Resumen de: US20260014259A1
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
Resumen de: US20260014281A1
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
Resumen de: US20260014274A1
The present invention relates to compositions and methods for delivering nucleic acids into cells.
Resumen de: WO2026013059A1
The present invention provides lipid nanoparticles and formulations comprising cationic lipids and helper phospholipid. These lipid nanoparticles may be formulated with therapeutic agents to facilitate their intracellular delivery for both in vitro and in vivo therapeutic applications. The present invention is specifically directed to nanoparticles and formulations that can target inflamed tissues and treat inflammatory diseases.
Resumen de: WO2026013635A1
The disclosure refers to a composition comprising poly(3-hexylthiophene) nanoparticles suspended in a saline solution comprising a stabilizing agent selected from a non-ionic surfactant, a water-soluble polymer having high intrinsic viscosity and a combination thereof. The preferred water-soluble polymer is hyaluronic acid. The saline solution is an aqueous solution comprising at least sodium ions and chloride ions. The saline solution may further comprise at least one of: magnesium ions, calcium ions, potassium ions, and combination thereof. The formulation has demonstrated an improved effectiveness in restoring a visual response in the RCS rat model of blindness and is potentially effective in the treatment of human retinal dystrophies such as retinitis pigmentosa (RP) and macular degeneration (AMD).
Resumen de: WO2026013203A1
The present invention relates to lipid nanoparticles (LNPs) comprising vitamin A or a vitamin A derivative. The LNPs according to the present invention show an improved stability and nucleic acid delivery efficacy.
Resumen de: US20260014197A1
A drug delivery system including a zeolitic imidazolate framework-8 (ZIF-8), silica, platinum nanoparticles, and polyethylene glycol. The silica penetrates the pores of the ZIF-8 and at least partially envelopes the ZIF-8 to form a ZIF-8/silica composite. The platinum nanoparticles are present on the surface of the ZIF-8/silica composite, and the polyethylene glycol surrounds the platinum nanoparticles present on the surface of the ZIF-8/silica composite.
Resumen de: US20260014229A1
Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2; or any combination thereof that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, an engineered protein selected from the group consisting of OCT4; KLF4; SOX2; or any combination thereof, an antibody capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, and methods of treating a (e.g., ocular disease), preventing a disease (e.g., ocular disease), regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, regulating tissue repair, regulating tissue regeneration, or any combination thereof).
Resumen de: US20260014090A1
Nanoparticles and formulations for non-surgical sterilization are disclosed herein. The nanoparticles for non-surgical sterilization contains a cage, such as a zeolitic imidazolate framework (“ZIF”), a surface modifying agent, a targeting ligand, and an active agent. The surface modifying agent is attached to the outer surface of the cage and the targeting ligand is exposed to the surrounding environment. The active agent is encapsulated in the cage. The targeting ligand binds to a reproductive hormone or a receptor of a reproductive hormone. The active agents can be a ribosome inactivating protein, an apoptosis inducer, a hormone, a receptor ligand, or a nucleic acid, or a combination thereof, that inactivates the ovaries or testes. Uses for the nanoparticles and formulations incorporating the nanoparticles for sterilizing a subject in need thereof are also disclosed.
Resumen de: US20260014186A1
The invention relates to polymer nanoparticle and DNA nanostructure delivery compositions for non-viral delivery, and methods therefor. More particularly, the invention relates to polymer nanoparticle delivery compositions, such as reversible addition-fragmentation chain transfer (RAFT) polymer compositions, and DNA nanostructure delivery compositions, such as DNA origami compositions, for the delivery of more than one payload, or for the delivery of a nucleic acid construct payload of 3 kB or more, and methods therefor.
Resumen de: US20260014258A1
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
Resumen de: CN120787151A
The present invention relates to a composition comprising nucleic acid-lipid particles, wherein the nucleic acid-lipid particles are characterized by encapsulating a nucleic acid in a lipid bilayer. The invention also relates to a composition for the prevention and/or treatment of a disease, in particular for the prevention and/or treatment of cancer. The invention also relates to a method for preparing a composition comprising the nucleic acid-lipid particles.
Resumen de: US20260014093A1
Embodiments of the present disclosure provide novel compositions and methods for making and using thermostable bacteriophage or bacteriophage-derived phage-like-particle (PLP)-containing formulations. In certain embodiments, compositions and methods are disclosed for embedding, decorating and/or associating at least one antigen or agent, or bioactive molecule on the surface of the bacteriophage or PLPs. In accordance with these embodiments, bacteriophage or PLPs harboring one or more antigen or agent, or bioactive molecule can further be thermostabilized and/or coated with one or more atomic layer deposition applied coating layer for control or timed release of the one or more antigen or agent when administered to a subject.
Resumen de: US20260014187A1
A composition includes a plurality of biocompatible metal nanospheres. the biocompatible nanospheres each having an outer surface with elemental carbon connected thereto. The composition also includes a plurality of single nucleic acids strands, wherein at least a portion of the respective individual single nucleic acid stands are in coordinated connection with the nanospheres, and a carrier medium.
Resumen de: US20260014094A1
A method is provided for producing a composition that includes a nanoparticle with at least one nucleic acid and at least one ionizable lipid. The method includes introducing a first composition that includes the at least one nucleic acid and a second composition that includes the at least one ionizable lipid into at least one reactor. The first composition and the second composition are introduced at a first flow rate and a second flow rate, respectively, therefore generating the nanoparticle in a reaction mixture. The method further includes filtering the nanoparticle from the reaction mixture via a single-pass tangential flow filter at a feed flux to provide a retentate, to produce the composition. The method may also include filtering the retentate through a sterile filtration membrane.
