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228
resultados
Última actualización
16/09/2025 [06:51:00]
Resumen de: WO2025189064A1
Provided are lipid nanoparticles, compositions, and methods of making and using the same. The lipid nanoparticles contain ionizable lipids, structural lipids, PEG lipids and specific amounts of helper lipids. The lipid nanoparticles may further contain a cell targeting group coupled to a PEG lipid. The lipid nanoparticles may carry a cargo, e g., a mRNA. The lipid nanoparticles may be used for transfection of cells, e.g., immune cells or hematopoietic stem cells.
Resumen de: CN120617307A
本发明属于医药制剂技术领域，具体涉及一种WZB117纳米制剂及其制备方法。在葡萄膜黑色素瘤的治疗中，缺少有效的治疗方案。本发明提供了一种新型眼用制剂及其制备方法，利用血红蛋白和多巴胺同时搭载葡萄糖转运蛋白1(Glut1)抑制剂WZB117和铜离子，该纳米制剂可通过铜死亡诱导的活性氧爆发与WZB117双硫死亡导致的抗氧化系统崩溃形成正反馈循环，加速肿瘤细胞死亡。
Resumen de: WO2025188636A1
Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases).
Resumen de: WO2025188185A1
The present invention provides a delivery system comprising of a first aqueous composition harbouring small particles and a second aqueous composition harbouring larger particles in which the particles of the first and second aqueous compositions may comprise the same or a different compound of interest, wherein said compound of interest preferably is a hydrophobic or amphiphilic compound. In both cases the small particles comprise a partly liquid oil phase at temperatures around about 4°C.
Resumen de: CN120617206A
本方案提供了一种负载麦角甾醇的工程化外泌体双载药系统及其制备方法与应用，属于纳米材料和纳米生物医药领域，基于创新的ROS响应型纳米材料CHPG设计，负载治疗活性成分麦角甾醇发挥治疗修复作用，其通过靶向功能修饰实现血脑屏障穿透和病灶区域精准释药；将其包裹于鼻粘膜来源的干细胞外泌体，形成载药递送体系Exo@Erg‑CHPG‑M，融合外泌体的天然生物学特性与纳米胶束的精准靶向优势，显著提升药物递送效率，体外及动物模型验证表明，该体系可通过协同调控炎症及神经修复通路，改善缺血性卒中后的神经功能缺损及脑组织损伤，本方案为脑卒中等中枢神经系统疾病的精准治疗提供了新的技术方案。
Resumen de: CN120617500A
本发明提出一种NK细胞制剂的制备方法及其应用，制备方法的步骤包括：采用密度离心从外周血中分离得到外周血单个核细胞，在外周血单个核细胞中加入白细胞介素‑2和白细胞介素‑15，辅以超顺磁性聚苯乙烯微珠进行免疫模拟刺激，在七天内培养活化，得到纯度≥90%的NK细胞；分别配制壳聚糖溶液和透明质酸钠溶液，先将NK细胞悬浮于壳聚糖溶液中，升温至37℃孵育30分钟，再置于透明质酸溶液中孵育15分钟，得到双层包覆NK细胞；将双层包覆NK细胞转入低氧培养箱，在含有5mM乳酸钠的培养基中孵育12小时，再复悬于2%人血清白蛋白的生理盐水，得到NK细胞制剂。从而提高制剂内细胞的存活率。
Resumen de: MY202837A
Compounds are provided having the following structure: Formula (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R, R, R, R4, R5, L, L, L, G, G, and G are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Resumen de: MX2025007398A
The present invention provides, in part, bis-ester and amide cationic lipid compounds of Formula (I):, or a pharmaceutically acceptable salt thereof, bis-ester and amide cationic lipid compounds of Formula (II):, or a pharmaceutically acceptable salt thereof, bis-ester and amide cationic lipid compounds of Formula (III):, or a pharmaceutically acceptable salt thereof, and bis-ester and amide cationic lipid compounds of Formula (IV):, or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
Resumen de: CN120617562A
本发明属于生物医药技术领域，具体涉及一种基于氮氧化物的磁共振/荧光双模态纳米诊疗剂的制备方法。本发明先利用TEMPO分别修饰二硬脂酰磷脂酰乙醇胺磷脂和IR780制备DSPE‑PEG‑TEMPO和ROS响应性荧光探针IT，再利用细胞膜流动性将磷脂嵌入巨噬细胞膜，并通过超声、挤压方式包裹荧光探针IT和姜黄素，进而制得基于MRI/FL双模成像的仿生纳米诊疗剂。其中的TEMPO既具有MRI成像性能，还能清除活性氧；ROS响应型荧光探针可通过荧光成像追踪炎症性肠病病灶并用于手术导航；抗炎药姜黄素能在病灶部位缓慢释放。三者协同作用，加上巨噬细胞膜的炎症趋势性，有望实现炎症性肠病的精准成像和药物释放治疗。
Resumen de: CN120617313A
本发明公开了一种用于治疗弱精症的药物，以脐带间充质干细胞UCMSC构建细胞外囊泡，并封装BITT或ATP；将UCMSC‑EVs与BITT或溶解于磷酸盐缓冲溶液PBS中的ATP混合，并在黑暗中于37℃孵育1小时，通过超速离心浓缩得到所述的用于治疗弱精症的药物。本发明采用了多功能脐带间充质干细胞外泌体，其中包裹了三磷酸腺苷，以协同恢复细胞能量并抑制铁死亡，通过结合成像精度与双重治疗模式，这种方法有望克服BTB的局限性，解决男性不育问题。
Resumen de: AU2023406303A1
Provided herein are lipid nanoparticle (LNP) compositions (e.g., pharmaceutical compositions) comprising a therapeutic nucleic acid (TNA), wherein the LNP comprises an ionizable lipid; a "helper" lipid, e.g., a ceramide or distearoylphosphatidylcholine (DSPC); a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, as well as uses thereof.
