Resumen de: US20260174820A1
The present invention provides an ionizable polymer comprising a constitutional unit according to formula (I). wherein R1 and R2 are as defined in the specification. The ionizable polymer of the invention finds use in the intracellular delivery of polynucleotides in vitro and in vivo. The present invention also provides compositions comprising the ionizable polymer of the invention and a polynucleotide. The compositions disclosed herein find utility as medicaments, in particular as medicaments for the prevention or treatment of an infectious disease, a cancer, or a protein-deficiency disease.
Resumen de: AU2024398058A1
The present disclosure describes improved LNP-based RNA vaccines, nucleobase editing systems, and therapeutics for use in treating and/or immunization against disease. In particular, the disclosure describes improved LNPs, including novel and improved ionizable lipids for making LNPs, that enhance the targeted delivery of LNP-based RNA vaccines and therapeutics based on linear and/or circular mRNAs. The improved LNPs protect linear and/or circular mRNA payloads from degradation and clearance while achieving targeted systemic or local delivery for use as enhanced vaccines and/or therapeutic agents.
Resumen de: US20260174733A1
A proliposomal delivery system and method for preparing the same is provided. The proliposomal delivery system involves a proliposomal formulation incorporating liposomal entrapment of vitamin C within a nano-emulsion that is stabilized using partially hydrolyzed guar gum. The proliposomal formulation includes ascorbic acid as the active ingredient, sunflower lecithin with a phosphatidylcholine content as an encapsulating material, and partially hydrolyzed guar gum as a dietary polysaccharide. The proliposomal formulation is a safer and more bioavailable form of vitamin C designed to minimize gastric discomfort. The proliposomal formulation offers a nutraceutical supplement aimed at managing oxidative stress, enhancing immune system function, supporting heart health, and improving skin. The process provided for preparing the proliposomal formulation is simple, cost-effective and scalable.
Resumen de: AU2026204465A1
COMPOSITIONS AND METHODS FOR TREATING OCULAR DISEASES The present invention relates to the treatment of ocular diseases in a human subject. In particular, the invention relates to an intracameral administration of a sustained release biodegradable intracameral implant.5 COMPOSITIONS AND METHODS FOR TREATING OCULAR DISEASES un u n
Resumen de: US20260174700A1
The invention relates to formulation for oral delivery of peptides and/or drug molecules and a method of manufacturing the same. The oral formulation is useful for delivering peptides to the gastrointestinal tract, preferably at the ileo-caecal junction of colon.
Resumen de: AU2026204376A1
Antibodies and antigen binding fragments thereof that specifically recognize and bind an epitope of elastin that is exposed and accessible in degraded elastic fiber are described. The antibodies and/or antigen binding fragments can be operably linked to a secondary component, including biologically active agents such as therapeutics and/or imaging agents. Optionally, the antibodies and/or antigen binding fragments thereof can be attached to a surface of a carrier, such as a particle, for specific binding and delivery of the carried agents to degraded elastic fiber. fiber. un u n
Resumen de: AU2024388909A1
The present invention relates to the technical field of medicine, and relates in particular to a molecule having a targeting function. The molecule having a targeting function can specifically deliver a drug to a specific cell, which facilitates precise programming of a specific cell in the body, a therapeutic effect of the drug is produced, and consequently drug dosage and side effects are greatly reduced, and the effect of precise treatment to cells in the body is achieved.
Resumen de: EP4763227A1
The present invention relates to a composition comprising a solid carrier, a protein branched-chain amino acid-degrading enzyme or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the protein branched-chain amino acid-degrading enzyme or a fragment thereof by embedding the protein branched-chain amino acid-degrading enzyme or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses of the composition for the prevention, delay of progression or treatment of branched-chain amino acid metabolism related diseases.
Resumen de: EP4763197A1
The present invention relates to a composition comprising a solid carrier, a protein ATP-hydrolyzing enzyme or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the protein ATP-hydrolyzing enzyme or a fragment thereof by embedding the protein ATP-hydrolyzing enzyme or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses of the composition for the prevention, delay of progression or treatment of dysbiosis or a dysbiosis-related disease. The present invention also relates to a pharmaceutical combination comprising (i) said composition; and (ii) an immune checkpoint modulator and uses thereof for the prevention, delay of progression or treatment of cancer and/or for use in adoptive (T) cell therapy.
Resumen de: EP4763189A1
The present invention relates to a composition comprising a solid carrier, a lipase or a fragment thereof immobilized on the surface of the solid carrier wherein the lipase or a fragment thereof is in the open conformation, a protease or a fragment thereof immobilized on the surface of the solid carrier, an amylase or a fragment thereof immobilized on the surface of the solid carrier, an agent which interacts with the lid domain of the lipase or a fragment thereof, a protective layer to protect the lipase or a fragment thereof, the protease or a fragment thereof and the amylase or a fragment thereof by embedding the lipase or a fragment thereof, the protease or a fragment thereof and the amylase or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group; b) an amino acid; c) a non-reducing sugar and/or a non-reducing sugar alcohol; and d) a surfactant. The present invention also relates to solid, lyophilized and liquid compositions of said nanoparticle composition, capsules comprising said nanoparticle composition, methods of producing said nanoparticle composition and uses thereof.
Resumen de: EP4763287A2
The invention relates to novel polymer conjugated lipids and to novel compositions comprising said novel polymer conjugated lipids useful for the delivery of nucleic acids into living cells.
Resumen de: WO2025038864A1
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use, including compounds of Formula (I) or a pharmaceutically acceptable salt thereof.
Resumen de: WO2025036861A1
The present invention relates to drug delivery, more specifically, to polymersomes used as a drug delivery systems. The present invention also relates to polymersomes comprising a composition for use in treating, for example, ocular disorders and pulmonary disorders.
Resumen de: WO2025038855A1
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use such as a compound of Formula (A) or a pharmaceutically acceptable salt thereof.
Resumen de: WO2025036956A1
The present invention relates to compositions designed for localized delivery within a subject's body. More particularly, the invention pertains to therapeutic compositions that remain localized to specific organs or tissues and do not exhibit systemic distribution. These compositions include specific carriers and therapeutic agents suitable for various medical applications.
Resumen de: WO2025040887A1
Described is a nanopore that is selectively convertible between an open form and a closed form using light, a method for producing such a nanopore, a method of modulating the flow of one or more substances through the nanopore, the use of the nanopore in modulating ionic flow across an amphiphilic membrane, a device comprising the nanopore, use of the nanopore as an ionotronic component, use of the nanopore in bio-computation and/or bioionotronics, a method of transmitting information using the nanopore, and method of receiving information comprising the nanopore, and an ionotronic component comprising the nanopore.
Resumen de: WO2025062064A1
The present disclosure provides a structural protein comprising an amino acid sequence unit comprising a motif (VPGVG)n, wherein n is 2 or more, and an amino acid sequence selected from SEQ ID NO:1–10. The present disclosure also provides a polynucleotide, an expression vector and a host cell, as well as a method for producing the structural protein. The present disclosure also provides a medical product, an electrospun filament comprising the structural protein, a protein-based micro-robot, photonic crystals, metamaterial and thermoresponsive glass comprising one or more of the structural proteins. The present disclosure also provides a method for preparing a product. The present disclosure also provides use of one or more of the structural proteins for preparing a product.
