Resumen de: CN121422199A
本发明提供了一种协同敲低PCSK9基因表达的mRNA疫苗,属于生物医药领域。所述mRNA疫苗包含靶向沉默PCSK9基因的siRNA、编码卵清蛋白(OVA)的mRNA和含氟化修饰的聚乙二醇化脂质的脂质纳米颗粒。所述siRNA通过下调PCSK9基因的表达量来提高MHCⅠ复合体的水平;所述mRNA则提高细胞内抗原的表达水平;所述脂质纳米颗粒则提高了mRNA的递送效率,为所述疫苗发挥疗效奠定了基础。本发明证明了所述mRNA疫苗能显著提高肿瘤细胞的抗原呈递效率,强烈诱导机体产生免疫反应,显著抑制肿瘤生长,延长荷瘤小鼠的寿命;且证实了所述siRNA和mRNA组合具有协同增效的作用。
Resumen de: WO2024163457A2
Artificial expression constructs including a cytokine transgene under the control of an inducible promoter are described. The artificial expression constructs can be used to enhance the function of a recombinant receptor-immune cell (e.g., CAR-T cell). The cytokine transgenes disclosed herein enhances the potency of the recombinant receptor-immune cell (e.g., CAR-T cell), resulting in potentiated killing, immune cell proliferation, and/or cytokine outputs.
Resumen de: NZ758037A
A compound of formula I or a pharmaceutically acceptable salt or ester thereof is provided for the treatment of cancer wherein (i) the cancer is one that has the characteristic of being a type prone to being or becoming refractory or resistant to platinum drug based therapy and (ii) the treatment is with a dose of between 1mg/m2and 30mg/m2of compound per patient body surface area per administration. Method of treatment and novel dosage forms are also provided. Particularly treated are ovarian cancers, particularly those expressing a-folate receptors, including epithelial ovarian, fallopian tube or peritoneal cancer.
Resumen de: NZ749229A
Oxidative stress is linked to several health conditions including, without limitation, cancer, aging, neurodegenerative and metabolic diseases. Increased levels of oxidative stress may result from increased exposure to radiation or other environmental stressors and/or diminished protective effects of cellular defenses against reactive oxygen species. The present disclosure provides compositions and methods comprising encapsulated cannabinoid compounds useful for reducing oxidative stress in a subject. In particular, the invention provides a use in reducing oxidative stress using an antioxidant and a terpene with a cannabinoid microencapsulated, wherein the microcapsules are substantially homogenous in size and the composition has improved stability, bioavailability and bioactivity.
Resumen de: MX2025013218A
Compounds are provided having the following Structure (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R<sup>1</sup>, R<sup>1</sup>, L<sup>1</sup>, L<sup>2</sup>, L<sup>2a</sup>, L<sup>2b</sup>, and A 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: AU2024303397A1
Provided is a rapidly-metabolized lipid compound. The present invention relates in particular to a compound represented by formula (I), or a pharmaceutically acceptable salt, an isotopic variant, a tautomer or a stereoisomer thereof. Also provided are a nanoparticle pharmaceutical composition comprising the compound, and a use of the compound and a composition thereof in delivering nucleic acids.
Resumen de: CN121426779A