Resumen de: AU2023406947A1
The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.
Resumen de: CN120618339A
本发明属于纳米材料制备技术领域，具体涉及一种纳米级超细粉体的防团聚制备装置及方法。纳米级羟基磷灰石简称nHA，是一种纳米级的超细粉，在常温常压的空气暴露环境，容易发生团聚现象，团聚成更大颗粒度的物质。本发明使用改性酪蛋白作为填充物，使nHA颗粒复合分散在填充物中，有效防止纳米团聚效应的发生。成本相对较低，适合大规模工业化应用。
Resumen de: CN120617204A
本发明公开了一种联合维奈克拉协同增强自然杀伤细胞免疫治疗的纳米接合器及在应用，属于生物医药与纳米技术交叉领域。本发明通过赋形剂分子与维奈克拉共沉淀制备纳米颗粒，改善药物疏水性，通过免疫激活‑靶点阻断的双功能抗体修饰，实现化疗药物与免疫细胞治疗的时空协同。本发明对于抗血液恶性肿瘤药物的研发、药效研究及白血病细胞耐药性研究等临床及科研领域具有极大的应用潜力。
Resumen de: CN120617201A
本发明公开了包裹卵黄抗体的植物外泌体样囊泡的制备方法，包括如下步骤：将稳定修饰后的植物源外泌体样囊泡复溶，得到复溶后的植物源外泌体样囊泡；将复溶后的植物源外泌体样囊泡进行冷冻；重复S1和S2操作多次，将植物源外泌体样囊泡再次复溶；取卵黄抗体，用PBS缓冲液超声溶解后备用，得到卵黄抗体溶液；将卵黄抗体溶液与再次复溶的植物源外泌体样囊泡混合后，进行孵育，得到包裹卵黄抗体的植物外泌体样囊泡；将包裹卵黄抗体的植物外泌体样囊泡复溶后，进行真空冻干，保存备用，制得包裹卵黄抗体的植物外泌体样囊泡。本发明用以解决幽门螺杆菌感染较多的技术问题。
Resumen de: WO2025188836A1
The present disclosure describes methods of using lipid nanoparticle (LNP) compositions for treating ocular- related disorders or diseases. In particular, methods are provided for using LNP compositions in gene therapy for targeting an ATP binding cassette subfamily A member 4 (ABCA4) or a functional fragment thereof, in a subject, thereby treating or mitigating Inherited Macular Degenerations including a Stargardt disease or other diseases that involve retinal degeneration.
Resumen de: WO2025189161A1
The disclosure provides Fab fragments that are site-selectively conjugated to the surface of a lipid nanoparticle (LNP) through the natural interchain disulfide bond between the heavy chain and the light chain (i.e., the CL-CH1 disulfide bond) to make targeted LNPs (also referred to herein as conjugates). The targeted LNPs (conjugates) produced by the methods disclosed herein can transduce specific targeted cells. Hence, the targeted LNPs are capable of delivering therapeutic payloads to cells to treat disease in a subject.
Resumen de: WO2025188498A1
Disclosed herein are cytokine constructs that include a delivery vehicle, and at least one cytokine or one decoy receptor. By adjusting the types and density of cytokines on the constructs, they can be tailored for both local and systemic treatment of patients with cancers and other immune-related diseases, such as autoimmune diseases. Additionally, the cytokine constructs can be used ex vivo to expand and activate cells from patients or healthy subjects before re-infusing the activated cells back into patients, which benefits adoptive and stem cell therapies. These constructs display improved half-life and stability of cytokines, enabling reducing doses, and minimizing toxicity from unintended effects of free cytokines. Furthermore, the constructs allow for co-delivery of other therapeutics, such as antibodies, small molecule inhibitors, chemotherapeutics, oligonucleotides, adjuvants, and antigens.