Resumen de: US20260014092A1
Provided are vaccines and immunomodulatory compositions containing extracellular vesicles from microalgae (MEVs) that are loaded with bioactive cargo, that includes antigens and/or immunomodulatory proteins, nucleic acids, and nucleic acid encoding the proteins. The MEVs are formulated and administered by a variety of routes of administration that are advantageous for modulating the immune systems. Vaccines include those that are therapeutic for treating a disease, disorder, or condition, those that elicit an immunoprotective response, and/or otherwise modulate the immune system. The compositions include MEVs containing cargos for modulating intracellular receptors.
Resumen de: US20260014175A1
A pharmaceutical composition in the form of a compressed tablet for oral administration. The composition has an intragranular portion including a first porous solid carrier with a physiologically acceptable salt that is soluble in gastric fluid/an acidic aqueous media, and a self-nanoemulsifying drug delivery system (SNEDDS) adsorbed on the carrier. The SNEDDS includes: at least one cannabinoid; one or more solubilizing agents selected from physiologically acceptable organic compounds including: alcohols C8-C18, cyclic alcohols, aromatic alcohols, glycerides, polyethylene glycols, polyethylene glycol esters, aromatic esters, phenols, tocopherols, phospholipids, polyoxylglycerides, or polyoxyethylene stearates; one or more emulsifying agents selected from physiologically acceptable nonionic surfactants; and one or more carrier oils. The composition has an extragranular portion including: an optional second porous solid carrier including a physiologically acceptable salt that is soluble in gastric fluid/an acidic aqueous media; one or more disintegrants; one or more diluents; and one or more lubricants.
Resumen de: AU2024234362A1
This disclosure is directed to a nanoparticle formulation for N,N-Diethyl-2-(N-(2-(4-hydroxyphenylamino)-2-oxoethyl)sulfamoyl)benzamide (SRP-3D (DA)), an active pharmaceutical ingredient that is insoluble in water and cannot be dissolved in any injection-safe vehicle to a sufficient concentration for dosing. Also encompassed by the disclosure are nanoparticulate suspension comprising a nanoparticulate SRP-3D (DA) (or SRP-3D (DA) nanoparticles), a wetting agent and a cryoprotectant. The nanoparticulate compositions comprise SRP-3D (DA) particles having an average particle size of less than about 500 nm or less than about 300 nm.
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: JP2025138725A
To provide nucleic acid based compounds or polynucleotides which encode a polypeptide of interest (for example, modified mRNA or mmRNA) and which have structural and/or chemical features that avoid one or more of the problems in the art, for example, features which are useful for optimizing formulation and delivery of nucleic acid-based therapeutics while retaining structural and functional integrity, overcoming the threshold of expression, improving expression rates, half-life and/or protein concentrations, optimizing protein localization, and avoiding deleterious bio-responses such as immune response and/or degradation pathways.SOLUTION: Compositions of polynucleotides, primary transcripts, and modified mRNA molecules (mmRNA), and methods, processes, kits, and devices for their design, preparation, manufacture, and/or formulation are provided.SELECTED DRAWING: None
Resumen de: CN120456919A
There is provided a composition for treating chronic hepatitis B infection comprising an mRNA encoding a hepatitis B virus antigen wherein the mRNA is encapsulated in lipid nanoparticles (LNPs).
Resumen de: CN120475964A
There is provided a composition comprising: (a) an active ingredient; and (b) a lipid mixture comprising: (i) a cationically ionizable lipid capable of forming lipid nanoparticles; (ii) a steroid; and (iii) a negatively charged amphiphile having a hydrophilic portion and a lipophilic portion; wherein the composition is a lipid nanoparticle composition and is substantially free of a polyethylene glycol conjugated lipid wherein the polyethylene glycol (PEG) moiety of the PEG conjugated lipid has at least 5 contiguous ethylene glycol repeat units.
Resumen de: AU2023407128A1
Provided herein is an ionizable, cationic amino lipid or a pharmaceutically acceptable salt thereof. The ionizable, cationic amino lipid has: a protonatable amino head group; two lipophilic chains, wherein the protonatable amino head group has a central carbon atom to which each of the two lipophilic chains are directly bonded; at least one of the two lipophilic chains has a structure of Formula C: Formula C wherein E is an ester in either orientation. The compounds may be formulated in a lipid nanoparticle for use in the delivery of charged cargo such as nucleic acid.
Resumen de: EP4678629A1
An ionizable lipid molecule, a preparation method therefor and a use thereof. Specifically, provided are a compound represented by formula I, and a stereoisomer, tautomer, pharmaceutically acceptable salt, prodrug or solvate thereof. The definition of each group in the formula is as described in the description. Also provided is a lipid nanoparticle, containing the ionizable lipid compound represented by formula (I). Further provided are a composition and a related use. The compound of formula I can be used to prepare the lipid nanoparticle for delivering a nucleic acid therapeutic agent or an active agent in vivo and in vitro.
Nº publicación: EP4678164A2 14/01/2026
Solicitante:
ACUITAS THERAPEUTICS INC [CA]
Acuitas Therapeutics Inc
Resumen de: EP4678164A2
Compounds are provided having the following structure:or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein a, b, c, d, G<sup>1</sup>, G<sup>2</sup>, L<sup>1</sup>, L<sup>2</sup>, R<sup>1a</sup>, R<sup>1b</sup>, R<sup>2a</sup>, R<sup>2b</sup>, R<sup>3a</sup>, R<sup>3b</sup>, R<sup>4a</sup>, R<sup>4b</sup>, R<sup>5</sup>, R<sup>6</sup>, R<sup>7</sup>, R<sup>8</sup> and X are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, nanoparticles comprising the compounds and methods for their use and preparation are also provided.