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174
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Última actualización
02/11/2025 [06:51:00]
Resultados 100 a 125 de 174
Resumen de: CN120112642A
The invention discloses gRNA (guide ribonucleic acid) targeting VEGFA (vascular endothelial growth factor A), a gene editing system and application of the gRNA. The gRNA of the present disclosure comprises a guide sequence having at least 80% sequence identity as compared to any of SEQ ID NO: 10-18, 40-230. The gene editing system disclosed by the invention comprises the gRNA disclosed by the invention and a coding sequence thereof, and RNA-guided nuclease and a coding sequence thereof.
Resumen de: AU2023368775A1
Provided herein is a lipid nanoparticle comprising an encapsulated oligonucleotide molecule, wherein the oligonucleotide molecule is single-stranded or double-stranded and has a length of between 5 and 500 nucleotides; and 20 to 70 mol% of a neutral lipid content relative to total lipid present in the lipid nanoparticle, an ionizable lipid; a sterol; and optionally a hydrophilic polymer-lipid conjugate.
Resumen de: CN120091992A
The present invention relates to a cationic lipid and a method for preparing the same, and more specifically, to a cationic lipid which can easily form a complex with an anionic drug and thus can be used for drug delivery, and a method for preparing the same.
Resumen de: CN120827534A
本发明公开了一种靶向PD‑L1蛋白降解的纳米颗粒及其制备方和应用。所述纳米颗粒包括透明质酸和BMS‑8分子的缀合物。本发明设计透明质酸和BMS‑8分子的缀合，构建一种新型的基于溶酶体降解体系(LYTAC技术)的双靶向纳米颗粒，能够特异性地与肿瘤细胞表面的PD‑L1蛋白结合，当纳米颗粒与肿瘤细胞表面的PD‑L1蛋白结合后，通过内吞作用被细胞摄取，随后被转运至溶酶体，触发PD‑L1蛋白的降解过程，高效降解PD‑L1蛋白，为肿瘤治疗提供了一种新的、有前景的方法。
Resumen de: CN120827557A
本发明提供基于硫辛酸的纳米酶及其制备方法与应用，首先利用硫辛酸的两亲性使其溶于有机溶剂并在水中自组装形成纳米聚集体，再由紫外光照引发硫辛酸开环聚合即得到稳定的硫辛酸纳米粒子，即基于硫辛酸的纳米酶。本发明的有益效果是：利用本发明方法制备得到的基于硫辛酸的纳米酶制备方法简单，且同时具备稳定的粒径、良好的活性氧清除能力以及跨越血脑屏障的能力，此外纳米酶内部的空腔适于负载疏水性的生物活性因子及药物，纳米酶表面丰富的羧基可与多种离子进行络合，可应用于治疗缺氧或缺血等引起的炎症类疾病以及脑部药物递送。
Resumen de: WO2024144009A1
The present invention relates to: ionized lipids comprising lipids with a branched structure; a lipid nanoparticle formulation using same; and use thereof. The ionized lipids of the present invention are a biodegradable lipid material with a lipid structure in which a heteroamine structure is branched, and the lipid nanoparticles using the ionized lipids can deliver a nucleic acid drug and the like with high efficiency, and thus can be effectively used in related technical fields such as mRNA vaccines and therapeutic agents.
