Resumen de: WO2025199302A1
A pH-sensitive cationic lipid, a lipid nanoparticle including the pH-sensitive lipid, and a method of delivering a nucleic acid encapsulated in the lipid nanoparticle to a cell or a subject. The lipid nanoparticle may include a compound represented by general formula (I) or pharmaceutically acceptable salt thereof: (R1)(R2)C(OH) -(CH2)a-(O-CO)b-X (I) wherein a represents an integer of 3-5; b represents 0 or 1; R1 and R2 each independently represent a group represented by general formula (A): (R11)(R12) - CH-(CH2)w - (CO-O)c - (CH2)v - (A), wherein R11 and R12 each independently represent C5-15 alkoxy group; each c independently represents 0 or 1; each v independently represents an integer of 4-12; each w independently represents an integer of 0-3; and X represents a 5- to 7-membered non-aromatic heterocyclic group, wherein a carbon atom of the heterocyclic group is bound to (O-CO)b- and one or two hydrogen atoms of the heterocyclic group may optionally be replaced with C1-4 alkyl group or C2-4 alkenyl group.
Resumen de: WO2025198369A1
The present invention relates to a novel provitamin B5-based lipid derivative compound, a lipid nanoparticle composition comprising same, and the like. More specifically, the novel provitamin B5-based lipid compound functions as an ionizable lipid in the formation of lipid nanoparticles and forms stable lipid nanoparticles, which can be used for delivering pharmaceutically active ingredients such as mRNA into the body.
Resumen de: WO2025197935A1
The present invention provides a drug-polymer complex that includes a drug and a drug delivery polymer having a phosphocholine group, and that exhibits a dissociation constant (KD) of 1.0×10-6M or less with respect to STARD7.
Resumen de: WO2025198324A1
The present invention relates to a polymer self-assembly nanocarrier and a method of preparing same, the nanocarrier binding a peptide that targets a specific cell and, simultaneously, carrying an active ingredient that has been verified. The present invention can exhibit various effects such as a moisturizing effect, an increase in filaggrin production, whitening, skin barrier reinforcement and the like by selectively and efficiently delivering a drug through a targeting peptide, can be applied to skin diseases such as atopy or psoriasis, and is expected to enable the maximum effect of an active substance in the pharmaceutical and cosmetic fields to be obtained.
Resumen de: WO2025196687A1
According to an aspect of the present disclosure, a composition for use in inhibiting viability of triple negative breast cancer (TNBC) cells contains 1,8-cineole as an active ingredient and β-cyclodextrin as a carrier, where the β-cyclodextrin encapsulates the 1,8-cineole forming a nanoformulation. According to another aspect of the present disclosure, a method of preparing a composition for use in inhibiting viability of TNBC cells includes forming a first solution by dissolving β-cyclodextrin in a solvent, forming a second solution by adding Elettaria cardamomum essential oil to the first solution, where the Elettaria cardamomum essential oil contains 1,8-cineole as an active ingredient. The method also includes sonicating the second solution to form a homogenized second solution, centrifuging the homogenized second solution to form a pellet, deep freezing the pellet, and lyophilizing the deep frozen pellet to form a nanoformulation, where the nanoformulation contains the 1,8-cineole encapsulated by the β-cyclodextrin.
Resumen de: WO2025196641A1
The invention relates to the field of medicine, and more particularly to bioengineering, genetic engineering, gene editing, molecular medicine, nanotechnology, biotechnology, nanoengineering and protein engineering. The invention can be used for packaging any RNA, DNA, or a mixture thereof into biological nanoparticles of any origin and composition, inter alia nanoparticles enriched with a protein of interest.
Resumen de: WO2025196660A1
The present disclosure provides nanoparticles having RGD-based peptides conjugated thereto. The nanoparticles may be formed one or more covalent organic frameworks (COFs) of the present disclosure. Also disclosed are methods of making the COFs and nanoparticles. The COFs have one or more binding partners and/or COF (following conjugation of the binding partner) may have an RGD-based peptide. The RGD- based peptide may be selected based on the desire to treat a specific cancer. COFs may have the following structure: Formula (I), where L is a linker group and Peptide is an RGD-based peptide.
Resumen de: WO2025195308A1
The present application provides new cationic lipid compounds and a composition containing the compounds. A nanoparticle composition contains new cationic lipid compounds and other lipids, such as phospholipids, structural lipids and PEG lipids. Additionally, the nanoparticle composition of the present application can be used to deliver a therapeutic agent and/or a prophylactic agent to mammalian cells or organs to, for example, regulate polypeptide, protein or gene expression.
