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一种跨血脑屏障递送蛋白药物的可降解球形核酸及其制备方法、应用

Resumen de: CN120053672A

本发明属于肿瘤药物研发领域，针对蛋白药物向脑部肿瘤递送存在的跨越血脑屏障困难、靶向性差等亟待解决的问题，本发明公开了一种跨血脑屏障递送蛋白药物的可降解球形核酸，是一种以包裹有蛋白药物的可降解纳米硅球作为内核，内核表面通过共价键连接有核酸适配体，形成的球形核酸结构。本发明通过球形核酸结构递送蛋白药物，得益于清道夫受体介导的转细胞作用，能够有效克服血脑屏障这一脑部递送中的主要障碍；同时对于蛋白药物的封装避免了其在血液中的提前释放、降低了其被提前清除的可能，也降低了蛋白药物引起不良免疫反应的风险。而且，本发明提出的跨血脑屏障递送蛋白药物的可降解球形核酸，不仅扩展了球形核酸的递送种类，并且通过响应性降解的构想，降低了其滞留风险，具有良好的应用前景。

STEALTH LIPID NANOPARTICLE COMPOSITIONS FOR CELL TARGETING

Resumen de: AU2023406483A1

The present disclosure provides stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types,

AEROSOLIZED LIPID NANOPARTICLES AND USES THEREOF

Resumen de: AU2023382622A1

Lipid nanoparticles (LNPs) containing particular cationic ionizable lipids with a biologically active polynucleotides (e.g., RNAs) are provided. In some aspects, the LNP complexes are provided as aerosols and/or dry powders, such as for delivery to the lungs. Methods of making and using such compositions are provided.

NOVEL LIPIDS USED FOR NUCLEIC ACID DELIVERY AND LIPID NANOPARTICLE COMPOSITION

Resumen de: AU2023383821A1

Provided are cationic lipid compounds represented by formula (I), which can be applied to lipid nanoparticles such that the lipid nanoparticles can target different tissue organs for drug delivery. The cationic lipid compounds or a lipid nanoparticle composition comprising same can specifically deliver a preventive/therapeutic agent, especially a nucleic acid component, to a target organ.

LIPID NANOPARTICLE DRUG CONJUGATES

Resumen de: AU2023375378A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

LIPID NANOPARTICLE DRUG CONJUGATES

Resumen de: AU2023375377A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

SYNTHETIC SINGLE STRANDED NUCLEIC ACID COMPOSITIONS AND METHODS THEREOF

Resumen de: AU2023406273A1

The present application discloses single stranded deoxyribonucleic acid (DNA) molecules comprising at least one nucleic acid sequence of interest flanked by a first at least one stem loop structure, methods of making and method of use for delivery and expression of a transgene in host cells.

LIPID NANOPARTICLES COMPRISING NUCLEIC ACIDS, IONIZABLE LIPIDS, STEROLS, LIPID ANCHORED POLYMERS AND HELPER LIPIDS, THEIR USES

Resumen de: AU2023406303A1

Provided herein are lipid nanoparticle (LNP) compositions (e.g., pharmaceutical compositions) comprising a therapeutic nucleic acid (TNA), wherein the LNP comprises an ionizable lipid; a "helper" lipid, e.g., a ceramide or distearoylphosphatidylcholine (DSPC); a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, as well as uses thereof.

PEPTIDE CONJUGATED PARTICLES

Resumen de: AU2025203403A1

0324 The present invention provides compositions comprising peptide-coupled biodegradable poly(lactide-co-glycolide) (PLG) particles In particular, PLG particles are surface-functionalized to allow for coupling of peptide molecules to the surface of the particles (e.g., for use in eliciting induction of immunological tolerance).

Cancer vaccines

Resumen de: AU2025203258A1

Abstract The disclosure relates to cancer ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.

