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216
resultados
Última actualización
27/04/2026 [07:00:00]
Resultados 50 a 75 de 216
Resumen de: WO2026083241A1
The present disclosure provides ionizable lipids that are particularly beneficial in formulations of lipid nanoparticles providing advantageous improvements in the delivery of nucleic acids to hepatic stellate cells with enhanced efficiency. The ionizable lipids are N,N-substituted hydroxylamine ester compound. The disclosure also provides lipid nanoparticles, pharmaceutical compositions, and methods including the provided ionizable lipids.
Resumen de: US20260108472A1
0000 Disclosed are methods and compositions for restoring Cyr61 in bone tissue. In certain aspects, the compositions comprise a recombinant Cyr61 carried in carbon nanotubes. The compositions may be used for various methods including restoring Cyr61 expression in bone, increasing bone density, or treating a bone disease in a patient.
Resumen de: US20260109984A1
0000 Therapeutic methods, systems, and compositions for treating a patient suffering from hemoglobinopathy include treating the patient to increase expression of noncoding RNA activated by DNA damage (NORAD). Overexpression of NORAD long non-coding RNA leads to an increase in fetal hemoglobin expression. The hemoglobinopathy may be β-thalassemia or sickle cell disease.
Resumen de: WO2026085500A1
Provided herein are compositions and methods for ablating intracellular signaling through specific cell surface receptors as means of treatment for various conditions of a pro-inflammatory character. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint. In some aspects, the compositions and method are for treating or preventing localized nociception, inflammation, degeneration, or morphological changes associated with back or spine conditions or disorders.
Resumen de: WO2026085291A1
Disclosed herein are metabolizable binary gold supraclusters, which, in some implementations, comprise cationic gold nanoclusters laden with a therapeutic nucleic acid, such as a small interfering RNA (siRNA), and are intertwined through bioresponsive crosslinkers within a hydrophilic polymer matrix. Also provided herein are uses of the supraclusters, e.g., in treatment of diseases such as cancers.
Resumen de: WO2026085498A1
The present disclosure provides compositions and methods for enhancing gene editing. In some aspects, the compositions and methods are used prevent the progression or treat diseases such as osteoarthritis and other joint disorders that are characterized by an inflammatory component.
Resumen de: WO2026082012A1
The present invention provides a drug-loaded nanoparticle composition suitable for drug delivery in vivo, wherein the drug-loaded nanoparticle composition is formed in the form of a non-covalent bond and comprises an active ingredient, cyclodextrin, and albumin. Drug-loaded nanoparticles or the composition thereof in the present invention are stable in a solution state, avoiding cumbersome operations of reconstitution and redissolution. Compared with a known freeze-dried powder of nanoparticles containing albumin, the nanoparticles in the present invention have a particle size of less than 150 nm, can remain stable for at least 7 days or more without change, and can be predicted to be stable for a long time.
Resumen de: WO2026082183A1
Disclosed in the present invention is a lyoprotectant for nucleic acid-lipid nanoparticles. The lyoprotectant of the present invention is formed by combining sucrose or trehalose with polyvinylpyrrolidone in a specific ratio, and provides a good lyoprotective effect for the nucleic acid-lipid nanoparticles, ensuring that there is no significant difference in particle size, uniformity, encapsulation efficiency, nucleic acid integrity, and expression performance of the nucleic acid-lipid nanoparticles before and after lyophilization. Furthermore, by adjusting the salt concentration in a mixed solution of the nucleic acid-lipid nanoparticles and the lyoprotectant, the lyoprotective effect on the nucleic acid-lipid nanoparticles is further improved, so that the performance of lyophilized nucleic acid-lipid nanoparticles is closer to that before lyophilization.
Resumen de: WO2026084314A1
The present invention relates to: lipid nanoparticles with improved genetic material delivery efficiency through the regulation of an extracellular matrix and pH control of intracellular organelles; and use thereof. A composition for delivering a genetic material, according to the present invention, comprises lipid nanoparticles and a proton pump inhibitor, whereby the genetic material can be effectively delivered by overcoming various intracellular and extracellular barriers and the limitations of existing delivery systems.
Resumen de: US20260108656A1
The present invention relates to a levan-catechol composite, and a tissue adhesion composition and nanocluster, including same. In particular, the levan-catechol composite is prepared by the conjugation of levan and catechol and is applicable, by hydrogelation or nanoclustering thereof, for use in tissue adhesion in wet environments, wound healing, hemostasis, or drug delivery.
Resumen de: KR102631907B1
The present invention relates to a device useful for manufacturing nanoparticles containing hydrophobic substances and hydrophilic substances. Specifically, the device of the present invention comprises: a plurality of inlet channels through which hydrophobic substances and hydrophilic substances are respectively introduced; a mixing channel through which the substances are mixed to manufacture nanoparticles; and an outflow channel through which the manufactured nanoparticles are discharged, wherein the mixing channel includes micro-pillars capable of increasing the mixing efficiency of the substances. Therefore, the nanoparticles manufactured by the device of the present invention have excellent particle uniformity and may be usefully used as drugs or drug delivery vehicles.
Resumen de: EP4729507A1
Provided is a rapidly-metabolized lipid compound. The present invention relates in particular to a compound represented by formula (I), or a pharmaceutically acceptable salt, an isotopic variant, a tautomer or a stereoisomer thereof. Also provided are a nanoparticle pharmaceutical composition comprising the compound, and a use of the compound and a composition thereof in delivering nucleic acids.
Resumen de: WO2025042791A1
The disclosure provides for compositions that comprise nanocomplexes formed by complexing one or more therapeutic agents with nucleic acid fragments of varying lengths and sizes that are coated or complexed with protamine sulfate, and uses thereof, including for the treatment of cancer in a subject in need thereof.
