of natural products, and specifically relates to a water-soluble ginger oleoresin microcapsule, and a preparation method therefor and the use thereof. In the present invention, by using modified starch, plant polysaccharide substances, etc., as wall materials and ginger oleoresin as the core material, a ginger oleoresin microcapsule product is obtained by means of the processes of molecular coating of the ginger oleoresin, emulsification, high-pressure homogenization, leaving to stand at a constant temperature, drying, etc. The product has the characteristics of a high gingerol content, good water solubility, and low spiciness in the form of a solution; moreover, the product has good stability in an acid solution environment, can meet the requirements of high stability, no precipitation, low spiciness and good thermal sensation in a low-pH environment, and is applicable to the fields of food products, beverages, health-care food products, medicines, cosmetics, etc.
Resumen de: WO2025077899A1
Provided are a lyophilized preparation of a lipid nanoparticle (LNP) containing a nucleic acid, and a preparation method therefor and a use thereof. A lyophilized preparation of an LNP containing a nucleic acid is provided, and the lyophilized preparation comprises: i) an LNP containing a nucleic acid; and ii) a buffer reagent containing a lyoprotectant. The prepared nucleic acid-LNP lyophilized preparation has a uniform sample particle size, a small polydispersity index, high entrapment efficiency, and high RNA integrity after reconstitution. Moreover, the nucleic acid is prevented from leaking from the nanoparticle, and a lyophilization preservation method is further provided, so that the nanoparticle can exist in a stable form under refrigeration conditions.
Resumen de: WO2025077709A1
A preparation method for a micro-vehicle formed by enveloping siRNA-loaded polypeptide dendrimer nanogel with a cell membrane of engineered MSCs, and the use of same. The method comprises: using lentivirus-mediated gene transfection technology for constructing MSCs that highly express CXCR4 and PSGL-1; using cytokinin B for stimulating a cell to generate vesicles, and then performing centrifugation to collect the vesicles; by means of the electrostatic attraction between polypeptide dendrimer nanoparticles and siRNAs, attaching the siRNAs of IL-6 and HIF-1α into the polypeptide dendrimer nanoparticles; and by means of using a membrane extruder, enveloping the siRNA-loaded polypeptide dendrimer nanogel with the cell membrane. The prepared micro-vehicle exhibits a good property of aggregating at inflammatory blood vessel sites and a targeting function for joint cavities with rheumatoid arthritis. In addition, the loaded siRNAs can inhibit the expressions of IL-6 and HIF-1α in the joint cavities and inhibit the cell migration and angiogenesis. The micro-vehicle targeting inflammation sites has the advantages of excellent biocompatibility, ideal siRNA loading ratio, good inflammation targeting function, etc.
Resumen de: WO2025081013A1
The disclosures provides compositions comprising nanoparticles and a junction opener protein; and nanoparticles comprising one or more bioactive agents and a junction opener protein that is conjugated to the surface of the nanoparticles. Various bioactive agents are described, such as squalene, squalane, and dehydroisosqualene, among others. Various nanoparticle compositions are provided, including lipid carriers comprising a hydrophobic core. Methods for treating a subject with cancer by administering the compositions provided herein are also described.
Resumen de: WO2025077347A1
The present invention relates to a multimeric fusion protein nano-vector and the use thereof, and belongs to the technical field of genetic vector materials. The present invention designs a fusion protein containing a cell-penetrating peptide, a linker peptide and a human ferritin light chain, and optionally further containing a T cell or B cell antigenic-determinant antigenic peptide. The fusion protein constructed by the present invention can be used as a nano drug vector to target to dendritic cells, thus improving the transfection efficiency of dendritic cells. The fusion protein nucleic acid vector prepared by the present invention has greatly improved biocompatibility, still has cell viability of 80% or above at high concentrations, and has good safety, and therefore has a wide application prospect in the field of pharmacy.
Resumen de: WO2025080854A1
The present disclosure provides lipid nanoparticles for delivery of a payload (e.g., messenger RNA (mRNA)) to the heart. For example, lipid nanoparticle comprises an acid-degradable polyethylene glycol (PEG)-lipid (ADP) having molecular weight in the range of 1000 to 4500 Da. In some embodiments, the acid-degradable polyethylene glycol (PEG)-lipid is present in a mole percentage of about 1% to about 25% of the total lipids. The disclosure also encompasses methods for delivery of messenger RNA (mRNA) for transfection of cells, methods of treatment, as well as kits thereof.
