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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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。
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: 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: 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: 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。
Nº publicación: CN120960449A 18/11/2025
Solicitante:
中国人民解放军空军军医大学
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。