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: WO2025080209A1
There is provided a compound represented by general formula (1) for preparing lipid nanoparticles encapsulating a therapeutic, prophylactic and/or biological agent: wherein AR comprises a unit from a poly(amino acid); R1 and R2 are each independently a hydrophobic group; R3, R4, and R5 are each independently H, optionally substituted alkyl, optionally substituted alkenyl or optionally substituted alkynyl; R7 is -H or -C(=O)R8, wherein R8 is optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl or optionally substituted alkoxy; m ≥ 1; and n ≥ 1.
Resumen de: WO2025076771A1
The present invention belongs to the technical field of biology. Disclosed are an ionizable lipid, a pharmaceutical composition comprising same, and the use thereof. Provided in the present invention is a compound as represented by formula (I), or a stereoisomer, tautomer, solvate, pharmaceutically acceptable salt or deuterated compound thereof, which serves as an ionizable lipid, overcoming the technical defect whereby other ionizable lipid compounds in the prior art are all mainly delivered to the liver for the expression of antigen proteins and cannot be delivered to the spleen. The compound represented by formula (I) provided by the present invention has the characteristics of good spleen-organ-targeting property, high delivery efficiency, etc. The compound represented by formula (I) provided by the present invention can be used for the preparation of liposomes, lipid nanoparticles, drug carriers or complexes, and used for the delivery of nucleic acid drugs.
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: 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: WO2025077836A1
Provided herein is novel ionizable cationic lipid compound that can be assembled with other helper lipids, such as phospholipids, structural lipids, and polymer conjugated lipids capable of reducing aggregation, to form lipid nanoparticles for delivery of therapeutic RNA both in vitro and in vivo. These compounds contain a thiourea group in the linker between the lipid group and the head group (-NRc-C(S)-NRd-).
Resumen de: WO2025080945A1
The present disclosure relates to lipid nanoparticles and methods of delivering active agents to target organs, tissues, or cells by utilizing the lipid nanoparticles.
Resumen de: WO2025081002A1
Novel ionizable lipids, compositions, and methods of using the novel lipids and compositions are disclosed. Lipid nanoparticle compositions include novel lipids as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticle compositions further including biologically active agents such as mRNA are useful in the delivery of therapeutics, diagnostics and/or prophylactics to cells or organs.
Resumen de: WO2025080572A1
A compound having the structure (I), (II), (III), or (IV) is provided: (I), (II), (III), (IV) ) where in these structures, (I), (II), (III), and (IV), each R1 is independently selected from aliphatic alkyl C4-C100 groups optionally substituted with one or more of alkenyl, alkynyl, hydroxyl, amide, ester, and/or ether groups; R2 is selected from -OCH2CH2-p or (A); R3 is selected from Formula (V) and Formula (VI): (V, (VI). M+ is selected from an alkali metal ion, an alkaline earth metal ion, or a primary, secondary or tertiary ammonium ion; and m, p, and s are independently selected from integers from 1 to 120. The compound is useful as a liver asialoglycoprotein receptor-targeted therapeutic agent in the form of a lipid nanoparticle, a liposome or a micelle including a drug or oligonucleotide.
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: 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: WO2023237842A1
The present invention relates to optimised microbubbles, in particular lipid microbubbles, which can be used in the prevention and treatment of diseases or else labelling. The invention relates in particular to a lipid microbubble comprising at least one cationic compound chosen from lipophosphoramidates, histidyl polyethyleneimines, and any mixture thereof. The microbubble preferably also comprises at least one agent chosen from a therapeutic agent, a targeting agent, a labelling agent, and any combination thereof. The invention also relates to a method for producing this microbubble. The present invention also relates to a composition, in particular a pharmaceutical composition, and a kit, comprising at least one of these microbubbles. The present invention also relates to the use of these microbubbles, composition, kit, as a medicament or as a labelling agent.
Resumen de: WO2023198857A1
The present invention provides, in part, second generation "good" buffer-based cationic lipids of Formula (I), and sub-formulas thereof: Formula (I), (I), or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
Resumen de: WO2023238060A1
The present patent application describes an injectable nanocomposite based on a thermo-responsive hydrogel for marking anatomical sites for surgical purposes.
Resumen de: EP4537852A1
Provided are a fusion protein including a glutathione-S-transferase and a protein having binding ability to a target cell or a target protein, and a drug complex thereof, and use thereof as a pharmaceutical composition. The fusion protein according to an aspect and the drug complex including the same can sustain a prolonged residence time in vivo, and can be effectively delivered to target cells due to an improved ability to target the target cells, and thus can be effectively used as a targeted therapeutic agent.
Resumen de: WO2023240261A1
The disclosure provides nucleic acid-containing lipid nanoparticle (LNP) compositions and methods relating to the delivery of TnpB nucleobase editing systems comprising TnpB polypeptides, engineered TnpB ncRNAs, and optionally one or more additional accessory functionalities (e.g., a deaminase, reverse transcriptase, recombinase, nuclease, a donor template, or combinations thereof) for use in applications such as precision gene editing.
Resumen de: AU2023285094A1
Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (
Resumen de: EP4537855A1
The present invention provides a nanoparticle comprising a scaffold comprising a biodegradable material, an antibody targeting a component of a Bruch's membrane, and an agent for use in a prophylaxis or in a treatment of a pathological change of Bruch's membrane and/or an adjacent tissue, including a retinal pigment epithelium and a choroid of an eye. Additionally, the present invention provides a pharmaceutical composition comprising said nanoparticle and pharmaceutical acceptable excipients for use in a prophylaxis or in a treatment of a pathological change of Bruch's membrane and/or adjacent tissues, including a retinal pigment epithelium and a choroid of an eye. Preferably said agent is an anti-calcifying agent and said pathological change is a calcification of Bruch's membrane and/or the adjacent tissue, including the retinal pigment epithelium and the choroid of the eye.
Resumen de: WO2023240213A2
A cerebrovascular unit tumor (CVU-T)-on-a-chip model that models both the blood brain barrier (BBB) and tumor is described. The model provided a physiologically relevant GBM tumor model including a corresponding microenvironment complete with a BBB component that is crucial in dictating drug bioavailability to the tumor. Additionally a novel biodegradable microcapsule for sustained local delivery of a therapeutic such as a connexin hemichannel inhibitor is provided. The therapeutic sensitizes malignant GBM populations to systemic TMZ chemotherapy.
Nº publicación: EP4536201A1 16/04/2025
Solicitante:
UNIV COLUMBIA [US]
The Trustees Of Columbia University In The City Of New York
Resumen de: US2025090489A1
PAMAM-based nanocarriers with high loading efficiency of chemotherapeutics and high cfNA binding ability enable sustained delivery of chemotherapeutics while inhibiting systemic inflammation caused by the chemotherapy. This is useful for treating both primary and metastatic tumors with attenuated cognitive impairment.