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219
resultados
Última actualización
11/10/2025 [07:12:00]
Resultados 125 a 150 de 219
Resumen de: US2025302761A1
EVs are being recognized as vectors for drug delivery. In particular. EV loading with targeting and therapeutic agents brings along an interesting opportunity to translate EVs into a bio-mimetic selective delivery system. Indeed. EVs constitute a physiological carrier being potentially less immunogenic than artificial delivery vehicles. The inventors now developed a novel method to control the loading of a cargo into EVs on demand. These EVs are equipped, if necessary, with non-viral fusogen, therefore enhancing EV-cargo delivery into acceptor cells. To acutely measure this process, they follow the fate of a luciferase-tagged cargo. Cargo loading was enabled through a drug-reversible inducible dimerization system. Briefly, donor cells were transfected with plasmids encoding for FKBP-tagged CD63, a classical membrane EV marker, and FRB-Nanoluciferase (NLuc) that is normally cytosolic. Upon addition of the dimerizing drug. FRB-Nluc interacts with FKBP-CD63 and is recruited into secreted EVs. This is accompanied by an enhanced delivery into acceptor cells. This phenomenon can be further enhanced when EVs are equipped with syncitin1, a mammalian fusogenic protein that trigger fusion between EV membrane and the plasma membrane of acceptor cells. Using this novel process, the inventors further demonstrated that the catalytic domain of the Diphteria toxin (DTA), that is responsible for protein synthesis inhibition and ultimately cell death, can be delivered to acceptor cells via fun
Resumen de: US2025302768A1
Provided herein are compositions, kits, and methods of making biodegradable compositions for localized drug delivery. The drug delivery compositions include one or more therapeutic agents that are dispersed within polymerized macromers of the drug delivery composition, loaded within biopolymeric nanoparticles within the drug delivery composition, or both. The release profiles of the one or more therapeutic agents are tunable based on the one or more therapeutic agents for a desired application.
Resumen de: US2025302987A1
The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention.
Resumen de: WO2025207803A1
Described are compounds, compositions, and methods for delivery of therapeutic, diagnostic, or prophylactic agents (for example, a nucleic acid).
Resumen de: WO2025206323A1
The present disclosure provides a novel modality for a new target disease (e.g. pancreatic cancer). The present disclosure applies a mRNA CAR-T vaccine to CAR-T for a new target disease (e.g., pancreatic cancer). In the present disclosure, it has been revealed, by in vitro and in vivo experiments, that a mRNA CAR-T vaccine that targets FAP as an antigen can achieve a potent anti-tumor effect in in vivo editing. As for a mechanism for inhibiting the effect of a mRNA CAR-T vaccine, the pathways involving regulatory T cells and the like have also been revealed. As for a synergistic effect with an immune checkpoint agonist, a "condition" for achieving a potent anti-tumor effect in vivo has also been revealed through much trial and error.
Resumen de: WO2025207986A1
The present invention provides delivery-enhancing polymers capable of being encapsulated in nanoparticles to enhance release of payload from the nanoparticle wherein the delivery-enhancing polymer comprises a polyamine comprising tertiary amine. The delivery-enhancing polymer may comprise methylated polyethylenimine (mPEI), branched PEI (bPEI), PAMAM dendrimer and/or histidine polymer.
Resumen de: WO2025199580A1
The present invention relates to formulations, devices and methods for coating surfaces of microprojections on microprojection arrays with nucleic acids. The present invention also relates to formulations and methods for coating surfaces of microprojections with nucleic acids where the nucleic acids are associated with lipid nanoparticles (LNP), in particular where the nucleic acid is mRNA.
Resumen de: WO2025206461A1
The present invention relates to a novel lipid derivative compound comprising an oligo-gamma-glutamic acid, a lipid nanoparticle composition comprising the same, and the like. According to the present invention, the compound can form lipid nanoparticles by replacing PEGylated lipids, and thus can prevent side effects such as anaphylaxis and has excellent in vivo stability.
Resumen de: WO2025207807A1
The disclosure provides a nanoparticle comprising a positively-charged surface and an interior comprising (i) a core and (ii) at least two nucleic acid layers, wherein each nucleic acid layer is positioned between a cationic lipid bilayer, and nucleic acid molecules in the nucleic acid layers comprise a sequence of a nucleic acid molecule expressed by a circulating tumor cell (CTC). The disclosure further provides a method of treating cancer in a subject need thereof, the method comprising administering to the subject the nanoparticle described herein, optionally wherein two or more administrations of the nanoparticle are provided, wherein each administration comprises a nanoparticle comprising RNA comprising a sequence of RNA expressed in a CTC isolated from the subject at different points in time.