NON-LINEAR PEGYLATED LIPID CONTAINING A TERTIARY AMINE AND APPLICATION THEREOF

Resumen de: US2025170062A1

A non-linear PEGylated lipid of the Formula (1) contains a tertiary amine; wherein, B1 and B2 are linking bonds or alkylene groups; L1, L2, Ld, and Lx are linking bonds or divalent linking groups; R1 and R2 are C1-50 aliphatic hydrocarbon groups or C1-50 residues of aliphatic hydrocarbon derivatives, each containing 0-10 heteroatoms; y is 2 or 3; X is C or CH; XPEG is a polyethylene glycol component. Compared with linear PEGylated lipids, the PEGylated lipid can realize better surface modification of LNP, better protective effects, extended systemic circulation of LNP, and significantly reduced biological toxicity of LNP. The LNP pharmaceutical composition shows excellent drug delivery capabilities. The lipid-nucleic acid pharmaceutical composition and its formulation exhibit higher pharmaceutical efficacy, especially the LNP-nucleic acid pharmaceutical composition.

TREATMENT OF SOLID TUMORS WITH NEGATIVELY CHARGED PARTICLES

Resumen de: US2025170066A1

The present disclosure generally relates to the methods of treating solid tumors using negatively charged particles.

COMPOSITIONS AND METHODS FOR LASER LITHOTRIPSY USING NANOPARTICLE FINE-TUNED NIR ABSORPTION

Resumen de: US2025169882A1

Systems and methods for performing laser lithotripsy include introducing a lithotripsy medium containing nanoparticles into a body cavity comprising target obstructions and applying laser energy through the lithotripsy medium to disrupt the target obstructions. The nanoparticles may have diameters configured to enhance absorption efficiency of the laser energy. The nanoparticles may include organic polymers such as PEDOT: PSS or inorganic compounds such as indium tin oxide. Systems may include a laser source, a fluid delivery component configured to deliver the nanoparticle-containing lithotripsy medium, and an optical fiber for delivering laser energy. Methods of manufacturing lithotripsy media include selecting target wavelengths, synthesizing nanoparticles with corresponding absorption characteristics, and dispersing the nanoparticles at selected concentrations.

NANOPARTICLES FOR DIRECT REGULATION OF MITOCHONDRIAL GENE EXPRESSION

Resumen de: US2025170269A1

Compositions comprising biologically active synthetic nanoparticle constructs and methods of use thereof to modify mitochondrial gene expression including transcriptional repression and transcriptional activation.

MESSENGER RIBONUCLEIC ACIDS FOR ENHANCING IMMUNE RESPONSES AND METHODS OF USE THEREOF

Resumen de: US2025170235A1

The disclosure features isolated mRNAs encoding a polypeptide that enhances immune responses to an antigen(s) of interest, such as polypeptides that activate Type I interferon pathway signaling or NFkB signaling, including mRNAs comprising one or more modified nucleobase. The disclosure also features methods of using the same, for example, for enhancing immune responses when administered with an antigen(s) of interest, such as to stimulate anti-cancer immune responses or anti-pathogen immune responses.

BIOMIMETIC VESICLES AND USES THEREOF

Resumen de: US2025170270A1

The present disclosure relates to unimolecular core-shell nanoparticle, nanoclusters thereof, and platelet biomimetic nanoclusters thereof. The disclosed compositions are useful for treating a subject with a disease or condition, such as a cardiovascular disease. In a further aspect, the cardiovascular disease can be a vascular stenosis or restenosis. Also described herein are methods of making and using the unimolecular core-shell nanoparticle, nanoclusters thereof, and platelet biomimetic nanoclusters thereof. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present disclosure.

COMPOSITION

Resumen de: WO2024133635A1

A composition comprising: (a) an active ingredient; and (b) a lipid mixture comprising: (i) a cationically ionisable lipid capable of forming a lipid nanoparticle; (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 consecutive ethylene glycol repeating units, is provided.

