Resumen de: WO2025232812A1
The present disclosure provides a biomolecule-conjugated lipid nanoparticle (LNP) and the method of preparing the biomolecule-conjugated LNP. Specifically, the present disclosure provides a nucleic acid delivery vector comprising a VHH-linker conjugate, wherein the VHH-linker conjugate comprises (a) a VHH, (b) a linker with a moiety Y and (c) a hinge, furthermore, the nucleic acid delivery vector comprises anchor-modified LNP, wherein the anchor-modified LNP comprises: (a) LNP; and (b) one or more anchor fragment, said anchor fragment comprises moiety X;wherein said moiety Y of the linker is capable of forming a linkage with said moiety X of the anchor fragment via bio-orthogonal click reaction.
Resumen de: WO2025232760A1
The present invention relates to a chimeric antigen receptor immune cell specifically binding to Claudin 18.2, and a use thereof. Specifically, provided is a CAR-T cell using a Claudin 18.2-targeting nanobody as an antigen-binding domain, capable of highly specifically targeting Claudin 18.2-positive tumor cells; the CAR-T cell co-expresses the extracellular domain of a TGF-β receptor, and optionally the intracellular signaling domain of an IL-7 receptor, which can effectively block or reduce TGF-β-induced inhibitory signals, and promote immune cell expansion, survival and persistence, thereby enhancing specific killing of tumor cells. The chimeric antigen receptor immune cell can be used for treating Claudin 18.2-positive tumors.
Resumen de: WO2025232448A1
Disclosed in the present invention are a conjugate comprising an N-oxidized tertiary amine polymer and a nucleic acid drug for improving nucleic acid drug delivery efficiency and nanoparticles thereof. A polymer comprising an N-oxidized tertiary amine group is conjugated to a nucleic acid drug strand to prepare a conjugate thereof. The conjugate has the advantages of being able to be directly used in cells and being able to be assembled with a cationic lipid or polymer to form complex nanoparticles with a dense core formed by complexing the nucleic acid drug/the cationic lipid or polymer and an electrically neutral polymer shell formed from the polymer comprising the N-oxidized tertiary amine group. The present invention can be used to efficiently deliver the nucleic acid drug in vivo or in vitro, with the toxic and side effects resulting from the cationic lipid being significantly reduced.
Resumen de: WO2025231560A1
An antigen (Ag) that elicits a polyclonal T cell receptor like antibody response from endogenous B cells. The Ag comprises a single chain peptide MHCI (pMHCI) complex, that directs B cell responses to the displayed peptide without reactivity to the rest of the pMHCI molecule. Also, methods of using the Ag to induce polyclonal T cell receptor like (TCRL) antibodies (Abs) and using the Ag to deliver the single chain peptide to a target cell.
Resumen de: WO2025231536A1
The present invention relates to a technology that aims to optimise the onset of action of a molecule having analgesic and anti-inflammatory properties, specifically celecoxib or derivatives thereof. The approach involves the production of nanocrystals of celecoxib or derivatives thereof stabilised with a low-viscosity polymer for a faster onset of action of the active ingredient, as well as the production process therefor, pharmaceutical compositions, uses and treatment methods comprising same.
Resumen de: US2025345481A1
Nanocomposite hydrogels suitable for bone tissue regeneration may include (i) a scaffold comprising serum albumin and a cell adhesion promoter crosslinked with polyethylene glycol and (ii) a nanoparticle dispersed in the scaffold. Said nanocomposite hydrogels may formed from an injectable composition that includes: a nanocomposite hydrogel precursor A comprising that comprises a polyethylene glycol with two or more N-hydroxysuccinimide-terminal groups (PEG-NHS); and a nanocomposite hydrogel precursor B comprising a serum albumin, a nanoparticle, and a cell adhesion promoter; wherein the nanocomposite hydrogel precursor A and the nanocomposite hydrogel precursor B are physically separated.
Resumen de: US2025345457A1
A functionalized diblock copolymer. The chemical structure of the functionalized diblock copolymer is shown in Formula II. The functionalized diblock copolymers or polymer particles can be widely used in tumor imaging, tumor treatment and other fields. It not only has good safety, realizes faster and adjustable (by changing the structure and number of functional groups) degradation and removal of polymers under acidic conditions, but also has excellent specificity and high-quality imaging effects at the target site, with high signal-to-noise ratio, clear boundaries, and long half-life. It solves the problem of fluorescence imaging technology in real-time intra-operative navigation, which has a good industrialization prospect.
