Resumen de: US2025186635A1
The present disclosure relates to a plurality of globular nanostructures. Each nanostructure comprises a central part comprising a polymer framework of monomer residues according to {(OR1)(OR2)PO}2—(C){(CH2)mSi(OR3)3}{(CH2)mSi(OR3)3}, wherein each R1 and R2 is independently selected from the group consisting of a negative charge and H; each R3 is independently selected from the group consisting of a negative charge, H and a covalent bond to the polymeric framework; wherein at least 3 R3 are bonds to the polymeric framework; and m is an integer between 1 and 5; and, wherein the central parts of the nanostructures have a volume average hydrodynamic diameter of 10 nm to 90 nm; and an anchoring layer surrounding the central part, wherein the anchoring layer comprises a polymer of monomer residues according to (RO)3Si(CH2)nSi(OR)3, wherein each R is independently selected from the group consisting of a negative charge, H and a covalent bond, wherein at least two R are independently selected from the group consisting of a covalent bond to a monomer residue of the central part and a covalent bond to a monomer residue of the anchoring layer; wherein represents a covalent bond; n is 1 or 2; and wherein the anchoring layer has a thickness of 1-5 nm. The present disclosure also relates to a method for producing such nanostructures as well as the use of the nanostructures and to pharmaceutical compositions comprising such nanostructures.
Resumen de: US2025188025A1
A nanosized complex includes a nucleic acid and a compound comprising formula (I):wherein R1 is an alkylamino group or a group containing at least one aromatic group;R2 and R3 are independently an aliphatic group or hydrophobic group;R4 and R5 are independently H, a substituted or unsubstituted alkyl group, an alkenyl group, an acyl group, or an aromatic group, or a polymer, a targeting group, a detectable moiety, or a linker, or a combination thereof, and at least one of R4 and R5 includes a retinoid or retinoid derivative that targets and/or binds to an interphotoreceptor retinoid binding protein;a, b, c, and d are independently an integer from 1 to 10; and pharmaceutically acceptable salts thereof.
Resumen de: US2025188016A1
Compounds are provided having the following Formula (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3a, R3b, R3c, R3d, L1, L2, and n are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Resumen de: US2025186431A1
Provided is a pharmaceutical composition for use in radiotherapy, comprising: a compound represented by formula (I) wherein R1 is an aryl group substituted with a substituent selected from an iodine atom and others, wherein the aryl group is optionally substituted with a substituent such as a hydroxy group, X represents a bond or the like and ring A is a group represented by formula (A), wherein R2 is a non-aromatic heterocyclic group optionally substituted with at least one C1-C4 alkyl group, or a pharmaceutically acceptable salt thereof; and a pharmaceutically acceptable carrier. Also, provided is a nanoparticle and a method for treating solid cancer using the compound.
Resumen de: US2025188140A1
The present disclosure relates to a lipid-like nanoparticle encapsulating a C—C motif chemokine ligand 5 (CCL5) mRNA and methods of use thereof.
Resumen de: US2025188191A1
The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses a known marker can be targeted for a therapeutic objective.
Resumen de: US2025188132A1
Aspects of the disclosure relate to nucleic acid vaccines. The vaccines include at least one RNA polynucleotides having an open reading frame encoding at least one varicella zoster virus (VZV) antigen. Methods for preparing and using such vaccines are also described.
Resumen de: US2025186358A1
The present disclosure provides bispecific stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., hematopoietic stem cells, to modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Resumen de: US2025186360A1
The present invention provides a composition comprising liquid-liquid phase separation (LLPS)-based compartments comprising a cationic peptide and an anionic polymer, variations and modifications thereof, methods for preparation of the LLPS-based compartments, and uses thereof for delivery or for controlling entrapping and releasing of a payload.
Resumen de: US2025186343A1
An emulsion comprises a first discontinuous phase comprising first droplets, a second discontinuous phase comprising second droplets, and a continuous aqueous phase. The first droplets include at least one first surfactant and at least one first fluorocarbon, and have a first diameter of more than 100 nm. The second droplets include at least one surfactant, at least one drug, and at least one solvent, and have a second diameter of no more than 100 nm.
