Resumen de: WO2025211353A1
Provided is a sheet exhibiting high drug delivery efficiency to the posterior part of the eye and excellent retentivity, which could not be achieved with a conventional method. The present invention provides a sheet containing nanofibers including a water-soluble polymer (A), wherein the water-soluble polymer (A) contains at least one selected from the group consisting of a polyvinyl alcohol-based resin, a hydroxyalkyl cellulose, and a polyethylene oxide; and the sheet comes into contact with at least one selected from the group consisting of the cornea, conjunctiva, and sclera, and maintains 50% or more of the mass after being immersed in phosphate-buffered saline at 37°C for 1 minute.
Resumen de: WO2025210606A1
A treatment for patients with GRIN disorder comprises overexpressing a GRIN1 gene without disrupting the genome and regardless of the type of mutation.
Resumen de: WO2025209612A1
The present invention relates to the modification of the surface of a silk-screen-printed carbon electrode by means of zinc oxide nanoparticles in the presence of carboxymethyl cellulose (ZnO-2), obtained using a new synthesis method. The ZnO-2 compound is selective and sensitive for the detection of hydrogen peroxide, and allows the adsorption of biomolecules, as well as electrochemical measurement in biosensors for the quantification of different analytes of diagnostic interest.
Resumen de: WO2025209535A1
Provided herein are compositions and associated methods for CH-based nano-carriers for therapeutic agents and their applications, a multi-step method for the synthesis of the pharmacologically stable and multifunctional CH-based nano-carriers, effectively carrying therapeutic or theranostic radioactive metal isotope, anionic immunotherapeutic agent, or other anionic therapeutic agents used as therapeutic drugs, including the one of "combined radio-and adjuvant immuno-therapy" for cancer treatment.
Resumen de: WO2025209581A1
Disclosed are a nitrogen-containing chain compound, a preparation method, a composition comprising the compound, and a use. Specifically, disclosed is a compound I or a pharmaceutically acceptable salt thereof. An LNP formulation prepared from the nitrogen-containing chain compound has relatively uniform nanoparticle size, high encapsulation efficiency, and high in-vivo expression activity.
Resumen de: WO2025209444A1
The present invention provides a compound represented by formula (I) or a pharmaceutically acceptable salt, isomer, isotope compound or solvate of the compound represented by formula (I). The present invention also provides a lipid nanoparticle comprising the compound represented by formula I or the pharmaceutically acceptable salt, isomer, isotope compound or solvate of the compound represented by formula I. The lipid nanoparticle can be used for nucleic acid delivery and has high organ targeting efficiency.
Resumen de: WO2025208337A1
Disclosed is a biomimetic nano-delivery system, comprising an inner core formed from a black phosphorus nanosheet and a drug, and a shell formed from an M1-type macrophage membrane. The biomimetic nano-delivery system has an anti-tumor effect, has a good photothermal effect under the irradiation of a near-infrared laser, and can actively recognize tumor cells and release the drug precisely and efficiently.
Resumen de: US2025312287A1
The present invention relates to compositions for in vitro and in vivo delivery of nucleic acids, in particular messenger RNAs (mRNAs), into a target cell and their applications. The present invention is directed to a composition comprising (A) at least one nucleic acid and (B) at least one lipid nanoparticle (LNP) comprising (i) at least one ionizable lipid; (ii) at least one phospholipid; (iii) at least one sterol, especially neutral sterol; (iv) at least one poly(ethyleneglycol)-lipid (PEG-lipid); and (v) an imidazolium-based cationic lipid of formula (I), wherein R1, R2, R3, R4, R5, R6, R7, R8 and R9, Y and A− are as defined in the description. The present invention also relates to a method for in vitro or in vivo transfection of live cells and uses of said composition.
Resumen de: US2025312411A1
Pharmaceutical compositions and methods provide treatment of solid tumors based on simultaneously preventing the development and spread of multi-drug resistance (MDR) and a metastatic phenotype. This technology operates through the inhibition of two types of intercellular communication within the tumor microenvironment—tunneling nanotubes and extracellular vesicles, both of which promote the spread of MDR. Nanoparticle delivery of both an inhibitor of tunnelling nanotubes and an inhibitor of extracellular vesicle release work synergistically to trap and improve the effectiveness of anticancer drugs in cells of the tumor and permit the use of lower doses of anticancer drugs, with reduction in harmful side effects.
Resumen de: US2025312289A1
This application relates in part to nanoparticles comprising a tobamovirus and nanoparticles comprising a tobamovirus and beta-cyclodextrin (β-CD or BCD). This application also relates in part to nanoparticles comprising tobamovirus and one or more active ingredients (AIs) that are non-covalently conjugated to the tobamovirus. The application also provides methods of making and methods of using such nanoparticles as well as compositions comprising the disclosed nanoparticles.
Resumen de: US2025312288A1
The present disclosure relates generally to lipids, lipid nanoparticle formulations, and methods of using the same for delivering nucleic acids, such as mRNA.
Resumen de: US2025313525A1
The present disclosure details various lipids, compositions, and/or methods of optimized systems and delivery vehicles for the delivery of nucleic acid sequences, polypeptides or peptides for use in vaccinating against infectious agents.
