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LastUpdate Última actualización 11/12/2025 [06:50:00]
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CAR mRNA送達のための脂質ナノ粒子とその製剤

NºPublicación:  JP2025175052A 28/11/2025
Solicitante: 
ザトラスティーズオブザユニバーシティオブペンシルバニア
JP_2025175052_A

Resumen de: US2022378700A1

The present invention relates to lipid nanoparticles (LNP) or compositions thereof for delivery of mRNA molecules encoding CAR, nucleic acid molecule, and/or therapeutic agents to selected targets, such as cells. Thus, in various aspects, the present invention also provides methods of preventing or treating diseases or disorders in a subject in need thereof using the said LNPs or compositions thereof.

イオン化脂質およびそれを含む脂質ナノ粒子

NºPublicación:  JP2025538495A 28/11/2025
Solicitante: 
サーテストバイオテック,エス.エル.
JP_2025538495_PA

Resumen de: CN120225501A

The present invention provides an ionizable lipid of formula (I) or a pharmaceutically acceptable salt thereof or a stereoisomer of any of them; a lipid nanoparticle comprising the ionizable lipid (in particular as an encapsulant), optionally comprising a pharmaceutically active agent; and a pharmaceutical composition comprising the lipid nanoparticles. The present invention also provides a lipid nanoparticle for a drug or a pharmaceutical composition comprising the same, and a use of the lipid nanoparticle as an encapsulant. # imgabs0 #

核酸送達のための新規脂質及び脂質ナノ粒子組成物

NºPublicación:  JP2025538636A 28/11/2025
Solicitante: 
ザミディアムセラピューティクスカンパニーリミテッド
JP_2025538636_PA

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.

항균용 인공 나노세포 및 이의 제조 방법과 응용

NºPublicación:  KR20250166873A 28/11/2025
Solicitante: 
더서드어필리에이티드호스피탈오브광저우메디칼유니버시티
WO_2025223307_PA

Resumen de: WO2025223307A1

An antibacterial artificial nano-cell and a preparation method therefor. The antibacterial artificial nano-cell is prepared by the following steps: mixing polyvinylpyrrolidone or an aqueous solution thereof, CuCl2·2H2O or an aqueous solution thereof, FeCl2·4H2O or an aqueous solution thereof, Elesclomol or a DMSO solution thereof, NaOH or an aqueous solution thereof, and H2O2 or an aqueous solution thereof, uniformly stirring the mixture, followed by washing, ultrafiltration and centrifugation to obtain nanoparticles, uniformly mixing the nanoparticles with cell membranes prepared from bone marrow stromal cells, and carrying out ultrasonic incubation and extrusion to obtain the antibacterial artificial nano-cells.

이온화 가능 지질 분자 및 그 제조 방법과 용도

NºPublicación:  KR20250166967A 28/11/2025
Solicitante: 
맥시알엔에이상하이파마슈티컬씨오엘티디맥시알엔에이저장테크놀로지씨오엘티디상하이셀테라피그룹씨오엘티디
AU_2024231082_PA

Resumen de: AU2024231082A1

An ionizable lipid molecule, a preparation method therefor and a use thereof. Specifically, provided are a compound represented by formula I, and a stereoisomer, tautomer, pharmaceutically acceptable salt, prodrug or solvate thereof. The definition of each group in the formula is as described in the description. Also provided is a lipid nanoparticle, containing the ionizable lipid compound represented by formula (I). Further provided are a composition and a related use. The compound of formula I can be used to prepare the lipid nanoparticle for delivering a nucleic acid therapeutic agent or an active agent in vivo and in vitro.

メッセンジャーRNAの乾燥粉末製剤の組成物及びその使用方法

NºPublicación:  JP2025538523A 28/11/2025
Solicitante: 
トランスレイトバイオ,インコーポレイテッド
JP_2025538523_A

Resumen de: CN120265279A

The present disclosure provides stable dry powder messenger RNA formulations for therapeutic use and methods of making and using the same.

