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一种预防白内障、黄斑病变和糖网病变的组合物以及制备方法和应用

Resumen de: CN121243255A

本发明提供了一种预防白内障、黄斑病变和糖网病变的组合物以及制备方法和应用，属于生物医药技术领域。本发明组合物，包括：纳米活性成分、植萃提取物、甘草酸二钾、烟酰胺、牛磺酸、依克多因、咖啡因和叶黄素；植萃提取物由野菊花提取物和龙胆提取物组成；纳米活性成分由纳米包裹虾青素和纳米视黄醇组成。本发明组合物通过抗氧化‑抗炎‑血管保护‑代谢调节的多维协同机制，有效抑制晶状体蛋白氧化变性、视网膜色素上皮细胞功能衰退及病理性血管生成等关键病理环节，从而实现对白内障、年龄相关性黄斑变性与糖尿病视网膜病变三种主要致盲眼病的预防与辅助治疗作用。

三螺旋结构元件在RNA药物设计中的应用

Resumen de: CN121249652A

本发明公开了一种三螺旋结构元件在RNA药物设计中的应用，属于生物医药领域。所述应用为：将ENE元件或其突变体作为核酸稳定元件，通过优化3’UTR与poly(A)的相互作用，增强mRNA稳定性并进一步提升蛋白翻译效率，从而有效解决RNA疗法中蛋白表达量低的问题。本发明提出的mRNA序列优化策略为传染病及肿瘤免疫治疗领域的疫苗设计提供了新方法，具有广泛的应用前景。

吸入药物组合物

Resumen de: AU2024279267A1

Disclosed is a pharmaceutical composition for inhalation, comprising lipid carriers comprising a pharmaceutical agent, the therapeutic uses thereof, and a method of making same.

用于递送核酸和其他治疗剂的含硫的可电离脂质

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.

含可裂解接头的可电离脂质和用于治疗性组合物的脂质载体

Resumen de: TW202438045A

The present disclosure relates to a lipid compound of formula (AL-GI):, having various cleavable linkers defined by the variables Z1 and Z2. The present disclosure also relates to a lipid carrier or lipid nanoformulation employing the lipid compound, and the use of the lipid compound in a pharmaceutical composition as well as for a method of delivering a therapeutic agent.

纳米颗粒组合物及相关方法

Resumen de: WO2024253582A1

There is provided a nanoparticle composition comprising a compound represented by general formula (1) or ionized form thereof, wherein R1, R2, and R4 to R8 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl, R3 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene, and R9 and R10 are each independently a hydrophobic tail or contains at least one of the groups defined above for R4 to R8; a therapeutic, prophylactic, and/or biological agent that is encapsulated by the compound of general formula (1) to form nanoparticles; and a cryoprotectant. There is also provided a method of preparing said nanoparticle composition.

一种基于N-乙酰半胱氨酸的可电离脂质、脂质纳米粒及制备方法与应用

Resumen de: CN121248455A

本发明提供一种基于N‑乙酰半胱氨酸的可电离脂质、脂质纳米粒及制备方法与应用。本发明所提供的全新结构的可电离脂质，其可形成粒径均一、结构稳定的LNP，转染效率高且生物相容性好。此外，所制备的LNP具有低免疫原性，能够引起免疫细胞(如巨噬细胞、树突状细胞)较低共刺激分子的表达。

LIPIDS AND LIPID NANOPARTICLES

Resumen de: WO2026003582A2

The disclosure provides for lipids that may be formulated in a delivery vehicle to facilitate the encapsulation of a wide range of single or multiple payloads including therapeutic, theragnostic, preventive, prophylactic, pre-emptive, and diagnostic agents, such as, without limitation, nucleic acids (e.g., RNA or DNA), proteins, peptides, and small molecule active pharmaceutical ingredients (APIs). Methods of delivering and/or producing a polypeptide of interest in a cell are also provided.

COMPOSITIONS AND METHODS FOR DELIVERY BY INHALATION

Resumen de: WO2026006634A1

The present disclosure provides, inter alia, compositions of LNPs that are suitable for nebulization, and associated methods of making and using the same. In certain aspects, the present disclosure provides LNPs, and compositions comprising them, comprising a simple block stabilizer, to form a LNP composition that is suitable for delivery of a nucleic acid therapeutic to a subject by nebulization, where the LNP remains stable post-nebulization. These compositions can be used to treat a variety of diseases and disorders by delivering a wide array of therapeutics, such as nucleic acid therapeutics, including nucleic acids encoding a protein sequence.

