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薬物を送達するための化合物、リポソーム及び薬物担体

Resumen de: US2025134813A1

The present invention provides a compound, which is a compound represented by formula (I) or a stereoisomer, tautomer, solvate, or pharmaceutically acceptable salt thereof represented by formula (I):X1, X2, and X3 are each independently a C1-C15 alkyl group optionally substituted; R1 and R2 are each independently a C1-C40 alkyl group optionally substituted, a C1-C40 heteroalkyl group optionally substituted, a C2-C40 alkenyl group optionally substituted, a C2-C40 heteroalkenyl group optionally substituted, a C2-C40 alkynyl group optionally substituted, or a C2-C40 heteroalkynyl group optionally substituted; R3, R4, R5, and R6 are each independently H, halogen, or a C1-C3 alkyl group optionally substituted; the substituent group is independently selected from halogen, —OH, —SH, —NH2, —NO2, cyano, or C1-C3 alkyl. This compound has the advantages of low cytotoxicity, strong delivery capability, and good immunostimulatory effect.

AMINO LIPID, AND LIPID NANOPARTICLES AND USE THEREOF

Resumen de: EP4685136A1

The present invention provides an amino lipid, and lipid nanoparticles (LNPs) and a use thereof, the amino lipid having a structure represented by general formula (I), or an isomer, pharmaceutically acceptable salt, prodrug or solvate of the amino lipid. The present invention further provides LNPs containing the amino lipid. According to the present invention, the amino lipid having a structure represented by general formula (I) is used as an ionizable lipid compound, and the LNPs are obtained by means of self-assembly of the ionizable lipid compound, a steroid, a neutral lipid, and a polymer-bonded lipid. The LNPs can further improve the translation expression level of a nucleic acid load in cells, improve the effect of a nucleic acid-LNP preparation, and enable the nucleic acid-LNP preparation to provide a theoretical basis for personalized treatment.

IN VIVO NICKASE-BASED EDITING OF THE LPA GENE FOR TREATMENT OF CARDIOVASCULAR DISEASE

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.

IONIZABLE ANIONIC LIPIDS

Resumen de: WO2024192528A1

The present disclosure provides lipid nanoparticle for delivery of nucleic acid having an ionizable anionic lipid Further provided are ionizable anionic lipid having the structure of Formula A described herein.

DNA NANODEVICES FOR SPECIFIC AND EFFICIENT DELIVERY OF FUNCTIONAL PAYLOADS TO THE CYTOPLASM AND METHODS OF THEIR USE

Resumen de: WO2024196422A1

A novel nanostructured nucleic acid platform that uniquely integrates the advantages of a disulfide moiety for the enhanced cytosolic uptake of a DNA or RNA nanostructure ("DNA origami") which can further comprise a therapeutic agent and a targeting moiety is described herein. The targeting moiety can be an affibody, in particular, a Her2 affibody. The disulfide moiety can be formed from a sulfide-modified oligonucleotide designed to target enhanced cystolic uptake.

LIPID COMPOUNDS, LIPID NANOPARTICLES, AND PHARMACEUTICAL COMPOSITIONS

Resumen de: CN120659770A

Provided herein are lipid compounds, such as compounds of Formula (I), Formula (II), Formula (III), Formula (IV), or Formula (V). Also provided are lipid nanoparticles and pharmaceutical compositions, each comprising a lipid compound, such as a compound of Formula (I), Formula (II), Formula (III), Formula (IV), or Formula (V). # imgabs0 #

ｍＲＮＡ担持脂質ナノ粒子の安定性組成物および作製のプロセス

Resumen de: US2025049954A1

The present invention provides an improved compositions and processes for preparing mRNA-loaded lipid nanoparticles (mRNA-LNPs). In some embodiments, the present invention provides mRNA-LNPs with exceptional stability and is particularly useful in cases where LNPs comprising low or no PEG-modified lipids are desired.

