Alerta

COMPOSITIONS AND METHODS FOR TREATING PULMONARY CONDITIONS

Resumen de: AU2024213596A1

A method of treating a pulmonary condition in a subject having or at risk of having the pulmonary condition generally includes administering to the subject a therapeutic composition in an amount effective to treat the pulmonary' condition. Generally, the therapeutic composition includes purified exosome product (PEP) exosomes and a pharmaceutically acceptable carrier. In one or more embodiments, the PEP exosomes are modified to include at least one exogenous active agent. In one or more embodiments, the therapeutic composition is formulated for delivery directly to a potion of tire pulmonary tract.

IN VITRO RECONSTITUTED ENVELOPED VIRUS-LIKE PARTICLES (EVLPS) FOR RNA GENE DELIVERY

Resumen de: WO2024163754A1

The invention provides compositions of matter comprising a lipid enveloped virus capsid protein and a ribonucleic acid, as well as methods for using such compositions. In illustrative compositions, the lipid enveloped virus capsid protein envelops the ribonucleic acid so as to for a capsid that can inhibit the degradation of the ribonucleic acid (e.g. by RNAses). A method of delivering a ribonucleic acid into the cytoplasm of a mammalian cell is also provided. Typically, the method comprises the steps of combining the mammalian cell with a composition of matter described herein under conditions selected to allow the lipid enveloped virus capsid to contact the mammalian cell and deliver the ribonucleic acid into the cytoplasm of a mammalian cell.

COMPOSITIONS AND METHODS FOR TREATING HYPERPROCALCITONEMIA

Resumen de: MX2025008776A

A composition for treating hyperprocalcitonemia is described. The composition comprises a lipophilic or hydrophobic component, an amphiphilic emulsifier, a polar liquid carrier, and with or without one or more electrolytes, where the amphiphilic emulsifier forms micelles having a lipophilic or hydrophobic core comprising the lipophilic or hydrophobic component in the polar liquid carrier, and /or liposomes organized as a lipid bilayer and/or other particle configurations.

CARBAMOYL LIPID OR UREA LIPID EACH HAVING CYCLIC AMINE, LIPID NANOPARTICLES CONTAINING SAME, AND PHARMACEUTICAL COMPOSITION

Resumen de: EP4660189A1

The present invention addresses the problem of creating a carbamoyl lipid or a urea lipid each having a cyclic amino and developing lipid nanoparticles, and thereby providing a pharmaceutical composition for nucleic acid therapeutics and others. The present inventors have discovered a compound that is a carbamoyl lipid or a urea lipid each having a cyclic amino or a salt of the compound, and have studied on lipid nanoparticles that can be used in various pharmaceutical compositions. As a result, it was revealed that lipid nanoparticles can be formed using the compound of the present invention, which is a lipid, or a salt of the compound. By using the lipid nanoparticles containing the carbamoyl lipid or the urea lipid each having a cyclic amino according to the present invention, it is expected that a pharmaceutical composition containing the lipid nanoparticles each encapsulating a nucleic acid therein can be used as a prophylactic or therapeutic agent for astrocyte-related diseases.

BIODEGRADABLE COMPOUNDS, METHOD OF PREPARATION AND USE THEREOF

Resumen de: WO2024161416A1

The present invention relates to a caprolactone based biodegradable compound(s) of formula (I) comprising photo-cleavable groups, photo-sensitive groups or combination thereof. The present invention also relates to a method of preparing the caprolactone based biodegradable compound(s) of formula (I). In addition, the present invention relates the caprolactone biodegradable compound(s) of formula (I) useful in the pharmaceutical field in controlled release systems, drug delivery system/carrier, bioimaging, implants, etc.

P-SELECTIN TARGETED NANOPARTICLES AND USES THEREOF

Resumen de: US2025352490A1

Particles made of a polymeric matrix having associated therewith a therapeutically active agent usable in treating a medical condition associated with an overexpression of P-selectin in a subject in need thereof and featuring a P-selectin selective targeting moiety represented by Formula I as defined and described in the specification and claims, compositions comprises these particles and uses thereof, are provided.

