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03/12/2025 [07:11:00]
Resumen de: CN121041418A
本发明公开了一种棘球蚴病SaRNA疫苗及其制备方法与应用。所述SaRNA疫苗的制备方法包括如下步骤：通过基因工程技术将改造的目标抗原蛋白进行密码子优化后，与自复制蛋白序列以及5'UTR、3'UTR和Poly(A)尾进行组装，并进行基因合成，然后将合成的基因克隆到质粒中，并将构建得到的重组质粒依次进行质粒线性化、体外转录和纯化，制备得到SaRNA分子，最后将SaRNA分子包裹于脂质纳米颗粒内形成棘球蚴病SaRNA疫苗。实验证明：SaRNA疫苗能同时激活小鼠的体液免疫和细胞免疫，且低剂量免疫就能产生较高水平的EG95特异性抗体和细胞因子，在预防和/或治疗棘球蚴病方面具有广阔的应用前景。
Resumen de: CN121041239A
本发明公开一种用于靶向蛋白降解的纳米颗粒及降解方法。本发明的纳米颗粒能够增强细胞的摄取，并对识别目标蛋白的特异性抗体实现有效地包封，同时干扰素组分可以诱导细胞内TRIM家族蛋白的表达。在被摄取入胞后，纳米颗粒释放的抗体可以结合目标蛋白，并结合TRIM家族蛋白介导目标蛋白的降解。在这一过程中，TRIM家族蛋白虽然被持续消耗，但可以被本发明的颗粒所诱导的TRIM家族蛋白表达所补偿，从而维持了高效的降解。
Resumen de: AU2024212425A1
Disclosed herein are modified lipid compositions comprising (a) a structural component comprising one or more lipids selected from the group consisting of soy-derived lipids, cardiolipin, sphingolipid, ceramide, glucosyl ceramide, lactosyl ceramide, galactosyl cholesterol, glucosyl cholesterol; and modified by (b) an ionizable lipid. The disclosure also includes a method for making a modified lipid composition, comprising reconstructing (a) a structural component comprising one or more lipids selected from the group consisting of soy-derived lipids, cardiolipin, sphingolipid, ceramide, glucosyl ceramide, lactosyl ceramide, galactosyl cholesterol, and/or glucosyl cholesterol in the presence of (b) an ionizable lipid, to produce the modified lipid composition, and loading into the modified lipid composition with one or more heterologous functional agents.
Resumen de: CN121046392A
本发明提供了一种编码PTEN的环状RNA及其组合物的应用。本发明进一步涉及前述环状RNA联合免疫检查点抑制剂的组合物在肿瘤治疗中的应用。体内外药效学实验证明编码PTEN的环状RNA及其组合物可以有效抑制肿瘤生长，并且在与免疫检查点抑制剂联用时可以实现更好的药效。
Resumen de: CN121041241A
本发明提供了一种双基因沉默、cRGD修饰的靶向LNP制备，及其在结直肠癌抗血管生成治疗中的应用；所述LNP由脂质原料和核酸原料制得；其中，脂质原料包括可电离阳离子脂质SM‑102、胆固醇、DOPE 磷脂、DMG‑PEG2000和DMG‑PEG2000‑cRGD，核酸原料包括siEIF3a与siVEGF；制备时，将脂质溶于乙醇、核酸溶于缓冲液后混匀静置。本发明通过cRGD靶向修饰结合双siRNA共递送实现，既依托cRGD提升LNP对结直肠癌细胞的靶向性与摄取效率，又通过双siRNA分别作用于VEGF通路与EIF3a‑ANG轴，针对性解决现有技术无法有效突破的结直肠癌抗血管生成治疗耐药问题。
Resumen de: CN121041305A
本发明涉及了骨关节炎药物技术领域，具体公开了DDX5‑K45mRNA在制备治疗和/或缓解骨关节炎药物中的应用，同时提供了一种用于靶向软骨细胞的递药系统，研究发现，DDX5‑K45mRNA加入软骨细胞，可恢复DDX5‑K45乳酸化同时阻断NF‑κB炎症信号、纠正代谢重编程(如抑制异常糖酵解)、并上调COL2A1等软骨保护因子。本发明利用LNP高效递送DDX5‑K45mRNA至软骨细胞，直接补充内源性乳酸化修饰缺陷，重建DDX5的软骨保护功能，抑制炎症反应并减少基质降解，从根源上维持软骨稳态，实现长效、安全的OA治疗。
Resumen de: CN121041306A
本发明提供一种核酸纳米药物的制备及增敏肝癌免疫治疗疗效的应用，涉及生物医药技术领域。该核酸纳米药物通过PLGA‑S‑S‑PEG载体包载TMCO1及LONP1s i RNA，可在肿瘤细胞内高GSH环境下释放s i RNA，沉默靶基因并诱导免疫原性死亡，显著增强免疫检查点抑制剂的治疗效果。体内外实验表明，该纳米药物具有靶向性强、增敏效果显著、制备成本低等优势，为肝癌免疫治疗提供了新策略。
Resumen de: CN121046378A
本发明提供了一种靶向Janus激酶1(JAK1)的干扰RNA，该干扰RNA可以降低JAK1的表达，进而抑制JAK/STAT信号通路的激活，从而达到治疗JAK/STAT信号通路失调的相关疾病。本发明还提供了一种包含干扰RNA的脂质纳米颗粒药物，用于治疗JAK/STAT信号通路失调的相关疾病。本发明还提供了一种抗体‑核酸偶联药物，其中包含干扰RNA，用于治疗JAK/STAT信号通路失调的相关疾病。
Resumen de: CN121045032A
本发明涉及含有精氨酸结构的可电离脂质分子、包含其的脂质纳米颗粒及其用途。具体地，提供一种式(1)所示的含有精氨酸及其衍生物结构的可电离脂质分子、包含其的脂质纳米颗粒、其制备方法和用途。与本领域常规使用的可电离脂质分子相比，本发明的式(1)所示的可电离脂质分子所制备得到的脂质纳米颗粒可实现核酸的高效率递送及表达。
Resumen de: CN121041238A
本发明涉及纳米药物技术领域，尤其涉及可用于治疗脓毒症的唾液酸修饰依鲁替尼磷脂复合物纳米粒。本发明制备了一种唾液酸修饰依鲁替尼磷脂复合物纳米粒，其包括依鲁替尼、蛋黄磷脂酰甘油以及唾液酸‑胆固醇衍生物，该纳米粒能够显著提高制剂在炎症部位的积累和对“炎症相关巨噬细胞”的靶向，从而极大地提高了BTK抑制剂治疗脓毒症的有效性与安全性，更为重要的是，能够帮助脓毒症幸存小鼠解除免疫抑制状态，并产生强免疫记忆，极大地降低再感染风险，对解决临床中脓毒症幸存者出院后死亡率较高的难题具有指导意义；同时，本发明的唾液酸修饰依鲁替尼磷脂复合物纳米粒也能够降低机体对含有LPS抗原的病原体所致疾病的二次感染的风险。
Resumen de: CN121041432A
本发明涉及近红外二区有机染料技术领域，尤其涉及一种负载近红外二区氧杂蒽染料和热休克蛋白抑制剂的人血清白蛋白纳米复合物、制备方法及温和光热治疗肿瘤应用。该纳米复合物包括人血清白蛋白、NIR‑II氧杂蒽染料LD和热休克蛋白90抑制剂格尔德霉素，三者质量比为20:1:0.65。该纳米复合物具有较高的NIR‑II荧光/光声成像分辨率和信噪比，可实现增强的温和光热治疗肿瘤，为肿瘤的温和光热治疗提供了一种安全、高效、精准的全新策略，具有显著的临床转化潜力。
Resumen de: CN121041240A
为本发明属于纳米生物材料技术领域，公开了一种负载二氢杨梅素的白桦脂酮酸自组装纳米复合体系的制备方法和应用，所述纳米复合体系为白桦脂酮酸为载体，二氢杨梅素为活性成分的纳米复合体系。本发明制备的纳米组装体系的组装机制为：二氢杨梅素分子的酚羟基与白桦脂酮酸分子中的羰基和白桦脂酮酸分子中的羧基中的羰基结合产生的氢键相互作用为主要的非共价相互作用力。该纳米复合体系具有良好的生物相容性，且HT‑29细胞对纳米复合体系具有良好的摄取效果。在纳米生物材料领域具有一定的研究和应用前景。
Resumen de: CN121040616A
本发明公开了一种用于番茄红素递送的淀粉样β‑乳球蛋白纤维‑岩藻多糖自组装复合物的制备方法及应用，包括以下步骤：S1.将β‑LG溶解在去离子水中，配制1‑20 mg/mL浓度的β‑LG溶液，并搅拌 2 小时，并用1 mol/L的HCl溶液将β‑LG溶液调节至pH 2.0，得A品；S2.将A品置于石英容器中的两个电极之间反应，在室温下进行CP处理，得B品；S3.将B品在85°C水浴中进一步处理10小时制得C品，并立即在冰水中冷却C品，C品在4°C的温度下保存。它制备的淀粉样β‑乳球蛋白纤维‑岩藻多糖自组装复合物能有效提高番茄红素递送时的热稳定性和紫外稳定性，提高番茄红素递送效果。
Resumen de: CN121045013A
本发明涉及一种可电离阳离子脂质化合物及其制备方法和应用，涉及药用化合物技术领域，本发明公开了一种新结构的可电离阳离子脂质化合物，该可电离阳离子脂质化合物与目前最高效的脂质纳米粒子(DLin‑MC3‑DMA)相比，本发明公开的脂质纳米粒子不仅制备过程简单，反应条件温和，产率高，成本低，而且递送效率更高。
Resumen de: CN121041417A
本发明公开了一种棘球蚴病mRNA疫苗及其制备方法与应用。所述棘球蚴病mRNA疫苗的制备方法包括如下步骤：将改造的目标抗原蛋白进行密码子优化后，与5'UTR、3'UTR和Poly(A)尾进行组装，并进行基因合成，然后将合成的基因克隆至pUC57质粒中，并将构建得到的重组质粒依次进行质粒线性化、体外转录和纯化，制备得到mRNA分子，最后通过微流控方法将mRNA分子包裹于脂质纳米颗粒内形成棘球蚴病mRNA疫苗，并对其进行了免疫效果评价。通过实验证明：本发明制备的棘球蚴病mRNA疫苗能同时激活小鼠的体液免疫和细胞免疫，可为攻虫小鼠提供有效的保护作用，在预防和/或治疗棘球蚴病方面具有广阔的应用前景。
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 #
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.
Resumen de: CN120265279A
The present disclosure provides stable dry powder messenger RNA formulations for therapeutic use and methods of making and using the same.
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.
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.
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.
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.
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.
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 #
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.
Resumen de: CN121015626A
本发明公开了一种基于花青素包覆的纳米粒子的制备方法及其应用，首先从蓝靛果中提取花青素以及细胞外囊泡（EVs），得到花青素粉末；然后向得到的花青素粉末中加入PBS、角鲨烯，得到角鲨烯@花青素；再将EVs加入到角鲨烯@花青素中，共孵育后离心处理，得到花青素包覆的纳米粒子。该纳米粒子具有粒径为180~220nm、Zeta电位为‑45~‑55mV、包封率≥85%的特征，稳定性与生物相容性优异，具有调节肠道菌群、抗抑郁、抗氧化、抗炎等功能，且生物相容性好、毒性低，解决了其稳定性和生物利用度问题，实现了对肠道菌群的精准调控，为抑郁症治疗提供了一种安全高效的新途径。
Resumen de: CN121015917A
本发明涉及生物医药技术领域，具体涉及诊疗一体化的脑靶向磁性粒子成像示踪剂及其制备方法和应用。本发明先采用纳米颗粒自组装技术，将四氢姜黄素装载于葡聚糖内，形成装载四氢姜黄素的葡聚糖纳米颗粒，然后采用化学沉淀法合成Fe3O4纳米颗粒，并使其镶嵌在葡聚糖纳米颗粒上，得到诊疗一体化的脑靶向磁性粒子成像示踪剂，记为THC@Dex‑Fe3O4纳米颗粒。THC@Dex‑Fe3O4纳米颗粒在脑部成像诊断的同时，发挥阿尔茨海默症的治疗作用，解决了磁性粒子成像无法应用于阿尔茨海默症的诊断以及阿尔茨海默症治疗困难的技术问题。
Resumen de: CN121015599A
本发明提供一种纳米载体递送石斛碱的POCD治疗药物组合物及应用，属于医药技术领域。该组合物由石斛碱、纳米载体材料和稳定剂组成，纳米载体为PLGA纳米粒或脂质体。组合物粒径50‑150nm，多分散指数＜0.3，石斛碱负载量5‑15%。本发明还提供了该组合物的两种制备方法：PLGA纳米粒采用乳化‑溶剂挥发法制备；脂质体采用薄膜水化‑超声挤压法制备，并具体限定了各工艺参数。该药物组合物能够显著提高石斛碱的脑靶向性和生物利用度，可用于制备预防或治疗术后认知功能障碍（POCD）的药物，给药途径可为静脉注射或鼻腔给药。
Resumen de: CN121015875A
本发明提供了一种诊疗一体化纳米平台、制备方法及其应用。所述诊疗一体化纳米平台通过结构优化与功能整合构建有机‑无机纳米杂化物，创新性地将PTT、PDT、SDT和CDT四种模式有机结合，通过光声成像实时监测，实现GBM的高效协同治疗。首先，dOMV包封策略显著提升穿透血脑屏障的能力。同时，IEICO‑4F具有强近红外吸收特性，与介孔铂纳米颗粒的高催化活性协同作用。在激光/超声激发条件下，该诊疗一体化纳米平台展现出卓越的光热转换性能，不仅能加速芬顿反应速率，还可持续催化内源性过氧化氢分解生成氧气，有效缓解肿瘤缺氧状态。这一系列反应进一步增强了ROS的生成，激活了Caspase‑1/GSDMD介导的细胞焦亡通路，最终实现高效的肿瘤抑制效果，成功突破了单一治疗模式的局限性。
Resumen de: CN121015598A
本发明公开了一种靶向肺泡微环境氧化还原的药物递送系统及其制备方法和应用，包括两亲性GSH‑(oxi)PEI‑PCL聚合物自组装形成的纳米粒子，纳米粒子内负载脂溶性药物或荧光染料，所述两亲性GSH‑(oxi)PEI‑PCL聚合物中GSH与(oxi)PEI‑PCL通过二硫键连接，所述(oxi)PEI‑PCL为经部分氧化的PEI‑PCL。经药效试验结果显示，本发明的药物递送系统能使药物实现病灶内被动靶向聚集和微环境氧化还原敏感分布，同时表现出病灶微环境和细胞内的抗氧化效果，实现较确切的药物分布效果和体内治疗效果，为早产肺发育不良、细菌性/病毒性肺炎或烧伤吸入性肺损害等肺部疾病的治疗提供了新的药物途径。
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.