Resumen de: CN122256328A
本申请适用于纳米材料技术领域,提供了一种CaPhy‑NAD纳米酶及其制备方法、应用与解酒制剂,所述CaPhy‑NAD纳米酶以植酸钙纳米颗粒为载体,原位负载乙醇氧化酶、乙醛脱氢酶、辣根过氧化物酶与辅酶NAD制得;该植酸钙纳米颗粒在酸性环境下少量酸溶释放的Ca2+可有效激活乙醛脱氢酶的催化活性,三种酶在植酸钙纳米颗粒的孔道内发生高效限域级联反应,既实现了辅酶NAD的循环再生,又能同步清除乙醇、乙醛及过氧化氢三类酒精代谢相关有毒物质。本申请制得的CaPhy‑NAD纳米酶兼具优异的催化活性与良好的生物安全性,且可实现口服给药,为酒精中毒的防治提供了全新的技术思路与解决方案。
Resumen de: CN122251565A
本发明涉及mRNA疫苗技术领域,具体为多抗原串联mRNA肺癌疫苗及其在制备抗肿瘤药物中的应用,包含mRNA分子与包封该mRNA的脂质纳米颗粒,mRNA自5'端至3'端依次为帽结构、5'非翻译区、开放阅读框、3'非翻译区、polyA尾,开放阅读框编码的融合抗原蛋白,依次包含信号肽、肺癌共享突变抗原模块、肿瘤相关抗原模块、MHC‑I定向转运结构域,各抗原片段间通过AAY连接肽连接,脂质纳米颗粒由离子化脂质、胆固醇、辅助磷脂、PEG‑脂质组成。本发明兼顾抗原特异性与覆盖广度,可高效释放抗原表位,提升抗原呈递效率,强效激活特异性抗肿瘤免疫,适配稳定递送体系,在肺癌治疗中具备良好应用前景。
Resumen de: CN122251364A
本发明公开了一种生物活性聚氨基酸纳米复合物、制备方法及用途,通过缩合反应合成透明质酸‑block‑聚两亲性嵌段共聚物,通过亲疏水、静电、金属配位等相互作用包覆CD73小分子抑制剂APCP和PD1/PD‑L1抑制剂S7911,制备HA@APCP/S7911。HA@APCP/S7911通过EPR效应富集在肿瘤区域后,受肿瘤区域透明质酸酶和低pH的共同作用下,实现对APCP和S7911在肿瘤组织内释放。APCP通过抑制CD73酶活性,恢复NK细胞的代谢和活性,启动NK细胞免疫应答;S7911在肿瘤组织释放后抑制PD‑L1与PD‑1的结合,阻滞免疫检查点启动T细胞免疫,结合放疗提高抗肿瘤免疫反应,放大了有效免疫应答的范围,为促进放射免疫联合治疗的联合作用提供了一种新的思路。
Resumen de: CN122251607A
本发明属于免疫学领域,涉及一种mRNA递送载体、口服疫苗的制备方法和应用。本发明的海藻酸钙微球与胆酸‑氟共修饰聚乙烯亚胺(DFP)形成双重保护体系,海藻酸钙微球的致密交联结构抵御胃肠道酸碱环境与核酸酶降解,DFP与mRNA形成的复合物可进一步防止mRNA被酶解。提供用于激活肠黏膜免疫的口服mRNA递送系统的制备及其在腹泻疫苗中的应用,针对猪腹泻型冠状病毒感染提供有效保护,为肠道黏膜疫苗的研发提供了新思路。
Resumen de: CN122251616A
本发明公开了一种核酸‑药物纳米颗粒及其制备方法和应用。所述核酸‑药物纳米颗粒含有核酸和AGEs交联断裂剂,所述AGEs交联断裂剂嵌入所述核酸的DNA双链结构中;所述核酸由四条脱氧核糖核酸单链自组装形成;所述脱氧核糖核酸单链的核酸序列包括如SEQ ID NO:1‑ SEQ ID NO:4所示的序列。本发明的纳米颗粒具有良好的生物相容性和载药性能,能够主动靶向脂肪组织,在脂肪组织中释放AGEs交联断裂剂,有效抑制糖基化终末产物介导的胶原交联,减轻脂肪组织纤维化,恢复脂肪细胞可塑性。
Resumen de: CN122255060A
本发明涉及生物医药技术领域,具体涉及一种维生素骨架可电离脂质及其制备方法与应用。本发明提供的维生素骨架可电离脂质包括亲水碱基头部以及疏水脂肪链尾部,具有两亲性,能够在水相中形成脂质体结构,维生素骨架可电离脂质的电荷可以随着pH的改变而改变,在酸性条件下可以带正电,与带负电的核酸分子通过静电作用结合,可有效递送核酸药物进入细胞内,提高细胞转染效率。
Resumen de: CN122251619A
本发明公开了一种辛伐他汀‑β‑环糊精纳米粒及其制备方法和应用,其属于生物医药技术领域,其中,所述辛伐他汀‑β‑环糊精偶联物具有活性氧响应性,其包含由β‑环糊精羟基、硫缩酮连接单元和辛伐他汀羟基形成的酯键连接结构,所述硫缩酮连接单元为3‑巯基丙酸与丙酮缩合形成的硫缩酮二羧酸残基;该辛伐他汀‑β‑环糊精偶联物能够自组装形成辛伐他汀‑β‑环糊精纳米粒。本发明提供的活性氧响应性辛伐他汀‑β‑环糊精偶联物可以在氧化应激相关环境中发生解离,实现抑制胆固醇合成和促进胆固醇外排的双路径胆固醇稳态调控功能,降低巨噬细胞泡沫化程度,进而减轻动脉粥样硬化斑块脂质负荷。
Resumen de: CN122251433A
本发明公开一种增强奥沙利铂临床疗效的复合纳米酶及其制备方法和应用,复合纳米酶由铟钌纳米酶和伊利司莫包埋锚定层组成;铟钌纳米酶由铟钌纳米颗粒和碳氮骨架组成,铟钌纳米颗粒负载在碳氮骨架表面和碳氮骨架孔道中;伊利司莫包埋锚定层由双巯基聚乙二醇包覆层和伊利司莫组成。制备方法:将锌盐甲醇溶液和2‑甲基咪唑甲醇溶液共沉淀制备ZIF‑8,焙烧得碳氮骨架;将碳氮骨架分散液与硝酸铟溶液和三氯化钌溶液油浴搅拌制备纳米酶前驱体,焙烧得铟钌纳米酶;将铟钌纳米酶分散至双巯基聚乙二醇溶液中并滴加伊利司莫溶液,将得到的固体产物干燥即得。本发明可以解决奥沙利铂治疗中药物耐受性强、胞内积聚不足以及肿瘤免疫抑制严重等问题。
Resumen de: WO2025116530A1
The present invention relates to nanoparticles comprising sirolimus and albumin, a pharmaceutical composition for subcutaneous administration, comprising same, and a preparation method therefor. If the preparation method of the present invention is used, nanoparticles comprising sirolimus and albumin and having excellent improved particle size distribution maintenance stability can be prepared, and the prepared nanoparticles can be prepared in a pharmaceutical composition suitable for subcutaneous injection, and thus dosage is increased and convenience of administration is improved.