カチオン性脂質及びその製造方法

NºPublicación:  JP2025538420A 28/11/2025
Solicitante: 
サムヤンホールディングスコーポレイション
JP_2025538420_PA

Resumen de: CN120129674A

The present invention relates to a cationic lipid and a method for preparing the same, and more particularly, to a cationic lipid which promotes the formation of a complex with an anionic drug material so as to be useful for drug delivery, and a method for preparing the cationic lipid.

阻害性核酸およびその使用方法

NºPublicación:  JP2025538360A 28/11/2025
Solicitante: 
ザリージェンツオブザユニバーシティオブカリフォルニア
JP_2025538360_A

Resumen de: CN120359038A

The present disclosure provides inhibitory nucleic acids, compositions comprising the inhibitory nucleic acids, and methods of using the inhibitory nucleic acids to treat various diseases.

核酸の送達用のイオン化可能な脂質および脂質ナノ粒子組成物

NºPublicación:  JP2025538390A 28/11/2025
Solicitante: 
シーウルフセラピューティクス、インコーポレイティッド
JP_2025538390_PA

Resumen de: MX2025005703A

Novel ionizable lipids are provided. Also provided are novel lipid nanoparticle compositions for the delivery of nucleic acid material to cells in vitro and in vivo with different and improved pharmacokinetic profiles as compared to what is typically observed in the art. Also provided are methods for using the compositions in research and as therapeutics.

トランスポーターペプチド及びその使用

NºPublicación:  JP2025538660A 28/11/2025
Solicitante: 
ヴァチーノバイオテックカンパニーリミテッド
JP_2025538660_A

Resumen de: CN120225539A

The present invention relates to a transporter peptide capable of binding to a transferrin receptor. The transporter peptide can be covalently or non-covalently conjugated with an effector to form a transporter peptide conjugate, or the transporter peptide and an effector form a recombinant transporter peptide conjugate in a manner that represents a nucleic acid encoding the transporter peptide and the effector. The transporter peptide and the recombinant transporter peptide transport the effector to a target by binding to a transferrin receptor. Binding of the transporter peptide to a transferrin receptor on a cell of a tissue barrier induces transendocytosis of the cell to transport the transporter peptide conjugate through the tissue barrier. The transport peptide can be used as a drug delivery system and can be used for related treatment of central nervous system (CNS) diseases.

口蹄疫ウィルス様粒子またはナノ粒子生産のための組換え発現ベクターおよびこれを用いたワクチン組成物

NºPublicación:  JP2025538585A 28/11/2025
Solicitante: 
オプティファームカンパニーリミテッド
JP_2025538585_PA

Resumen de: WO2024112115A1

The present invention relates to a recombinant expression vector for preparing foot-and-mouth disease virus-like particles or nanoparticles, and a vaccine composition using same. The present invention provides virus-like particles or nanoparticles, and a preparation method therefor, the particles or nanoparticles being produced by expressing, simultaneously, VP4 with VP1, VP2 and VP3 or expressing VP1, VP2 and VP3 while excluding VP4, from among FMDV structural proteins. The virus-like particles or nanoparticles, prepared according to the method of the present invention, can be effectively used in the prevention of diseases caused by foot-and-mouth disease viral infection.

イオン化脂質及びその応用

NºPublicación:  JP2025538515A 28/11/2025
Solicitante: 
艾斯拓康医薬科技(北京)有限公司
JP_2025538515_PA

Resumen de: CN118125994A

The present invention provides an ionizable lipid, and a drug delivery system comprising the ionizable lipid. Specifically, the invention provides an ionizable lipid with a structure as shown in a formula (I), or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof. The lipid nanoparticles constructed by using the ionizable lipid can realize safe and efficient delivery of nucleic acid drugs, small molecule drugs, peptide drugs and protein drugs. # imgabs0 #

双脂质结构

NºPublicación:  CN121039138A 28/11/2025
Solicitante: 
特恩生物技术公司
CN_121039138_PA

Resumen de: TW202438044A

A compound having the following structure of Formula (I): or a stereoisomer, salt, or tautomer thereof, wherein R1, R2, R3, R4, R5, R6, G1, G2, x, y, n, z, and w are as defined herein. Compositions comprising the compounds, and their use in methods of treating diseases, are also described.