Resumen de: CN121022891A
本发明提供了一种FAP CAR mRNA、聚合物脂质纳米颗粒递送系统及其制备方法和应用，属于生物医药技术领域。本发明FAP CAR mRNA聚合物脂质纳米颗粒递送系统包括FAP CAR mRNA、RP182多肽和脂质纳米颗粒。本发明成功制备出粒径均匀、稳定性好的FAP CAR mRNA的聚合物脂质纳米颗粒递送系统，且mRNA包封率高，能够高效转染小鼠RAW264.7细胞，使巨噬细胞表达FAP CAR，转染效率显著高于单独使用FAP CAR mRNA或其他对照载体。经LNP递送FAP CAR mRNA转染的巨噬细胞(CAR‑M细胞)能够特异性识别并杀伤活化的成纤维细胞，显著抑制成纤维细胞的增殖。
Resumen de: CN121015595A
本发明涉及生物医药领域，具体涉及一种α‑生育酚乙酸酯‑透明质酸钠纳米粒子的制备方法及其应用。本发明首先将α‑生育酚乙酸酯丙酮溶液与透明质酸钠溶液进行剪切乳化处理，得到均匀分散的溶液，然后利用低剂量电子束辐射作用，避免透明质酸钠过多降解的前提下，打断透明质酸钠糖链上的部分化学键，产生大量高分子自由基。这些透明质酸钠自由基具有高反应活性，与生育酚乙酸酯分子形成共价键。本发明选取生育酚乙酸酯作为透明质酸钠的接枝改性材料，同时包裹β‑谷甾醇等活性成分，可作为头皮填充剂中的主要成分，为治疗雄激素脱发提供新的思路。
Resumen de: TW202438043A
A compound having the following structure of Formula (I): or a stereoisomer, salt, or tautomer thereof, wherein R1, R2, R3, R4, X, Y1, and Y2 are as defined herein. Compositions comprising the compounds, and their use in methods of treating diseases, are also described.
Resumen de: CN121021329A
本发明提供了两类功能性肽脂质化合物及三组分纳米颗粒与应用，属于新型药物制剂技术领域。本发明设计了两类功能性肽脂质，其中，第Ⅰ类功能性脂质通过改变氨基酸的数量，种类及其构建的多肽的性状，得到不同亲水头部结构的肽脂质；第Ⅱ类功能性脂质通过改变碳链数量、碳链上碳的数量及其饱和度，得到不同疏水尾结构的肽脂质。两类可调、多样的功能性肽脂质以用于各项需求。同时，本发明通过将第I类功能肽脂质、第II类功能肽脂质、磷脂‑PEG及mRNA层层组装构建了三组分mRNA脂质纳米颗粒；所述三组分脂质纳米颗粒具有较好的器官靶向性、生物降解性及基因递送效率。
Resumen de: CN121015832A
本发明公开了一种治疗口腔念珠菌的口腔喷雾剂及其制备方法，涉及治疗口腔念珠菌技术领域；所述治疗口腔念珠菌的口腔喷雾剂由兼具磁性和活性氧双响应性的载药纳米粒、改性碳纳米管水凝胶载体、木糖醇、柠檬酸钠、PEG‑60氢化蓖麻油和去离子水组成；将兼具磁性和活性氧双响应性的载药纳米粒附着在改性碳纳米管水凝胶载体上，形成了基于改性碳纳米管凝胶复合纳米粒的双载药材料，提高了载药量，能够以磁性和活性氧双响应的高度靶向性识别念珠菌寄生的黏膜部位，并与之有效结合，同时在感染的黏膜部位缓慢释放负载的药物成分，以化学作用和物理作用协同发挥抑菌性，提高了治疗口腔念珠菌的有效性。
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
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。
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。
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.
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：
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。
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。
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。
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。
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.
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。
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。
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.
Resumen de: US2025360213A1
Materials and methods for enhancing the effectiveness of proton radiation therapy (e.g., high linear energy transfer (LET) proton radiation therapy) against tumor cells are provided herein.
Resumen de: WO2025245481A1
Provided herein are methods and materials for methods and materials for treating cardiac conditions in a subject in need thereof. In some cases, provided herein are methods and materials for modulating the antithetical expression of MYH6 and MYH7 in a subject (e.g., a mammal). For example, pharmaceutical compositions (e.g., nucleic acid molecules, genetically modified cells, vectors, or combinations thereof) provided herein can be used to treat a subject having a cardiac condition.
Resumen de: US2025360089A1
Aspects of the disclosure relate to particle formulations, e.g., nanoparticle formulations, methods of making particle formulations, and methods for delivery of oligonucleotides and/or synthetic RNA, e.g., for increasing gene expression in a targeted manner. In some embodiments, compositions and methods are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the disclosure described herein provide compositions and methods that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).
Resumen de: US2025360087A1
Drug delivery systems are needed to assist in improving the therapeutic characteristics of pharmaceutical agents. Provided is a dry composition, comprising a polysaccharide, such as inulin, and lipid droplets, wherein the lipid droplets are encapsulated within polymeric chains of the polysaccharide, and wherein the polysaccharide is not in a nano-particulate form. The use of such compositions enables efficient delivery of agents, such as poorly-water soluble drugs and antibiotics.
Resumen de: US2025360176A1
A medicinal chewing gum has an inner core containing a first gum base and a first cannabinoid in a lipophilic nanosized form and an outer layer containing a second gum base and a second cannabinoid in a hydrophilic nanosized form, thereby providing quick release of the second cannabinoid in the outer layer and sustained release of the first cannabinoid in the inner layer. At least one of the inner core and the outer layer contains a synergistic compound having a synergistic effect with at least one of the first and second cannabinoids in the treatment of a medical condition. At least one of the first cannabinoid and the second cannabinoid are a cannabinoid other than cannabidiol (CBD).
Resumen de: US2025360202A1
The present disclosure belongs to the technical field of mRNA vaccines, and specifically relates to a herpes zoster mRNA vaccine, a preparation method therefor, and a use thereof. The herpes zoster mRNA vaccine provided by the present disclosure comprises an RNA encoding a varicella-zoster virus gE glycoprotein or a variant thereof. The vaccine can prevent herpes zoster infection and its complications.
Resumen de: US2025360140A1
The disclosure provides the use of particular substituted heterocycle fused gamma-carboline compounds as pharmaceuticals for the treatment of residual symptoms of psychosis or schizophrenia. The disclosure also provides novel long acting injectable formulations of particular substituted heterocycle fused gamma-carboline compounds and use of such long acting injectable formulations for the treatment of residual symptoms of psychosis or schizophrenia.
Resumen de: US2025360197A1
The Human Immunodeficiency Virus (HIV) infection and recurrent infection prevention mRNA vaccine comprising epitopes of HIV-1 and HIV-2 viruses and their corresponding Nef proteins.
Resumen de: US2025361270A1
Disclosed are peptides and peptidomimetics that in some embodiments include the amino acid sequence KRGARST or (SEQ ID NO: 1), AKRGARSTA or (SEQ ID NO: 2), or CKRGARSTC (SEQ ID NO: 3). Also disclosed are conjugates and compositions that include the peptides and/or peptidomimetics, methods for directing a moiety to tumor lymphatic vasculature, methods for imaging tumor lymphatic vasculature, methods for reducing or inhibiting tumor metastasis, methods for reducing the number of tumor lymphatic vessels, methods for treating cancer, methods for treating a disease or disorder associated with a gC1q/p32 receptor biological activity, methods for detecting the presence of a gC1q/p32 receptor, methods for detecting interactions between gC1q/p32 receptors and the presently disclosed conjugates and compositions, methods for delivering the presently disclosed conjugates and compositions to gC1q/p32 receptors, methods for assessing gC1q/p32 receptor levels in cells, methods for identifying subjects having diseases associated with gC1q/p32 receptor biological activities, and methods for screening for compounds that interact with gC1q/p32 receptors.
Resumen de: US2025361208A1
Provided herein are lipids having the Formula (I):and pharmaceutically acceptable salts thereof, wherein R1, R2, a, and b are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising lipid having the Formula (I) and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).
Resumen de: US2025361511A1
Provided here are nanoparticle compositions containing siRNA to disrupt the signal regulatory protein-α (SIRPα) signaling pathway. Embodiments include methods for treating a subject diagnosed as having ovarian cancer by administering to the subject the nanoparticle composition containing SIRPα siRNA. Other methods include administering to the subject the nanoparticle composition containing SIRPα siRNA in addition to a platinum-based chemotherapeutic agent.
Resumen de: WO2025242834A1
The invention disclosed herein provides a delivery system for active molecules formed by nanoassemblies with a structure of a hybrid bilayer comprising an amphiphilic polymer, which may be functionalized with some moieties, and a phospholipid. This bilayer delimits an aqueous core in which an active molecule of aminoacidic nature has been incorporated. Furthermore, the nanoassemblies may incorporate a targeting moiety to direct the system to the target tissue. Moreover, the present invention also relates to a pharmaceutical composition comprising these nanoassemblies and to the therapeutic use of these nanoassemblies, as they are able to achieve an intracellular delivery and distribution of the active molecule into the target tissues.
Resumen de: US2025360203A1
Disclosed is an mRNA composition containing: an mRNA sequence comprising, in order from 5′ to 3′, a 5′ cap region, a 5′ UTR region, a start codon region, a 3′ UTR region, and a poly (A) tail region; and an antisense oligonucleotide containing a region complementary to the 5′ cap region. The mRNA composition containing the mRNA sequence and the antisense oligonucleotide containing a region complementary to the 5′ cap region of the mRNA sequence may regulate the translation rate of the mRNA, enables selective protein expression based on the type of nucleotide modification and DNA repair mechanism, and may improve the stability of the mRNA against RNA-degrading proteins, thereby improving the stability and efficiency of mRNA vaccines or therapeutics.
Resumen de: AU2024252577A1
Disclosed herein is a polysialic acid (PSA)-polymer conjugate compound represented by the structural formula (I): or a pharmaceutically acceptable salt thereof, wherein P is a poly(lactide-co-glycolide)-poly(ethylene glycol) copolymer (PLGA-PEG) and p is an integer from 4 to 200, nanoparticles comprising same, and methods of treating ophthalmic diseases using same.
Resumen de: US2025360086A1
Provided herein is a device for producing an aqueous-core polymeric-shell particle as described herein. Also provided are methods of preparing such particles, as well as the particles themselves. The particles are useful in medicine, particular in the context of vaccines.
Resumen de: AU2023447567A1
An oil-in-water nanoemulsion comprising a continuous aqueous phase, a surfactant, and a particulate oil phase, wherein the particulate oil phase is dispersed within the aqueous phase, wherein the particulate oil phase comprises particles comprising extracted agarwood oil having a diameter in the range of about and including 160 nm to about 200 nm.
Resumen de: AU2024256347A1
The present disclosure is directed to the use of reconstituted mRNA dry powder particles for parenteral administration. The present disclosure is also directed to a method of generating dry powder particles supplemented with appropriate excipients for optimal thermostability and in vivo expression.
Resumen de: AU2025263792A1
COMPOSITIONS AND METHODS FOR TREATING NON-AGE- ASSOCIATED HEARING IMPAIRMENT IN A HUMAN SUBJECT Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject. COMPOSITIONS AND METHODS FOR TREATING NON-AGE- ASSOCIATED HEARING IMPAIRMENT IN A HUMAN SUBJECT Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject. ov - - o v
Resumen de: US2025360083A1
Disclosed herein are lipid nanoparticles comprising plurality of lipids, a targeting moiety for an HIV-1 chemokine receptor, and a CRISPR nucleic acid complementary to an HIV-1 gene, pharmaceutical compositions and methods of use thereof.
Resumen de: WO2025241798A1
The present invention relates to the technical field of pharmaceutical formulations, and provides a dual-targeted nanoparticle, a preparation method therefor, and a use thereof. The dual-targeted particle comprises a browning inducer, i.e., Chiglitazar sodium, a photothermal reagent, i.e., indocyanine green, an adipocyte homing peptide, a cell penetrating peptide, a DSPE-PEG polymer, and a lipid matrix. The browning inducer and the photothermal reagent are encapsulated into a nanoparticle having excellent biocompatibility, and the surface of said nanoparticle is coated with the functionalized adipose homing peptide and the cell penetrating peptide to construct the dual-targeted nanoparticle. The combination of the dual-targeted nanoparticle and photothermal-drug therapy can effectively promote "browning" of white adipose, reduce weight and enhance glycolipid metabolism, and lay a foundation for developing safe and effective drugs against obesity and related metabolic disorders.