本发明公开了一种香豆素衍生物、超声响应性药物及其制备方法和应用,涉及生物医用材料领域。超声响应性药物组成包括载体聚谷氨酸、香豆素衍生物、含羟基或氨基的拓扑异构酶抑制剂和端氨基聚乙二醇单甲醚,所述药物经酰胺缩合或酯化反应制备得到。所述香豆素衍生的氨基甲酸酯可在超声下触发化学键断裂,具有超声响应特性。本发明将具有超声响应性的香豆素衍生物引入制备治疗肿瘤的高分子纳米药物中,在超声条件下,直接实现纳米颗粒表面电荷的反转,这种超声响应机制不受肿瘤微环境的动态变化的影响,同时可以提高药物在组织中的穿透深度。
Resumen de: CN121421988A
本发明属于生物医学工程领域,具体涉及一种干细胞膜仿生纳米系统的制备方法及其应用。本发明提供了一种干细胞膜仿生纳米系统的制备方法及其应用,干细胞膜的包裹赋予了系统优异的血脑屏障穿透能力和肿瘤主动归巢靶向性;使用三苯基膦修饰的碳点在干细胞膜仿生纳米系统中能实现精准的线粒体靶向,增强凋亡诱导;干细胞膜仿生纳米系统能够实现整合光热治疗、光动力治疗、纳米酶催化治疗及线粒体靶向治疗,多维度杀伤肿瘤细胞并破坏肿瘤微环境稳态。本发明在制备肿瘤预防或治疗药物上有很好的应用前景。
Resumen de: CN121426768A
本发明是关于一种化合物、LNP组合物、药用组合物及其应用,涉及医药生物技术领域,解决的问题是目前亟需开发一种新型的阳离子脂质。主要采用的技术方案为:所述化合物为化合物1或化合物2;其中,化合物1和化合物2的化学结构式如下: 化合物1; 化合物2;本发明涉及一种化合物,其可以作为阳离子脂质,由其制备的LNP组合物及药用组合物,可以用于体内核酸递送,具有良好的脾脏靶向性,还可作为传染病疫苗和肿瘤疫苗的递送系统。
Resumen de: CN121421948A
本发明提供了病毒载体高分子基质水凝胶冻干制剂及其制备方法和应用,涉及生物医药技术领域,本发明实施例提供的病毒载体高分子基质水凝胶冻干制剂包括:高分子基质水凝胶,所述病毒载体加载于交联后的高分子基质水凝胶的三维网络中。病毒载体高分子基质水凝胶冻干制剂,使病毒载体能够在4℃下稳定储存至少12周。通过利用水凝胶的局部保留和持续释放特性,病毒载体高分子基质水凝胶可以在病变区域实现高药物浓度,同时有效避免了全身毒性和抗病毒载体中和抗体反应。在小鼠黑色素瘤和癌症术后切除模型中,联合治疗发挥了强大的抗肿瘤作用,并显著抑制了肿瘤复发。
Resumen de: CN121422201A
本发明涉及mRNA疫苗研发技术领域,且公开了一种基于脂质纳米颗粒递送的Tp0136T细胞表位mRNA疫苗及其在梅毒预防中的应用,该疫苗含mRNA序列与LNP递送系统:mRNA序列含Tp0136T1T细胞表位编码区(氨基酸L477-S486,经密码子优化)、Cap 1结构、优化UTR及65-76个腺苷的poly(A)尾,且用ψ或m5C替代天然碱基;LNP由SM‑102、DSPC、胆固醇、DMG‑PEG按50:10:38.5:1.5摩尔比组成。疫苗粒径≤150nm、PDI<0.2、Zeta电位‑5~‑15mV、包封率≥93.47%。其制备含mRNA设计合成、mRNA‑LNP制备表征、体外表达验证步骤,通过肌肉注射接种,能诱导强效Th1型与CD8+CTL免疫应答,降低梅毒螺旋体负荷与皮肤溃疡率,可用于梅毒预防。
Resumen de: CN121421989A
本发明属于药物递送技术领域,涉及一种基于生物正交反应线粒体靶向递送药物的仿生纳米制剂及其制备方法和应用。所述仿生纳米制剂包含载药粒子以及包覆所述载药粒子的线粒体靶向仿生细胞膜;所述载药粒子包含药物以及负载所述药物的金属有机骨架材料;所述线粒体靶向仿生细胞膜的外表面标记有线粒体靶向基团。本发明将线粒体靶向基团标记至仿生细胞膜表面,利用仿生细胞膜和线粒体靶向基团(例如TPP)的联合靶向性,能够将仿生纳米制剂级联递送至病灶(如肿瘤)部位和病灶部位的细胞线粒体,不仅能够实现药物精准递送至病灶部位,同时也延长了仿生纳米制剂在体滞留时间,改善了药物的生物分布,提高了用药安全性。
Resumen de: AU2023457271A1
Provided is a lipid formulation which includes cholesteryl hemisuccinate as a cholesterol substitute and which is mixed with nucleic acid to form lipid nucleic acid nanoparticles which are capable of transfecting cells of the immune system.
Resumen de: AU2023457271A1
Provided is a lipid formulation which includes cholesteryl hemisuccinate as a cholesterol substitute and which is mixed with nucleic acid to form lipid nucleic acid nanoparticles which are capable of transfecting cells of the immune system.
Resumen de: CN121421179A
本发明属于生物材料技术领域,提供了一种pH响应型载营养因子脂质体的多糖修饰金属有机框架载体的制备方法,包括以下步骤:通过薄膜水合法制备载有营养因子的脂质体载体并与金属有机框架相结合得到载有营养因子脂质体的金属有机框架;将载有营养因子脂质体的金属有机框架分散到多糖的复合溶液中,缓慢搅拌,离心、洗涤、干燥后得到pH响应型载营养因子脂质体的多糖修饰金属有机框架载体,既可以避免胆固醇摄入过多对人体产生不良影响,同时在脂质体、金属有机框架和多糖的协同增效下提高营养因子在胃肠道环境中的稳定性,提高其生物可及性。
Resumen de: FR3164913A1
------ NANOPARTICULES DE FLUORURE DE GADOLINIUM ET DE TERBIUM, MODIFIEES EN SURFACE, LEUR PREPARATION ET LEUR UTILISATION EN IMAGERIE ET TRAITEMENT D’UNE TUMEUR Nanoparticules de GdaTbbF3, où a = 0,50 à 0,95, b = 0,05 à 0,50 et a+b = 1, modifiées en surface par au moins un ligand de biocompatibilité et par au moins un photosensibilisateur absorbant dans la même bande de longueur d’onde que la bande de longueur d’onde d’émission du terbium à savoir entre 520 et 560 nm.