LIPID COMPOUNDS AND USES THEREOF

Resumen de: WO2024161249A1

Compounds are provided having the following structure Formula (I) or a pharmaceutically acceptable salt, N-oxide, tautomer or stereoisomer thereof, wherein G1, G2, G3, L1, L2, R1, R2, R3, W and Y are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a nucleic acid, compositions comprising the compounds and methods for their use and preparation are also provided.

HSC-SPECIFIC ANTIBODY CONJUGATED LIPID NANOPARTICLES AND USES THEREOF

Resumen de: AU2024214423A1

Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., HSC), and methods of making and using such lipids and targeted lipid. nanoparticles.

RNA FORMULATION

Resumen de: WO2024160936A1

The present invention provides improved RNA-LNP formulations with lower amounts of RNA adduct, As well as methods and uses to reduce the amount of RNA adduct in RNA- LNP formulations, in particular mRNA-LNP formulations.

COMPOSITIONS AND METHODS

Resumen de: CN120603581A

An aqueous dispersion having an aqueous mobile phase and a dispersed phase is described; wherein the dispersed phase comprises a lipid mixture comprising a cationically ionizable lipid; and the aqueous mobile phase comprises anions of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents, and RNA. Methods of making the aqueous dispersions, nucleic acid-lipid particles and methods of making them using the aqueous dispersions, and their use in medicine are disclosed.

COMPOSITIONS AND METHODS

Resumen de: CN120603581A

An aqueous dispersion having an aqueous mobile phase and a dispersed phase is described; wherein the dispersed phase comprises a lipid mixture comprising a cationically ionizable lipid; and the aqueous mobile phase comprises anions of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents, and RNA. Methods of making the aqueous dispersions, nucleic acid-lipid particles and methods of making them using the aqueous dispersions, and their use in medicine are disclosed.

COMPOSITIONS AND METHODS

Resumen de: CN120603581A

An aqueous dispersion having an aqueous mobile phase and a dispersed phase is described; wherein the dispersed phase comprises a lipid mixture comprising a cationically ionizable lipid; and the aqueous mobile phase comprises anions of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents, and RNA. Methods of making the aqueous dispersions, nucleic acid-lipid particles and methods of making them using the aqueous dispersions, and their use in medicine are disclosed.

NANOPARTICLES DIRECTED TO CXCR4 AND USE THEREOF

Resumen de: WO2024161391A1

Nanoparticles comprising an outer surface covalently conjugated to 1,4-Bis(1,4,8,11-tetraazacyclotetradecan-l-yl)methylbenzene (AMD3100) or a derivative thereof capable of binding to C-X-C chemokine receptor type 4 (CXCR4) are provided. Methods for of treating a CXCR4 positive cancer, such as multiple myeloma or acute myeloid leukemia, in a subject in need thereof, methods of determining suitability for treatment and methods of covalently linking a molecule comprising a secondary amine to a lipid nanoparticle, are also provided.

一种用于猪繁殖与呼吸综合征防控的可吸入式重组小RNA-脂质纳米递送系统及其制备方法和应用

Resumen de: CN121081429A

本发明涉及生物医药技术领域，具体涉及一种用于猪繁殖与呼吸综合征防控的可吸入式重组小RNA‑脂质纳米递送系统及其制备方法和应用。通过微流控技术将溶于有机相的脂质与溶于水相筛选得到的重组小RNA进行混合，得到具有治疗效果的重组小RNA‑脂质纳米递送系统。本发明用于猪繁殖与呼吸综合征防控的可吸入式重组小RNA‑脂质纳米递送系统，可以通过抑制猪蓝耳病病毒在细胞内复制，降低蓝耳病病毒滴度以及PRRSV蛋白水平，且脂质纳米颗粒可以稳定包封重组小RNA，为猪蓝耳病防控药物研发提供了新策略和吸入式给药的脂质纳米递送系统。