Resumen de: US2025345442A1
Provided are compositions, kits, and methods for delivering a proteinaceous cargo, or a protein or a peptide, or a drug or a marker, to or into a cell or to an individual in need thereof. In alternative embodiments, products of manufacture as provided herein comprise: (a) a recombinant bacterial Contractile Injection System (CIS) or a Metamorphosis Associated Contractile structure (MAC) formed or configured to comprise a tube having an inner core, (b) a Metamorphosis-Inducing Factor 1 (Mif1) protein positioned in the inner core of the tube of the CIS or MAC, (c) a chaperone 605 protein non-covalently associated with the Mif1 protein positioned in the inner core of the tube of the CIS or MAC, and (d) a proteinaceous cargo, or a heterologous protein or peptide, or compound, non-covalently associated or covalently associated or linked to the Mif1.
Resumen de: US2025345455A1
The present invention relates to nanoparticle bioconjugates comprising binding proteins for binding to a cancer cell and an antigen of an immune cell, and uses and methods of treatment comprising administration thereof.
Resumen de: US2025345453A1
Disclosed are compounds and compositions that preferentially target cancer cells with a warhead that comprises a chemotherapeutic agent releasably bound to a targeting agent where the chemotherapeutic agent is released upon cellular absorption. Also disclosed are methods of use.
Resumen de: US2025345454A1
Disclosed are compounds and compositions that preferentially target cancer cells with a warhead that comprises paclitaxel releasably bound to a targeting agent where the chemotherapeutic agent is released upon cellular absorption. Also disclosed are methods of use.
Resumen de: US2025345456A1
Nontoxic, targeted poly beta-amino esters (PBAEs) are synthesized by using click chemistry to attach a targeting moiety. The click chemistry uses a dienophile, such as a strained alkene ring, and a diene, such as tetrazine, to provide rapid attachment of targeting moieties to PBAE polymers and nanoparticle surfaces containing them. Targeting moieties such as aptamers, antibodies or antibody-like proteins can be quickly and safely coupled to PBAEs to provide highly specific localization of nanoparticles for gene therapy or targeted delivery of therapeutics.
Resumen de: US2025345462A1
The present disclosure relates, in part, to a composition comprising a nucleic acid, wherein the nucleic acid encodes a protein which has a reduced abundance in a GTPase IMAP family 5 (GIMAP5) deficient subject or at least partially inhibits expression of a protein which has an increased abundance in a GIMAP5 deficient subject, as compared to a healthy subject. In certain embodiments, the composition is a nucleic acid-lipid particle. In certain embodiments, the composition is a polymer-based vehicle. In another aspect, the present disclosure relates to a recombinant viral vector comprising a nucleic acid encoding a protein which has a reduced abundance in a GIMAP5 deficient subject as compared to a healthy subject. In yet another aspect, the present disclosure provides a method of treating, ameliorating, and/or preventing liver disease and/or portal hypertension in a subject with administration of one or more compositions and/or vectors of the present disclosure.
Resumen de: US2025345285A1
The invention provides polymeric particles comprising a matrix of a biocompatible polymer and polyethylene imine, said matrix having incorporated therein an anionic or hydrophobic sonosensitiser and, optionally, an immunomodulatory agent and/or an imaging agent. Such particles find use in methods of sonodynamic therapy, in particular in methods of combined sonodynamic therapy and immunotherapy, for example in the treatment of cancer, metastasis or micrometastasis derived from cancer. The invention is particularly suitable for the treatment of deep-sited, hard to treat tumours such as pancreatic cancer.
Resumen de: US2025345289A1
The present application discloses compositions comprising nanoparticles of vitamin K2, and their methods of use.
Resumen de: US2025345284A1
The present disclosure relates, in part, to lipid nanoparticles (LNPs) comprising cholesterol substitutes (i.e., cholesterol analogs and/or derivatives) and methods of use thereof for in vivo delivery of nucleic acid molecules and/or therapeutic agents to a target cell. In certain embodiments, the nucleic acid molecules encode chimeric antigen receptors (CARs). In certain embodiments, the target cell is a T cell. In certain embodiments, the LNPs of the present disclosure are anti-inflammatory. In certain embodiments, the present disclosure relates to the use of the LNPs described herein for the treatment, prevention, and/or amelioration of diseases and/or disorders in a subject, including but not limited to cancer.