Resumen de: WO2025242083A1
Provided in the present invention is a functional material capable of self-assembling into nanoscale dimensions, with an amino acid or polypeptide as a core backbone, a hydrophobic segment at one end and a hydrophilic segment at the other end. The material can be used in fields such as drug delivery through self-assembly into nanoscale forms. Formula (I).
Resumen de: WO2025242219A1
Disclosed are a chloroquine structure-containing compound, a composition containing same, and an application thereof. The present invention provides a compound as represented by formula (I) or a pharmaceutically acceptable salt thereof. The chloroquine structure-containing compound provided by the invention is a nano material obtained from the synthesis of chloroquine, a derivative thereof, and a lipid tail. The nano material can be applied to gene therapy, drug delivery, and the like. A lipid nanoparticle (LNP) formed from the nano material and mRNA has a novel nanoscale spatial structure different from that of a traditional LNP, and compared to a traditional LNP, the nano material has better pharmaceutical properties. The LNP exhibits an immunosuppressive function, avoids triggering an excessive inflammatory response, and has a higher safety profile.
Resumen de: WO2025242194A1
A latanoprost ophthalmic gel, comprising the following raw materials: 0.001-0.01% of latanoprost, 0.01-0.1% of a gel matrix, 0.1-5.0% of a nonionic surfactant, 0.1-5.0% of an osmotic pressure regulator, 0.01-1.0% of a pH regulator, and the balance of water for injection. The raw materials do not comprise a bacteriostatic agent. Not adding the bacteriostatic agent can fundamentally eliminate toxic side effects caused by the bacteriostatic agent, avoid adverse reactions to users, and improve product safety; and the non-ionic surfactant can form nanomicelles during the preparation process of the gel to exert a solubilizing effect, so as to overcome the problems of reduced solubility and poor stability of latanoprost caused by the absence of the bacteriostatic agent.
Resumen de: WO2025242237A1
An engineered cell membrane nanovesicle and a preparation method therefor and use thereof. A use of normal fibroblasts in the preparation of a drug or tumor diagnostic reagent or nano-drug delivery system or engineered vesicle for treating tumors. The vesicle prepared using the normal fibroblasts has high specificity and the ability to quickly target various types of tumor tissues, and can achieve tumor targeted diagnostic imaging or drug delivery; also, the accumulation in normal organs and non-targeted tissues is greatly reduced, the toxic side effects in vivo are minimized, and safety and effectiveness are greatly improved.
Resumen de: WO2025243932A1
The present invention provides a polyion complex comprising: a block copolymer that has a hydrophilic polymer segment and a cationic poly(amino acid) segment; and a nucleic acid. The cationic poly(amino acid) segment includes an amino acid residue A that is selected from an amino acid residue having an achiral carbon atom as a carbon atom constituting the main chain and a polar non-charged amino acid residue, and also includes an amino acid residue B having a cationic group in a side chain.
Resumen de: WO2025244940A1
Disclosed herein are compositions and methods for the treatment of hepatitis B infection, including chronic hepatitis B (CHB).
Resumen de: WO2025245275A1
A surfactant reagent, comprising about 1 to about 10 wt% of surfactant polypeptide and about 90 to 99 wt% of a lipid mixture and methods for the preparation and use thereof. The surfactant polypeptide is cationic and the lipid mixture consists of 1,2-dipalmitoyl-sn-glycero-3-phosphatidylcholine (DPPC), and one or both of 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol sodium salt (POPG-Na), and one or both of 1-palmitoyl-2-oleoyl-glycero-3-phosphatidylcholine (POPC) or 2-dioleoyl-sn-glycero-3-phosphatidylcholine (DOPC); or the surfactant polypeptide is anionic and the lipid mixture consists of POPC, DPPC, and 1,2-dioleoyl-3-trimethylammonium propane (DOTAP).
Resumen de: WO2025244582A1
The present disclosure provides a metal oxide nanoparticle comprising a metal oxide, the metal capable of being a Lewis acid; a polymer comprising an ionisable moiety; and an amino acid; wherein a weight ratio of amino acid to 5 polymer is about 0.1:99.9 to about 99.9:0.1; wherein the amino acid is electrostatically interacting with the polymer; wherein the metal oxide comprises metal ions in a 3+ and 4+ valence state; and wherein the metal oxide is selected from lanthanide oxide and/or zirconium oxide. The present disclosure also provides a method of fabricating a metal oxide particle and use thereof.
Resumen de: US2025360199A1
This disclosure generally relates to methods and compositions for eliciting broad and robust immune responses to a protein of interest. The methods employ both DNA and RNA-based vaccines that encode at least a portion of the protein of interest.
Resumen de: CN120500498A
The present invention provides, in part, protein-drug conjugates for delivering a molecular cargo (e.g., a polynucleotide, a polypeptide, a liposome, or a lipid nanoparticle) to a targeted tissue (e.g., brain), the protein-drug conjugates comprising an anti-fibroblast growth factor receptor 3 (FGFR3) (e.g., a polypeptide, a liposome, or a lipid nanoparticle) conjugated to the molecular cargo. The present invention relates to human FGFR3 (e.g., human FGFR3) antigen binding proteins (e.g., scFv, Fab). The invention provides methods for treating various diseases or conditions, such as neurological diseases, with the conjugates.
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.
Resumen de: US2025269035A1
Disclosed herein are a lipid-charged molecule conjugate, an inhalable lipid nanoparticle, a preparation method therefor, and use thereof. The lipid-charged molecule conjugate of the present invention comprises a lipid unit and a charged unit. The charged unit is selected from a negative charge unit and/or a positive charge unit. The lipid nanoparticle of the present invention comprises an ionizable lipid, an auxiliary phospholipid, cholesterol, a pegylated lipid, and the lipid-charged molecule conjugate. After the lipid nanoparticle of the present invention is administered by means of atomization inhalation, the anti-atomization stability of the lipid nanoparticle and the nucleic acid delivery efficiency are improved. When the lipid nanoparticle was used to encapsulate mRNA vaccine, inhaled mRNA-LNP can activate humoral, cellular, and mucosal immune responses.
Resumen de: US2025347665A1
The present disclosure provides a method of detecting size, density, or size and density of the lipid nanoparticles. The detection may be performed by centrifugal field flow fractionation using a carrier solution comprising a monosaccharide.
Resumen de: JP2025172276A
【課題】アルツハイマー病などのアミロイドβ関連またはタウ・タンパク質関連の認知症や脳炎症に関連するタンパク質の発現を制御（抑制や阻害など）できる、新規な遺伝子の発現制御剤の提供。【解決手段】アミロイド前駆体タンパク質（ＡＰＰ）、β－セクレターゼ（ＢＡＣＥ１）、ＮＭＤＡ活性化タンパク質、グリコーゲン合成酵素キナーゼ－３β（ＧＳＫ－３β）、およびポリグルタミン結合タンパク質－１（ＰＱＢＰ１）のうち少なくとも１種類のタンパク質の発現に関する遺伝子を標的遺伝子とするｍｉＲＮＡを含む微小粒子である、遺伝子の発現制御剤；アルツハイマー病の予防薬または治療薬；認知症の改善方法。【選択図】図１９
Resumen de: EP4653462A2
Certain embodiments of the invention provide a method for treating a Hepatitis B virus infection and/or ameliorating one or more symptoms associated with a Hepatitis B virus infection in a mammal, the method comprising the step of administering to the mammal a therapeutically effective amount of an anti-PD-1 antibody, or fragment thereof.
Resumen de: EP4652991A1
The invention disclosed herein provides a delivery system for active molecules formed by nanoassemblies with a structure of a hybrid bilayer comprising an amphiphilic polymer, which may be functionalized with some moieties, and a phospholipid. This bilayer delimits an aqueous core in which an active molecule of aminoacidic nature has been incorporated. Furthermore, the nanoassemblies may incorporate a targeting moiety to direct the system to the target tissue. Moreover, the present invention also relates to a pharmaceutical composition comprising these nanoassemblies and to the therapeutic use of these nanoassemblies, as they are able to achieve an intracellular delivery and distribution of the active molecule into the target tissues.
Resumen de: WO2025029323A1
Embodiments of the present disclosure provide novel compositions and methods for making and using thermostable polynucleotide-containing formulations. In certain embodiments, compositions and methods are disclosed for creating thermostable polynucleotides and/or thermostable polynucleotides encoding at least one therapeutic agent for use in therapies for the treatment of health conditions in a subject. In some embodiments, compositions and methods are disclosed for creating thermostable polynucleotides for use in therapeutics, vaccines, and targeted gene therapies. In other embodiments, compositions and methods are disclosed for creating thermostable polynucleotides capable of being coated or encased for prolonged storage and/or timed-delivery.
Resumen de: CN120603809A
Disclosed are: a composition comprising a lipid-like compound; methods of making such compositions; and the use of these compositions in gene delivery applications.
Resumen de: CN120603580A
Therapeutic compositions and methods. Disclosed is a composition comprising: a particle comprising hydrolysable doped silicon; one or more lipids; and an active pharmaceutical ingredient (API). Also disclosed are related products, methods and uses thereof.
Resumen de: WO2024153954A1
Disclosed is a composition comprising: particles comprising one or more metals and hydrolysable silicon; one or more lipids; and an active pharmaceutical ingredient (API). Also disclosed are related products, methods and uses.
Resumen de: CN120603600A
The present invention relates to formulations comprising miRNAs having improved stability for use in the treatment of diseases, including neurodegenerative diseases, such as spinal cerebellar ataxia type 3.5.
Resumen de: CN120712078A
Provided herein is a composition comprising a lipid and a copolymer in the form of a nanodisk assembly. The copolymer comprises monomeric units of methacrylic acid and styrene. Also provided herein are aqueous solutions comprising the compositions of the invention, and methods of making the nanodisk assemblies. Further, provided herein are methods of solubilizing a hydrophobic component (e.g., a membrane protein) in an aqueous solution, comprising forming nanodisk assemblies of lipids, hydrophobic components, and copolymers of the invention.
Resumen de: WO2024155885A1
Compositions that facilitate therapeutic delivery, as well as methods of making and using the same, are described herein. In particular, the composition including a cross-linked network of cyclic macromolecules and a plurality of functional groups, wherein the cyclic macromolecules are covalently cross-linked to one another by a plurality of cross-linking agents, and the plurality of functional groups are covalently associated with the cross-linked network of cyclic macromolecules, wherein the plurality of functional groups include (i) a proximal end covalently associated with the cross-linked network of cyclic macromolecules, and (ii) a distal end protruding out of the cross-linked network of cyclic macromolecules, wherein the distal end comprises a charge-imparting group.
Resumen de: AU2023425729A1
A method of targeting antigen-presenting cells and delivering an encapsulated payload with lipid nanoparticles including a POZ-lipid conjugate. The encapsulated payload may include, but is not limited to, a nucleic acid payload such as mRNA or modified mRNA. These LNPs are not subject to accelerated blood clearance and they have a low or reduced immunogenicity profile in vivo.
Resumen de: EP4652988A1
The present invention relates to a cyclosporine nanomolecular association and an eye-drop composition containing the same. More specifically, the present invention relates to a cyclosporine nanomolecular association and an eye-drop composition containing the same, which has excellent storage stability and can be delivered to target tissues (cornea and conjunctiva) more efficiently at lower concentrations compared to conventional formulations.
Resumen de: MX2025008266A
A mixture for treating a tumor, which includes an agent which turns into a hydrogel by addition of calcium ions, a vehicle carrying the agent in a manner allowing injection of the mixture into a tumor; and radium radionuclides bonded to the agent in a concentration sufficient to treat the tumor by radiotherapy.
Resumen de: WO2024153122A1
Nanoparticles encapsulating core particles with attached photothermal agents, and therapeutic agents. The methods of imaging a tumor tissue and/or treating a subject suffering from tumor with the nanoparticles provided herein..
Resumen de: AU2024209240A1
The invention relates to a composition comprising or essentially consisting of (i) nanoparticles comprising or essentially consisting of Enzalutamide in crystalline form and (ii) one or more physiologically acceptable polymers and/or copolymers. The invention also relates to processes for the preparation of such compositions, pharmaceutical dosage forms comprising or made from such compositions, and uses of such pharmaceutical dosage forms for medical purposes.
Resumen de: CN120712350A
The present disclosure relates to an RNA comprising one or more miRNA binding sequences, wherein the one or more miRNA binding sequences bind to a miRNA present in a cell in which expression of the RNA is not desired. After administration, in particular after intramuscular or intravenous administration, the RNA is delivered to a cell such that the polypeptide encoded by the RNA is expressed in certain cells while inhibiting expression in other cells. In some embodiments, such cells comprise endothelial cells. The RNA compositions described herein allow for expression of a pharmaceutically active peptide or polypeptide in a subject by the RNA while reducing or avoiding the risk of undesirable effects caused by expression of the pharmaceutically active peptide or polypeptide in certain cells or tissues.
Resumen de: US2024011003A1
The present invention encompasses engineered meganucleases that bind and cleave a recognition sequence within a TTR gene. The present invention also encompasses methods of using such engineered meganucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or nucleic acids encoding engineered meganucleases of the invention, and the use of such compositions for treatment of TTR-associated diseases, such as transthyretin amyloidosis.
Resumen de: US2023092885A1
A method of inducing cell death in a subject includes administering to the subject a plurality of cell targeted nanobubbles that are internalized by the target cell and insonating nanobubbles internalized into the target cell with ultrasound energy effective to promote inertial cavitation of the internalized nanobubbles and apoptosis and/or necrosis of the target cell.