CONICAL SHAPED DIACYL GLYCEROETHYLPHOSPHOCHOLINES AS CATIONIC LIPIDS AND USES THEREOF

Resumen de: WO2026006196A1

A compound having the structure (I) is provided. The compound is useful as a therapeutic agent in the form of a lipoplex, or in a form of a polyplex, in a form of a lipid nanoparticle, or in a form of a liposome, or in a form of a micelle, or in a form of an emulsion including a drug, vaccine, oligonucleotide, antibody, enzyme, protein, small molecule, or nucleotide.

TARGETED POLYMERIC NANOPARTICLES LOADED WITH DUAL THERAPEUTIC AGENTS AND METHODS THEREOF

Resumen de: WO2026006790A2

Provided herein are nanoparticles comprising a triblock copolymer and a targeting moiety for targeted delivery of one or more therapeutic agents, and formulations thereof. Also provided herein are layered drug delivery vehicles. Further provided herein are methods, or uses thereof, for treating cancers, preferably pancreatic and brain cancers, by administration of the nanoparticles disclosed herein.

PHARMACEUTICAL COMPOSITIONS, DOSAGE FORMS, AND METHODS OF MAKING AND USING SAME

Resumen de: WO2026006759A1

Pharmaceutical compositions of Compound (I) including solid dispersions of Compound (I) and nanocrystalline powder forms of Compound (I). Related unit dosage forms, and methods of making and using the same are also provided.

ETHER-CONTAINING NOVEL IONIZABLE LIPIDS AND THEIR USES THEREOF

Resumen de: WO2026006579A1

Compounds are provided having the following structure: (I), or a pharmaceutically acceptable salt, prodrug or stereoisomer thereof, wherein Y, Q, R, Ri, R2, m, n and o 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. Formula (I).

LIPID NANOPARTICLE SPHERICAL NUCLEIC ACIDS FOR GENOME ENGINEERING

Resumen de: WO2026006618A1

Spherical nucleic acids (SNAs) are an attractive platform for therapeutic delivery due to their chemically tunable structures, biocompatibility, and ability to rapidly enter cells without transfection reagents. The present disclosure provides SNAs and strategies for delivering genome editor proteins into cells. Concurrent delivery of genome editor proteins and a single-guide RNA in an SNA platform allows for rapid entry into mammalian cells and effective genome modification.

METHOD FOR PRODUCING LIPID NANOPARTICLES

Resumen de: WO2026006555A1

The invention relates to a method for producing lipid nanoparticles (LNPs), comprising the steps of (a) performing a reference method for the preparation of LNPs, the reference method comprising the steps of (a1) introducing a first inlet stream of an aqueous solution via a first inlet port of a first mixing chamber, and introducing a second inlet stream of a lipid solution via a second inlet port of the first mixing chamber, thereby mixing the aqueous solution and the lipid solution so as to produce LNPs, and (a2) recovering a first outlet stream comprising the produced LNPs via an outlet port of the first mixing chamber, and (b) producing LNPs according to the reference method in a second mixing chamber, wherein the inner diameters of the first inlet port, second inlet port and outlet port of the second mixing chamber are each by the same factor proportionally bigger by at least 5% than the inner diameters of the first inlet port, second inlet port and outlet port of the first mixing chamber, wherein the linear flow rate of the first outlet stream and the second outlet stream are essentially identical.

BIS-ESTER AND AMIDE CATIONIC LIPIDS

Resumen de: WO2026006524A2

The present invention provides, in part, cationic lipid compounds of Formulae (I), (II), (III), (IV), (V), or (VI) or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

LYOPHILIZED AND FROZEN MRNA-LNP FORMULATIONS

Resumen de: WO2026006617A1

The present invention relates to lyophilized and frozen pharmaceutical formulations comprising a nucleic acid (NA)-lipid nanoparticle (LNP) particle, wherein the NA-LNP particle comprises a nucleic acid encapsulated in the lipid nanoparticle, wherein the formulation further comprises an excipient comprising a lyoprotectant or cryoprotectant and a buffer, and wherein the lyoprotectant or cryoprotectant is a polyvinylpyrrolidone (PVP) or a polyethylene glycol (PEG). Methods of making the lyophilized and frozen formulations are also provided.

NANOENCAPSULATION OF OLIVE PHENOLIC COMPOUNDS IN PROTEIN MATRICES

Resumen de: WO2026003770A1

A formulation for nutraceutical and/or pharmaceutical use based on protein nanoparticles is described, including a bioactive compound chosen from hydroxytyrosol, tyrosol, olive phenolic compounds, and mixtures thereof, and a zein protein matrix stabilized with sodium deoxycholate.