Resumen de: WO2025080672A1
The present invention relates to methods and compositions comprising a delivery vehicle conjugated to a chemokine or cytokine ligand, wherein the delivery vehicle comprises at least one agent, and wherein the targeting domain specifically binds to the surface of a target immune cell. The invention also relates to methods for treating or preventing diseases and disorders, including cancers, infectious diseases, and immunological disorders, using the described delivery vehicle for delivery of a therapeutic agent.
Resumen de: WO2025092744A1
Disclosed are an aminocholesterol derivative and a composition comprising same. Nanoparticles containing the aminocholesterol derivative are used for delivering nucleic acid drugs to improve the delivery efficiency for the nucleic acid drugs, thereby enhancing the targeting and therapeutic effect of the nucleic acid drugs. Thus, the nanoparticles are of great significance for the development and application of nucleic acid prophylactic and therapeutic agents.
Resumen de: CN122251578A
本发明属于医药材料领域,具体涉及一种金属核增强双势垒压电纳米系统及其制备方法与应用。所述制备方法包括:先通过分步反应制备“金属@介孔二氧化钛@介孔钛酸钡”核壳结构,再经脂质包裹得到目标纳米颗粒。该系统创新的构建了双势垒结构,可在4mmHg生理压力下自触发产生活性氧,实现恶性腹水的内源性压力自驱动治疗,兼具低压高催化效率、长期结构稳定及良好生物相容性优势,解决了现有治疗手段侵入性强、依赖外源刺激的问题,为恶性腹水治疗提供了新型安全高效的技术方案。
Resumen de: WO2025061153A1
Provided are RNA-lipid particles and formulations comprising novel lipids and therapeutic agents, methods of preparing RNA-lipid particles, and methods of delivering and/or administering RNA-lipid particles. In particular, provided are RNA-lipid particles comprising a polysarcosine-based lipid conjugate or a conjugate of polysarcosine and lipid-like material, and RNA such as single-stranded messenger RNA which encodes a peptide or protein of interest, and methods of preparing the RNA-lipid particles. The particles comprising mRNA and polysarcosine are delivered to target tissues after administered via intramuscular, intravenous, or subcutaneous route.
Resumen de: WO2025062315A2
This disclosure relates to methods of manufacturing cardanols and/or derivatives thereof, cardols and/or derivatives thereof, lipids and/or derivatives thereof, and lipid nanoparticles and/or derivatives thereof. The disclosure extends to the cardanols and/or derivatives thereof, the cardols and/or derivatives thereof, the lipids and/or derivatives thereof, and the lipid nanoparticles and/or derivatives thereof. The disclosure further extends to use of lipids, preferably ionizable lipids, in the manufacture of lipid nanoparticles, wherein said lipid nanoparticles are employed in the manufacture of delivery means for active pharmaceutical ingredients (API).
Resumen de: CN122251575A
本发明公开了一种抗肿瘤免疫治疗纳米粒及其制备方法和应用,涉及纳米制剂技术领域,其技术方案要点所述核壳结构包括核和壳两部分,所述核为氧化锰纳米粒,所述壳为携载有声敏剂的高分子聚合物,所述氧化锰纳米粒的水力学半径为179.0±10.8nm,表面电位为9.45±1.73mV,还包括氧化锰佐剂,其中所述氧化锰佐剂为二氧化锰纳米粒,所述声敏剂为卟啉及其衍生物中的一种;所述高分子聚合物为聚乳酸羟基乙酸共聚物,所述二氧化锰纳米粒的粒径为20.7±2.5nm,表面电位为17.34±0.66mV,效果是为肿瘤的个性化免疫治疗提供了一种简单有效的策略。
Resumen de: CN122251335A
本发明涉及一种o/w乳液,其包括油相、水相和分散在水相中的颗粒,其中,所述的颗粒是含有核酸成分的脂质纳米颗粒(LNP),所述颗粒能够稳定油水界面。本发明还公开了一种含该o/w乳液的药物组合物,例如疫苗。
Resumen de: CN122251633A
本发明公开了一种金属有机框架/过氧化钙异质结复合纳米材料及其制备方法与应用,通过一步配位自组装法将钒(V)掺杂到四(4‑羧基苯基)卟啉(TCPP)与钴(Co)离子配位形成的CoTCPP配合物中,构建V‑CoTCPP金属有机框架基体,利用V元素的成骨及降糖功能,为材料赋予额外的生物活性;在此基础上,通过原位沉淀法将过氧化钙纳米颗粒负载于V‑CoTCPP表面,形成具有非对称结构的无机‑有机Janus异质结纳米平台,在微波触发下,实现微波热疗‑动力协同杀菌、调节局部糖代谢及促进骨组织修复的功能协同。
Resumen de: CN122251365A
本发明属于生物材料制备技术领域,具体涉及一种基于巨噬细胞膜的复合递药系统及其制备方法与应用,本发明的复合递药系统以脂质纳米粒为药物靶向递送载体,通过主动吞噬方式包覆槲皮素,再将巨噬细胞膜包覆在脂质纳米粒表面,构建巨噬细胞膜包覆的槲皮素脂质纳米粒MCM@QU@LNP;本发明提供的复合递药系统可促进药物在RA病灶富集,提高局部药物暴露、实现药物RA部位精准递送,具有良好的生物安全性,降低系统毒性。
Resumen de: CN122251614A
本发明公开了一种EcN@BEVs益生菌复合物及其制备方法和应用,属于生物技术领域。包括大肠杆菌Nissle 1917 EcN以及附着在大肠杆菌Nissle 1917 EcN外表面的白及胞外囊泡BEVs。本申请为益生菌制剂构建了一种先进的靶向递送系统,成功将EcN与BEVs定向连接,构建出工程化益生菌复合物EcN@BEVs;附着在大肠杆菌 Nissle 1917 EcN外表面的纳米涂层BEVs作为一个刚性屏障,增强对胃酸和胆盐的抵抗,同时通过改善与粘膜上皮的粘附促进肠道的长期滞留,提高了益生菌口服给药在消化道各种极端条件下的存活率,有效促进其在肠道内的定植能力;得益于白及来源的纳米颗粒的抗炎特性和封装益生菌的高生存率及强大肠道粘附能力,本EcN@BEVs益生菌复合物相对于未涂层的EcN,在治疗肠炎方面具有更优的治疗效果。
Resumen de: CN121466034A
The invention belongs to the technical field of micro-robots, and particularly relates to a pulse laser responsive colloid micro-nano robot and a preparation method and application thereof.The micro-nano robot is of a spherical structure and comprises a core layer and a functional layer from inside to outside, and the functional layer wraps the core layer; the core layer is made of a thermal response phase change material, the functional layer is made of a noble metal nano material, and the noble metal nano material of the functional layer can absorb and convert near-infrared pulse laser into heat, so that the core layer is subjected to phase change, and the micro-nano robot is driven to realize stepping motion and penetrate through a physical barrier. The micro-nano robot is prepared through the steps of high-energy ultrasonic mechanical emulsification, chemical reduction and the like. The micro-nano robot can be used as a universal carrier, is used for loading chemotherapeutic drugs, gene drugs or photo-thermal therapeutic agents, realizes an integrated function of barrier penetration-targeted drug delivery-collaborative treatment for tumor deep delivery, blood brain barrier penetration and the like, and provides a brand new technical path for diagnosis and treatment of brain glioma and other diseases.