Resumen de: CN121005655A
本发明公开了一种含氯喹结构的化合物、含其的组合物和其应用。本发明提供了一种如式I所示化合物或其药学上可接受的盐。本发明提供的含氯喹结构的化合物是一种由氯喹及其衍生物与脂质尾部合成所得的纳米材料，所述纳米材料可用于基因治疗、药物递送等应用。所述纳米材料与mRNA形成的脂质纳米粒(LNP)具有有别于传统LNP的新型纳米空间结构，较传统LNP具有更优的制剂学性质。所述LNP具有免疫抑制功能，可避免引发冗余的炎症反应，具有更高的安全性。
Resumen de: CN121005773A
本发明公开了用于抗炎的多肽纳米颗粒及其制备方法，属于生物活性肽技术领域。所述用于抗炎的多肽纳米颗粒是采用壳聚糖和三聚磷酸钠包覆具有抗炎作用的多肽获得的纳米颗粒；所述具有抗炎作用的多肽中包含氨基酸序列为YAADQ、FPYTT、YVTYA的生物活性肽。藻蓝蛋白抗炎肽来源天然，纳米包埋技术可显著提升其性能，相较于传统药物，具有更低的毒副作用和潜在的成本优势。本发明制备的多肽纳米颗粒能够有效的防止藻蓝蛋白抗炎肽在消化道中的降解，对溃疡性结肠炎小鼠具有显著的治疗效果，在溃疡性结肠炎防治产品的制备中具有重要的应用前景。
Resumen de: CN121003597A
本发明提供一种甘露糖功能化超分子纳米粒及其制备方法与应用，所述纳米粒由水溶性两亲性化合物MaCa在水相中通过超分子自组装形成，用于三阴性乳腺癌（TNBC）的靶向治疗和早期诊断。该纳米粒的表面甘露糖单元特异性结合TNBC标志物α‑甘露糖苷酶2C1，疏水内核负载药物（如紫杉醇）或成像试剂（如DiR、MRI造影剂）。实验表明，该颗粒在肿瘤部位富集量达对照组的9.3倍，治疗7天使肿瘤体积消失，且无显著毒性。本发明工艺简便，为TNBC诊疗一体化提供了创新解决方案。
Resumen de: AU2024237452A1
To provide lipid particles that can be produced without relying on PEG-modified lipids, that are superior in medicinal effect, safety, or stability to lipid particles that rely on PEG-modified lipids, or that have different immunodynamics than lipid particles that rely on PEG-modified lipids. Provided is a lipid particle, wherein the lipid particle contains an ionized lipid, a phospholipid, and a sterol as lipids constituting the lipid particle and contains a modified polysaccharide that includes a hydrophobic group.
Resumen de: CN121003583A
本发明公开了一种青光眼手术术中应用的毛蕊异黄酮苷PLGA水凝胶，涉及生物医药技术领域，按重量份计，包括如下组分制成：毛蕊异黄酮苷1~5份、PLGA 20~40份、乳化稳定剂0.1~3份、水凝胶基质3~8份、交联剂3~8份。本发明通过双重缓释体系的构建，释放持续时间长，可覆盖术后瘢痕增生高峰期；半固态凝胶形态贴合巩膜瓣，提升手术部位黏附性，可降低全身副作用和二次手术率；生物相容性优异，无明显毒性或刺激，并可规避传统抗代谢药物的并发症（如低眼压和炎症）。
Resumen de: CN121003598A
本发明公开了一种用于鼻黏膜mRNA疫苗递送的纳米颗粒及其制备与应用，属于生物医药技术领域。所述纳米颗粒由季铵化壳聚糖、低分子量聚乙烯亚胺、醛基化聚乙二醇与mRNA通过自组装形成。QCS和PEI通过静电作用吸附mRNA，OPEG通过席夫碱键与PEI/QCS交联，形成pH响应型纳米颗粒。该颗粒在生理pH下稳定，保护mRNA免受降解；在内涵体酸性环境下解离，快速释放mRNA并促进内体逃逸。QCS赋予颗粒优异的黏膜黏附性和佐剂效应。本发明还提供了该颗粒的制备方法及在制备鼻黏膜mRNA疫苗中的应用。该疫苗能有效诱导黏膜sIgA和全身IgG/IgA应答，用于预防病毒性传染病和肿瘤。
Resumen de: CN121003596A
本发明提供了一种载药聚合物纳米胶囊及其制备方法。通过超分子自组装的方式，在亲疏水的作用下将难溶性药物负载在聚合物胶束中，在超分子静电引力和氢键等非共价作用力下，使带电单体富集在载药胶束的表面，再加入交联剂、引发剂在胶束表面引发原位自由基聚合反应，可以在不改变内部胶束的结构的前提条件下，在表面形成一层聚合物壳层，实现对内部疏水性药物的保护。经过修饰后的载药胶束在稳定性及细胞摄取方面展现出良好的性质，同时聚合物外壳可控释放药物，即外壳可以在特定条件下溶胀、降解，释放出内部负载的药物，在实验中展现出良好的效果，本发明解决了难溶性药物的稳定递送和可控释放。
Resumen de: CN121005730A
本发明提供了一类单硅氧烷醚修饰的双羟基脂质分子、以及包含该脂质分子的纳米颗粒及其制备方法和应用。这一系列结构新颖的单硅氧烷醚修饰的双羟基可离子化的脂质分子制备方法具有合成简单、产率高的优点。同时，包含单硅氧烷醚修饰的双羟基可离子化脂质的LNP粒径均一且分散性好，对于核酸的包封率高，可以高效地递送核酸药物使其具有更好的进细胞效率、核酸的表达效率，溶酶体逃逸效率，并具有脾脏靶向性，在免疫治疗中具有重大的应用前景。
Resumen de: AU2023392764A1
There is provided a pharmaceutical formulation that is useful in the treatment of metabolic disorders or conditions, comprising a plurality of particles suspended in a carrier system, which particles: (a) have a weight-, number-, or volume-based mean diameter that is between amount 10 nm and about 700 µm; and (b) comprise solid cores comprising at least one glucagon-like peptide-1 receptor agonist, or a pharmaceutically-acceptable salt thereof, coated, at least in part, by a coating of inorganic material comprising mixture of: (i) zinc oxide; and (ii) one or more other metal and/or metalloid oxides, wherein the atomic ratio ((i):(ii)) is at least about 1:10 and up to and including about 10:1. Said mixed oxide coated particles are preferably synthesized via a gas phase coating technique, such as atomic layer deposition. The formulation may provide for the delayed or sustained release of glucagon-like peptide-1 receptor agonists to treat metabolic disorders or conditions, such as type 2 diabetes and/or obesity without a burst effect. The glucagon-like peptide-1 receptor agonist is preferably liraglutide.
Resumen de: AU2024241934A1
Described herein are lipid nanoparticle (LNP) formulations for the delivery of active agents, including Archexin.
Resumen de: CN120983384A
本发明涉及靶向SPP1的RNAi剂、包含其的脂质纳米颗粒及其医药用途。具体地本发明涉及靶向SPP1的RNAi剂、IFN‑γmRNA以及包含其的脂质纳米颗粒在癌症治疗中的用途。
Resumen de: CN120983383A
本发明公开了一种用于逆转肺癌耐药的纳米递送系统及其制备方法和应用，属于药物递送技术领域。该系统包括：由透明质酸修饰的聚β‑氨基脂嵌段聚合物形成的载体，所述载体同时包载吉非替尼和靶向间质‑上皮细胞转化因子基因的siRNA（siMET）；其中，所述嵌段聚合物通过二硫键连接HA与PBAE，形成具有pH响应和谷胱甘肽响应双重响应特性的共聚物HA‑ss‑PBAE。本发明为吉非替尼耐药的非小细胞肺癌患者提供新型联合治疗策略，克服单一用药局限性；载体可降解、免疫原性低，具备转化潜力。
Resumen de: CN120983431A
本发明属于医药领域，公开了POCM‑NP@A939572在制备治疗骨质疏松药物中的应用，所述POCM‑NP@A939572包括破骨前体细胞膜囊泡和负载于破骨前体细胞膜囊泡的NP@A939572，所述NP@A939572为包载A939572的PLGA纳米粒。本发明通过将负载A939572小分子药物的纳米粒载入破骨前体细胞膜囊泡中得到的POCM‑NP@A939572具有缓解骨质疏松的效果，可以抑制破骨细胞分化，能够用于骨质疏松的防治和缓解。
Resumen de: CN120988041A
本发明属于生物医药领域，具体涉及式(I)所示氘代的核苷酸及其应用，
Resumen de: CN120983382A
本发明公开了一种具有抗肿瘤作用的杂化细胞膜修饰的仿生纳米粒子及其制备方法和应用，属于药物制剂技术领域，该仿生纳米粒子包括纳米粒子内核及包覆该纳米粒子内核的杂化细胞膜；纳米粒子内核包括两亲性聚合物药物载体和活性药物，活性药物包括光热激动剂TPT和STING激动剂DMXAA；杂化细胞膜由癌细胞膜和巨噬细胞膜共挤出杂化而成。STING激动剂DMXAA与光热激动剂TPT协同发挥作用，可以在近红外激光照射下，产生热量杀伤癌细胞诱导癌细胞免疫原性死亡，释放出损伤相关分子模式，从而激活DMXAA介导的免疫疗法，实现抗肿瘤的效果。
Resumen de: CN120983390A
本发明公开了一种柚皮素载药体系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)将铁皮石斛叶进行组织破碎，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铁皮石斛叶外泌体；(2)将柚皮素溶于二甲基亚砜，将铁皮石斛叶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柚皮素铁皮石斛叶外泌体；(3)重悬于磷酸盐缓冲液，冷冻保存，即得。本发明利用铁皮石斛叶外泌体的肠道菌群调控作用，以及铁皮石斛叶外泌体口服结肠递送功能和细胞内质网定位能力提高柚皮素靶向抑制STING通路治疗炎症作用，通过口服给药治疗炎症性肠病。
Resumen de: CN120989110A
本发明公开了一种用于调控动物生殖能力的mRNA疫苗及其应用，所述mRNA疫苗的抗原为GnRH与Fc蛋白或Folden蛋白偶联成GnRH融合蛋白抗原，增加GnRH了的免疫原性和稳定性，其制成的mRNA疫苗通过实验证明能够很好地控制性激素的水平，进而调控其生殖能力，且安全性良好；并通过实验进一步表明，GnRH‑Fc mRNA疫苗诱导产生的抗体水平要高于GnRH‑Folden mRNA疫苗组，且GnRH‑Fc mRNA疫苗相比GnRH‑Folden mRNA疫苗能够更有效地降低激素水平，从而能够更好地控制动物的生殖能力。
Resumen de: CN120988876A
本发明公开了一种植物乳杆菌，其保藏于中国典型培养物保藏中心(CCTCC)，保藏号为CCTCC NO:M 20241272的菌株BM‑5。本发明菌株在硒添加浓度<0.5g/L亚硒酸钠时可被制备成结合富硒和益生的多功能菌株，在硒添加浓度0.5g/L～40g/L亚硒酸钠时可获得高富硒的冻干菌粉，利用本发明菌株的胞外提取物可以体外还原无机硒获得零价纳米细颗粒，具备应用于生产安全的富硒发酵食品以及其它人体硒补充剂的潜力。
Resumen de: CN120983619A
本发明属于蛋白清除技术领域，公开了一种阿尔茨海默病β淀粉样蛋白清除系统，包括：跨血脑屏障递送模块、Aβ特异识别模块、免疫调控模块、降解与代谢模块；本发明所述复合式Aβ清除系统通过多模块集成，实现了高效穿越血脑屏障、精准识别病灶、快速水解Aβ及炎症控制的协同治疗效果。该系统能够显著降低阿尔茨海默病的病理负担，延缓认知功能退化，体现出相较现有单一抗体或酶类清除剂更为显著的技术进步和应用前景。
Resumen de: CN120987906A
本发明提供了一种基于硫辛酸衍生物的脂质纳米颗粒的制备方法及其应用。本发明所提供的硫辛酸衍生物依靠硫辛酸的环戊烷结构以及二硫键，使得脂质化合物更容易被细胞内化，提高核酸递送的有效性。本发明脂质纳米颗粒有优异的脾脏靶向性，优先将药物递送到脾脏。
Resumen de: CN120989099A
本发明公开了一种用于预防猫传染性疾病的四联mRNA疫苗及其应用，该四联mRNA疫苗能够通过一次性免疫，同时预防猫狂犬病、猫杯状病毒病、猫泛白细胞减少症和猫鼻气管炎，并且通过实验表明该疫苗相对安全、效力强，降低了注射成本，优化了免疫程序，对预防猫传染性疾病具有重大意义。
Resumen de: CN120983385A
本发明公开了一种中药工程化的核‑壳结构金纳米粒及制备方法和应用，制备方法包括：将D1‑miR155修饰的金纳米粒、D2‑miR155修饰的金纳米粒、聚乙二醇修饰的去甲斑蝥素和肿瘤细胞膜混匀，通过脂质体挤出机进行挤压，得到中药工程化的核‑壳结构金纳米粒。本发明制备的金纳米粒能够捕获肿瘤细胞内过表达的致癌基因miR155来激活光热转换效应，诱导肿瘤细胞凋亡并引起免疫原性死亡(ICD)过程。同时，还能通过降低miR155来调控肿瘤的关键信号分子STAT3以增强肿瘤的免疫原性，并有效地递送Tregs抑制剂——去甲斑蝥素(DMC，中药小分子)来抑制肿瘤的免疫逃逸，与光热引起的ICD过程共同实现更强劲的光热‑免疫治疗活性。
Resumen de: AU2024219315A1
The present invention relates to polysaccharide cross-linked colloidal particles and a use thereof as a modifier for in vivo injection. The present invention provides a polysaccharide cross-linked colloidal particle platform technology that can be designed to not only act as a drug delivery carrier for functional nanoparticles or drugs for in vivo introduction, but also control in vivo distribution and excretion.