Resumen de: CN120827844A
本发明涉及生物技术和基因工程技术领域，其公开了用于脂质纳米颗粒合成的被动式微通道反应器及其应用，其包括反应器本体和反应器盖板，反应器本体内嵌设有凹陷呈Y字型的流道，包括供第一种液体进入的第一进液口、第一进液口通道、供第二种液体进入第二进液口、第二进液口通道、混流通道、在混流通道末端用于形成稳态出液流的出液口通道和出液口；混流通道内设有用于螺旋扰流的螺旋混流芯片；反应盖板上设有上设有三个接口，第一进液转接头与第一进液口对应连通；第二进液转接头与第二进液口对应连通；出液转接头与出液口对应连通。本发明的流道结构简单，利于大规模制造易清洁，且产物粒径分布均一性好，灵活地适配各种类型的动力系统。
Resumen de: CN120827533A
本发明公开了一种靶向β淀粉样蛋白降解的纳米颗粒及其制备方法和应用。所述纳米颗粒包括共价连接的透明质酸、聚乙二醇和GKLVFFK多肽。GKLVFFK多肽的结构相对稳定，有利于与Aβ形成稳定的复合物，同时具备低免疫原性和毒副作用；透明质酸同时作为纳米颗粒的骨架和靶向单元，通过靶向细胞表面的CD44受体，可以将目标蛋白介导进入细胞的溶酶体降解通道；基于透明质酸骨架上缀合的PEG的亲疏水作用自主形成纳米颗粒；各单元整体配合，实现抑制和减缓致病蛋白Aβ的积累，有望为AD患者带来实质性的治疗效果。
Resumen de: CN120829373A
本发明提供了一种用于肺部选择性药物递送的脂质化合物及其制备方法和应用。本发明的脂质化合物的结构通式如式Ⅰ所示。本发明的脂质化合物可用于制备脂质纳米颗粒或用于构建生物大分子递送体系。本发明构建的生物大分子递送体系可用于肺部选择性多种大分子如蛋白质递送的通用平台，且构建的生物大分子递送体系的工艺简单，原料廉价易得，通过投射电镜观察可见所形成的脂质纳米颗粒的粒径均一。
Resumen de: CN120829386A
本发明涉及局部递送药物的材料及其应用。具体提供包含脂质组分的纳米颗粒组合物，所述脂质组分包含式(I)化合物。本发明纳米颗粒组合物仅在施用部位递送。
Resumen de: US2025325695A1
The present invention provides a method of a prophylaxis or a treatment of a pathological change of Bruch's membrane and/or an adjacent tissue, including a retinal pigment epithelium, a choroid, and an optic nerve head of an eye, e.g. a calcification of Bruch's membrane and/or the adjacent tissue, using a nanoparticle comprising a scaffold comprising a biodegradable material, an antibody targeted to a component of a Bruch's membrane, a component of a sub-retinal pigment epithelial deposit, or a component of an optic nerve head, and an anti-calcifying agent. Additionally, the present invention provides a pharmaceutical composition comprising said nanoparticle and one or more pharmaceutical acceptable excipient(s). Said pharmaceutical composition could be used in a method of prophylaxis or treatment of a pathological change of Bruch's membrane and/or adjacent tissues, including a retinal pigment epithelium and a choroid of an eye and/or a calcified sub-retinal pigment epithelium deposit and/or a calcified drusen.
Resumen de: US2025325699A1
The invention features polypeptides containing nuclear localization sequences that can be used to deliver polynucleotides to a cell. The polypeptides can be formulated with a polynucleotide and a lipid.
Resumen de: US2025325655A1
A vaccine adjuvant, and a preparation method therefor and a use thereof. The vaccine adjuvant is a MA105 immunologic adjuvant, and comprises (1) QS-21:50 μg/ml to 300 μg/ml; (2) Poly I:C: 400 μg/mL to 3000 μg/mL; and (3) lipid molecules constituting a vector, the vector being a mixture of a cationic liposome and a neutral liposome.
Resumen de: US2025325513A1
The present invention relates to a cabazitaxel prodrug anti-tumor preparation, designs and synthesizes a small molecule cabazitaxel prodrug with branched fatty alcohol involving formulas (I), (II) and (III) and containing different fatty alcohol side chains and different linking chains, and prepares a self-assembled nanoparticle. Results showed that the self-assembled nanoparticle of the small molecule cabazitaxel prodrug with branched fatty alcohol can effectively improve the efficacy of cabazitaxel, reduce toxic and side effects. The length of branched fatty alcohol side chains, the structure of the fatty alcohol side chains, the elemental composition of the linking chains and the length of the linking chains significantly affect preparation properties, in vivo fate and anti-tumor activity of the cabazitaxel-branched fatty alcohol prodrug self-assembled nanoparticle, which exhibits higher anti-tumor activity and lower toxicity compared with the self-assembled nanoparticle of small molecule cabazitaxel prodrug with straight-chain fatty alcohol.
Resumen de: US2025325491A1
The invention relates to nanoparticles particularly for nanoparticles suitable for the delivery of a nucleic acid to a cell. The nanoparticles comprise polyvalent molecules to stabilise the nucleic acid molecules in the nanoparticles. Particularly the polyvalent molecules have a dendrimer like structure. The invention further relates to manufacturing nanoparticles, and uses of such nanoparticles in the treatment of a disease.