Resumen de: WO2025200185A1
An astragaloside nanoformulation for the treatment of hepatitis B and a preparation method therefor. The astragaloside nanoformulation for the treatment of hepatitis B comprises astragaloside and an auxiliary material. The auxiliary material comprises at least one of a polyoxyethylene-polyoxypropylene ether triblock copolymer, phospholipid, albumin, and casein. The compounding of these auxiliary materials and astragaloside can result in an astragaloside nanoformulation with good water solubility and high bioavailability, which exhibits higher safety and efficacy for the treatment of hepatitis B.
Resumen de: WO2025206286A1
The present invention addresses the problem of providing a composition for freeze-drying cells with which it is possible to suppress a decrease in survival rate or a decrease in particle size when the cells are reconstituted after freeze-drying. The composition for freeze-drying cells contains (a) a hydrophobic substance such as (a-1), (a-2), etc., and (b) nanoparticles formed from monolayers or bilayers of amphiphilic molecules, where: (a-1) is one or more hydrophobic amino acids selected from the group consisting of phenylalanine, leucine, glycine, valine, isoleucine, tryptophan, and alanine; and (a-2) is a dipeptide configured from one or two hydrophobic amino acids selected from the group consisting of phenylalanine, leucine, glycine, valine, isoleucine, tryptophan, and alanine. The composition is characterized by being used added to the cells.
Resumen de: WO2025207828A1
This disclosure is directed to payload bioactive agents (PBAs) that include modified exatecans. This disclosure is also directed to bioactive compositions and pharmaceutical compositions for treating cancer. The pharmaceutical compositions comprise a polymer, a targeting bioactive agent (TBA), a PBA comprising a modified exatecan that is covalently linked to the TBA directly or indirectly, a linker that can comprise a cleavable linker, and a pharmaceutical suitable carrier. The pharmaceutical compositions can be antibody-drug conjugates (ADCs) for treating cancers, with the potential for treating tumors having negative or low (AgLow) tumor antigens.
Resumen de: WO2025207378A1
Surface modification of nanoparticles (NPs) via the layer-by-layer (LbL) technique is a approach to generate targeted drug delivery vehicles. A simple and scalable synthesis method for LbL-NPs that can be adapted for clinical translation is of great interest. Presented herein is a robust and scalable method of polymer deposition onto nanoparticles.
Resumen de: WO2025200270A1
A cationic lipid compound, a preparation method therefor, a composition comprising same, and a use thereof, relating to the technical field of biomedicine. The cationic lipid compound has relatively high transfection efficiency and relatively low cytotoxicity by introducing at least one protonatable polar headgroup containing a secondary or tertiary amine and at least two hydrophobic tails containing a specified number of carbons, and connecting the polar headgroup and the hydrophobic tails by means of a specific linker chain.
Resumen de: WO2025207519A1
Embodiments of the present disclosure pertain to an active agent carrier that includes a disc-shaped membrane with a plurality of self-assembled amphiphilic block copolymers encased by membrane stabilizing agents, where the amphiphilic block copolymers include hydrophilic blocks and hydrophobic blocks, and where at least one active agent is associated with the disc-shaped membrane. Tunable numbers of targeting agents may also be associated with the disc-shaped membrane. Additional embodiments pertain to methods of delivering one or more active agents to a subject by administering to the subject an active agent carrier of the present disclosure. Further embodiments pertain to methods of making an active agent carrier of the present disclosure.
Resumen de: WO2025200113A1
Provided is a superoxide dismutase-based nanoscale transdermal delivery system, which is a capsule composite structure consisting of a small molecule active ingredient, superoxide dismutase, and a polymer coating. The small molecule active ingredient is loaded in the superoxide dismutase, and the surface of the superoxide dismutase is coated with the polymer coating. The superoxide dismutase-based active ingredient transdermal delivery system has a diameter of 20-100 nm, and the polymer coating has a thickness of 7.5-52.5 nm. In the delivery system, the polymer coating protects the superoxide dismutase and the small molecule functional substance having an antioxidant effect, and the capsule composite structure is in the form of nanogel. The nanogel not only promotes the penetration depth of the delivery system, but also mitigates the stimulation of the delivery system to the skin. The delivery system features biofriendly starting materials, simple preparation method, and high yield, and thus can be produced in large scale industrially.