Resumen de: MX2025011322A
The present invention relates to antisense oligonucleotides (AONs) that can mediate RNA editing by binding to a target RNA nucleic acid molecule, preferably an RNA transcript molecule, in a cell and recruiting an endogenous deaminating enzyme in the cell to deaminate one or more target adenosine nucleotides in the target RNA molecule to an inosine. The target RNA molecule is a transcript molecule form the SLC10A1 gene that encodes the Na<sup>+</sup>/Taurocholate Co-transporting Polypeptide (NTCP). The RNA editing of the one or more target adenosines will result in a loss-of-function of the NTCP protein, which will result in lowered uptake of bile acids from the portal circulation into the liver, thereby lowering the risk of suffering from disorders related to bile accumulation in the liver.
Resumen de: CN120983386A
本发明公开仿生矿化纳米颗粒及其制备方法和用途。本发明的仿生矿化纳米颗粒表面为矿化病毒外壳，能够模拟病毒感染，增强细胞的摄取，并对识别目标蛋白的特异性抗体实现有效地包封，同时病毒膜组分可以诱导细胞内TRIM家族蛋白的表达。在被摄取入胞后，仿生矿化纳米颗粒释放的抗体可以结合目标蛋白，并结合TRIM家族蛋白介导目标蛋白的降解。在这一过程中，TRIM家族蛋白虽然被持续消耗，但可以被本发明的颗粒所诱导的TRIM家族蛋白内源性表达所补偿，从而维持了高效的降解。
Resumen de: CN120983388A
本发明公开了一种靶向涎腺腺样囊性癌肿瘤的双金属复合纳米材料的制备方法及应用。所制备双金属复合纳米材料具有SACC同源靶向能力，用于系统性激活"冷"肿瘤的抗肿瘤免疫反应。该纳米材料在808 nm激光照射下，凭借多重类酶活性并通过光热治疗/光动力治疗/化学动力治疗促进肿瘤细胞内活性氧生成、谷胱甘肽耗竭并加剧氧化应激，同时产生氧气维持PDT/CDT疗效，有效诱导肿瘤细胞免疫原性细胞死亡，显著增强适应性抗肿瘤免疫应答与细胞毒性T淋巴细胞浸润，从而抑制原发灶和转移灶的生长。本发明为逆转SACC"冷"肿瘤表型及抑制全身转移提供了一种有效的NIR触发时空调控系统。
Resumen de: CN120983391A
本发明属于纳米载药系统技术领域，尤其涉及两亲性级联响应壳聚糖纳米载药系统及其制备方法与应用，纳米载药系统为酶响应内层与pH响应外层组成的多层结构；酶响应内层由磺丁基醚‑β‑环糊精与硫酸鱼精蛋白通过静电吸附作用形成，疏水性抗癌药物负载于磺丁基醚‑β‑环糊精的疏水空腔内形成酶响应超分子纳米颗粒；pH响应外层由丁二酸酐亲水改性与脱氧胆酸疏水改性的两亲性壳聚糖构成，两亲性壳聚糖包覆于酶响应超分子纳米颗粒表面。本发明利用这种结构的设置，提供了一种适用于疏水性抗癌药物的肠道靶向递送与控释治疗的基于两亲性壳聚糖纳米载药系统。
Resumen de: CN120987783A
本发明提供了一种酯酶响应可电离阳离子脂质及其在提高核酸药物递送效率方面的应用。本发明提供的脂质在生理条件下呈电中性，具有优异的生物安全性，在偏酸性的内体/溶酶体环境中呈正电荷，通过“质子海绵效应”促进载体及核酸药物逃逸，在胞内酯酶作用下水解继而转变为电中性或负电性化合物，与带负电荷的核酸药物完成解离，最大化发挥核酸药物的生物学功能。因此，本发明提供的脂质可用于体内外核酸药物的高效递送，应用前景广阔。
Resumen de: CN120983389A
本发明属于药物技术领域，具体涉及一种萝卜硫素‑壳聚糖纳米粒及其制备方法和应用。制备为采用离子交联法，以壳聚糖为载体通过滴加带负电的阴离子交联剂，发生静电相互作用，使萝卜硫素负载于壳聚糖形成纳米粒。本发明制备的萝卜硫素‑壳聚糖纳米粒粒径分布均匀，稳定性好，载药量高，包封效果好；制备工艺简易可行，重复性高，极大提高难溶性药物的生物利用度。
Resumen de: CN120983621A
本发明涉及纳米材料制备及生物医药技术领域，公开了一种用于光敏剂与过氧化氢酶共递送的复合制剂及其制备方法，复合制剂包括贴片基底、设置在贴片基底上的微针以及分散在微针内部的纳米颗粒，纳米颗粒包括过氧化氢酶以及原位负载在过氧化氢酶上的疏水光敏剂。该MN贴片可实现PS与CAT的靶向共递送，在肿瘤酸性微环境触发下，NPs发生可控降解并恢复CAT酶活性，同步释放PS。释放的CAT高效催化TME中过表达的H2O2生成O2，缓解肿瘤缺氧并增强PDT疗效；同时酸响应“关闭‑开启”机制避免PS对正常组织的早释损伤。本发明突破传统PDT中PS递送效率低与肿瘤缺氧的双重局限，为实体瘤治疗提供一体化解决方案。
Resumen de: CN120983639A
本发明公开了一种基于多肽修饰的寡核苷酸胞内纳米递送体系及其应用。所述体系由含骨膜蛋白靶向序列（SDSSD）、基质金属蛋白酶2（MMP2）响应序列（GPAGLLG）、细胞穿膜序列（RRRRRRRRR，R9）、活性氧（ROS）清除暨黏附增强基团(Gly‑DOPA))以及末端二苯并环辛炔（DBCO）修饰的工程化多肽SDSSD‑PEG5‑YGFGG‑GPAGLLG‑R9‑(G‑DOPA)3‑K4‑C‑DBCO，与经5聚腺苷酸（AAAAA）、叠氮基团（Azido）修饰的目标寡核苷酸miRNA‑26a‑A5‑Azido，通过点击化学反应及非共价作用组装形成。该纳米体系具备良好的骨靶向性、酶响应性、胞内递送效果与生物安全性，能够在体内实现对治疗性寡核苷酸的稳定高效递送，显著提升寡核苷酸的利用效率与治疗潜能。所述寡核苷酸胞内纳米递送体系在寡核苷酸药物的临床转化与精准治疗领域具有广阔应用前景。
Resumen de: CN120983387A
本发明涉及一种仿生响应性的多功能纳米剂及其制备方法与应用，属于生物医药领域。本发明的纳米剂由抗炎药物、造影剂金属离子和多巴胺自组装形成颗粒核心，外层包裹细胞膜作为壳层。该膜具有优异的生物相容性，可在体内实现长期循环，逃避免疫清除，并选择性靶向炎症部位。该纳米剂创新性地整合了细胞膜靶向、ROS响应性、多功能治疗及成像监测等特性，不仅为AP提供了多靶点协同治疗与精准诊断策略，更为其他炎症相关疾病的纳米药物开发开辟了新的思路。
Resumen de: WO2025237387A1
The present disclosure relates to a lipid composition, including a polymeric lipid, and the polymeric lipid is a compound with a structure shown in formula (I), or a pharmaceutically acceptable salt, an isotopic variant, a tautomer, or a stereoisomer thereof. The present disclosure further provides a nano-particle composition and a pharmaceutical composition including the lipid composition, and an application of the lipid composition in a delivery load. The polymeric lipid compound in the lipid composition of the present disclosure may replace a polyethylene glycol (PEG) lipid and has the advantages of biocompatibility, readily decomposable in vivo, and non-toxic, thus a new lipid delivery system is obtained.
Resumen de: WO2025237388A1
The present disclosure relates to a polymeric lipid compound, in particular to a compound in formula (I), or a pharmaceutically acceptable salt, an isotopic variant, a tautomer, or a stereoisomer thereof. The present disclosure further provides a nano-particle pharmaceutical composition including the compound, and an application of the compound and its composition in a delivery load. The polymeric lipid compound of the present disclosure may replace a PEG lipid and has the advantages of biocompatibility, readily decomposable in vivo, and non-toxic, thus a new lipid delivery system is obtained.
Resumen de: WO2025240833A1
Compounds having the structure of Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof; wherein R1, R2, R3, R4, R5, L1, L2, a and z are as defined herein, are disclosed. Use of these 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.
Resumen de: WO2025238385A1
The present invention is referred to a method for production of a pharmaceutical composition, said composition comprises: A) naturally secreted exosomes from normal human embryonic lung fibroblast cells (MRC-5 cell line), wherein said exosomes comprise a lipophilic and/or hydrophilic active substance, said method comprises the following steps in the following order: a) separation of exosomes by ultrafiltration, b) collection of exosomes by membrane-based affinity binding method, c) loading of exosomes with active substance, or B) artificial exosomes, wherein said exosomes comprise a lipophilic and/or hydrophilic active substance, said method is a microfluidic method and said method comprises the following steps in the following order: a) extraction of lipids from a cell line of normal human embryonic lung fibroblast cells (MRC-5 cell line) to obtain a lipid film, b) resuspending of the lipid film in an organic solvent and mixing with the aqueous phase in a microfluidics device to obtain exosomes, wherein the aqueous phase comprises Tween 20 and wherein in the microfluidics device I) the flow rate ratio of the organic phase to the flow rate of the aqueous phase (flow rate ratio, FRR) is set from 1:5 to 1:3, ii) the organic phase flow rate (OPFR) is preferably set at 200 pL/min or 400 pL/min and ill) the aqueous phase flow rate (APFR) is preferably set at 100 pL/min or 1000 pL/min, c) purification of the obtained exosomes using size exclusion chromatography, d) loading of the e
Resumen de: WO2025238502A1
In one aspect, the present disclosure relates to adjuvant nanoparticles comprising aluminum and/or CpG oligodeoxynucleotides. In particular embodiments, the nanoparticles have an average size of less than about 100 nm. Also provided herein are methods of making the nanoparticles using a microfluidic device.
Resumen de: WO2025237830A1
The present invention refers to novel polyoxyalkylene based compounds and their manufacturing method as well as compositions comprising at least one novel polyoxyalkylene based compound and at least one active agent. Furthermore, the present invention refers to the manufacture of the compositions of the present invention as well as their use for the treatment of an illness in mammals or humans.
Resumen de: US2025352666A1
The present disclosure relates to pharmaceutical compositions comprising a lipid nanoparticle (LNP), a therapeutic nucleic acid (TNA) and at least one pharmaceutically acceptable excipient, wherein the LNP comprises at least one lipid, and wherein the LNP is capable of delivering the TNA to a retinal cell. Methods of treating ocular diseases and disorders are also provided.
Resumen de: US2025352575A1
A method of manufacture and compositions and uses of a multifunctional bioinorganic theranostic nanoconstruct are disclosed. Nanoconstructs described herein contain manganese dioxide in a biocompatible matrix and are useful for, e.g., MRI contrast imaging of tumor environments and enhancement of radiation therapy in cancer. Some nanoconstructs described herein incorporate targeting agents for specific targeting of cancer cells.
Resumen de: US2025352579A1
Provided are an anti-CD7 nanobody and a derivative thereof. The derivative comprises a humanized anti-CD7 nanobody, a chimeric antigen receptor based on a single nanobody, a chimeric antigen receptor based on a double nanobody, a recombinant expression vector, an engineered host cell, a conjugate, a pharmaceutical composition, a kit, and a reagent for detecting CD7 on the cell surface. The nanobody has a good affinity to CD7, and the prepared CAR-T cells target and recognize tumor antigens and have high killing activities against tumor cells.
Resumen de: US2025352637A1
The invention relates to a method of treating a disease or disorder in a patient in need thereof that includes providing an active pharmaceutical ingredient (API) to the patient by administering more than one split-dose of the API over a pre-determined period of time. In embodiments of the invention, the API is an mRNA encoding an antigen. The attractiveness of mRNA as a vaccine modality is supported by several advantages. As a non-infectious agent that does not require incorporation into the host's genome to confer activity along with its well-defined chemical composition, mRNA is regarded as a relatively safe vaccine modality.
Resumen de: US2025352664A1
Methods of administering lipid nanoparticle compositions including poly(ethyloxazoline)-lipid conjugates (PEOZ-lipid LNPs) without triggering a PEOZ-associated immune response. In some aspects, the PEOZ-lipid LNPs trigger a protective response based on the encapsulated payload but do not trigger an IgM or IgG response. also provides an absent or. In other aspects, the PEOZ-lipid LNPs trigger a protective response based on the encapsulated payload but trigger an IgM and IgG response that is markedly reduced (as compared to a comparable PEG-lipid currently used in LNP vaccine delivery systems). PEOZ-DMA LNPs incorporating payloads such as oligonucleotides payloads mRNA, DNA, and siRNA for delivery into living cells is also contemplated.
Resumen de: US2025352663A1
Compositions, methods, and kits are provided for treating infections and cancer with metallic nanoclusters. In particular, metallic nanoclusters having a size of less than 10 nm that are conjugated to adenosine triphosphate (ATP) or an analogue thereof can be used to eradicate a cell in a growth arrest phase such as infectious bacterial or fungal cells. Such nanoclusters can also induce endoplasmic reticulum stress and inhibit growth of cancerous cells. Additionally, such metallic nanoclusters can be used to inhibit a purinergic P2X7 receptor and FtsH protease.