Resumen de: US2025186390A1
Compositions are provided that include having at least 95% by weight of a taxane, or a pharmaceutically acceptable salt thereof, where the particles have a mean bulk density between about 0.050 g/cm3 and about 0.15 g/cm3, and/or a specific surface area (SSA) of at least 18 m2/g, 20 m2m/g, 25 m2/g, 30 m2/g, 32 m2/g, 34 m2/g, or 35 m2/g. Methods for making and using such compositions are also provided.
Resumen de: US2025186609A1
The present disclosure provides methods and compositions for modulating the activity of self-associated molecular pattern recognition receptors such as for example, Siglec (sialic-acid-binding immunoglobulin-type lectins) and complement factor H (CFH). Modulating the activity of infectious organisms such as viral influenza A, B, C, SARS-CoV1, 2, and cancer/tumor cells such as lung, breast and skin cancers. The compositions comprise a particle, comprising a molecule represented by the following structural formula:P-L-G,wherein P is a biocompatible polymer scaffold comprising at least one biocompatible polymer defined herein, G is a polysialic acid (PSA) comprising from 5 to 200 repeat units of sialic acid; and L is a covalent linker, or a pharmaceutically acceptable salt thereof.
Resumen de: US2025186608A1
Detonation nanodiamond nanocarrier platforms to transport quaternary oxime antidotes into the central nervous system have been developed. The nanodiamond-based AChE reactivators contain an organophosphorus poisoning antidote (e.g., a 4-hydroximinopyridinium moiety) bound to a biocompatible linker covalently attached to the nanodiamonds. These functionalized nanodiamonds successfully cross the layer of Madin-Darby Canine Kidney (MDCK) cells, the epithelial cell surrogate BBB model, and demonstrate a measurable dose-independent in vitro reactivation capacity towards human AChE inhibited by toxic organophosphorus compounds.
Resumen de: US2025186607A1
A method for functionalizing plant viral nanoparticles (VNPs) includes selecting a plant VNP. Additionally, a metal ion and a phenolic compound that form a metal-phenolic network (MPN), and at least one functional component that adheres to the MPN are also selected. A nanohybrid structure is synthesized from a solution of the selected metal, the selected phenolic compound and the selected functional component such that the synthesized nanohybrid structure has an MPN coating encapsulating the plant VNP with the functional component being embedded in the MPN coating.
Resumen de: US2025186524A1
A method of preparing a carbon quantum dots (CD) and Moringa oleifera silver nanoparticle (CD-MOE-AgNP) composition includes providing a Moringa oleifera extract, combining the Moringa oleifera extract with silver nitrate to provide silver nanoparticles, and combining the silver nanoparticles with the carbon quantum dots (CD) to provide the CD-MOE-AgNP composition. In an embodiment, combining the carbon quantum dots (CD) with the Moringa oleifera silver nanoparticle (CD-MOE-AgNP) composition can include ultra-sonification of the carbon quantum dots (CD) with the Moringa oleifera silver nanoparticles.
Resumen de: US2025188013A1
The present application provides a novel lipid compound, a lipid nanoparticle comprising the same, and use thereof in targeted delivery of a drug. Specifically, the present application provides a compound of Formula Ior a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof. Furthermore, the present application provides a lipid nanoparticle comprising the compound of Formula I or a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof; still further, the lipid nanoparticle may further comprise a cationic lipid; yet still further, the lipid nanoparticle may further comprise a helper lipid. A composition comprising the above compound or a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof, or the lipid nanoparticle can achieve the organ-targeted delivery of a therapeutic/prophylactic agent.
Resumen de: WO2024028492A1
The present invention relates to methods of determining the efficiency of RNA encapsulation in lipid nanoparticles (LNPs). In some embodiments, methods according to the present invention comprise a step a) of contacting a sample comprising RNA encapsulated in LNPs with a first fluorophore and a second fluorophore, thereby forming fluorophore-RNA complexes, and a step b) of detecting the fluorescence signals of the complexed first and second fluorophore, wherein the first fluorophore permeates the LNPs and wherein the second fluorophore does not permeate the LNPs.