Resumen de: WO2025219707A1
Drug-eluting device The present invention relates to: (i) a drug delivery device comprising a fibrous material, said fibrous material comprising: (a) a first fibre which comprises: - a first polymeric material comprising a carbon nanoparticle covalently bonded to a polymer; and/or - a first acidic polysaccharide; and/or - a first protein; and (b) a drug, and a method of manufacture thereof, as well as said drug delivery device for use in administering a drug.
Resumen de: WO2025219528A1
Provided is a lipid mix composition for forming lipid nanoparticles (LNP) in association with a DNA, for use in transfecting a cell of hematopoietic lineage, the lipid mix composition comprising an ionizable lipid, a phospholipid, a stabilizer, and cholesterol. The LNP, a method of modifying cells of hematopoietic lineage using the LNP, a cell of hematopoietic lineage modified by the method, and a method of treatment for immune deficiency, cancer, autoimmune disease or genetic insufficiency are also provided.
Resumen de: WO2025219491A1
Provided are compositions and methods for transfecting a cell of hematopoietic lineage, using a lipid nanoparticle (LNP) that includes a lipid mix composition including an ionizable lipid, the lipid mix composition encapsulating an HDR template DNA comprising a gene of interest for insertion into a desired chromosomal locus.
Resumen de: WO2025219613A1
The present invention relates to particles comprising a polymer and DNA, wherein the DNA comprises at least one binding site capable of binding to RNA, in particular miRNA. Further, the invention is directed to a pharmaceutical composition comprising the particles and a pharmaceutically acceptable carrier. Moreover, the particles may be used in the treatment disease associated with RNA-overexpression.
Resumen de: WO2025219465A1
The invention relates to a hemoglobin-based oxygen carrier where the carrier has a metal-organic framework to entrap hemoglobin and is provided as nanoparticles; as well as a method for preparing the nanoparticles. The invention further relates to a pharmaceutical comprising the hemoglobin-based oxygen carrier.
Resumen de: WO2025221702A1
Provided is a nanoparticle including: a solid core comprising an active ingredient, a wax, and a carrier oil; a first shell surrounding the core, wherein the first shell comprises one or more proteins and one or more carbohydrates; and a second shell surrounding the first shell.
Resumen de: WO2025221757A1
The present invention relates to a carbohydrate-polymer conjugate comprising a repeat unit, a divalent linker, and a carbohydrate and compositions thereof, wherein the repeat unit comprises, in part, a linker and a carbohydrate, and a method comprising the step of administering a composition comprising the carbohydrate-polymer conjugate, wherein the composition can cross the blood-brain barrier.
Resumen de: WO2025221606A1
The present disclosure relates to compounds of the various formulas disclosed herein, including Formula (I) and Formula (II), and uses thereof in hyperactivating mammalian dendritic cells, such as human dendritic cells or canine dendritic cells. The present disclosure also relates to compositions comprising a compound of the various formulas disclosed herein, including Formula (I) and Formula (II), and one or more of a pathogen recognition receptor agonist, an antigen, and mammalian dendritic cells, as well as methods for production and use of the compositions.
Resumen de: WO2025221397A1
The present disclosure provides a protein-based nanoparticle made of a protein and an amine for the delivery of a nucleic acid molecule, such as an mRNA vaccine, to a cell. The present disclosure further provides systems and methods for producing such protein-based nanoparticle including a nucleic acid molecule, and methods of delivering the nucleic acid molecule to a cell, and treating a subject with the compositions as disclosure herein.
Nº publicación: WO2025218536A1 23/10/2025
Solicitante:
RONGCAN SHANGHAI BIOTECH CO LTD [CN]
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Resumen de: WO2025218536A1
The present invention relates to lipid nanoparticles for targeted high-efficiency delivery of nucleic acid to the lung, an inhaled formulation comprising the lipid nanoparticles, and use of the lipid nanoparticles and the inhaled formulation in the preparation of a pharmaceutical composition for targeted delivery of the nucleic acid to the lung. The inhaled formulation of the lipid nanoparticles for targeted high-efficiency delivery of the nucleic acid to the lung can withstand shearing damage generated during atomization and overcome multiple physiological barriers to deliver the lipid nanoparticles to the lung, and it has high transfection efficiency.
BOPI
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