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.
Resumen de: US2025352487A1
Provided herein are compositions and methods of delivering nucleic acids, such as therapeutic mRNAs and DNA, to the central nervous system by delivery, e.g., intrathecally or intracerebrally, of nucleic acid containing lipid nanoparticles using sphingolipids as helper lipids.
Resumen de: US2025352489A1
A gene delivery system and applications thereof in the technical field of biological medicines that is particularly useful in the preparation of drugs for treatment of tumors are disclosed. The gene delivery system and applications relate to technology for inducing the differentiation of malignant tumor cells into mature cells, in which regulating the expression of HNF4 alpha protein in the malignant tumor cells using messenger ribonucleic acid, the malignant phenotype of the malignant solid tumor cells is inhibited, and the effective treatment of the malignant solid tumors is achieved. The gene delivery system and applications are therefore applicable to a method of preparing a drug for treating malignant solid tumors and to a method of treating a patient having a malignant solid tumor.
Resumen de: US2025352507A1
The present invention relates generally to hybrid polymer (e.g., polyphosphazene) based drug delivery platforms and to methods of producing, evaluating, administering, and treating subjects with the same. More particularly, the present invention provides polyphosphazene based drug delivery platforms comprising one or more polyphosphazenes with controlled molecular weights and/or polydispersities as well as selective methods for associating one or more therapeutic drug (or prodrug) substances to the polyphosphazenes.
Resumen de: US2025352491A1
The present disclosure is drawn to nanoparticles, method of using and making thereof. The disclosed nanoparticles comprise nanoparticles, that may be rod-shaped and/or coated on the outer surface with albumin, that are designed to effectively cross lymphatic barriers as an effective strategy to improve the efficacy and translatability of putative immunotherapies. Delivery systems or pharmaceutical compositions comprising the disclosed nanoparticles are also provided.
Resumen de: US2025352488A1
One or more ionizable lipid(s) and lipid nanoparticles comprising same are provided. Pharmaceutical compositions comprising the lipid nanoparticles encapsulating an active agent are also provided.
Resumen de: US2025352543A1
A nanocrystalline preparation and a preparation method therefor, the nanocrystalline preparation containing a ROCK2 inhibitor and a stabilizer. The present invention also relates to use of the nanocrystalline preparation in the prevention, alleviation, and/or treatment of selected diseases and medical conditions, especially diseases such as idiopathic pulmonary fibrosis, fatty liver disease and/or steatohepatitis, post-hematopoietic stem cell transplantation graft versus host disease or viral infection.
Resumen de: CN120569192A
The invention relates to an iron nanocluster having the following features:-the surface of which is covered with a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc),-which has a spherical shape,-the hydrodynamic diameter of which is 0.6-2.0 nm, preferably less than 1.0 nm,-the metal core diameter of which is 0.5-1.5 nm, preferably less than 1.0 nm,-the metal core diameter of which is 0.5-1.5 nm, preferably less than 1.0 nm,-the surface of which is coated with a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc),-which has a spherical shape. The nanocluster has a stability duration of 5 to 20 weeks when the nanocluster is in liquid form and stored at a temperature of 4 DEG C, a stability duration of at least 12 months, preferably 12 to 18 months, when the nanocluster is in dry form and stored at a temperature of 4 DEG C and under nitrogen gas, and exhibits spectrophotometric properties, and the nanocluster has a stability duration of 5 to 20 weeks when the nanocluster is in liquid form and stored at a temperature of 4 DEG C and under nitrogen gas, preferably 12 to 18 months when the nanocluster is in dry form and stored at a temperature of 4 DEG C and under nitrogen gas. The iron nanocluster is characterized in that the shoulder peak in the UV-visible spectrum is located at 300 +/-15 nm, the excitation wavelength of the fluorescence spectrum is 364 +/-15 nm, the emission wavelength is 415 +/-15 nm, and the formul
Resumen de: CA3273638A1
The invention relates to methods and materials for use in treating neurodegenerative diseases associated with pathological protein aggregates and provides fusion proteins comprising: (i) a first portion comprising a sequence of a protein which aggregates pathologically in neurodegenerative disease, such as tau protein, and (ii) a second portion comprising a RING-type E3 ubiquitin ligase, a component of a RING-type E3 ubiquitin ligase complex, or a domain of either. These fusion proteins have utility in selectively or preferentially targeting pathogenic protein aggregates for degradation.
Resumen de: MX2025005463A
Compounds, compositions, uses, and methods for reducing obesity in a subject, or for preventing or treating obesity, are provided herein. In certain examples, methods for reducing obesity in a subject and/or for preventing or treating obesity in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1aGDP), one or more expressible nucleic acids encoding Rab1aGDP, or a combination thereof.
Resumen de: CN120359023A
Disclosed herein are RNA compositions comprising one or more polynucleotides encoding one or more gene editing systems, the one or more polynucleotides being formulated within a lipid reconstituted native messenger packet (LNMP) comprising a native lipid and an ionizable lipid. The disclosure also includes a method for preparing an RNA composition comprising reconstituting a membrane comprising a purified NMP lipid in the presence of an ionizable lipid to produce an LNMP comprising the ionizable lipid, and loading one or more polynucleotides encoding one or more gene editing systems into the LNMP. The present disclosure also includes RNA compositions that can be repeatedly administered.
Resumen de: US2025352473A1
Disclosed herein are compositions comprising nanoliposomes useful for the treatment and prevention of cerebrovascular and aging-related degenerative diseases.
Resumen de: US2025354174A1
Nanoparticle compositions for delivery of nucleic acids to subjects including carriers comprising sugar functionalized nucleic acid carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the delivery molecules are described. Methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects are provided.
Resumen de: US2025353931A1
The present disclosure relates to a molecular delivery system that facilitates the internalization of LNPs into a specific target of choice, such as a specific cell type, ex vivo and/or in vivo. The present disclosure also relates to methods, molecules, and compositions for enhancing the targeted delivery of compounds within a living system. In particular, embodiments provided herein relate to methods, molecules, and compositions for the targeted delivery of lipid nanoparticles containing therapeutic molecules into a cell or system of choice, such as a T cell. The present disclosure also relates to methods of administering the enhanced targeting system to a patient or system, compositions for use in such methods, and further methods of use of the targeting system as part of T cell-based immunotherapy.
Resumen de: US2025352537A1
The present invention relates to solid phase pharmaceutical compositions of 6′-fluoro-N-(4-fluorobenzyl)-4′-oxo-3′,4′-dihydro-1′H-spiropiperidine-4,2′-quinoline-1-carboxamide that is useful as a AKR1C3 dependent KARS inhibitor. The present invention also relates to processes for the preparation of said pharmaceutical compositions of said compound, methods of using said pharmaceutical compositions in the treatment of various diseases and disorders, and their use in diseases and disorders mediated by an AKR1C3 dependent KARS inhibitor.
Resumen de: US2025354140A1
The disclosure is generally related to calcium salted spherical nucleic acids (SNAs). SNAs comprise a nanoparticle core surrounded by a shell of oligonucleotides. In some aspects, the disclosure provides a spherical nucleic acid (SNA) comprising: (a) a nanoparticle core; and (b) a shell of oligonucleotides attached to the external surface of the nanoparticle core, wherein one or more oligonucleotides in the shell of oligonucleotides comprises a phosphate backbone; the SNA comprising Ca2+ ions adsorbed to the phosphate backbone of one or more oligonucleotides in the shell of oligonucleotides. Methods of making and using the SNAs are also provided herein.
Resumen de: US2025354138A1
This disclosure provides compositions and methods for the editing of a SERPINA1 gene with prime editing.
Resumen de: US2025353809A1
Disclosed are a nitrogen-containing chain compound, a preparation method, a composition containing said compound, and a use thereof. Provided is a nitrogen-containing chain compound represented by formula (I) or a pharmaceutically acceptable salt thereof. The nitrogen-containing chain compound represented by formula (I) may be used for preparing a lipid carrier. The prepared lipid carrier can encapsulate a nucleic acid drug, and may be used for delivering a nucleic acid prophylactic agent and/or therapeutic agent to mammalian cells and organs and producing an effect.
Resumen de: US2025352486A1
The present invention is based on a nanodevice comprising a nanoparticle comprising a therapeutic agent, a molecular drill comprising a compound with catalytic activity, and a self-propulsion system. The molecular drill in combination with the self-propulsion system allows breaking the matrix of biofilms formed by infectious microorganisms with high efficiency and the molecular drill, in turn, allows the therapeutic agent to be released from the nanoparticle under specific conditions, preferably in the acidic environment of an infection. The invention relates to the nanodevice, to the nanodevice used in the treatment of an infection caused by biofilms, and to the use of the nanodevice to disinfect samples in vitro or inert materials ex vivo.
Resumen de: WO2024229321A1
This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects.
Resumen de: AU2025260005A1
Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. ov o v
Resumen de: US2025352665A1
The present invention relates to compositions comprising particles, each of said particles comprising a complex of at least one double-stranded polyribonucleotide, such as polyinosinic-polycytidylic acid poly(IC), and at least one linear polyal-kyleneimine. The particles are also characterized by their monomodal diameter distribution and z-average diameter within specific ranges. The present invention additionally relates to the use of said compositions as medicaments, in particular for the treatment of a cell growth disorder characterized by abnormal growth of human or animal cells, as well as to processes for the preparation of said compositions.
Resumen de: AU2025259971A1
A formulation comprising an oligonucleotide selected from the group consisting of an oligonucleotide having one of SEQ ID No.:1 through SEQ ID No.:42 or a variant thereof. Also, a method of treating anesthesia-induced neurotoxicity. The method may comprise administering the formulation. The formulation may be administered prior to, concomitant with, subsequent to, or combinations thereof administration of a general anesthetic comprising a fluorinated compound. The oligonucleotide may be incorporated into a carrier system, for example, a liposome, a biodegradable polymer, a hydrogel, or a cyclodextrin, a nucleic acid complex, a virosome, or combinations thereof. Also, a method of treating anesthesia-induced neurotoxicity. A formulation comprising an oligonucleotide selected from the group consisting of an oligonucleotide having one of SEQ ID No.:1 through SEQ ID No.:42 or a variant thereof. Also, a method of treating anesthesia-induced neurotoxicity. The method may comprise administering the formulation. The formulation may be administered prior to, concomitant with, subsequent to, or combinations thereof administration of a general anesthetic comprising a fluorinated compound. The oligonucleotide may be incorporated into a carrier system, for example, a liposome, a biodegradable polymer, a hydrogel, or a cyclodextrin, a nucleic acid complex, a virosome, or combinations thereof. Also, a method of treating anesthesia-induced neurotoxicity. ct c t
Resumen de: AU2024283556A1
The present invention addresses the problem of providing a technology for improving poor water solubility of a compound of general formula (1), and suppressing excessive release of cytokines and myelotoxicity of the compound of general formula (1). The present invention solves the problem by a complex comprising: a modified polysaccharide containing a hydrophobic group; and a compound represented by general formula (1).
Resumen de: AU2024272346A1
The present technology relates to recombinant polypeptides, or nucleotides encoding the same, comprising tissue plasminogen activator (tPA) fragments and uses thereof for treating cardiovascular diseases. In some embodiments, the tPA fragments comprise the kringle 2 domain of tPA.
Resumen de: AU2024267555A1
The invention relates to the production, modification, and use of extracellular vesicles (EVs), particularly for the delivery of payloads of nucleic acids. The EVs can contain retroviral capsids and can be coated with polymers. The EVs can be treated with a protease to remove proteins on the outer surface of the EV to create shaved EVs. The shaved EVs can be coated with a polymer and used for the delivery of payloads of nucleic acids to cells.
Resumen de: AU2024243584A1
Mucosal patches are disclosed that have a support layer supporting an active layer that has a collagen carbon dot nanocomposite carrying an active agent. The collagen carbon dot nanocomposite is absorbable through a mucosa. The mucosal patches are part of various methods of treatment.
Resumen de: AU2024269428A1
The invention provides submicronic liposomal formulations in a dry powder form characterized in that they can incorporate various types of lipophilic and hydrophilic actives while maintaining their original structure and size under various production conditions, reconstitution mediums, varying pH, osmolarity and storage conditions.
Resumen de: WO2025240555A1
The present invention relates to compositions comprising nanoparticles associated with (e.g., complexed, conjugated, encapsulated, absorbed, adsorbed, admixed) celastrol and/or rapamycin. In particular, the present invention relates to nanoparticles (e.g., sHDL nanoparticles) associated with celastrol and/or rapamycin, and related methods of use (e.g., in diagnostic and/or therapeutic settings), and optionally, a plurality of tolerogenic antigens.
Resumen de: WO2025240771A1
In accordance with at least one aspect of this disclosure, there is provided a small zwitterionic multimeric targeted anti-cancer drug which effectively addressed tumor cell heterogeneity and off-target/non-specific binding and uptake. The small zwitterionic multimeric targeted anti-cancer drug includes a nexus, three different targeting ligands conjugated to the nexus, a payload of a zwitterionic diagnostic or therapeutic agent, and zwitterionic flexible linkers connecting each of the three different targeting ligands and the pay load to the nexus.
Resumen de: WO2025240356A1
Provided herein are lipid nanoparticles composition comprising a lipid component which comprises: (i) a first lipid wherein the first lipid is an ionizable cationic lipid, (ii) a second lipid wherein the second lipid is separate from the first lipid, (iii) a phospholipid, and (iv) a PEG-lipid wherein the second lipid is a lipid having a structural formula S-I'a, S-I'b, or S-I'c. Also, provided herein is a method of treating or preventing a disease or disorder in a subject in need thereof, the method comprising administering an effective amount of the lipid nanoparticle composition disclosed herein.