Resumen de: WO2025080867A1
Provided herein are ionizable cationic lipids and lipid nanoparticle compositions comprising a lipid component comprising the same. Also, provided herein is a method of treating or preventing a disease or disorder in a subject in need thereof, the method comprising administering an effective amount of the lipid nanoparticle composition disclosed herein.
Resumen de: WO2025080649A1
Disclosed herein are POEGMA-based block copolymers that have phase transition and self-assembly properties. The disclosed block copolymers can take advantage of these properties to form particles that can effectively encapsulate and deliver drugs. An example block copolymer includes a first block that includes POEGMA with ethylene glycol side chains of 2 monomers, 3 monomers, or combinations of both; and a second block that includes POEGMA with ethylene glycol side chains of 1 monomer, 2 monomers, or combinations of both. Also disclosed herein are compositions that include the block copolymers, methods of treating a disease or disorder, and methods of delivering a drug.
Resumen de: WO2025080770A1
Methods are disclosed for increasing retinal ganglion cell (RGC) survival and/or RGC axon regeneration and/or optic nerve survival and/or optic never regeneration in a subject in need thereof. These methods include locally administering to an eye of the subject a therapeutically effective amount of (a) isolated nanovesicles derived from an extracellular matrix and (b) a statin or a pharmaceutically acceptable salt thereof. Combination therapy is also disclosed for use in increasing RGC survival and/or RGC axon regeneration and/or or optic nerve survival and/or optic nerve regeneration in a subject. Disclosed is therapeutically effective amount of (a) isolated nanovesicles derived from an extracellular matrix and (b) a statin or a pharmaceutically acceptable salt thereof, for use in increasing RGC survival, RGC axon regeneration and/or optic nerve survival and/or optic never regeneration in a subject in need thereof in a subject, wherein the isolated nanovesicles and the statin are formulated for ophthalmic administration.
Resumen de: WO2025080869A1
Drug delivery nanoparticle compositions and methods for the treatment of a cancer are provided that can be used individually or in combination. These compositions and methods include: (1) exogenous vaccination using neoantigens or tumor-associated antigens to potentiate an anti-tumor immune response; (2) nanoparticle-based drug delivery targeting the liver, aimed at reprogramming the hepatic immune environment to reduce or eliminate tumor antigen tolerance, thereby enhancing the immune system's ability to combat metastatic cancer; and (3) expansion of the liver metastasis treatment platform to target tumor-associated lymphoid structures, supporting the immune response at the primary tumor site. Any of these methods may be optionally combined with chemo-immunotherapy for enhanced efficacy.
Resumen de: WO2025080555A1
In one aspect, the present disclosure relates to certain ionizable lipid compounds and methods of preparation thereof. In another aspect, the present disclosure relates to lipid nanoparticles (LNPs) comprising certain ionizable lipid compounds of the present disclosure and methods of use thereof.
Resumen de: WO2025079599A1
The present invention addresses the problem of providing: a carrier that allows selective delivery thereof to a cell at a subset level, which cannot be realized by a conventional technique; and a method for producing said carrier. The present invention relates to: a carrier for realizing selective delivery thereof to a target cell having a plurality of target receptors on a surface thereof, wherein the carrier has individual ligands that respectively bind to the plurality of receptors, and is incorporated into target cells having all of the plurality of receptors, but not into cells that do not have at least one of the plurality of receptors; and a method for producing said carrier.
Nº publicación: WO2025079114A1 17/04/2025
Solicitante:
VYTALS WELLBEING INDIA PVT LTD [IN]
VYTALS WELLBEING INDIA PVT. LTD
Resumen de: WO2025079114A1
A self-nano emulsifying drug formulation for cholecalciferol by a method of spontaneous emulsification and spray drying, the self-nano 5 emulsifying drug formulation for cholecalciferol comprising of one active ingredient cholecalciferol, powder substrates, oil phases, surfactants and co-surfactants, wherein, the active ingredient having a loading capacity range from 100 IU/g to 10,00,000 IU/g and has an increased rate of dissolution, increased rate of permeability and increased bioavailability of the at least one active ingredient, wherein, the method for adsorption of the self-nano emulsifying drug on the substrate to obtain a powder formulation is spontaneous emulsification and spray drying method (SESDM) where the homogenous mixture of self-nano emulsifying drug and the aqueous dispersion of solid substrate is prepared and atomized into fine droplets and kept in a drying chamber for rapid evaporation of liquid to form a powder formulation.