Resumen de: WO2025194234A1
The present invention relates to a method for preparing nano-structured lipid carriers that are nanometric systems compatible with hydrophobic drug products and that do not exhibit toxicity due to their excipients. These nano-structured lipid carriers contain co-encapsulated cannabidiol and paclitaxel and have applications in the pharmaceutical field, more specifically in antitumour therapy.
Resumen de: WO2025194409A1
The present disclosure relates to novel lipids for delivering one or more biologically active molecules to a subject. The present application also relates to a composition or a nanoparticle comprising said lipid, methods of preparing said lipid, use of said lipid and method of using said lipid.
Resumen de: WO2025195465A1
Provided is use of a magnetoelectrically driven magnetostrictive piezoelectric nanoparticle in antiviral therapy. The magnetostrictive piezoelectric nanoparticle comprises a shell structure with piezoelectric properties and a core structure with magnetoelectric response properties. The magnetostrictive piezoelectric nanoparticle, by means of up-regulating multiple immunomodulatory pathways, effectively enhances the expression of type I interferon and a host's innate immune response, ultimately inhibiting the viral activity, ameliorating hepatic and pulmonary tissue damage, and improving the survival rates. The magnetostrictive piezoelectric nanoparticle has the advantages such as high controllability, deep penetration, and non-invasiveness, and offers broad application prospects in the antiviral field.
Resumen de: US2025295818A1
The present invention relates to calibrated dual-phase nanodroplets comprising an outer layer and an inner core, said outer layer comprising a biocompatible fluorinated surfactant and said inner core comprising a fluorinated compound and a biocompatible oil. The invention further relates to a method of preparation of said calibrated dual-phase nanodroplets through microfluidic technique, and to their use for in vivo or in vitro diagnostic and/or for therapy.
Resumen de: US2025295606A1
The present invention refers to a method for producing a polymer in form of a gel or a particle, and to the resulting polymer, gel and particle, respectively. The polymer comprises a carbon donor and a metal oxide precursor, a metal oxide or a combination thereof and optionally an active agent. The invention is further directed to a composition and film comprising such polymer, and their use as a medicament for example in treating diabetes, obesity, neuronal disease, viral infection or cancer.
Resumen de: WO2024197065A2
Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the LPA gene. Treatment or prevention of cardiovascular disease through disruption of the production of apo(a) through genetic editing and the reduction of the blood lipoprotein(a) Lp(a) concentration is disclosed herein. Disclosed are nickase-based gene editing systems designed to effectuate the installation of insertions and/or deletions (indel variants) and/or non-synonymous variants in the coding sequence of LPA. The nickase-based gene editing systems generally comprise one or more mRNAs that encode one or more nickases and a plurality of guide oligonucleotides (e.g., gRNAs) and may be delivered in vivo to a mammalian subject in need thereof via a suitable delivery system, such as lipid nanoparticles (LNPs) (with or without GalNAc targeting moieties) intravenously, or otherwise, administered to a patient as potentially a once-and-done therapeutic. The manufacturing, use, and formulation of the gene editing systems and compositions are also disclosed.
Resumen de: US2025295612A1
The present invention comprises: a novel molecule capable of disaggregating amyloid-beta plaques; and a brain-targeting amyloid-beta plaque disaggregation nano platform loaded with the molecule. An amyloid-beta plaque disaggregating agent developed according to the present invention exhibits high amyloid-beta plaque disaggregation efficacy, and the brain-targeting amyloid-beta plaque disaggregation nano platform shows high potential in the medical field on the basis of the effects of effectively targeting the brain and disaggregating amyloid-beta plaques present in the brain.
Resumen de: AU2024261935A1
A nano-size controllable and stable polymer-drug conjugate, an intermediate thereof, and a use thereof. Provided is a polymer-drug conjugate as represented by formula (I) and having different polymer bodies, a controllable coupled group quantity, controllable group coupling sites and a controllable nano-size. The drug conjugate has one or more of the following advantages: a low renal clearance rate, a low liver-spleen clearance rate, long plasma half-life, strong drug accumulation capability at lesion tissues, strong drug permeability at lesion tissues, low toxic and side effects, and an excellent treatment effect.
Resumen de: AU2024233180A1
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: AU2024228558A1
This disclosure provides the use of nanoparticles of self-assembled acidic molecular clusters (NP-AMCs) of ammonium salts to lower the pH of skin and underlying tissue of a human or other mammal as a means of stimulating the localized immune response to a wound.