一种基于NK细胞膜包裹碳酸锰的免疫增敏IRE治疗平台

Resumen de: CN121401233A

本发明涉及一种基于NK细胞膜包裹碳酸锰的免疫增敏IRE治疗平台，所述碳酸锰复合纳米材料为碳酸锰纳米颗粒表面包覆细胞膜。本发明实现了更高的肿瘤特异性富集，降低了非靶组织蓄积与系统毒性风险，实现了肿瘤靶向递送、免疫激活和IRE消融的协同整合，较单一IRE显著增强抗肿瘤免疫反应。

一种含STING佐剂的mRNA疫苗及其组合物和应用

Resumen de: CN121401406A

本发明提供了一种含STING佐剂的mRNA疫苗及其组合物和应用，通过胆固醇偶联的方式构建了一种含STING佐剂的mRNA疫苗，不仅改善了STING佐剂的体内稳定性和生物利用度，还能与mRNA协同诱导强效免疫反应。将该疫苗与TCR/CAR融合的工程化T细胞联合治疗实体肿瘤，能够实现TCR/CAR‑T细胞的体内大量扩增，增强TCR/CAR‑T细胞的肿瘤杀伤功能，提高抗肿瘤效果，弥补了现有TCR/CAR融合的工程化T细胞在体内稳定性、长效性及抗实体肿瘤等方面的不足，显示出其作为工程化T细胞增强性疫苗的巨大潜力，为实体肿瘤的治疗提供了新的技术方案，具有显著的临床应用潜力。

一种含纳米颗粒-药物偶联物的药物及其制备方法、应用

Resumen de: CN121401234A

本发明涉及纳米医学和免疫治疗领域。具体涉及一种含纳米颗粒‑药物偶联物的药物及其制备方法、应用。所述的纳米颗粒‑药物偶联物其包含：金纳米颗粒核心、聚合物涂层、以及至少一个通过接头共价连接至所述聚合物涂层的有效载荷分子，本发明首次提出了利用纳米颗粒固有靶向性来递送细胞毒性药物以选择性消除ABCs的构思以及药理学实验，临床价值较高。

具有广谱抗菌作用的复合物、纳米制剂及其制法与应用

Resumen de: CN121401235A

本发明属于纳米生物医药技术领域，具体涉及一种具有广谱抗菌作用的复合物、纳米制剂及其制法与应用。该复合物包括抗菌组分、载体和表面正电荷修饰剂。其中，抗菌组分为镓，载体包括2,3‑二羟基苯甲酸，表面正电荷修饰剂优选聚乙烯亚胺。本发明采用镓离子为核心抗菌组分，以细菌铁载体前体DHBA为“木马”载体，并利用PEI进行表面正电荷修饰，通过一步水热法或溶液自组装法，合成镓‑酚酸复合纳米颗粒。该镓‑酚酸复合纳米颗粒具有广谱高效、成本低廉、制备简便、不易产生耐药性、生物相容性好等优点，在抗菌药物和医用敷料等领域具有广阔的应用前景。

共载丙酮酸脱氢酶激酶抑制剂和米托蒽醌的纳米载药系统及其制备方法和应用

Resumen de: CN121401448A

本发明涉及一种共载丙酮酸脱氢酶激酶抑制剂和米托蒽醌的纳米载药系统及其制备方法和应用，属于药物制剂技术领域。通过简单快速的方法先后将丙酮酸脱氢酶激酶抑制剂和米托蒽醌共同荷载于金属有机框架ZIF‑90纳米材料，制备纳米载药系统。该纳米载药系统能够靶向肿瘤细胞线粒体，在酸性肿瘤微环境中响应释放药物，通过代谢重编程及氧化还原失衡的协同作用，实现化疗‑糖代谢治疗的多模式协同治疗，显著增强抗肿瘤效果。

一种CXCL13 mRNA药物及其在肿瘤治疗中的应用

Resumen de: CN121401451A

本发明涉及一种CXCL13mRNA药物及其在肿瘤治疗中的应用。本发明提供一种脂质颗粒，其包括可电离脂质、磷脂、胆固醇、PEG‑脂质和包封在所述脂质颗粒内的CXCL13mRNA。本发明还提供包括所述脂质颗粒的药物组合物及其用途。本发明提供的包含CXCL13mRNA药物尤其适于治疗包括肺癌、结直肠癌、乳腺癌、肝癌、胰腺癌、头颈癌、脑胶质瘤和黑色素瘤等在内的肿瘤。