Resumen de: WO2025188061A1
The present invention relates to a method for manufacturing porous silicon nanoparticles by means of ultrasonic treatment. A novel ultrasonic treatment apparatus using a duty cycle scheme was fabricated, and physical properties such as optimized conditions of the apparatus, yield, size, size distribution, surface state, shape, pore size, and drug delivery capacity of the porous silicon nanoparticles manufactured by the duty cycle scheme were analyzed. The duty cycle scheme was confirmed to exhibit a significantly higher yield of porous silicon nanoparticles compared to conventional schemes, and the porous silicon nanoparticles manufactured by the duty cycle scheme exhibited similar physical properties to those of porous silicon nanoparticles manufactured by the conventional schemes. Therefore, it was confirmed that the method for manufacturing porous silicon nanoparticles by means of ultrasonic treatment using the duty cycle scheme of the present invention is suitable for mass production.
Resumen de: WO2025187760A1
The purpose of the present invention is to provide a lipid nanoparticle that makes it possible to efficiently introduce a drug into a cell, and a constituent lipid of this lipid nanoparticle. Provided are: a compound represented by general formula (I), (II), or (III); a salt or stereoisomer thereof; and a lipid nanoparticle containing the same as a constituent lipid.
Resumen de: WO2025184767A1
A composition that includes a stem cell and a coated iron oxide nanoparticle. The coated iron oxide nanoparticle, being present in the cytoplasm of the stem cell, contains a superparamagnetic iron oxide core that is coated with one or more biocompatible polymers, each of which has a polyethylene glycol group, a silane group, and a linker covalently linking the polyethylene glycol group and the silane group. Also provided is a method for treating an inflammatory disorder in which stem cells are cultured in the presence of coated iron oxide nanoparticles and the cultured stem cells are administered to a subject suffering from an inflammatory disorder. Further disclosed is a method for tracking stem cells in vivo by labeling stem cells with coated iron oxide nanoparticles, administering the labeled stem cell to an individual, and obtaining one or more T2 weighted magnetic resonance images of the individual to track the stem cells.
Resumen de: WO2025184750A1
A lipid nanoparticle composition for use in the delivery of a self-amplifying RNA (saRNA) construct in one or more target cells is disclosed. The lipid nanoparticle composition comprises a lipid mixture comprising at least one (ionizable) cationic lipid, at least one helper lipid, a sterol, a corticosteroid such as Dexamethasone, and a least one lipid-polyethylene glycol conjugate. The saRNA construct comprises a first open reading frame which encodes one or more non-structural proteins, and a second open reading frame operatively linked to the first open reading frame. The second open reading frame comprises a coding region which encodes one or more target proteins. In some embodiments, the target proteins comprise one or more therapeutic proteins. In some embodiments, the target proteins comprise one or more one or more Cas proteins and/or variants thereof for use in CRISPR-based gene editing.
Resumen de: WO2025184694A1
The present disclosure relates generally to nanoparticles and compositions comprising the same, and their use for transfecting transfection-recalcitrant cells, wherein the nanoparticle is tethered to an antigen-binding moiety having binding specificity for an antigen expressed on the surface of the cell, such as cluster of differentiation 2 (CD2) or cluster of differentiation 7 (CD7), wherein the nanoparticle comprises at least an ionisable lipid and a sterol.
Resumen de: CN120617207A
本发明公开了一种仿生纳米囊泡制备方法，包括以下步骤：将表没食子茶素没食子酸酯和硒代蛋氨酸溶于去离子水中，加入甲醛溶液在黑暗环境中搅拌，离心纯化，得球形纳米颗粒；从干细胞中提取外泌体；将球形纳米颗粒与外泌体加入去离子水中搅拌得仿生纳米囊泡。本发明制备的仿生纳米囊泡可对巨噬细胞和T细胞的进行调控，具有抗炎和调控免疫网络作用，应用于对急性肾损伤的保护作用。
Nº publicación: WO2025185570A1 12/09/2025
Solicitante:
THE INST OF MEDICINAL PLANT DEVELOPMENT THE CHINESE ACADEMY OF MEDICAL SCIENCES [CN]
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Resumen de: WO2025185570A1
A polysaccharide composition, a nano polysaccharide, a preparation method therefor, and use thereof. The polysaccharide composition comprises a polysaccharide and a lipid material. The weight ratio of the polysaccharide to the lipid material is 2-50:1.
BOPI
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