Resumen de: WO2024258949A2
Disclosed herein are immunosorbent nanoparticles, devices, and methods for selective removal of a target protein such as beta-2 microglobulin (B2M) from a liquid such as blood.
Resumen de: WO2024259421A2
A nanoparticle generally comprising a targeting peptide-lipid conjugate, wherein a targeting peptide moiety of the targeting peptide-lipid conjugate comprises a GRP78 targeting peptide, a polyethylene glycol (PEG)-lipid conjugate, a drug-lipid conjugate comprising a prodrug moiety, wherein the drug-lipid conjugate comprises one or more of a mertansine (DM1) prodrug, a doxorubicin prodrug, and a bortezomib (BTZ) prodrug; and wherein the prodrug is linked to a lipid moiety of the drug-lipid conjugate via a phosphodiester bond or a boron ester bond; cholesterol comprising about 1 mol% to about 10 mol% of the nanoparticle; and distearoylphosphatidylcholine (DSPC).
Resumen de: US2024417680A1
0000 The application pertains to compositions and methods useful for growing living cells such as stem cells. The compositions employ a mixture of an extracellular matrix and discrete carbon nanotubes. The extracellular matrix may also comprise components selected from the class of proteins, proteoglycans, polysaccharides, lipids, peptides, messenger molecules, signaling molecules, or any mixture thereof. The discrete carbon nanotubes are usually less than about 1% by weight of the dry weight of the total composition.
Resumen de: WO2024254709A1
The present disclosure provides a therapeutic nanodisk, the therapeutic nanodisk comprising: a lipid-binding polypeptide; a lipid bilayer and a therapeutic agent, wherein the therapeutic agent may be of use for treating, preventing a central nervous system disease, disorder, trauma or injury; or as a diagnostic agent for diagnosing a central nervous system disease, disorder, trauma or injury. The lipid bilayer may be 1,2-Dimyristoyl-sn-glycero-3-phosphocholine (DMPC) and the therapeutic agent may be a nucleic acid polymer. Further provided are methods for administration of the therapeutic nanodisk to treat, prevent or diagnose the central nervous system disease, disorder, trauma or injury and uses of such therapeutic nanodisks.
Resumen de: WO2024254703A1
The blood-brain barrier (BBB) and the blood-cerebrospinal fluid (CSF) barrier (BCSFB) create an obstacle for effective systemic drug delivery to the CNS. This application provides compounds and nanoparticles for increasing the penetration of drugs through the BBB. Specifically, this application provides nanoparticles for the diagnosis and treatment of central nervous system (CNS) diseases and preparation methods therefor. These nanoparticles are polymer-lipid based nanoparticles (PLNPs) functionalized to facilitate blood brain barrier (BBB) penetration and accumulation in a disease area of the CNS. Notably, said nanoparticles target an LDL receptor and/or glucose transporter. In various embodiments, the nanoparticles comprise terpolymers which comprise polysorbates (such as polysorbate 80), poly acrylic acids (such as poly methacrylic acid (PMAA)) and various polysaccharides (including maltodextrin) and the nanoparticles also comprise cholesterol and lipids. The nanoparticles encapsulate a pay load which is a therapeutic drug molecule, biomolecule, contrast agent or nucleotide.
Resumen de: WO2024259281A2
Poly(oxazoline) conjugates with pendant cationic groups (cationic POZ) and lipid nanoparticles (LNPs) including cationic POZ used to facilitate delivery of an encapsulated payload. LNPs and polyplexes including cationic POZ and a nucleic acid payload such as, but not limited to, mRNA or modified mRNA are disclosed. Such LNPs have no immunogenicity or reduced immunogenicity as compared to a corresponding LNP containing an ionizable lipid.
Resumen de: WO2024259374A2
To introduce material to cells, contemporary medicinal constructs rely on the uptake mechanisms of the cell membrane. This puts major restrictions on the types of utilizable materials (e.g., charge compatible), specifications (e.g., 100 nanometer scale or less) and organizations (mostly simplistic spheroids); this is the regime of nanoparticles, protein/peptide conjugates etc. However, the focus and novelty of the innovation presented are constructs which can still achieve this membrane interaction to connect to cells yet the constructs themselves remain outside of the cell, thus establishing a network by which to transfer materials. These can surpass the aforementioned limitations as well as create entirely new application spaces as these new constructs enable different desired distribution patterns and exchanged material.
Resumen de: WO2024259175A2
Provided herein is a system for inhibiting a miRNA-17 family miRNA from binding to the 3'UTR of PKD1, where the system includes: a gRNA; and a polynucleotide-programmable nucleotide-binding domain, where the system modifies a binding site of a miRNA-17 family miRNA in the 3'UTR of PKD1, thereby preventing binding of the miRNA-17 family miRNA and de-repressing PKD1 mRNA.
Resumen de: WO2024259373A1
The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel cationic lipid as well as additional lipids such as ionizable lipids, phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
Resumen de: WO2024259356A1
Compounds are provided having the following Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein G1, G2, R1, R2, R3, L1a, L1b, and L2 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: EP4729075A1
0001 The present application relates to the field of biomedicine, and provides an epigenetic editing tool for targeting a hepatitis B virus gene and a use thereof.
Nº publicación: EP4728076A1 22/04/2026
Solicitante:
PFIZER [US]
Resumen de: WO2024256962A1
The invention relates to RNA molecules encoding an E. coli fimbrial H antigen (FimH). The present disclosure further relates to compositions comprising the RNA molecules formulated in a lipid nanoparticle (RNA-LNP). The present disclosure further relates to the use of the RNA 5 molecules, RNA-LNPs and compositions for the prevention of E. coli infection, including urinary tract infection.
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