Resumen de: WO2025240355A1
Provided herein are lipid nanoparticles composition comprising a lipid component which comprises: (i) a first ionizable cationic lipid, (ii) a PEG-lipid, wherein the PEG-lipid comprises a PEG-Ceramide. Further provided are lipid nanoparticle compositions comprising a lipid component which comprises: (i) a first ionizable cationic lipid, (ii) a PEG-lipid, wherein the PEG-lipid comprises a PEG-Ceramide selected from: N-octanoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)5000} (C8 PEG5000-Ceramide), N-octanoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)2000}(C8 PEG2000-Ceramide), N-octanoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)750} (C8 PEG750-Ceramide), N-palmitoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)5000} (C16 PEG5000-Ceramide), N-palmitoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)2000} (C16 PEG200-Ceramide), and N-palmitoyl-sphingosine-1-{succinylmethoxy(polyethylene glycol)750} (C16 PEG750-Ceramide) (iii) a phospholipid, and (iv) a permanently cationic lipid, an anionic lipid, or a second ionizable cationic lipid separate from the first ionizable cationic lipid.
Resumen de: WO2025240477A1
This document provides methods and materials for expressing one or more polypeptides in immune cells (e.g., one or more splenic immune cells such as B cells and T cells). For example, CpG-oligodeoxynucleotides (CpG-ODNs) that can be used to target lipid nanoparticles (LNPs; e.g., LNPs including a nucleic acid having the ability to encode a polypeptide) to immune cells (e.g., one or more splenic immune cells such as B cells and T cells) are provided. In some cases, one or more CpG-ODNs provided herein can be administered to a mammal (e.g., a human) together with one or more LNPs including a nucleic acid having the ability to encode a polypeptide to express that polypeptide in immune cells (e.g., one or more splenic immune cells such as B cells and T cells) within the mammal.
Resumen de: WO2025240485A1
The disclosure is directed at methods for reducing the bioburden of both particles comprising an active pharmaceutical ingredient (API) and the equipment used to process such particles. A method of applying a coating to particles comprising an API comprises: (a) loading the particles into a chamber; (b) applying a vaporous or gaseous metal or metalloid precursor to the particles in the reactor by pulsing the vaporous or gaseous aluminium precursor into the reactor; (c) performing one or more pump-purge cycles using an inert gas; (d) applying a vaporous or gaseous oxidant to the particles in the reactor by pulsing the oxidant into the reactor; (e) performing one or more pump-purge cycles using an inert gas; (f) repeating steps (a)-(e) at least once to provide a metal oxide or metalloid oxide coating layer on the particles; and a sterilization step before step (a), between step (a) and step (b), and/or after step (f).
Resumen de: WO2025238646A1
Particulate compositions comprising magnesium and low molecular weight chitosan and ss or dsRNA partially complementary to, binding to or at least 90% identical to mRNA targets of microsporidia and viruses pathogenic in farmed crustaceans, compositions and farmed aquatic crustaceans comprising the same, and methods for their use in treating or preventing parasitic microsporidia and viral infection in aquaculture are provided.
Resumen de: WO2025238578A1
The present disclosure provides gRNAs for allele-selective gene editing, nucleic acids encoding such a gRNA, vectors comprising such a nucleic acid, RNPs comprising such a gRNA and a Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition using such gRNAs. The present disclosure further provides lipid-based TCVs.
Resumen de: WO2025238579A1
The present disclosure provides dual gRNAs for allele-selective or specific gene editing, nucleic acids encoding such gRNAs, vectors comprising such nucleic acids, RNPs comprising such gRNAs and Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective or specific gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition, containing. The present disclosure further provides lipid-based TCVs.
Resumen de: WO2025237276A1
Provided is an improved mRNA molecule, containing a UTR that enhances mRNA stability and/or the translation efficiency, and preferably further comprising an optimized coding sequence for a polypeptide of interest.
Resumen de: WO2025237331A1
Provided is use of annexin in improving the tumor penetration efficiency of a nanocarrier with a phospholipid surface. The present invention innovatively explored a protein capable of promoting the tumor penetration efficiency of a phospholipid-based nanocarrier, and found that coating the phospholipid-based nanocarrier with annexin can improve the tumor penetration efficiency, thereby improving the bioavailability of a drug loaded with the nanocarrier and improving the therapeutic effect. In addition, it is also found that the efficiency-enhancing ability of annexin is positively correlated with the expression level of integrin α5 or β1. By means of pre-detecting and analyzing the expression level of integrin α5 or β1 in a patient, the ability of annexin coating technology to promote the transport of nanocarriers can be predicted, and the beneficiary population suitable for the use of the annexin coating technology can be screened.
Resumen de: WO2025237108A1
Disclosed in the present invention are a polysulfide polymer, and a preparation method therefor and a use thereof, relating to the field of pharmaceutical chemistry. In the present invention, the polysulfide polymer, or a pharmaceutically acceptable salt thereof, or a stereoisomer thereof, or a derivative thereof has the following repeating unit structure. Disclosed in the present invention is a method for preparing the polysulfide polymer or the derivative thereof. Further disclosed in the present invention is a nano-micelle or vesicle, comprising the polysulfide polymer or the derivative thereof. Disclosed in the present invention is a use of the polysulfide polymer or the derivative thereof and the nano-micelle or vesicle in the preparation of an H2S donor or/and a drug carrier. The polysulfide polymer or the derivative thereof of the present invention can be used as an H2S donor to achieve effective H2S treatment, and can also be used as a drug carrier to improve delivery and improve the therapeutic effect.
Resumen de: WO2025236837A1
The present invention pertains to the technical field of biomedicine, and specifically relates to a gene delivery system and use thereof in the preparation of a tumor therapeutic drug. The present invention relates to a technical means for inducing differentiation of tumor cells into mature cells, which utilizes messenger ribonucleic acid to regulate the expression of HNF4α in tumor cells, inhibits the malignant phenotype of malignant solid tumor cells, and achieves the effect of treating malignant solid tumors, thereby being applied to a preparation method and use of a solid tumor drug.
Resumen de: WO2025237069A1
Provided in the present invention are an ionizable lipid compound, a liposome, a lipid nanoparticle, and the use thereof. The ionizable lipid compound is selected from the structure as shown in the following formula (I). A lipid nanoparticle prepared using the ionizable lipid compound provided in the present invention has uniform particle size distribution and low cytotoxicity, and is capable of specifically targeting and delivering an active substance to cells, tissue, or organs, thereby realizing the targeted delivery of the active substance.
Resumen de: WO2025236311A1
The present invention relates to the field of lipid nanoparticle biological application technology, and in particular, to a composite lipid nanoparticle and a method for preparing same, an RNA vaccine, a medicament, and use. The provided composite lipid nanoparticle comprises a lipid composition and a streptavidin-labeled recombinant antibody protein; and the lipid composition comprises an ionizable lipid, a helper lipid, cholesterol, a polyethylene glycol lipid, and a biotinylated polyethylene glycol lipid in a mass ratio of (40-90):(0.1-20):(20-50):(0.5-10):(0.5-10). The composite lipid nanoparticle, by means of strong interaction force between the streptavidin-labeled recombinant antibody protein and the biotinylated polyethylene glycol lipid in the composite lipid nanoparticle, can achieve safe, convenient, rapid, efficient, and stable connection between the LNP and the recombinant antibody protein, and the recombinant antibody protein can be adaptively replaced according to the target cells and/or tissues, resulting in high flexibility and applicability. The recombinant antibody protein on the obtained composite lipid nanoparticle still has good specific recognition and binding capabilities to the corresponding protein.
Resumen de: WO2025236040A1
Disclosed herein are solid lipid nanoparticles comprising a fatty acid esterified with polyethylene glycol. The polyethylene glycol is functionalised with an aptamer (i.e. targeting ligand). The nanoparticle may be loaded with an antibiotic or an antifungal, and a fluorescent compound. Also provided are their methods of preparation and their use in drug delivery and biosensing.
Resumen de: WO2025236353A1
The present application discloses a three-dimensional stent, a preparation method therefor, and use thereof. Starting materials for preparing the three-dimensional stent provided by the present application include a carbon-inorganic oxide hybrid micro-nanofiber and carrageenan. The three-dimensional stent provided by the present application not only enables photo-thermal treatment, photo-thermal antibacterial effects, controlled drug release, and bone repair, but also has excellent biocompatibility and biological activity.
Resumen de: WO2025240946A1
Lipid nanoparticles comprising ionizable lipids, helper lipids, neutral lipids, structural PEG-lipids, and anchor PEG-lipids are provided herein, together with targeted lipid nanoparticle compositions, and uses thereof. Methods of administering targeted lipid nanoparticles and targeted lipid nanoparticle compositions for the delivery of biologically active agents, are also provided. Such compositions and methods can be used, for example, to deliver a cargo (e.g., a mRNA cargo), to a cell population of interest.
Resumen de: EP4650349A1
The invention provides a compound, its preparation method, and lipid nanoparticles, as well as a pharmaceutical composition. The compound includes at least one structural formula (I-1) and may be provided as a pharmaceutically acceptable salt, prodrug, or isomer. It increases structural diversity among lipid molecules and is biodegradable. The L1a, L1b, and L1c groups in the compound can be interchanged with L2, enabling the integration of other molecules like steroids or peptides. This interchangeability expands the functionality of cationic lipid compounds, allowing for the direct coupling of functional molecules to the lipid compound to enhance its functional scope. The replaceable L groups offer greater flexibility in selecting L1a, L1b, L1c, and L2.
Resumen de: EP4650343A1
An amino lipid compound, and a preparation method therefor and the use thereof. The present invention further relates to a lipid nanoparticle and a pharmaceutical composition containing the amino lipid compound, and the use thereof.
Resumen de: US2025090472A1
The present application relates to lipid nanoparticles having a diameter above certain value, as well as the preparation and uses of such compositions. Lipid nanoparticles presented in the application generally have a diameter of at least 160 nm. Such lipid nanoparticles are useful in the delivery of therapeutic agents, such as nucleic acids, in vivo to none-hepatic organs (e.g., lung) for the treatment or prevention of certain diseases or disorders.
Resumen de: AU2024207086A1
A miRNA-mimic based therapeutic particle is disclosed herein. The particles comprise a synthetic miRNA or mimic of miR-187-3p encapsulated in a lipid nanoparticle (LNP) carrier or synthetic miR-193b-5p inhibitor encapsulated in a lipid carrier or their combination encapsulated in a lipid carrier. The lipid nanoparticle carrier is made up of at least four (4) types of lipids, in which the four (4) types of lipids include a) an ionizable cationic lipid selected to be positively charged in a formulation buffer (pH 4), which binds and protects the negatively charged miRNA, and facilitates endosomal escape, and is neutral in a storage buffer, b) a sterol in the structure of the lipid nanoparticle (LNP)., c) a structural helper lipid selected to contribute to lipid nanoparticle stability and/or enhances endosomal release, and d) a PEGylated-lipid selected such that it stabilizes the therapeutic particle and protects it from opsonization.
Resumen de: AU2024265712A1
Suspension formulations of nanoparticles of clobetasol propionate are described. The suspensions can be used therapeutically to treat skin and ocular burns; to enhance wound healing; to prevent or reduce hypertrophic scarring/keloids; to treat allergic rhinitis/sinusitis, asthma, inner ear disorders including hearing loss, tinnitus, or vertigo, tenosynovitis, tendinitis, enthesitis or arthritis.
Resumen de: EP4650358A2
The present disclosure provides a ribose-modified cap analog and a use thereof, and belongs to the technical field of chemical and biological engineering. The ribose-modified cap analog has a structure of formula (I). The ribose-modified cap analog described herein can improve the stability of mRNA and/or the translation efficiency of mRNA.
Resumen de: EP4649964A1
The present disclosure belongs to the technical field of lipid nanoparticle biological application, and discloses a composite lipid nanoparticle and a preparation method thereof, an ribose nucleic acid (RNA) vaccine, a drug and use. The composite lipid nanoparticle provided by the present disclosure includes a lipid composition and a recombinant antibody protein containing a streptavidin tag; and the lipid composition comprises an ionizable lipid, an auxiliary lipid, cholesterol, a polyethylene glycol lipid and a biotinylated polyethylene glycol lipid in a mass ratio of (40-90):(0.1-20):(20-50):(0.5-10):(0.5-10). Through strong interaction between the recombinant antibody protein with the streptavidin tag and the biotinylated polyethylene glycol lipid in the composite lipid nanoparticle, the composite lipid nanoparticle achieves safe, convenient, rapid, efficient, and stable linkage between a lipid nanoparticle (LNP) and a recombinant antibody protein, and the recombinant antibody protein can be adaptively replaced according to target cells and/or tissues, with high flexibility and applicability. Moreover, the recombinant antibody protein on the obtained composite lipid nanoparticle still has a good ability to specifically recognize and bind a corresponding protein.
Resumen de: WO2024150222A1
One or more ionizable lipid(s) and lipid nanoparticles comprising same and further encapsulating a polynucleic acid are provided. Pharmaceutical compositions comprising a therapeutically effective amount of the lipid nanoparticles encapsulating a therapeutically active polynucleic acid are also provided. Pharmaceutical compositions are used in delivering of the polynucleic acid to a lung tissue of a subject are also provided.
Resumen de: MX2025009500A
The invention relates to the field of vaccine compositions. The invention more particularly relates to a prophylactic vaccine composition that is intended for mammals and birds and comprises a killed whole bacterium, said bacterium being covered with a cationic agent, in particular cationic nanoparticles.