Resumen de: WO2025200517A1
The present invention provides a lipid material for nucleic acid delivery, wherein the lipid material comprises a compound having structure I. The present invention also provides use of the lipid material for nucleic acid delivery in the preparation of a therapeutic drug for one or more selected from an infectious disease, a tumor disease, a congenital hereditary disease, and an immune disease. By means of the lipid material provided in the present invention and adopting a nucleic acid drug carrier strategy with high efficiency and low toxicity, a novel ionizable lipid and an auxiliary lipid material are mixed to encapsulate nucleic acid drugs, so that efficient and safe delivery of the nucleic acid drugs in vivo is achieved, and the druggability of the nucleic acid drugs is improved.
Resumen de: WO2025203087A1
The present disclosure discloses a recombinant construct including a vector and a recombinant nucleic acid molecule (1). The vector including at least one promoter region (13). The recombinant nucleic acid molecule (1) is encoded at least by SEQ ID No. 1. The recombinant nucleic acid molecule (1) is disposed downstream of the at least one promoter region (13) to enable transcription of the recombinant nucleic acid molecule (1) by the promoter region (13) to a plurality of messenger ribonucleic acid (mRNA) molecules encoded by SEQ ID No. 9.
Resumen de: MX2025010536A
The present invention provides novel ionizable lipids and novel lipid nanoparticles comprising messenger RNA (mRNA) useful for the delivery of nucleic acids, related pharmaceutical compositions or vaccines as defined herein for use in human or veterinary medicine, in particular for use in the treatment and/or prophylaxis of cancer diseases.
Resumen de: MX2025008776A
A composition for treating hyperprocalcitonemia is described. The composition comprises a lipophilic or hydrophobic component, an amphiphilic emulsifier, a polar liquid carrier, and with or without one or more electrolytes, where the amphiphilic emulsifier forms micelles having a lipophilic or hydrophobic core comprising the lipophilic or hydrophobic component in the polar liquid carrier, and /or liposomes organized as a lipid bilayer and/or other particle configurations.
Resumen de: CN120051455A
Novel sulfur-containing lipids and nanoparticles containing the lipids and cargo molecules, such as nucleic acids, methods of formulating the lipids with nucleic acids to produce lipid nanoparticles, and chemical pathways for preparing the lipids, are provided. The lipid may have a structure of Formula A as defined herein. # imgabs0 #
Resumen de: CN120239748A
The present invention encompasses systems, kits, and compositions comprising two RNA molecules wherein a first RNA molecule comprises an open reading frame encoding a functional RNA dependent RNA polymerase (replicase), and wherein a second RNA molecule is a replicable RNA molecule comprising at least one miRNA sequence that, when present in a cell, is capable of being cleaved from the second replicable RNA, and wherein the second RNA molecule is a replicable RNA molecule that comprises at least one miRNA sequence that, when present in the cell, is capable of being cleaved from the second replicable RNA. The first RNA molecule is capable of replication and is capable of modulating gene expression in a cell, and the replicable RNA molecule is capable of trans-replication by a replicase encoded by the first RNA molecule. The invention also encompasses methods of treating or preventing cancer or infections or other diseases and disorders with such systems and compositions, and the use of such systems and compositions in such methods of treatment and prevention.
Resumen de: WO2024054855A1
The present disclosure relates to a combination therapy comprising an anti-VEGF antibody, a nanoparticle formulated plasmid comprising an IL-12 coding nucleic acid, and, optionally, at least one adjunctive chemotherapeutic drug, and methods of treatment using such combination therapies and/or compositions.
Resumen de: AU2023385865A1
Disclosed herein are dense nanolipid fluid (DNLF) dispersions comprising desirable characteristics for incorporating bioactive agents such as peptides into lipid phase of the dispersion for biodelivery of the agents for their typical purpose. Continuous methods for preparing the DNLF dispersions are also disclosed herein to include formation of a crude mill base and passing the base through a twin screw extruder. Dispersions disclosed herein can express a particle size of less than 150 nm under stable storage conditions, while forming lamellar structures after exposure to heat and/or evaporation of the aqueous components of the dispersion.
Nº publicación: EP4622651A1 01/10/2025
Solicitante:
UNIV CALIFORNIA [US]
UNIV AALBORG [DK]
The Regents of the University of California,
Aalborg Universitet
Resumen de: CN120359038A
The present disclosure provides inhibitory nucleic acids, compositions comprising the inhibitory nucleic acids, and methods of using the inhibitory nucleic acids to treat various diseases.
BOPI
Sede Electrónica