基于中药与铜离子自组装的抗氧化纳米材料及其制备方法

Resumen de: CN121081418A

本发明属于纳米医学与中药领域，具体公开一种基于中药与铜离子自组装的抗氧化纳米材料及其制备方法，该纳米材料由小檗碱和原花青素B2在铜离子诱导下自组装形成的BPCu纳米颗粒，进一步通过聚乙烯吡咯烷酮进行表面修饰，得到稳定分散的PVP@BPCu纳米结构。该材料粒径小、分布均一、水溶性和储存稳定性良好，具有优异的自由基清除能力及类过氧化氢酶活性，适用于氧化应激相关疾病的辅助治疗，且本发明制备方法操作简便、无需有机溶剂，具有良好的产业化及疾病治疗潜力。

一种RSV-流感联合疫苗及其应用

Resumen de: CN121081615A

本申请涉及生物医药领域，具体公开一种RSV‑流感联合疫苗及其应用。所述RSV‑流感联合疫苗包括呼吸道合胞病毒mRNA疫苗和流感疫苗，所述呼吸道合胞病毒mRNA疫苗中还包括脂质纳米颗粒，所述mRNA装载于所述脂质纳米颗粒中。所述RSV‑流感联合疫苗用于预防多种呼吸道病毒的感染(RSV病毒和流感病毒)，并且RSV mRNA疫苗的LNP佐剂可大幅增加流感疫苗的免疫应答，增强其在免疫低下人群中的防护效率。

一种犬尿氨酸酶-Fc融合蛋白、其纳米药物制剂、制备方法和应用

Resumen de: CN121087011A

本发明属于生物医药和药物制剂领域，涉及一种犬尿氨酸酶‑Fc融合蛋白、其纳米药物制剂、制备方法和应用。犬尿氨酸酶‑Fc融合蛋白由犬尿氨酸酶（KYNase）和人免疫球蛋白G的Fc结构域连接组成。相较于PEG修饰犬尿氨酸酶，犬尿氨酸酶‑Fc融合蛋白可在保持原有KYNase活性的同时，显著延长KYNase在全血中的半衰期，增加药物在肿瘤部位的浓度，提高KYNase的抗肿瘤效果。基于犬尿氨酸酶‑Fc融合蛋白的纳米药物制剂由犬尿氨酸酶‑Fc融合蛋白、光敏剂组成。该纳米药物制剂通过非共价键结合方式包载药物光敏剂，提高光敏剂在肿瘤部位富集，不改变光敏剂在全血中的半衰期。本发明提供的犬尿氨酸酶‑Fc融合蛋白可用于抗肿瘤免疫治疗，增强了肿瘤光动力治疗联合犬尿氨酸酶的免疫治疗效果。

纳米复合物颗粒及其制备方法，在制备治疗癌症药物中的应用

Resumen de: CN121081431A

本发明公开了一种纳米复合物颗粒及其制备方法，在制备治疗癌症药物中的应用。纳米复合物颗粒，包括：由大麻二酚形成的核和包覆在所述核外部的包覆层，所述包覆层由氧化锌纳米粒子形成。通过绿色合成工艺制备ZnO纳米粒子，并进一步对其进行稀释和包封CBD，形成药物负载型ZnO/CBD复合物。通过动态光散射(DLS)测定其粒径分布，并利用Zeta电位分析其表面电荷特征，从而表征其稳定性。同时，采用透射电子显微镜(TEM)成像对所合成的ZnO/CBD进行结构分析，确认其纳米颗粒形貌及药物包封成功。制备后的ZnO/CBD复合物可用于后续的细胞实验，包括表型分析、细胞活性检测及3D球体模型观察等。