Resumen de: US2025345286A1
The present disclosure belongs to the technical field of lipid nanoparticle biological application, and discloses a composite lipid nanoparticle and a preparation method thereof, an ribose nucleic acid (RNA) vaccine, a drug and use. The composite lipid nanoparticle provided by the present disclosure includes a lipid composition and a recombinant antibody protein containing a streptavidin tag; and the lipid composition comprises an ionizable lipid, an auxiliary lipid, cholesterol, a polyethylene glycol lipid and a biotinylated polyethylene glycol lipid in a mass ratio of (40-90):(0.1-20):(20-50):(0.5-10):(0.5-10). Through strong interaction between the recombinant antibody protein with the streptavidin tag and the biotinylated polyethylene glycol lipid in the composite lipid nanoparticle, the composite lipid nanoparticle achieves safe, convenient, rapid, efficient, and stable linkage between a lipid nanoparticle (LNP) and a recombinant antibody protein, and the recombinant antibody protein can be adaptively replaced according to target cells and/or tissues, with high flexibility and applicability.
Resumen de: US2025345283A1
Compositions and methods described in this document make use of macrophage-derived engineered vesicles (MEV) having specificity for delivery to a target environment, for use in modifying macrophage phenotype and/or treating a condition.
Resumen de: US2025345277A1
An ionizable liposome, preparation thereof, and an application thereof in gene delivery. Specifically, provided are an ionizable lipid compound of the following formula I, and a stereoisomer, tautomer, pharmaceutically acceptable salt, prodrug, or solvate thereof, wherein the definition of each group in the formula is as stated herein. Further provided are lipid nanoparticles, comprising the ionizable lipid compound of formula (I). Further provided are a composition and a related use. The compound of formula (I) can be used for preparing the lipid nanoparticles for delivering nucleic acid-type therapeutic agents or active agents in vivo and in vitro.
Resumen de: US2025345280A1
The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.
Resumen de: US2025345278A1
Nanoparticles suitable for delivery of a linear DNA molecule are provided. Nanoparticles suitable for delivery of mRNA or DNA are provided. Further provided are uses of the nanoparticles including the use of the nanoparticles for treating disease and the use of the nanoparticles in vaccines.
Resumen de: US2025345416A1
The present disclosure provides pharmaceutical compositions for delivery of HSV antigens (e.g., an HSV vaccine) and related technologies (e.g., components thereof and/or methods relating thereto).
Resumen de: US2025345248A1
Core-shell particles and methods of making and using thereof are described herein. The core is formed of or contains one or more hydrophobic materials or more hydrophobic materials. The shell is formed of or contains hyperbranched polyglycerol (HPG). The HPG coating can be modified to adjust the properties of the particles. Unmodified HPG coatings impart stealth properties to the particles which resist non-specific protein absorption and increase circulation in the blood. The hydroxyl groups on the HPG coating can be chemically modified to form functional groups that react with functional groups and adhere the particles to tissue, cells, or extracellular materials, such as proteins.
Resumen de: US2025346904A1
The present invention pertains to the nanoencapsulation of siRNA and other biologics in phospholipid nanosomes for the improved delivery of siRNA and other biologics to targeted diseased human or animal organs and human or animal cells and apparatus and methods for making the same. In embodiments of the present invention, novel siRNAs were designed to down regulate CCR5 and CD4, based on an analysis of all known alternative transcripts for each gene from both human and monkey (Macaca mulatta) genomes. Embodiments of the present invention feature supercritical, critical and near critical fluids. Embodiments of the present invention also pertain to down regulation of CXCR4 receptor and targeting of nanosomes containing specific siRNA sequences to cells expressing those receptors on the cell surface by coating them with specific ligands. These include ligands for the receptors CCR5, CD4 and CXCR4.
Nº publicación: WO2025232898A1 13/11/2025
Solicitante:
YOLTECH THERAPEUTICS CO LTD [CN]
YOLTECH THERAPEUTICS CO., LTD
Resumen de: WO2025232898A1
Provided herein are lipids that can be used in combination with other lipid components, such as neutral lipids, cholesterol and polymer conjugated lipids, to form lipid nanoparticles for delivery of therapeutic agents (e.g., nucleic acid molecules encoding gene editing machinery) for therapeutic or prophylactic purposes.