Resumen de: CN122251362A
本发明用于生物医药与功能食品技术领域,公开了基于黑果枸杞多糖微环境释放的肠道免疫调节系统,所述系统包括:用于递送和释放黑果枸杞多糖的微环境响应型递送单元,所述递送单元由黑果枸杞多糖核心和包覆所述核心的响应层构成,所述响应层包含至少一种能够响应肠道靶部位微环境信号的响应性材料,以使所述递送单元在到达肠道靶部位时,能响应所述微环境信号而发生结构变化,从而实现所述黑果枸杞多糖的定位释放。该基于黑果枸杞多糖微环境释放的肠道免疫调节系统,通过由响应性材料构成的“响应层”对黑果枸杞多糖(LBPs)活性核心进行包覆,构建了一个物理屏障,该系统能够有效抵御上消化道恶劣环境对LBPs结构的破坏。
Resumen de: CN122251406A
本发明涉及药物制剂与纳米医学领域,尤其涉及一种纳米药物组合物及其联合给药系统和应用。所述纳米药物组合物包含α1‑AR阻断剂和两亲性纳米载体;所述α1‑AR阻断剂包载于由两亲性纳米载体形成的疏水核心中;所述α1‑AR阻断剂选自哌唑嗪、特拉唑嗪、多沙唑嗪中的一种或多种;所述纳米药物组合物的平均粒径为220‑260 nm,聚合物分散指数<0.3,包封率>50%。本发明的纳米药物组合物兼具抗肿瘤增殖与免疫微环境重塑双重功能,提升药物在生理介质中的分散稳定性,有效降低体内清除率、延长循环时间,实现药物的高效体内递送。该纳米制剂的制备工艺稳定、可规模化生产。
Resumen de: CN122255484A
本发明提供了一种用于奥沙利铂传输的聚赖氨酸纳米材料及其制备方法,涉及生物制药技术领域。本申请的合成方法合成路线明确、可控性强。通过模块化设计,依次制备炔基化透明质酸、叠氮化聚赖氨酸‑维生素E琥珀酸酯及MMP酶敏感肽段连接体,最终采用点击化学高效偶联,产物结构明确、纯度高、批次间重现性好,克服了传统方法偶联效率低、副产物多的问题。制备得到的药物具备主动靶向与双重响应释药能力,透明质酸可特异性识别肿瘤细胞表面高表达的CD44受体,实现主动靶向;MMP酶敏感肽段可在肿瘤微环境中被过度表达的MMP‑9酶切,实现酶响应释药;同时,载体中引入的二硫键结构可在高浓度谷胱甘肽环境中断裂,实现还原响应释药。
Resumen de: CN122255146A
本发明公开了一种D–A–D型有机小分子、包含其的纳米颗粒及它们的制备方法与应用。所述D–A–D型有机小分子及其纳米颗粒,特别适用于白血病、淋巴瘤、多发性骨髓瘤等血液系统恶性肿瘤的精准光热治疗与实时成像引导。所述D–A–D型有机小分子,构建了D–A–D型共轭结构,通过增强分子内电荷转移(ICT)效应,实现近红外吸收红移至685nm,并利用高激发态重组能(λ=1.29eV)促进非辐射跃迁,光热转换效率超过35%。该小分子不含金属与卤素,分子量约878Da,具备良好生物相容性与代谢潜力。通过自组装形成粒径约130nm的纳米颗粒,具备良好胶体稳定性与被动靶向能力。同时,其高摩尔吸光系数与高效光热转换特性赋予其强光声成像能力,可实时监测药物分布,实现“诊疗一体化”。
Resumen de: WO2025124548A1
The present disclosure relates to an imidazole lipid and a use thereof. Specifically, provided are a compound represented by formula I or a salt thereof, and a lipid particle or a pharmaceutical composition containing the compound. The compound can be used as a delivery system, and is used for preparing drugs for preventing and/or treating cancers, infectious diseases, autoimmune diseases, neurodegenerative diseases, inflammation, etc. The definition of each group in formula (I) is as described in the description.
Resumen de: KR20250076429A
The present invention relates to a use of a gene carrier comprising metal nanoparticles and a double-helical nucleic acid molecule bound to the surface of the nanoparticles as a drug carrier, in particular, a therapeutic use of the carrier for metabolic diseases, or a pharmaceutical composition for preventing or treating metabolic diseases comprising the same.
Resumen de: CN122229808A
本发明属于医药领域,具体涉及一种磷酸钙矿化载抗生素纳米血小板囊泡复合凝胶及其制备方法和应用。本发明通过非接触式超声和膜挤出法制备负载抗生素的纳米血小板囊泡,随后在磷酸盐与钙离子存在下进行矿化,最后将其嵌入由纤维蛋白原和凝血酶形成的纤维蛋白水凝胶网络中,制得复合凝胶。该复合凝胶结合了纳米血小板囊泡的天然细菌靶向、免疫调节与药物递送功能,以及磷酸钙矿化的离子缓释与促成骨活性。体外实验表明,该凝胶能有效杀灭金黄色葡萄球菌并破坏其生物膜,同时显著促进骨髓间充质干细胞的增殖、迁移及成骨分化。体内动物模型证实,该凝胶能高效清除骨感染部位的细菌,并显著促进新骨生成,实现感染性骨缺损的一体化治疗。
Resumen de: CN122229800A
本发明公开一种MSN核层双载药结构的纳米药物递送系统、应用及制备方法,能够实现铜死亡激活与能量阻断的协同治疗效果,有效克服单一疗法的局限性,保证药物能够高浓度地富集在肿瘤细胞内部,从而增强治疗效果,能够特异性识别并结合肾癌细胞表面的CD44受体,显著提高药物在肿瘤部位的富集效率,实现药物的触发式释放,有效避免药物在血液循环中的泄露,降低对正常组织的全身毒性,具有良好的生物安全性。这种MSN核层双载药结构的纳米药物递送系统,其包括:介孔二氧化硅MSN、药物层、透明质酸层,药物层负载在介孔二氧化硅上,透明质酸层包裹药物层;药物层中的药物包括葡萄糖代谢抑制剂和铜死亡诱导剂。
Resumen de: CN122229998A
本发明公开了一种神经肿瘤串扰阻断凝胶协同增效mRNA疫苗的组合物及其制备与应用,属于生物医药技术领域。该组合物包括制剂A和制剂B,制剂A为基于纳米铝类脂质的mRNA制剂,具有优异的细胞摄取效率和溶酶体逃逸能力,制剂B为神经肿瘤串扰阻断凝胶,可实现小分子药物的原位长效缓释,二者协同作用,能双信号激活T细胞并阻断神经肿瘤免疫串扰,有效逆转肿瘤免疫抑制微环境。本发明制备方法反应原料易得,所制备的联合组合物安全性高,在实体瘤免疫治疗中具有广阔应用前景。
Resumen de: CN122234006A
本申请公开了含胺基甲酸酯的多胺基多酯键阳离子脂质、包含其的组合物及用途,具体公开了一种式(I)所示的阳离子脂质。本申请提供的阳离子脂质可用于核酸靶向递送,同时显著增强核酸药物的脾靶向性。
Resumen de: WO2025102670A1
A nanoparticle adiposome encapsulating a hydrophobic small-molecule drug, a preparation method therefor, and a use thereof. The nanoparticle adiposome encapsulating the hydrophobic small-molecule drug comprises a monomolecular phospholipid membrane and hydrophobic small-molecule drug-containing neutral lipids encapsulated within the monomolecular phospholipid membrane. The nanoparticle adiposome has the hydrophobic small-molecule drug-containing neutral lipids as a hydrophobic core, and nanobeads wrapped by the monomolecular phospholipid membrane can efficiently dissolve and encapsulate a hydrophobic small-molecule compound. In addition, the adiposome encapsulating the hydrophobic small-molecule drug has bioactivity, can remarkably kill cancer cells, and has a killing effect superior to that of a free drug. In addition, the preparation method is simple and efficient. Therefore, the adiposome is a prospecting hydrophobic small-molecule drug delivery platform, and can be widely applied to the treatment of diseases such as cancer, infectious diseases and metabolic diseases.