一种负载姜黄素的玉米醇溶蛋白-天然胶纳米复合物的制备方法

Resumen de: CN121400573A

本发明提供一种负载姜黄素的玉米醇溶蛋白‑天然胶纳米复合物的制备方法，涉及食品科学与工程技术领域，包括以下步骤：(1)玉米醇溶蛋白(zein)和虫胶(Shellac)共架载体复合物的制备；(2)负载姜黄素的纳米复合物的制备：将姜黄素通过pH循环法负载入玉米醇溶蛋白‑虫胶共架载体中，得到复合纳米颗粒溶液。本发明基于玉米醇溶蛋白在纳米包封技术方面的巨大潜力，运用相对温和的pH循环法引入天然胶对其进行共组装，保留蛋白质的营养特性的同时实现姜黄素的有效包埋，提高姜黄素的水溶性及稳定性。本发明无需引入有机溶剂，具有操作简单、绿色安全、效果显著、成本低廉的优点。

一种超声响应ZnS@Lf压电纳米颗粒及其制备方法和应用

Resumen de: CN121401437A

本发明公开了一种超声响应ZnS@Lf压电纳米颗粒及其制备方法和应用，涉及压电材料技术领域。该超声响应ZnS@Lf压电纳米颗粒包括ZnS纳米粒子和负载于ZnS纳米粒子表面的乳铁蛋白。本发明以乳铁蛋白作为强效的脑靶向配体，通过脑毛细血管内皮细胞的转胞作用穿过血脑屏障积聚于脑实质。ZnS纳米粒子在超声作用下释放电荷，电信号激活电压门控钙离子通道，胞外钙离子内流，钙离子/钙调蛋白激酶诱导酪氨酸羟化酶（TH）磷酸化，增强酪氨酸羟化酶的活性，而酪氨酸羟化酶是生成多巴胺的关键酶，因此体内酪氨酸在该酶的作用下更多地转化成神经递质多巴胺。最终实现多巴胺能神经元的再生，使帕金森病得到有效改善。

用于预防肠道病毒A71感染的mRNA疫苗组合物及其制备方法与应用

Resumen de: CN121401402A

本发明涉及一种用于预防肠道病毒A71（EV‑A71）感染的mRNA疫苗组合物。该组合物包括编码EV‑A71的VP1蛋白的mRNA以及含特定阳离子脂质的脂质纳米颗粒（LNP）。与常规阳离子脂质体系相比，本发明采用具有强膜融合能力的咪唑并吡啶基阳离子脂质A5，能够在单次、低剂量免疫条件下诱导显著的中和抗体及细胞免疫反应，表现出优越的安全性与儿童适用性。

一种杂化仿生纳米递送系统及其制备方法

Resumen de: CN121401236A

本申请提供了一种杂化仿生纳米递送系统及其制备方法，所述杂化仿生纳米递送系统由碳点掺杂的碳化氮基纳米粒内核以及包裹碳化氮基纳米粒内核的细胞膜组构成。本发明构建了一种杂化仿生纳米递送系统，具有良好生物相容性和生物代谢性，有利于在生物医学领域应用，可实现肝纤维化的低温光热治疗。本申请所构建的氮化碳基纳米仿生系统主要由高温煅烧、超声处理和杂化细胞膜包裹合成。本发明的制备方法简单，具有良好的可重复性，具有较好的工业化生产前景。

一种花生四烯酸脂质纳米递药系统的制备方法

Resumen de: CN121401237A

本发明公开了一种花生四烯酸脂质纳米递药系统的制备方法，涉及生物技术领域，所述脂质纳米递药系统包括以下组分：（1）可电离脂质；（2）花生四烯酸；（3）非阳离子脂质或可电离脂质的抑制颗粒聚集的缀合的脂质；（4）除（3）以外的非阳离子脂质或非可电离脂质；（5）金属离子；（6）核酸药物；所述脂质纳米递药系统由至少包括上述（1）至（6）组分混合后自组装形成。本发明提供了一种新的脂质纳米递药系统的制备方法，通过脂质替换及同时使用核核酸药物和金属离子，特异性杀伤癌细胞，提升治疗精准度，能增加稳定性以及溶酶体逃逸率。

负载黄芪甲苷的黄芪外泌体、其制备方法、以及其在制备治疗脑缺血再灌注损伤药物中的应用

Resumen de: CN121401238A

本发明涉及一种负载黄芪甲苷的黄芪外泌体，破碎黄芪，过滤获得汁液；多步差速离心得到上清液；超滤、离心，收集沉淀；加至蔗糖阶梯梯度液上层，离心，收集部分条带；稀释后离心并弃上清，沉淀、去除蔗糖，得到黄芪外泌体；与黄芪甲苷混合，超声或共孵育后得到负载黄芪甲苷的黄芪外泌体，可用于制备特异性抑制脑缺血再灌注损伤中cGAS/STING通路过度激活及其下游NLRP3炎症小体介导的细胞焦亡的药物，以及制备保护、修复脑缺血再灌注损伤中线粒体的药物。本发明利用同源的黄芪外泌体作为天然载体，有效克服血脑屏障，显著提高了黄芪甲苷在脑缺血区域的富集浓度和生物利用度。