一种胡柚皮外泌体载药靶向制剂及其制备方法和应用

NºPublicación:  CN121015596A 28/11/2025
Solicitante: 
杭州医学院
CN_121015596_PA

Resumen de: CN121015596A

本发明公开了一种胡柚皮外泌体载药靶向制剂及其制备方法和应用,属于生物医药技术领域。该制备方法具体包括以下步骤:(1)将胡柚皮进行组织破碎,高速离心除去破碎组织及细胞,超高速离心收集外泌体粗提物,利用蔗糖密度梯度离心纯化胡柚皮外泌体;(2)将川陈皮素溶于乙醇,将胡柚皮外泌体溶于磷酸盐缓冲液,混合,搅拌,利用蔗糖密度梯度离心分离负载川陈皮素胡柚皮外泌体;(3)重悬于磷酸盐缓冲液,冷冻干燥,即得。本发明利用胡柚外泌体的抗炎作用,以及胡柚外泌体内质网定位提高川陈皮素靶向抑制STING通路治疗炎症作用,通过吸入给药治疗肺炎。

一种共递送铜离子和铜离子螯合前药的纳米药物组合物及其制备方法和用途

NºPublicación:  CN121015593A 28/11/2025
Solicitante: 
中国科学院上海药物研究所
CN_121015593_A

Resumen de: CN121015593A

本发明提供了一种共递送铜离子和铜离子螯合前药的纳米药物组合物及其制备方法和用途。本发明所述纳米药物组合物通过使用含邻苯二酚结构的多酚对二价铜盐中的铜离子和铜离子螯合前药进行负载,并与苯硼酸修饰的透明质酸组装形成纳米粒而得到。本发明所述纳米药物组合物解决了铜离子和铜离子螯合前药共递送的问题,有助于在肿瘤微环境氧化条件下,选择性地触发高细胞毒性的8‑羟基喹啉铜络合物的生成,实现高效低毒的抗肿瘤策略。此外,本发明所述纳米药物组合物使用溶剂交换法制备,制备操作简单,条件温和。

用于PCSK9基因修饰或编辑的组合物及其使用方法

NºPublicación:  CN121022805A 28/11/2025
Solicitante: 
深圳信立泰药业股份有限公司
CN_121022805_PA

Resumen de: WO2025180418A1

Provided are a composition for PCSK9 gene modification or editing, and a method for using same. Specifically, provided is a composition for editing a PCSK9 gene target, comprising a base editor for carrying out base editing on a PCSK9 gene target, and a guide RNA. The composition can effectively edit a target gene, and can be used for treating diseases caused by the abnormality of a polynucleotide which codes a PCSK9 protein.

可离子化脂质及其用途

NºPublicación:  CN121021427A 28/11/2025
Solicitante: 
广州市恒诺康医药科技有限公司
CN_121021427_A

Resumen de: CN121021427A

本发明公开了一种可离子化脂质及其用途。具体的,本发明设计合成了一类可离子化脂质,由其组成的脂质纳米颗粒递送系统可用于高效安全地递送DNA、RNA等核酸药物或小分子药物,不仅具有较好的包封率和稳定性;而且在动物体内具有很好的安全性、清除速率,对于核酸类治疗剂或预防剂具有较好的递送作用,在药物递送相关领域具有广泛的应用前景。

一种用于治疗炎症性肠病的多功能化硒纳米粒及其制备方法和应用

NºPublicación:  CN121015594A 28/11/2025
Solicitante: 
中国医学科学院生物医学工程研究所
CN_121015594_PA