Resumen de: CN122233962A
本发明属于生物医药技术领域,具体涉及一种新型可电离阳离子脂质化合物、纳米颗粒及其制备方法和应用。所述可电离阳离子脂质化合物的结构如式(Ⅰ)所示,其中取代基R1、R2、G1、G2、G3、G4的定义如文中所示。采用本发明提供的新型可电离阳离子脂质,与中性辅助脂质、胆固醇和聚乙二醇化脂质组成的脂质纳米颗粒,通过微流控技术高效包封治疗性蛋白的mRNA。该体系具有包封率高、递送效率优异及生物安全性好的特点,在体内展现出优于基准脂质DLin‑MC3‑DMA的蛋白表达或基因编辑效果,例如在TTR基因编辑治疗中实现了显著的蛋白敲低。
Resumen de: CN122235184A
本发明涉及一种新型mRNA治疗性肿瘤疫苗设计,具体而言,涉及一种编码融合蛋白的多核苷酸,所述融合蛋白包含肿瘤抗原和免疫增强元件。本发明还涉及一种多核苷酸的组合,其包含编码肿瘤抗原的至少一种多核苷酸以及编码免疫增强元件的至少一种多核苷酸。本发明还涉及包封有本发明的编码融合蛋白的多核苷酸或多核苷酸的组合的LNP,使用本发明的编码融合蛋白的多核苷酸、多核苷酸的组合或LNP治疗癌症的用途以及方法。
Resumen de: CN122235141A
本发明涉及一种治疗神经衰老和神经退行性疾病的基因治疗药物及其制备方法,所述药物包括包载靶向受体相互作用蛋白激酶3的小干扰RNA的工程化外泌体,具有高效穿过血脑屏障(BBB)和靶向神经元的递送能力。通过将所述工程化外泌体递送至患者脑部,实现对RIPK3基因的特异性沉默。本发明所提供的外泌体递送siRNA的治疗策略具有靶向性强、生物相容性好和安全性高的优点,为神经衰老及神经退行性疾病的治疗提供了一种新的、有效的技术方案。
Resumen de: CN122229804A
一种三七多糖铁纳米制剂及其制备方法与应用,可有效制备三七多糖铁纳米制剂,解决制备治疗缺铁性贫血、炎症性肠病相关贫血、缺铁性贫血相关结肠炎、炎症性贫血或炎症性肠病药物中的应用问题,具体步骤是:将三七粗多糖溶液用常规纯化方法提纯,得纯化的三七多糖,用超纯水溶解,在惰性气体的保护下,加入铁源,经过共沉淀、化学还原反应,调节pH值,将铁纳米粒负载于三七多糖上,得三七多糖包覆的铁纳米粒;本发明制备方法简单,原料丰富,生产成本低廉,是治疗缺铁性贫血、炎症性肠病相关性缺铁性贫血药物上的一大创新,有实际的推广应用价值。
Resumen de: CN122229807A
本公开属于生物制剂技术领域,特别涉及一种靶向活化中性粒细胞的仿生纳米颗粒及其制备方法与应用。本公开提供一种靶向活化中性粒细胞的仿生纳米颗粒,所述仿生纳米颗粒由纳米核芯和包覆在所述纳米核芯表面的内皮细胞膜组成,所述内皮细胞膜为有ICAM‑1表达的内皮细胞膜。本公开提供的仿生纳米颗粒具有活化中性粒细胞特异性靶向、生物相容性好、循环时间长、炎症富集效率高、诊疗一体化等优势,能够精准调控中性粒细胞过度活化,显著提升对急性感染、慢性创面等疾病的治疗效果与安全性。
Resumen de: CN122229797A
本发明公开了一种连续式制备双层包埋脂溶性营养素微粒的方法,包括以下步骤:(1)将脂溶性营养素芯材乳化,形成纳米级营养素乳化液;(2)疏水性辅料通过热风气流送入造粒塔内,在送风和引风系统作用下悬浮形成辅料气溶胶;通过离心喷雾装置将营养素乳液雾化成微细液滴喷入辅料气溶胶中,液滴表面吸附一层疏水性辅料,干燥定型,形成双层包埋脂溶性营养素微粒;(3)双层包埋脂溶性营养素微粒和部分疏水性辅料由造粒塔落入内置流化床内干燥。经气旋筛分离辅料,进入振动流化床和立式流化床进一步干燥。同时,通过引风系统分离回收辅料。该方法简化了产品制备工艺,降低了设备要求和能耗,实现了连续自动化生产,提升了产业化优势。
Resumen de: CN122229997A
本申请涉及一种用于阻断日本血吸虫病传播的保虫宿主疫苗组合物。本发明通过明确组分配方、优化工艺参数,最大化发挥抗原‑递送系统‑佐剂的协同作用,兼具免疫强效、工艺可控、安全适配等优势。动物实验表明,该疫苗尤其适用于保虫宿主,显著降低肠道虫卵负荷(盲肠降卵率达98.68%),有效阻断虫卵经粪便排入水体,切断关键传播环节,为实现"以传染源控制为主"的血吸虫病防治策略和“健康中国2030”消除血吸虫病目标提供关键技术支撑。
Resumen de: CN122234143A
本发明涉及一种用于基因递送的多肽及由其制备的多肽/核酸分子复合物及其应用。本申请的多肽具有下式I所示的结构,其中,通式I中含有由Y表示的免疫细胞靶向基团。本申请开发的多肽作为基因递送载体具有高效的核酸包载能力,此外,其在体外和体内对免疫细胞具有高效的递送效率,因此,在基因的免疫细胞靶向递送中具有广阔的应用前景。I
Resumen de: CN122229803A