Resumen de: US2025313531A1
Formulated and/or co-formulated liposomes comprising IDO prodrugs and methods of making the liposomes are disclosed herein. The IDO prodrug compositions comprise a drug moiety, a lipid moiety, and linkage unit that inhibit IDO-1. The IDO prodrugs can be formulated and/or co-formulated into a liposome to provide a method of treating cancer, immunological disorders, and other disease by utilizing a targeted drug delivery vehicle.
Resumen de: US2025313835A1
The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
Resumen de: US2025313675A1
Described herein are flash nanoprecipitation methods capable of encapsulating hydrophobic molecules, hydrophilic molecules, bioactive protein therapeutics, or other target molecules in amphiphilic copolymer nanocarriers.
Resumen de: US2025312498A1
Advantage is taken of the enhanced permeability and retention effect (EPR effect) to shield normal tissue from exposure to combinations of chemotherapeutic agents. Imaging agents that exhibit the enhanced permeability and retention (EPR) effect in solid tumors are useful in mimicking the behavior of chemotherapeutic or other drugs for treatment of said tumor conjugated to carriers of similar size and shape to the carriers of said imaging agents.
Resumen de: US2025312485A1
Compositions and methods for effecting base editing to correct mutations in the phenylalanine hydroxylase gene, thereby curing phenylketonuria, are disclosed.
Resumen de: US2025312482A1
Described herein are lipid nanoparticle (LNP) formulations with demonstrated tropism towards smooth muscle cells. Also described herein are LNPs conjugated with peptides that can target tissue or cell surface receptors. The formulations of the disclosure include amounts of DOTAP, an ionizable lipid, amounts of a neutral lipid; amounts of cholesterol; and amounts of one or more PEG-lipids with preferential tropism towards vascular smooth muscle cells (vSMCs). Also described herein are peptides that target receptors highly expressed on the surface of vSMCs (IL-6R, CD63 and GAL-3) or that target proteins in the extracellular matrix adjacent to vSMCs (Col-IV) increasing the uptake into these cells.
Resumen de: US2025312481A1
A genetic delivery nanoparticle includes gelatin, a lipidoid, and a genetic molecule payload. The genetic molecule payload can be an RNA, DNA or Crispr system payload. SiRNA is an example payload and can be encapsulated with the gelatin and lipidoid and covalently conjugated to a surface of the nanoparticle. The nanoparticle surface can also include an antibody and PEG conjugated to it. A method for forming genetic delivery nanoparticle includes forming an adduct of gelatin, the lipidoid and a genetic payload; and cross-linking the gelatin to form the genetic delivery nanoparticle. Varying the size of a carbon chain in the lipidoid controls the size of the formed genetic delivery nanoparticle.
Resumen de: US2025312455A1
The present invention relates to plasmonic metal-collagen hybrid nanoparticles that have excellent protein loading efficiency and excellent biocompatibility and can be utilized as protein delivery vehicles. In the present invention, a plasmonic metal-collagen hybrid nanoparticle for protein delivery, comprising a collagen of 1 kDa to 30 kDa, a thermosensitive polymer monomer, a plasmonic metal nanoparticle, and a photoinitiator, and a method for preparing the same are provided.
Resumen de: US2025312430A1
Lipid nanoparticle formulations with cell type specific transfection activity and capable of producing Th1 and/or Th2 response in vivo and their use for plasmid DNA or mRNA delivery is disclosed.
Resumen de: US2025312442A1
The disclosure relates to EBV ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.
Resumen de: US2025313857A1
Provided herein are compositions and methods for gene therapy for disorders of arterial calcification as well as Generalized Arterial Calcification of Infancy (GACI). The methods include a gene addition strategy to deliver a DNA construct to target tissues (such as liver and smooth muscle cells) to express soluble recombinant ENPP1 (srENPP1) or transmembrane full-length recombinant ENPP1 (rENPP1).
Resumen de: US2025313830A1
The disclosure features a polynucleotide encoding a polypeptide, which polynucleotide comprises a 5′ UTR, a coding region encoding a polypeptide, and a 3 UTR, and lipid nanoparticles comprising the same. The polynucleotides and/or lipid nanoparticles of the present disclosure can increase the level and/or activity of the polypeptide by increasing the half-life and/or duration of expression of the polynucleotide encoding the polypeptide. Also disclosed herein are methods of treating a disease or disorder in a subject using the lipid nanoparticles of the present disclosure.
Nº publicación: US2025312483A1 09/10/2025
Solicitante:
FUNDACION INSTITUTO DE INVESTIG MARQUES DE VALDECILLA [ES]
FUNDACI\u00D3N INSTITUTO DE INVESTIGACI\u00D3N MARQU\u00C9S DE VALDECILLA
Resumen de: US2025312483A1
The present invention relates to silica particles comprising nucleic acids encapsulated inside the same. Furthermore, the present invention relates to a method for producing said particles and the uses thereof in gene transfer or cell marking and as a medicinal product, specifically as a medicinal product for protein/enzyme replacement therapy.