一种用于预防或治疗癌症及细胞过度增殖相关疾病的包含17-AAG和星形孢菌素的药物组合物，以及含有该组合物的聚合物-脂质纳米颗粒

Resumen de: WO2024228630A1

The description of the invention discloses a composition comprising 17- AAG and staurosporine for use in the treatment of melanoma, non-small cell lung cancer, pancreatic cancer, mammary gland tumours or gliomas, and a stable polymer-lipid nanoparticle for drug binding and delivery, consisting of a core containing polylactic-co-glycolic acid (PLGA) and polyvinyl alcohol (PVA) and a core envelope containing a mixture of lipids: 1,2-dipalmitoyl-sn-glycero-3-phosphocholine (DDPC), cholesterol and the ammonium salt 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N - amino(polyethylene glycol)-2000 (DSPE-PEG(2000)NH2J.

用于核酸递送的基于二脱水己糖醇的可电离脂质

Resumen de: TW202517243A

The present invention provides, in part, dianhydrohexitol-based cationic lipids of Formula (I), and sub-formulas thereof:, or a pharmaceutically acceptable salt thereof. The present invention also provides, in part, dianhydrohexitol-based cationic lipids of Formula (II), and sub-formulas thereof:, 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.

ヒトに予備保存されている抗ＰＥＧ抗体を回避する方法

Resumen de: CN120712105A

The invention relates to a method for avoiding a human body pre-stored anti-PEG antibody and application of a terminal hydroxyl group-containing PEGylated nano-carrier in preparation of a medicine for avoiding the human body pre-stored anti-PEG antibody. The composition contains a terminal hydroxyl group-containing PEGylated nano-carrier and a nano-preparation. The terminal hydroxyl group-containing PEGylated nano-carrier and the nano-preparation are low in combination with the anti-PEG antibody pre-stored in the human body, so that the anti-PEG antibody can be prevented from being quickly cleared in the blood of the human body, and the treatment effect can be better exerted. Besides, the PEGylated nano-carrier containing terminal hydroxyl and the nano-preparation can relieve complement activation by avoiding combination with a pre-stored anti-PEG antibody in human blood, so that side effects such as clinical injection reaction and the like are relieved.

融合前安定化ヒトパラインフルエンザウイルス３Ｆタンパク質

Resumen de: CN120569211A

Provided herein are engineered parainfluenza virus fusion protein (PIV F) polypeptides. In some aspects, the engineered PIV F polypeptides exhibit enhanced conformational stability and/or antigenicity. Also provided are methods of using the engineered PIV F polypeptides as diagnostic agents, using the engineered PIV F polypeptides at screening platforms, and/or using the engineered PIV F polypeptides in vaccine compositions.

具有GSH/ROS双响应的聚合物纳米粒和其制备方法及应用

Resumen de: CN121405930A

本发明涉及一种具有GSH/ROS双响应的聚合物纳米粒和其制备方法及应用。本发明GSH/ROS双响应型聚合物纳米粒用于载药体系，能使载药纳米粒具有载药量高、响应性高、高肿瘤靶向、高肿瘤细胞抑制且无需稳定剂即可稳定的特点，能够在肿瘤微环境类似环境中快速崩解、迅速释放包载药物，降低了化疗药物对正常组织的毒性，绿色安全。本发明提供了一种稳定性好且具有GSH/ROS双响应的聚合物纳米粒的制备方法。此外，本发明还提供了载药体系的冻干方法，提高了制剂的稳定性。该技术解决了现有纳米药物载体合成复杂、成本高的问题，具有广泛的应用前景。

一种心脏归巢肽协同秋水仙碱磷脂复合物及其制备方法和应用

Nº publicación: CN121401232A 27/01/2026

Solicitante:

新疆医科大学第一附属医院

Resumen de: CN121401232A

本发明提供了一种心脏归巢肽协同秋水仙碱磷脂复合物及其制备方法和应用。其中，本发明制得的心脏归巢肽协同秋水仙碱磷脂复合物纳米粒子对心脏组织修复具有积极效果。