本发明公开了一种可穿透肌腱的脂质纳米颗粒(LNP)及其应用,该 LNP 针对性设计为 50~90nm 粒径与 0~20mV 弱正电位,精准匹配肌腱胶原网格孔径(<100nm),中和负电蛋白聚糖排斥作用,实现对肌腱深层的高效渗透。经实验验证,该 LNP 可穿透至跟腱约 2/3 深度。其包载编码 FGF7 的修饰 mRNA 后,在肌腱损伤部位原位长效表达 FGF7 蛋白持续大于等于14 天,既能促进腱系分化、增加胶原沉积,又能抑制纤维化与肌腱异位骨化。本发明通过 LNP 的高效穿透特性,突破肌腱递送瓶颈,为跟腱炎、肩袖损伤等肌腱病提供新型高效治疗策略。
Resumen de: CN122229166A
本发明公开了一种基于MLCT的油体纳米结构脂质载体及其制备方法和应用,包括,将长链油与癸酸甘油三酯混合后加入固定化脂肪酶真空干燥、搅拌过滤后脱酸、真空蒸发得到MLCT结构脂;将单硬脂酸甘油与MLCT结构脂混合加热作为油相,将瓜蒌籽油体与超纯水混合加热作为水相,水相与油相混合后加热超声破碎、冰浴得到油体纳米结构脂质载体。本发明通过将天然油体(OB)作为乳化剂替代传统小分子乳化剂,在契合消费者健康需求的同时,展现出卓越的稳定性与安全性;降低了化学合成物残留风险。其次,MLCT 的引入从代谢机制层面革新 NLC 的消化性能。体外模拟消化实验数据显示游离脂肪酸释放率高于80%,为功能性食品与药物递送系统的开发提供了新解决方案。
Resumen de: CN122234232A
本发明公开了一种登革病毒环状mRNA疫苗;属于核酸疫苗技术领域。本发明提供的表达登革病毒四价EDⅢ串联抗原的环状mRNA分子,均一表达DENV‑1至DENV‑4四种血清型EDⅢ结构域;主要通过四种血清型EDⅢ结构域多重组合、T2A/P2A自剪切肽、Furin酶切位点、和柔性连接肽(GGGS)4排列组合设计使四种血清型EDⅢ结构域表达均一,进一步形成包含该环状mRNA分子的登革病毒四价mRNA疫苗的免疫应答均衡高效,减少ADE表位免疫应答,能有效规避ADE效应风险,工艺可实现工业化放大。
Resumen de: CN122234152A
本发明公开了一种PIV3病毒F蛋白、mRNA疫苗及其应用。所述PIV3病毒F蛋白的氨基酸序列如SEQ ID NO:5或6所示。本发明提供的mRNA疫苗能够促进抗PIV3病毒抗体的产生,有效地控制PIV3病毒感染以及在肺部的扩增,用于预防和/或治疗PIV3病毒感染所致疾病。
Resumen de: CN122229805A
本发明公开了一种丙泊酚纳米制剂及其制备方法,解决了传统乳剂依赖大豆油等脂质载体,导致脂质过载(高甘油三酯血症)、突释率高(注射痛)及初始血药浓度过高(血压下降)等问题发生。本专利采用多种生物可降解聚合物,如PLA(聚乳酸),PLGA(聚乳酸‑羟基乙酸共聚物)等纳米颗粒作为丙泊酚载体,通过调节载体粒径及聚乙二醇修饰(提高循环稳定性),实现丙泊酚的有效封装和持续缓释。
Resumen de: CN122229799A
本发明公开了一种光热与自电泳双驱动Janus纳米马达及制备方法和应用。本发明在光热金纳米棒一端选择性沉积SiO2绝缘层;NIR‑II光照下,Au NR两端形成热梯度,产生自热泳高速定向运动。为进一步实现持续推进且不改轨迹,本申请将实心SiO2蚀刻为中空hSiO2,原位合成Pt纳米粒。放射性皮炎病灶富含ROS,Pt催化H2O2分解,导致Au‑Pt端局部H+浓度升高,形成自建电场,驱动纳米马达自电泳前进。NIR‑II瞬态推进与H2O2持续推进协同,使马达快速穿透表皮,直达真皮及皮下。负载的单宁酸可清除辐射产生的ROS,缓解氧化应激。本发明为放射性皮炎提供新的治疗策略。
Resumen de: CN122229764A
本发明涉及生物医药与材料科学技术领域,尤其是涉及一种局部缓释免疫调节水凝胶及其制备与应用。本发明首先将表面活性剂、助表面活性剂溶解后与无机前驱体(过硫酸盐)混匀,得到过硫酸盐纳米颗粒;然后将其分散后加入盐酸多巴胺溶液和油胺,混匀得到过硫酸盐@P核壳纳米颗粒;将透明质酸和普朗尼克F127混匀,得到HF水凝胶预聚液;最后将过硫酸盐@P核壳纳米颗粒与HF水凝胶预聚液混匀,自组装后后处理得到局部缓释免疫调节水凝胶制剂。本发明提供的局部缓释免疫调节水凝胶能够长效滞留于手术创面、同步实现清除肿瘤微残留与逆转免疫抑制微环境。
Resumen de: WO2025065012A1
Disclosed herein are Clostridium neurotoxin (BoNT) or SNARE cleaving homologue variants, constructs, and methods of use. The BoNT or SNARE cleaving homologue variants can include one or more amino acid or domain substitutions, deletions, or insertions in the light chain.