Resumen de: WO2024244304A1
The present disclosure relates to a nucleic acid-lipid nanoparticle composition and a lyophilized preparation thereof, and further relates to a preparation method for the composition and a use of the composition. The nucleic acid-lipid nanoparticle composition of the present disclosure comprises lactate, so that the stability of nucleic acid-lipid nanoparticles is improved in a lyophilization process and a prepared lyophilized preparation. According to the present disclosure, the total time of a freeze-drying process for the nucleic acid-lipid nanoparticles can be shortened, and energy consumption and the time cost of product scale-up production can be greatly reduced; the rehydration of lyophilized nucleic acid-lipid nanoparticles is quick, and the total amount of nucleic acids, the encapsulation efficiency, and the nucleic acid integrity are high; in addition, a lyophilized and rehydrated preparation has cell transfection efficiency not significantly different from that of a non-lyophilized nucleic acid-lipid nanoparticle stock solution, and has a high in vivo immune response, even exceeding that of the non-lyophilized nucleic acid-lipid nanoparticle stock solution.
Resumen de: WO2026022743A1
The subject of the present invention is N,N'-bis(acryloyl)selenocystine represented by formula 1, a method of its manufacturing, and its use as a crosslinking agent in polymerisation reactions, preferably in nanogel polymerisation reactions. Another subject of the invention is a method of manufacturing a p(NIPA-BISeSe) nanogel, and the use of the p(NIPA-BISeSe) nanogel obtained by this method as a carrier of drugs, preferably anticancer drugs.
Resumen de: WO2026022261A1
The present invention relates to a lipid system comprising: a) at least one oil, b) at least one ionizable lipid, c) at least one sphingolipid, and d) at least one lipid-polymer, with the proviso is that the lipid system does not comprise cholesterol. The present invention also uses of the lipid system in therapy.
Resumen de: WO2026022713A1
The invention relates to nanoparticles of GdaTbbF3, where a = 0.50 to 0.95, b = 0.05 to 0.50 and a + b = 1, which are surface-modified by at least one biocompatibility ligand and by at least one photosensitizer that absorbs in the same wavelength band as the emission wavelength band of terbium, namely between 520 and 560 nm.
Resumen de: WO2026022656A2
The present disclosure provides lipid nanoparticles that are beneficially effective in the targeting of hepatic stellate cells with enhanced specificity. The lipid nanoparticles include five lipid components, including a permanently charged cationic lipid that is a quaternary ammonium lipid. The disclosure also provides pharmaceutical compositions and methods including the provided lipid nanoparticles.
Resumen de: WO2026021698A1
The present invention generally relates to nucleic acid particles, in particular nucleic acid-lipid particles, comprising targeting moieties that are bound to the particles, and to pharmaceutical compositions containing the nucleic acid particles and their uses in medicine.
Resumen de: US20260028323A1
The present application disclosed a compound of formula (I) and a salt and a stereoisomer thereof, wherein each variable is as defined in the specification. The present application also disclosed a nanoparticle composition comprising the compound or a salt or a stereoisomer thereof, and the use of the nanoparticle composition for delivering active agents.
Resumen de: US20260028349A1
Photodynamic therapy (PDT) is a minimally invasive treatment that involves the administration of a light-activatable drug followed by light activation of the lesion to produce reactive oxygen species that kill cancer cells. VISUDYNE®, a liposomal formulation of benzoporphyrin derivative (BPD) photosensitizer, is clinically approved for PDT of ocular diseases and is now being tested for PDT and imaging of pancreatic, brain, and other cancers. While VISUDYNE® improves the pharmacokinetics of BPD, it lacks treatment selectivity. This present disclosure is directed to dBPD, dBPD nanoparticles, and preparation and use thereof that provide cancer treatment selectivity for cancers characterized by overexpression of folate receptor (FR).
Nº publicación: US20260028406A1 29/01/2026
Solicitante:
MASSACHUSETTS INSTITUTE OF TECH [US]
CHILDRENS MEDICAL CENTER CORP [US]
WHITEHEAD INSTITUTE FOR BIOMEDICAL RES [US]
Massachusetts Institute of Technology,
Children's Medical Center Corporation,
Whitehead Institute for Biomedical Research
Resumen de: US20260028406A1
Methods for developing disease-related nanobodies and related products and kits are provided. The disease-specific proteins are extracellular matrix (ECM) proteins, domains or epitopes that are associated with various aspects of disease and are not present, or are present in very low quantities, in non-diseased individuals. Highly effective nanobodies capable of specifically binding to these ECM protein epitopes useful in in vivo imaging assays, the detection, diagnosis and treatment of diseases as well as monitoring therapeutic progress in a patient with a disease are provided herein.