一种基于线型-树枝状聚合物的肿瘤微环境响应型纳米药物

Resumen de: CN121081664A

本发明属于生物医药技术领域，具体涉及一种基于线型‑树枝状聚合物的肿瘤微环境响应型纳米药物。本发明将树枝状片段通过酶可切割的肽链连接至聚合物骨架，化疗药物通过pH不稳定性腙键偶联至树枝状外围，获得两亲性聚合物前药；通过筛选聚合单体，获得具有稳定的纳米粒，其可被肿瘤细胞高效摄取，并在肿瘤组织穿透和抗肿瘤方面具有优势；该聚合物前药还可装载腺苷受体拮抗剂药物，制得纳米药物，该纳米药物逆转腺苷介导的免疫抑制，实现腺苷受体拮抗剂药物与化疗药物的协同效应；纳米药物与PD‑1抗体协同，进一步改善了体内的免疫应答和抗TNBC的效果。本发明合成的聚合物前药和纳米药物在治疗TNBC方面前景良好。

脂质纳米颗粒制剂及其制备方法

Resumen de: WO2025214340A1

A lipid nanoparticle formulation having remarkably improved stability and a preparation method therefor. The lipid nanoparticle formulation comprises a pharmaceutically acceptable buffer solution, lipid nanoparticles and a nucleic acid encapsulated in the lipid nanoparticles.

用于体内递送的递送载体高通量筛选平台

Resumen de: WO2024235034A1

Provided is a method for characterizing delivery vehicles for in vivo delivery of an agent. The methods can be used to characterize various properties of a wide variety of delivery vehicles.

预活化小胶质细胞膜包被负载人参皂苷的纳米颗粒和制备方法及其在抑郁症中的应用

Resumen de: CN121081415A

本发明公开了预活化小胶质细胞膜包被负载人参皂苷的纳米颗粒和制备方法及其在抑郁症中的应用，包括如下步骤：将预活化的小胶质细胞膜包被于人参皂苷Rb1的纳米颗粒表面制备得到预活化小胶质细胞膜包被负载人参皂苷的纳米颗粒。本发明能将GRb1@PLGA和GRb1@PLGA@AM招募至抑郁小鼠的脑部，从而增加GRb1@PLGA@AM在炎性病变部位的积蓄，实现高效抑郁症炎症部位的靶向功能，不但能够有效抑制抑郁的进展，且具有良好的生物安全性。

一种微环境响应型释放硫化氢的多功能靶向纳米粒子及其制备方法和应用

Resumen de: CN121081411A

本发明公开了一种微环境响应型释放硫化氢的多功能靶向纳米粒子及其制备方法和应用，步骤：(1)制备Poly‑Lys‑Cbz；(2)制备α‑PLL；(3)制备PB‑TCD；(4)取α‑PLL、SPDP‑PEG‑NHS、DMSO和三乙醇胺反应；加入PB‑TCD和4‑二甲氨基吡啶反应；反应液透析，干燥，获得聚合物1；(5)将聚合物1和CGRGDS多肽溶于DMSO中，透析，获得聚合物2；(6)将聚合物2溶解在DMSO中，得到溶液1，透析，得到一种微环境响应型释放硫化氢的多功能靶向纳米粒子；本发明的纳米粒子具有良好的生物相容性，有效靶向到气体爆炸造成的急性肺损伤处，并有效下调ROS，清除细胞外游离DNA。

阳离子脂质材料在TLR9激动剂组合物中的应用

Resumen de: CN121081409A

本发明涉及生物医药领域，特别是一种可用于核酸递送的阳离子脂质及其在TLR9激动剂组合物中的应用，以及所述组合物的制备方法和应用。通过本发明的阳离子脂质所制备的TLR9激动剂LNP佐剂配伍疫苗与单独疫苗相比有较为显著的免疫增强效果，且与市售的疫苗佐剂以及由市售的LNP产品中所用的磷脂制备的佐剂相比，有着更强的免疫效力、更长的持续时间，以及对毒性及其分布控制上的优势。

AAV PIGGYBAC转座子多核苷酸组合物及其使用方法

Nº publicación: CN121100184A 09/12/2025

Solicitante:

波西达治疗公司

Resumen de: MX2025009590A

The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV â¿¿ piggyBac transposon polynucleotide vectors and LNP compositions comprising a nucleic acid encoding a transposase, and methods of using the compositions for treating PKU.