Resumen de: CN122230042A
本发明涉及一种巨噬细胞膜包被的梓醇纳米颗粒及其制备方法与应用,采用巨噬细胞膜包被聚乳酸‑羟基乙酸共聚物纳米粒作为梓醇的递送载体,将其靶向递送至脑缺血区域,制备得到巨噬细胞膜包被梓醇纳米粒。与现有技术相比,本发明MpM/CNPs具备免疫逃逸潜力,能够减少纳米粒的非特异性清除,延长其体内血液循环时间;可与活化的血管内皮细胞竞争性结合,从而减少血管内皮细胞活化引发的炎症细胞迁移,即能延缓炎症细胞向炎症内皮的浸润,对抑制炎症反应的扩散具有重要意义;可显著缓解缺血再灌注引发的脑组织超微结构损伤,调控血管内皮细胞通透性,可修复血管内皮细胞的紧密连接,实现血脑屏障的结构重建,促进血管内皮细胞的增殖、迁移。
Resumen de: CN122229802A
本发明涉及生物医药领域,具体涉及一种仿生压电纳米粒及其制备方法和在制备治疗抑郁症的药物中的应用。所述的仿生压电纳米粒(称为NKM@ZnO‑PDA)由具有压电效应的纳米氧化锌为核心,分别包裹聚多巴胺(PDA)和自然杀伤细胞膜(NK细胞)。其制备方法包括:将压电纳米氧化锌分散在多巴胺的碱性水溶液中自氧化制备仿生压电纳米粒的内核(称为ZnO‑PDA);ZnO‑PDA在超声孵育和共挤出下包裹NK细胞膜制备仿生压电纳米粒。所述仿生压电纳米粒具有优异的血脑屏障透过性和生物安全性,被星形胶质吞噬后可改善线粒体功能。所述仿生压电纳米粒被细胞吞噬后可在低功率超声条件下极化,刺激细胞钙内流,促进含有线粒体的细胞外囊泡释放,向神经元转移健康线粒体从而恢复神经元功能。
Resumen de: CN122234121A
本发明公开了一种地塞米松前药可电离脂质分子的制备方法及应用。所述脂质分子(DPIL)的结构式表示为T‑A‑Linker‑Dex;T代表脂质尾部,A代表氨基核心,Linker代表连接基团,Dex代表地塞米松。基于DPIL建立出地塞米松脂质前药LNP平台,简称SHILD LNP。该SHIELD LNP能显著降低炎症不良反应,包括显著下调促炎性细胞因子IL‑1β、IL‑6、TNF‑α、趋化因子CCL2等,且实现对mRNA的高效递送。该SHILD LNP具有普适性规律,将DPIL引入其他种类LNP体系,同样可以显著降低炎症反应和提高mRNA表达水平,实现针对肝脏、脾脏、肺脏、胰腺等器官的靶向mRNA递送。
Resumen de: CN122229747A
本发明公开了一种负载人参皂苷Rg1工程化囊泡的仿生复合微针贴片和制备方法。仿生复合微针贴片包括微针基底和微针阵列,所述微针阵列设置在所述微针基底上;所述微针基体包括脱细胞柚皮基质和聚乙烯醇形成的复合水凝胶材料,所述复合水凝胶材料具有三维连通的多孔网络结构;所述微针阵列中均匀分散有载药工程化细胞膜囊泡,所述载药工程化细胞膜囊泡包括杂合细胞膜和被所述杂合细胞膜包裹的人参皂苷Rg1;所述杂合细胞膜包括由免疫细胞膜和心肌细胞膜融合形成的杂合膜组分;所述免疫细胞膜采用巨噬细胞膜。本发明能够突破免疫清除与靶向不足的双重屏障,实现药物向受损心肌的精准递送与长期局部缓释。
Resumen de: CN122235043A
本发明公开了一种富含胁迫诱导因子的植物外泌体及其制备方法和应用。本发明通过以下步骤制备植物外泌体:首先对活体植物母株施加非生物胁迫处理,所述非生物胁迫包括低温、盐渍、干旱或机械损伤中的一种或多种组合;然后从经过胁迫处理的植物组织中提取汁液,进行差速离心得到粗提液;再采用双水相萃取技术对所述粗提液进行纯化,得到植物外泌体纯品。本发明通过胁迫处理诱导植物产生应激反应,使外泌体产量提高3‑5倍,同时富集具有抗炎活性的微小RNA miR‑159a。所得外泌体粒径分布均匀,膜结构完整,在抑制炎症因子TNF‑α和促进细胞迁移方面表现出显著活性。本发明还提供了所述外泌体在制备载药系统中的应用。
Resumen de: CN122230003A
本发明涉及一种自佐剂型仿生脂质纳米颗粒及其微流控组装方法与抗肿瘤应用。所述自佐剂型仿生脂质纳米颗粒具有在负载mRNA的脂质纳米颗粒(LNP)的表面包覆有活化树突状细胞膜(ADCM)的结构。本发明相较于传统LNP的进步体现在:本申请的自佐剂型仿生脂质纳米颗粒(ADCM‑LNP)表现出更快的细胞摄取速率和更高的转染效率,从而诱导产生强烈的Th1型免疫反应以及强效的CTL介导的肿瘤杀伤效应。体内生物分布研究表明该系统具有显著的脾脏靶向性(Splenic Tropism),并在HER2阳性乳腺癌模型中展现出优异的治疗效果。
Resumen de: CN122229798A
本发明公开了一种高稳定性麦角甾醇脂质纳米分散体及其制备方法,包括:按质量百分比计,由以下组分组成:麦角甾醇0.5‑5%、复合脂质载体5‑20%、乳化剂2‑8%、稳定剂0.1‑2%、抗氧化剂0.05‑1%,余量为去离子水;所述复合脂质载体为固态脂质与液态脂质的复配物,所述固态脂质与液态脂质质量比为1:0.2‑1:1,涉及食品添加剂与生物活性物质递送技术领域,通过采用复合脂质载体对麦角甾醇进行包埋,结合了固态脂质稳定性好和液态脂质分散性优的优点,能够为麦角甾醇提供稳定的包埋环境,同时改善纳米分散体的分散性,通过复配乳化剂和添加稳定剂、抗氧化剂,进一步提高了纳米分散体的稳定性,有效抑制了麦角甾醇的氧化降解,延长了产品的保质期。
Resumen de: CN122245505A
本发明涉及生物技术领域,具体为基于可电离脂质库筛选高效低毒mRNA递送载体的方法,包括S1.提供包含至少100种结构多样化的可电离脂质的脂质库;S2.采用自动化微流控平台,将所述脂质库中各可电离脂质分别与辅助脂质、胆固醇、PEG‑脂质及报告基因mRNA混合,并行制备成脂质纳米颗粒(LNP)库;S3.对所述LNP库进行高通量体外筛选;S4.选取效能评分排名前10%且细胞活力值大于80%的LNP对应的可电离脂质作为Hit(初筛阳性候选物);S5.对所述Hit进行体内快速验证;S6.基于体内肝表达强度>阳性对照200%且ALT小于100 U/L的标准确定终选脂质。基于可电离脂质库筛选高效低毒mRNA递送载体的方法,可以更有效地筛选无毒的mRNA递送载体。
Resumen de: CN122234220A
本发明属于纳米载体及免疫治疗技术领域,具体涉及一种靶向肿瘤相关巨噬细胞的脂质纳米颗粒、联合递送方法及在肿瘤免疫治疗方面的应用。本发明构建的靶向肿瘤相关巨噬细胞CD206的脂质纳米颗粒,用于体内高效递送多功能核酸分子,实现免疫调控、基因编辑及抗肿瘤治疗。本发明通过脂质颗粒设计、核酸序列设计及体内外功能验证,实现了靶向递送和多功能协同作用。本发明通过ipLNP 的体内递送,巨噬细胞可同时实现:1)表达抗 PD‑L1 抗体,阻断免疫抑制信号;2)表达 HER2‑CAR 结构,增强对肿瘤的吞噬和杀伤能力;3)通过 CRISPR/Cas9 介导的 FN1 敲除,重塑肿瘤免疫微环境。利用本发明构建的脂质纳米颗粒,能够解决传统 LNP 递送系统在肿瘤微环境中免疫细胞靶向不足的问题。
Resumen de: CN122229806A
本发明公开了ARP1@BTZ@PtS在制备多发性骨髓瘤治疗药物中的应用,通过水热合成法得到了分散性好、光热性能优异的PtS,利用了生物膜‑癌细胞膜来包裹BTZ和PtS,不仅尽可能减少了治疗剂递送至肿瘤部位前的损失,还实现了BTZ‑温和光热协同治疗。本发明使用了多发性骨髓瘤细胞系ARP1细胞膜作为仿生材料,具有肿瘤细胞膜本身的同源靶向和骨髓归巢能力。与BTZ和温和光热同剂量单独治疗相比,ARP1@BTZ@PtS实现BTZ‑温和光热协同治疗,在细胞、皮下瘤和静脉移植瘤模型中具有更优的抑制增殖、诱导凋亡、消融肿瘤效果。
Resumen de: CN122235044A
本发明涉及一种从黄花倒水莲植物组织中分离外泌体与大囊泡的方法,属于生物医药领域,方法:采用含4%纤维素酶、2%果胶酶和0.6 mol/L甘露醇的酶解液在50℃下酶解黄花倒水莲组织6小时;通过1000×g离心去除碎片后,以17,000×g离心沉淀大囊泡;上清液经滤膜过滤后,于100,000×g超速离心两次纯化外泌体。通过透射电镜、粒径分析、Zeta电位检测及BCA蛋白定量进行多维度鉴定。所得外泌体平均粒径175.3 nm,电位‑37.78 mV;大囊泡平均粒径188.5 nm,电位‑28.27 mV。实现了植物源外泌体与大囊泡的高效同步分离,所得囊泡结构完整、性质稳定,细胞实验显示其具有促进肾细胞增殖活性,动物实验证实其对糖尿病肾病具有治疗作用,可在制备药物递送系统或治疗糖尿病肾病的药物中应用。
Resumen de: CN122229801A
本发明公开了一Treg细胞来源外泌体载药系统及其制备方法和应用,属于生物医药技术领域。本发明通过T细胞来源外泌体超声负载miR‑142‑5p得到载药系统。本发明超声加载法具有更高的加载效率,确保每个外泌体携带足够剂量的药物;更好的膜完整性,超声结束后膜孔可以自行修复,最大程度地保留了外泌体的天然结构和功能;操作相对简单、可控。T细胞来源外泌体与携带的药物(miR‑142‑5p)可以产生协同增效作用,在治疗心肌病尤其是心肌梗死方面具有良好的效果。
Resumen de: WO2024238942A2
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: WO2026125793A2
The present invention relates to nanostructured lipid carriers comprising a lipid core of liquid and solid lipids, particularly a triglyceride having a fatty acid chain of 12 to 16 carbon atoms and a mixture of triglycerides of medium-chain saturated fatty acids and covered by a surfactant layer, in which at least one of said surfactants is phosphatidylcholine. Said nanostructured lipid carriers are characterised in that they do not comprise any active ingredient or agent. The invention also relates to the (non-therapeutic) use of the nanostructured lipid carriers for inhibiting the formation of biofilms, as well as their therapeutic use as a medicament for treating or preventing diseases caused by biofilm-forming bacteria.