Resumen de: AU2024219315A1
The present invention relates to polysaccharide cross-linked colloidal particles and a use thereof as a modifier for in vivo injection. The present invention provides a polysaccharide cross-linked colloidal particle platform technology that can be designed to not only act as a drug delivery carrier for functional nanoparticles or drugs for in vivo introduction, but also control in vivo distribution and excretion.
Resumen de: AU2024217151A1
The present invention relates to a non-vascular injection formulation containing nanoparticles of which the hydrodynamic diameter is adjusted to 2 to 20 nm and the surface charge is adjusted to -20 mV to 0 mV through the hydration of hydrophilic functional groups exposed to the surface of the nanoparticles, so that the nanoparticles are selectively discharged only into peripheral lymphatic vessels without infiltrating or being discharged into the capillaries at an injection site, wherein the nanoparticles (i) themselves are a drug and/or (ii) a carrier for other drugs, and the nanoparticles are discharged only into peripheral lymphatic vessels when injected into tissue areas and not intravenously administered, and thus drug modality in which residence time at the injection site is extended is provided. Nanoparticles of which the hydrodynamic diameter and the surface charge are adjusted, according to the present invention, can be excreted from the body within 1 week after administration without any residue at the administration site, and, when the nanoparticles are designed as a contrast agent exhibiting T1-MRI contrast effects and are injected into tissue areas adjacent to blood vessels, peripheral or central lymphatic vessels can be selectively imaged without venous contamination.
Resumen de: US2025297256A1
A composition for treating sickle cell disease includes a cGMP exosome having a size range between 60 nm to 120 nm, which may be extracted from a human mesenchymal stem cell (hMSC) or human PBMC at 320,000 g, wherein the cGMP exosome may be loaded with a Hemoglobin Subunit Beta (HBB) DNA plasmid carrying a gene encoding a normal beta chain of hemoglobin and alone or in combination with a short interference RNA (siRNA) that silences the translation of the SNP rs334 (A) mutation of the beta chain of a Hemoglobin A protein.
Resumen de: US2025296932A1
The present disclosure relates generally to pharmaceutical compositions (e.g., solid oral dosage forms) of the compound of Formula I:Also disclosed are methods of treating or preventing human immunodeficiency virus (HIV) infection in a human, including orally administering to the human the solid oral dosage forms or pharmaceutical compositions disclosed herein.
Resumen de: US2025295788A1
The present disclosure relates, in part, to siloxane-based lipids or lipidoids, lipid nanoparticles (LNPs) comprising the same, and pharmaceutical compositions thereof. In certain embodiments, the LNPs of the present disclosure selectively target cells (e.g., hepatocytes, epithelial cells, endothelial cells, and immune cells, inter alia) and/or organs of interest (e.g., liver, spleen, heart, and lungs, inter alia). In another aspect, the present disclosure relates to methods of treating, preventing, and/or ameliorating one or more diseases and/or disorders in a subject, the method comprising administering to the subject at least one LNP of the present disclosure and/or at least one pharmaceutical composition of the present disclosure.
Resumen de: US2025295806A1
An immunostimulatory nanoparticle comprising a biocompatible lipid shell that defines an outer surface of the nanoparticle and a core, which is loaded with a Toll-like Receptor 9 (TLR9) agonist and a nucleic acid inhibitor of V domain Immunoglobulin Suppressor of T cell activation (VISTA), and optionally a plurality of targeting moieties linked to the outer surface, wherein the optional targeting moieties are configured to direct the nanoparticle to tumor-resident myeloid cells in a tumor microenvironment upon administration of the nanoparticle to a subject with cancer.
Nº publicación: US2025295805A1 25/09/2025
Solicitante:
THE REGENTS OF THE UNIV OF MICHIGAN [US]
The Regents of the University of Michigan
Resumen de: US2025295805A1
The present invention relates to nanoparticles complexed with biomacromolecule agents configured for treating, preventing or ameliorating various types of disorders, and methods of synthesizing the same. In particular, the present invention is directed to compositions comprising nanoparticles (e.g., synthetic high density lipoprotein (sHDL)) carrying biomacromolecule agents (e.g., nucleic acid, peptides, glycolipids, etc.), methods for synthesizing such nanoparticles, as well as systems and methods utilizing such nanoparticles (e.g., in diagnostic and/or therapeutic settings).