COMPOSITION FOR TARGETED DELIVERY OF THERAPEUTIC COMPOUNDS TO BONE TISSUE

Resumen de: WO2026002313A1

A system and method are disclosed for the production of drug delivery vesicles formed from hydroxyapatite (Ca10(PO4)6(OH)2), called apasomes, using a microfluidic device. The device comprises a first inlet with a bifurcated tube and a second inlet with an inlet tube. Hydroxyapatite nanoparticles and, optionally, therapeutic agents suspended in a second fluid are introduced into the device via the second inlet, while a miscible first fluid is introduced via the first inlet. The two fluids are combined at a mixing point within the device and the combination fluid travels through an elongate tube before exiting the device. Shear forces cause self-assembly of vesicles, the size of which can be modified by adjusting the flow rates, the shapes and dimensions of the tube, and the composition of the fluids. The method may also include refluxing, filtration and drying steps to enable scalable production of customizable drug delivery vesicles for therapeutic applications.

IONIZABLE LIPIDS AND LIPID NANOPARTICLES CONTAINING THEM FOR EXTRAHEPATIC DELIVERY

Resumen de: WO2026003403A1

It is provided a novel lipid of formula (I) or a pharmaceutically acceptable salt thereof, or a stereoisomer of any one of them, and a nanoparticle comprising the ionizable lipid, particularly as encapsulating agent, optionally comprising a molecule or a pharmaceutically active inside. It is also provided a lipid nanoparticle or a pharmaceutical composition comprising thereof for use in medicine, and the use of the lipid nanoparticles as encapsulating agents. Formula (I)

PHENOLIC ACID LIPID BASED CATIONIC LIPIDS

Resumen de: WO2026003373A1

The present invention provides lipids of Formula (I) to (IX) or subformulae thereof, or pharmaceutically acceptable salts thereof. Said lipids can be useful for the delivery and expression of mRNA and encoded protein, e.g. as a component of liposomal delivery vehicles, and accordingly can be useful for treating various diseases, disorders, and conditions, such as those associated with deficiency of one or more proteins. Compositions comprising lipid compounds of Formulae (I) to (X) and subformulae thereof, and mRNA encoding a peptide or protein may be administered to subjects in need thereof intranasally.

PIPERAZINE BASED DIVALENT IONIZABLE LIPIDS AND THEIR USES

Resumen de: WO2026005944A1

A compound having the structure (I) is provided. The compound is useful as a therapeutic agent in the form of a lipoplex, or in a form of a polyplex, in a form of a lipid nanoparticle, or in a form of a liposome, or in a form of a micelle, or in a form of an emulsion including a drug, vaccine, oligonucleotide, antibody, enzyme, protein, small molecule, or nucleotide.

IN PLANTA NANO-DELIVERY OF NUCLEIC ACIDS INTO WHEAT AND OTHER CEREAL CROP SEED

Resumen de: WO2026006565A1

A method of introducing a polynucleotide into wheat seed using nanoparticles, e.g., mesoporous silica nanoparticles or silica nanoparticles comprising a polynucleotide, e.g., a polynucleotide for use in gene editing, transgene insertion, upregulation of gene expression, or downregulation of gene expression.

ALDEHYDE DEHYDROGENASE 2 ENZYMES, ALCOHOL DEHYDROGENASE 1 ENZYMES, NUCLEIC ACIDS ENCODING ENZYMES, LIPID NANOPARTICLES, AND METHODS OF USE

Resumen de: WO2026006513A2

Alcohol dehydrogenase 1 enzymes, aldehyde dehydrogenase 2 enzymes, nucleic acids encoding alcohol dehydrogenase 1 enzymes and aldehyde dehydrogenase 2 enzymes, lipid nanoparticles, lipid nanoparticles comprising nucleic acids encapsulated therein, pharmaceutical compositions comprising lipid nanoparticles which comprise nucleic acids encapsulated therein are useful for treating alcohol-related conditions, such as alcohol poisoning.

BRAIN TARGETED METABOLIC INHIBITION OF CANCER STEM CELLS TO DESTROY THEIR STEMNESS INDUCED TUMORIGENICITY

Nº publicación: WO2026006393A1 02/01/2026

Solicitante:

UNIV OF MIAMI [US]
UNIVERSITY OF MIAMI