Resumen de: AU2023317842A1
The present invention relates to compositions that can deliver therapeutic molecules such as nucleic acids to mammalian cells, and to the human and animal body and methods of preparing and using the same. The compositions comprise nanoparticles comprising a peptide dendrimer, a nucleic acid and a lipid. The compositions of the invention find utility in the field of medicine, such as for treating cancer and autoimmune diseases.
Resumen de: CN119654167A
The present disclosure relates generally to the field of nucleic acid (e.g., DNA or RNA, particularly mRNA) compositions comprising amphiphilic oligomeric ethylene glycol (OEG) conjugated compounds (as substitutes for PEG lipids); uses of such compositions, in particular for delivering nucleic acids to cells of a subject or for therapy; to an amphiphilic OEG conjugated compound of this type; and conjugates of such amphiphilic OEG conjugated compounds.
Resumen de: EP4566672A2
Methods and devices are provided for targeted non-surgical administration of a drug formulation to the suprachoroidal space (SCS) of the eye of a human subject for the treatment of a posterior ocular disorder or a choroidal malady. In one embodiment, the method comprises inserting a hollow microneedle into the eye at an insertion site and infusing a drug formulation through the inserted microneedle and into the suprachoroidal space of the eye, wherein the infused drug formulation flows within the suprachoroidal space away from the insertion site during the infusion. In one embodiment, the fluid drug formulation comprises drug nanoparticles or microparticles.
Resumen de: GB2636076A
A lipid nanoparticle (liposome) comprising: (i) an outer shell comprising a lipid formulation; and (ii) one or more therapeutic agents which prevent apoptosis of a target cell is provided. The therapeutic agent may comprise one or more caspase inhibitors, such as Emricasan, or may comprise one or more siRNA which prevents or reduces the expression of a protein which induces or enhances apoptosis, such as NR3C1 or ADRB2. The lipid formulation may comprise hydrogenated soy phosphatidylcholine (HSPC), cholesterol (Chol) and 2-distearoyl-sn-glycero-3-phosphoethanolamine-N-methoxy(polyethylene glycol)-2000 (DSPE-PEG2000). The outer shell may further comprise 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N-diethylenetriaminepentaaceticacid (PE-DTPA). The lipid nanoparticle may further comprise a spleen-targeting moiety. The lipid nanoparticle (liposome) for use in treating or preventing a disease or disorder associated with spleen abnormality is provided. The disease or disorder is preferably a post post-brain injury infection, such as a stroke-associated infection. A method of treating a stroke-associated infection in a subjection by administrating the lipid nanoparticle is provided.
Resumen de: EP4566594A1
A long-chain ceramide such as an exogenous C18-ceramide has very low solubility in an aqueous solution and thus has limited use, and when even used as a drug, has had poor cell delivery. In this regard, the prevent invention provides lipid nanoparticles comprising a long-chain ceramide and a preparation method therefor. Through the present invention, by delivering long-chain ceramides such as C18-ceramides to an in vivo target through lipid nanoparticles, cell death may be effectively induced according to the purpose, and utilization as a cell therapy product with high water solubility and high water dispersibility may be increased.
Resumen de: EP4566606A1
A specific silicon compound is represented by the following formula:wherein each symbol is as defined in the specification. A composition comprises the silicon compound and cetylpyridinium chloride hydrate and is in the form of nanoparticles. The composition can be easily attached to or carried by a medical material or a medical device in a favorable manner.
Resumen de: WO2024031051A1
Provided herein are ionizable lipids, compositions comprising the ionizable lipids, and methods of making and using the same. The ionizable lipids provided herein can be formulated in lipid compositions for the delivery of macromolecules, such as nucleic acids, in vitro, ex vivo, or in vivo.
Nº publicación: EP4566618A1 11/06/2025
Solicitante:
ADVANTAGE THERAPEUTICS INC [US]
ADvantage Therapeutics, Inc
Resumen de: EP4566618A1
The present disclosure relates to therapeutic and non-therapeutic methods for obtaining or promoting weight loss in a mammal, especially in a human individual, comprising supplementing a cell of the mammal with a Klotho protein by administering to the cell a nucleic acid encoding the Klotho protein such that the nucleic acid is expressed within the cell to produce the Klotho protein so as to obtain weight loss in said mammal.