Resumen de: EP4649962A1
The present invention discloses a method for circumventing pre-existing anti-PEG antibodies in the human body and the use of a terminal hydroxyl-modified PEGylated nanocarrier in the preparation of a drug that circumvents pre-existing anti-PEG antibodies in the human body. The present invention also discloses a terminal hydroxyl-modified PEGylated nanocarrier and a nanoformulation comprising the same. The terminal hydroxyl-modified PEGylated nanocarrier and nanoformulation according to the present invention have a low binding to pre-existing anti-PEG antibodies in the human body, so that they can circumvent being rapidly cleared in the human blood and better exert the therapeutic effect. In addition, the terminal hydroxyl-modified PEGylated nanocarrier and nanoformulation can reduce complement activation by circumventing a binding to pre-existing anti-PEG antibodies in human blood, thereby alleviating side effects such as clinical injection reactions.
Resumen de: MX2025005156A
The present disclosure provides methods for eliciting an immune response against respiratory syncytial virus (RSV) in a subject. The present disclosure also provides methods for preventing an RSV infection or reducing one or more symptoms of an RSV infection in a subject.
Resumen de: AU2024219315A1
The present invention relates to polysaccharide cross-linked colloidal particles and a use thereof as a modifier for in vivo injection. The present invention provides a polysaccharide cross-linked colloidal particle platform technology that can be designed to not only act as a drug delivery carrier for functional nanoparticles or drugs for in vivo introduction, but also control in vivo distribution and excretion.
Resumen de: CN120960169A
本发明公开了一种基于聚硫辛酸的纳米制剂药物的制备方法及纳米制剂药物，所述方法包括：将硫辛酸与药物溶于有机溶剂形成均相溶液，在搅拌下滴加去离子水后加入还原剂，于20～40℃原位引发硫辛酸聚合与药物共组装，形成载药胶束溶液，经透析纯化后制得基于聚硫辛酸的纳米制剂药物。本发明创新性在于利用硫辛酸兼具"药物载体基质"与"动态交联单体"的双重特性，通过一步原位聚合‑共组装同步实现载体合成与药物高效包封，突破传统纳米载体需预合成及分步载药的技术瓶颈。实用性表现为：（1）工艺简捷高效，无需复杂设备，反应条件温和；（2）载药性能优异，粒径均一可调控（50～300纳米）；（3）聚硫辛酸骨架赋予纳米药物制剂环境响应性，可实现智能释药；（4）适用于疏水药物及复方制剂。本方法为疏水药物递送提供了高载量、高稳定性、刺激响应的纳米化解决方案，在纳米医药领域具有应用价值。
Resumen de: CN120324608A
The invention discloses a Mo-PDA-coated GOx nano particle and a preparation method and application thereof, and relates to the technical field of medicine preparation, the nano particle is a spherical structure Mo-PDA-coated GOx nano particle which is formed by mixing and reacting phosphomolybdic acid Mo-POM and dopamine to prepare a composite nano particle Mo-PDA and then loading glucose oxidase (GOx), the mixing reaction comprises the steps of taking the phosphomolybdic acid Mo-POM and the dopamine to prepare the Mo-PDA-coated GOx nano particle, and then loading the composite nano particle Mo-PDA and the glucose oxidase (GOx) to prepare the Mo-PDA-coated GOx nano particle. Adding deionized water, then adding dopamine, continuously stirring for 1 hour, and continuously reacting for 14-18 hours at 150-170 DEG C; according to the invention, PDA is combined with Mo-containing polyacid salt and externally loaded with glucose oxidase (GOx), so that the nano-particle Mo-PDA-GOx for treating tumors through synergism of photothermal treatment, chemical kinetics and hunger is synthesized, and the nano-particle Mo-PDA-GOx has excellent photothermal conversion efficiency which reaches 67.2%. The nanoparticles effectively exert photo-thermal-chemical kinetics-hunger synergistic treatment, and the treatment effect on tumors is improved.
Resumen de: CN120965588A
本发明公开了基于不对称尾链的可离子化脂质及其组合物在核酸递送中的应用。本发明公开的可离子化脂质，其结构以天然氨基酸(谷氨酸或天冬氨酸)为骨架，以叔胺基团作为亲水头基，以具有不对称性的烷烃作为疏水尾链，并以生物可降解的酰胺键或酯键作为连接键，其合成简单，易于大规模生产。基于本发明的可离子化脂质构建的脂质纳米粒能稳定荷载包括siRNA、mRNA与pDNA在内的多种核酸药物，在体外对多种细胞系具有优异的转染效率，并在体内可以特异性的靶向脾脏免疫细胞，在一定程度上解决现有LNP递送系统内体逃逸效率低下与肝外靶向性不足的问题。
Resumen de: CN120960248A
本发明提供了一种负载miR‑146a和miR‑124的铁皮石斛外泌体组合物及其制备方法和应用。铁皮石斛外泌体具有抗氧化功能，miR‑146a和miR‑124具有协同抗炎功能，铁皮石斛外泌体为miR‑146a和miR‑124提供了递送载体并且提高了miR‑146a和miR‑124的稳定性和抗炎功能，最终开发出一种治疗神经炎及其相关疾病的药物组合物EXE‑miR‑146a‑124。本发明不仅提供了一种安全高效的miRNA递送系统，而且赋予其神经保护和抗炎效果，为神经系统炎性疾病的治疗提供了一种全新的、天然来源的干预方案。
Resumen de: CN120960449A
本发明公开了一种肝实质细胞靶向基因递送系统的制备方法及其应用，涉及肝实质细胞靶向脂质材料GalNAc‑PEG2000‑DSPE修饰的负载MKK4‑siRNA（siMKK4）的脂质纳米粒基因递送系统GalNAc‑LNP‑siMKK4的构建及其制备，可用于肝衰竭的主动靶向基因治疗。该肝实质细胞靶向基因递送系统选用siMKK4作为促肝再生基因治疗药物，具有靶点明确，能精准沉默靶基因的优势，脂质纳米粒作为基因递送载体可保护siMKK4抵抗细胞外核酸酶的降解，选用N‑乙酰半乳糖胺（GalNAc）作为靶头可将治疗基因精准靶向递送至肝实质细胞，在促进肝再生的同时可减少机体其他部位成瘤的风险，为肝衰竭患者的肝再生治疗提供了新的思路和方法。
Resumen de: CN120960250A
本发明提供miR‑494‑3p在制备治疗修复跟腱损伤药物中的应用，涉及生物医学技术领域，其技术要点在于：本申请提供了miR‑494‑3p在制备治疗修复跟腱损伤药物中的应用，并提供了将miR‑494‑3p制备成载miR‑494‑3p纳米球水凝胶缓释系统在制备修复跟腱损伤药物中的应用。本申请中通过具体实施例验证了miRNA纳米球水凝胶递送系统能够有效释放miR‑494‑3p，并满足肌腱损伤修复的需要。研究中发现miR‑494‑3p通过促进肌腱细胞增殖、抑制炎症和凋亡，同时增强细胞外基质(尤其是I型胶原)合成，进而改善肌腱愈合，提高肌腱的最大张力强度。
Resumen de: CN120960413A
本发明公开了一种基于不同形态纳米铝佐剂的核酸疫苗及其应用，从内到外依次由内核、中间层和外壳组成，所述内核为阳离子聚合物和核酸的复合物，所述中间层为阴离子聚合物，所述外壳为连续或不连续的纳米铝包覆层。本发明提供的纳米铝包覆的核酸递送系统用于制备疫苗能有效激活抗原呈递细胞，促进抗原递呈，从而同时激活机体的体液免疫和细胞免疫过程，增强肿瘤免疫治疗效果，具有较高的临床使用价值。
Resumen de: CN120960172A
本申请提供一种脑靶向递送系统，包括：作为药物载体的介孔普鲁士蓝纳米酶、包裹于所述药物载体表面的红细胞膜；所述红细胞膜经过狂犬病毒糖蛋白多肽修饰。本申请提供一种还提供一种药物，还包括负载于所述药物载体上的活性物质，所述活性物质包括L‑精氨酸和丁苯酞中的至少一种。该脑靶向递送系统用于协同治疗缺血性脑卒中时，免疫逃逸能力显著增强、脑靶向效率较未修饰纳米粒提高10倍以上、脑内滞留时间≥24h。
Resumen de: CN120960136A
本发明提供了一种利丙双卡因纳米乳膏及其制备方法，属于药物制剂技术领域。本发明提供的利丙双卡因纳米乳膏中，包载利丙双卡因纳米粒分散在乳膏基质中，包载利丙双卡因纳米粒能够控制利多卡因和丙胺卡因具有稳定的比例，并且能够使利丙双卡因纳米乳膏具有良好的缓释效果；本发明利用乳膏基质的作用提高利多卡因和丙胺卡因的透皮渗透性。实施例结果显示，本发明提供的利丙双卡因纳米乳膏具有良好的稳定性、渗透性和缓释效果。
Resumen de: WO2024194454A1
The present disclosure relates generally to the field of stabilized compositions comprising particles dispersed in an aqueous phase, wherein the aqueous phase comprises a buffer system and has a pH of about 4.0-5.5 and wherein the particles contain (i) nucleic acid (such as DNA or RNA, in particular mRNA or inhibitory RNA, e.g., siRNA); and (ii) a cationic or cationically ionizable lipid, methods for preparing and storing such compositions, and the use of such compositions in therapy.
Resumen de: CN120960443A
本发明涉及一种脂质组合物，其中包含聚合物脂质，所述聚合物脂质为式(I)所示结构的化合物，或其药学上可接受的盐、同位素变体、互变异构体或立体异构体。本发明还提供了包含所述脂质组合物的纳米颗粒组合物和药物组合物，以及所述脂质组合物在递送荷载中的应用。本发明脂质组合物中的聚合物脂质化合物可替代PEG脂质，具有生物相容性、易于在体内分解、无毒的优点，从而得到了一种新的脂质递送系统。
Resumen de: US2025230471A1
The present disclosure provides compositions and methods for inserting heterologous payload sequences into a target-site in a host cell genome. The compositions and methods use non-LTR retrotransposon reverse transcriptase proteins that bind template RNAs comprising a payload sequence that encodes a protein or regulatory RNA. The template RNA can comprise modified uridines that are not cleavable by a ribozyme. The incorporation of modified uridines increases the efficiency of integration and expression of the payload sequence and decreases cellular toxicity.
Resumen de: CN120960170A
本发明公开了一种双金属基纳米复合材料及其制备方法和应用，制备方法包括：将硝酸铁和硝酸铋分散于去离子中，搅拌至溶解，调节溶液pH至碱性，超声分散均匀后倒入聚四氟乙烯釜中进行水热反应，获得双金属基纳米材料FBO，分散到Tris缓冲液中，并加入盐酸多巴胺，避光条件下搅拌，离心、洗涤、干燥，获得黑色的纳米复合材料FBO@PDA，即得。本发明的双金属基纳米复合材料具有抗炎、促成骨和促血管化性能，制备方法简单，安全环保，成本低，易于操作，可作为骨修复材料用于颌骨坏死的再生修复领域，具有广阔的应用前景。
Resumen de: CN120965668A
本发明提供了一种具有光热产生能力的近红外二区聚集诱导发光型光敏剂及其制备方法和应用，属于生物化学材料领域。本发明提供的有机荧光化合物的分子结构中存在电子给体‑电子受体相互作用，其中受体部分中R基团的引入，有利于促进D‑A相互作用，促使吸收波长发生红移，增加对生物组织的穿透深度，提供具有更高分辨率的荧光成像结果；此外，本发明提供的有机荧光化合物给体部分为噻吩基乙烯偶联的久洛尼定以及共轭二烯偶联的4,4'‑二(N,N‑二甲氨基)二苯甲酮，其中4,4'‑二(N,N‑二甲氨基)二苯甲酮具有高度扭曲的空间结构，更有利于非辐射跃迁过程的发生，从而促进了光热性能的提升。
Resumen de: CN120965753A
本发明公开了一种可离子化脂质以及由其制备的脂质载体及用途。具体地，本发明公开了一种如式(I)所示的可离子化脂质化合物、其立体异构体、互变异构体或药学上可接受的盐。本发明的化合物是一类兼具磷酸基团与阳离子头基的新型脂质分子，具有生产工艺简单、转染效率高、细胞毒性低和适用范围广等优点。
Resumen de: CN120965996A
本发明涉及一种聚合物脂质化合物，具体涉及一类式(I)的化合物，或其药学上可接受的盐、同位素变体、互变异构体或立体异构体。本发明还提供了包含所述化合物的纳米颗粒药物组合物，以及所述化合物及其组合物在递送荷载中的应用。本发明的聚合物脂质化合物可替代PEG脂质，具有生物相容性、易于在体内分解、无毒的优点，从而得到了一种新的脂质递送系统。
Nº publicación: CN120960171A 18/11/2025
Solicitante:
青岛瑞斯凯尔生物科技股份有限公司
Resumen de: CN120960171A
本发明提供一种外囊泡药物及生物标志物装载系统制备方法，属于外囊泡药物及生物标志物装载系统技术领域，本发明首先通过差速离心和超速离心分离纯化外囊泡，采用聚乙二醇修饰提高稳定性，经动态光散射法筛选合适粒径的外囊泡，然后通过配体修饰赋予靶向能力，采用电穿孔法进行药物及生物标志物装载，装载过程中基于系统平衡方程组和多维指标向量实现定量优化，最后通过冷冻干燥制备成品，并通过结晶度控制确保制剂性能。建立了完整的数学描述体系和多维参数优化方法。通过系统平衡方程组的求解，实现了对装载过程的精确控制；通过多维指标向量的构建，实现了装载参数的定量优化，解决了现有技术中存在的外囊泡药物及生物标志物装载系统缺乏难以实现定量优化的技术问题。
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