CABAZITAXEL PRODRUG ANTI-TUMOR PREPARATION

Resumen de: AU2022412640A1

The present invention relates to a cabazitaxel prodrug anti-tumor preparation, designs and synthesizes a cabazitaxel-fatty alcohol small molecule prodrug containing different fatty alcohol side chains and different connecting chains of general formulas (I), (II) and (III), and prepares a self-assembly nanoparticle. A result shows that the cabazitaxel-fatty alcohol small molecule prodrug self-assembly nanoparticle can effectively improve a curative effect of cabazitaxel and reduce toxic and side effects thereof. The length of a side chain of a branched chain fatty alcohol, the structure of the side chain of the fatty alcohol, the composition of a connecting chain element and the length of a connecting chain can significantly affect the pharmaceutical property and the anti-tumor activity of the cabazitaxel prodrug self-assembly nanoparticle. The prodrug self-assembly nanoparticle has higher anti-tumor activity and lower toxicity than a cabazitaxel-linear fatty alcohol small molecule prodrug self-assembly nanoparticle.

SYNTHETIC SINGLE-STRANDED DNA MOLECULES AND METHODS OF PRODUCING AND USING THE SAME

Resumen de: AU2023406947A1

The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.

OPTIMIZED ENGINEERED MEGANUCLEASES HAVING SPECIFICITY FOR A RECOGNITION SEQUENCE IN THE HEPATITIS B VIRUS GENOME

Resumen de: US2025171757A1

The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and/or efficiency of indel formation, when compared to the first-generation meganuclease HBV 11-12x.26. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or hepatocellular carcinoma.

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE SIGNAL REGULATORY PROTEIN ALPHA (SIRPa) GENE

Resumen de: US2025171786A1

Provided herein, in various embodiments, are methods and compositions comprising a Signal Regulatory Protein Alpha (SIRPα) therapeutic. In certain embodiments, the disclosure provides for methods and compositions for modulating SIRPα expression. In certain embodiments, the methods and compositions are useful in the treatment of a SIRPα-mediated disease or condition.

POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE

Resumen de: US2025170070A1

This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

NANOPREPARATION FOR JOINT ANALGESIA, AND PREPARATION METHOD AND USE THEREOF

Resumen de: US2025170069A1

The present disclosure discloses a nanopreparation for joint analgesia, including glucocorticoid and a nanocarrier, where the nanocarrier is mainly composed of phospholipid and an auxiliary agent, and the auxiliary agent includes polyoxyethylene castor oil. The nanopreparation of the present disclosure can be used for joint analgesia, has a large drug load, and can be retained in the joint cavity for at least 4 weeks. Its analgesic effect on OA is significantly better than that of a commercially available GC injection and can be maintained for at least 4 weeks. Further disclosed is a preparation method and use of the nanopreparation. The process is simple, and the particle size is less than 200 nm. Terminal sterilization can be achieved by probe ultrasound, high-pressure homogenization or sterilization through a microfiltration membrane, and the like, and the preparation cost is relatively low.

NOVEL POLYGLYCEROL-CONJUGATED LIPIDS AND LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME

Resumen de: AU2023406321A1

The present disclosure provides novel polymer-conjugated lipids, e.g., comprising DODA conjugated to a polyglycerol or a polyglycerol derivative. The present disclosure also provides lipid nanoparticles (LNPs) formulation using the polymer-conjugated lipids and methods of treating a disease by administering the LNP formulations.

ULTRASOUND RESPONSIVE LIPID NANOPARTICLE CARRYING GENES AND CELL MEMBRANE, ITS PREPARATION METHOD AND APPLICATION

Nº publicación: US2025170071A1 29/05/2025

Solicitante:

HARBIN MEDICAL UNIV [CN]
Harbin Medical University

Resumen de: US2025170071A1

An ultrasound responsive lipid nanoparticle carrying genes and a cell membrane is provided, which can simultaneously carry small molecule nucleic acids and a cell membrane, and can be used as a gene transfection carrier in vivo. Its surface is connected with small molecule nucleic acids based on the principle of charge adsorption, a component of a cell membrane is mixed in a surface lipid layer, PFP is encapsulated inside the nanoparticle, liquid gas phase transition occurs under ultrasonic irradiation, and blasting occurs under continuous ultrasonic irradiation, the blasting energy generated forms reversible acoustic holes on the surface of the cell membrane, and a drug released after blasting is driven by a blasting driving force to directly enter an interior of the cell through the acoustic holes, realizing an efficient release and transfection of nucleic acids in the cell.