Resumen de: CN121015594A

本发明公开了一种用于治疗炎症性肠病的多功能化硒纳米粒及其制备方法和应用,涉及生物医药技术领域。该多功能化硒纳米粒为核‑壳结构,包括内核和包覆所述内核的外层;该内核为硒纳米粒;该外层为嵌入壳寡糖的聚多巴胺外壳。本发明提供的多功能化硒纳米粒具有良好的稳定性与分散性,能够靶向肠道炎症部位,延长纳米粒在体时间,在细胞、动物层面显示出强大的抗氧化和抗炎活性。纳米粒整体基于天然材料的低毒性和结肠靶向性,较传统糖皮质激素/生物制剂安全性更高,通过调节氧化还原稳态、延长药物作用时间等方式减轻肠道炎症,有望成为兼具高效性、靶向性和安全性的结肠炎治疗新策略。

一种氧化还原响应的载HE4 siRNA纳米粒、其制备方法及在治疗顺铂耐药卵巢癌的应用

NºPublicación:  CN121015597A 28/11/2025
Solicitante: 
青岛市妇女儿童医院(青岛市妇幼保健院、青岛市残疾儿童医疗康复中心、青岛市新生儿疾病筛查中心)青岛科技大学

Resumen de: CN121015597A

本发明提供一种氧化还原响应的载HE4siRNA纳米粒,该纳米粒由氧化还原响应的肿瘤靶向载体材料和HE4siRNA组成。氧化还原响应的肿瘤靶向载体材料为c(RGDfK)肽修饰的聚(乙烯亚胺)‑双硒键‑聚(乙二醇)(PEI‑SeSe‑PEG)。该纳米粒将载体材料和siRNA按N/P比为10:1混合,自组装成纳米粒,粒径在116nm左右,体外释放具有氧化还原响应性,在高浓度谷胱甘肽(GSH)的肿瘤微环境实现siRNA的释放,有利于在肿瘤部位发挥作用。本发明制备的氧化还原响应的载HE4siRNA纳米粒,实现溶酶体逃逸,又具有肿瘤靶向性,可抑制顺铂耐药卵巢癌细胞的增殖、迁移和侵袭,且在动物体内具有良好的肿瘤靶向性、抗肿瘤活性和生物相容性。该纳米粒制备方法简单,抗癌效果显著,具有良好的应用价值和市场前景。

金属配位脂质化合物及其组合物和应用

NºPublicación:  CN121021379A 28/11/2025
Solicitante: 
浙江大学

Resumen de: CN121021379A

本发明涉及一种金属配位脂质化合物,化学式Ⅰ所示的化合物、或其立体异构体、或其互变异构体、或其药学上可接受的盐:

一种基于分步酶解的防脱植物组合物及其制备方法

NºPublicación:  CN121015734A 28/11/2025
Solicitante: 
湖州嘉亨实业有限公司
CN_121015734_PA

Resumen de: CN121015734A

本发明涉及防脱技术领域,具体涉及一种基于分步酶解的防脱植物组合物及其制备方法。该组合物由芍药提取物、侧柏叶提取物经丝素蛋白纳米粒负载而成,其中芍药提取物采用纤维素酶与果胶酶分步酶解工艺制备,与侧柏叶提取物按特定比例复配后,通过丝素蛋白纳米粒实现高效包载与缓释。实验验证表明,该组合物在5α还原酶抑制、人毛乳头细胞增殖及ALPL、TGF‑β基因表达调控方面具有协同增效作用,防脱效果显著优于单一成分及传统提取工艺制备的组合物。本发明通过工艺创新与配方优化,解决了现有防脱植物制剂功效单一、透皮吸收差的问题。

含有可电离脂质的肺部递送系统、其制备方法和应用

NºPublicación:  CN121015605A 28/11/2025
Solicitante: 
晟迪生物医药(苏州)有限公司
CN_121015606_PA

Resumen de: WO2025153098A1

A pulmonary delivery system containing an ionizable lipid, and a preparation method therefor and the use thereof, which belong to the technical field of biomedicine. The pulmonary delivery system containing an ionizable lipid comprises an ionizable lipid, an auxiliary lipid, and a bioactive substance, and does not contain polymer-lipid conjugates. The pulmonary delivery system containing an ionizable lipid can deliver the bioactive substance to the lungs. The pulmonary delivery system has the characteristics of uniform particle size and high safety, and can efficiently deliver a therapeutic agent to the lungs. Before and after atomization, the particle properties such as the particle size, particle size distribution coefficient, potential, and encapsulation efficiency remain unchanged. The pulmonary delivery system provides a safe and effective solution for the treatment of pulmonary diseases, and has broad application prospects.

双功能白蛋白纳米颗粒、制备方法、药物组合物及应用

NºPublicación:  CN121015873A 28/11/2025
Solicitante: 
南京鼓楼医院

Resumen de: CN121015873A

本发明公开了一种双功能白蛋白纳米颗粒、制备方法、药物组合物及应用,该纳米颗粒以白蛋白为载体,表面修饰有螯合钆离子的大环配体螯合剂,内部包裹疏水性含硼化合物。该双功能白蛋白纳米颗粒具有良好的T1加权磁共振成像能力,可用于实时追踪含硼药物在体内的分布与富集,实现与硼中子俘获疗法的协同应用,有助于评估治疗窗口,提升治疗的精准性;并且其粒径均一、zeta电位稳定,具备良好的载药能力和组织穿透性,有利于靶向富集于肿瘤组织;该双功能白蛋白纳米颗粒优选人血清白蛋白作为纳米粒载体,具有优异的生物相容性和良好的体内稳定性,安全性高,利于临床转化。

新型树枝状大分子衍生物的合成及应用

NºPublicación:  CN121021828A 28/11/2025
Solicitante: 
上海交通大学
CN_121021828_PA

Resumen de: CN121021828A

本发明涉及医药技术领域,公开了新型树枝状大分子衍生物的合成方法,新型树枝状大分子和1H‑吡唑‑1‑甲脒盐酸盐溶于去离子水中在室温下搅拌,最后通过透析纯化产物,冻干获得黄色粘稠液体,双胍新结构的的树枝状大分子的制备方法,步骤:树枝状大分子和双氰胺溶于2 M HCl溶液中,反应物加热反应,最后通过透析纯化产物,冻干获得黄色粘稠液体。新型树枝状大分子衍生物在在药物递送载体以及治疗和预防肥胖和Ⅱ型糖尿病中的应用。通过对树枝状大分子进行胍基化修饰,成功制备出一种结构明确、性能优良的胍修饰树枝状大分子,并进一步构建相应的纳米颗粒体系。在改善Ⅱ型糖尿病小鼠的肥胖、脂肪肝及胰岛素抵抗等方面表现出显著效果。

来自微藻的细胞外囊泡、它们在鼻内施用时的生物分布及其用途

Nº publicación: CN121038801A 28/11/2025

Solicitante:

AGS\u6CBB\u7597\u7B80\u6613\u80A1\u4EFD\u516C\u53F8

CN_121038801_PA

Resumen de: US2025339483A1

Provided are compositions containing microalgae extracellular vesicles (MEVs) formulated for intranasal delivery, whereby, upon intranasal administration the MEVs traffic through specific routes following intranasal administration to specific regions in the brain via the olfactory nerve and throughout the lateral olfactory tract (LOT) to interconnected brain regions. The MEVs traffic via neuronal axonal transport. The MEVs have the ability to cross-over synapses including: (i) the synapses between the olfactory sensory neurons (OSN) and the mitral/tufted neurons; (ii) the synapses between the mitral/tufted neurons and the local neurons in the various brain regions colonized by the lateral olfactory tract (LOT); and (iii) the synapses between the neurons in the brain regions colonized by the LOT and neurons from the frontal cortex, the hippocampus, the thalamus, and the hypothalamus. The compositions contain extracellular vesicles from microalgae (MEVs) that are loaded with bioactive cargo for treating, detecting, diagnosing, or monitoring a disease, disorder, or condition of the brain or involving the brain, particularly providing neuronal delivery of the cargo. The compositions and methods have a variety of applications as therapeutics and diagnostics for treating, diagnosing, and monitoring a disease, disorder, or condition of the brain or involving the brain. The compositions can be used in methods and uses for treating cancers involving the brain, and can be used, for exa

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