Resumen de: US20260165977A1
0000 The present disclosure relates to supraparticles loaded with high levels of payload and having controlled payload release profiles. Such supraparticles may be used in a range of therapeutic applications.
Resumen de: AU2025344428A1
The present disclosure relates to ionisable lipids of Formula I and related structures that comprise a headgroup, core, and hydrophobic substituents. The lipids are capable of forming lipid particles, including lipid nanoparticles (LNPs), in combination with additional lipids such as neutral lipids, charged lipids, structural lipids, or PEGylated lipids. The lipid particles are suitable for delivery of therapeutic agents, including polynucleotides, peptides, antibodies, and small molecules. Compositions comprising the ionisable lipids and methods of forming and using the lipid particles are also provided.
Resumen de: AU2024414252A1
The present invention pertains to the technical field of pharmaceutical preparations, and provides a novel solution-type quetiapine composition for transnasal administration. In the provided composition, quetiapine is completely dissolved therein in the form of a solution and administered transnasally. Compared with oral dosage forms on the market, the provided quetiapine solution-type transnasal preparation has particularly useful pharmacokinetic and pharmacodynamic profiles. Compared with other disclosed transnasal nano-emulsion forms, the composition of the present invention still has surprising pharmacokinetic and pharmacodynamic profiles, shows good clinical application prospects, and is expected to meet unmet clinical needs. On this basis, the present invention also provides a further improved high-saturation dissolution concentration technical solution and a further improved good chemical stability technical solution for preparing the quetiapine solution-type transnasal preparation.
Resumen de: AU2024401935A1
Aspects of the disclosure relate to compositions and methods for improving the stability of lipid nanoparticles (LNPs). In some embodiments, the LNPs comprise one or more active pharmaceutical ingredients (API) encapsulated therein. The disclosure is based, in part, on compositions that directly or indirectly reduce the degradation (e.g., oxidation, hydrolysis, etc.) of one or more lipids components of the lipid nanoparticle. In some embodiments, the compositions comprise a citrate drug product matrix, EDTA drug product matrix, methionine drug product matrix, and/or a tryptophan drug product matrix. The disclosure also provides methods for storing compositions contemplated herein as well as methods for improving the stability of the API.
Resumen de: US20260166176A1
Alterations of the 5′GT splice motif or 3′AG splice motif result in severe missplicing and are prevalent in many genetic diseases, including cystic fibrosis (CF). While protein-targeted modulator therapies are currently available for treatment of CF, individuals with these canonical splice site variants (CSSVs) are among the ˜10% who remain untreated. The most common CF-causing variant in individuals of African descent is a CSSV, c.2988+1G>A. While >75% of individuals with a CSSV have a modulator eligible in trans allele, only 50% of the 450 individuals bearing c.2988+1G>A are eligible. Thus, there is a particular unmet need for a treatment for these individuals. CRISPR/Cas9-mediated adenine base editing (ABE) is an efficient and targeted genome editing method correct G>A variants. We electroporated NRCH-ABE8e mRNA and a previously optimized sgRNA to non-differentiated human primary nasal (HNE) or bronchial (HBE) epithelial cells from individuals with CF compound heterozygous for c.2988+1G>A. After differentiation of edited cells genomic editing and recovery of CFTR channel function were assessed. In primary HBEs and primary HNEs, we observed an allelic conversion to WT of 74.7% and 81.3%, respectively, at the +1 site. Interestingly, we also observed high levels of editing at adjacent adenines (+3, +7), which would have a modest effect on mRNA splicing (˜20% reduction). However, this did not preclude recovery of CFTR channel function. Compared to WT/WT HBEs and HNEs, un
Resumen de: US20260166175A1
The present disclosure provides for a polymer comprising repeating units of a cyclodextrin comprising α-cyclodextrin (α-CD), γ-cyclodextrin (γ-CD), or any combination thereof, and a crosslinker. Further provided herein are a particle and cryogel comprising the same. Also provided herein are methods of using and making thereof.
Resumen de: US20260165980A1
0000 The present disclosure relates to lymphatic endothelial cell (LEC)-specific lipid nanoparticle comprising a sterol, an ionizable lipid, a PEGylated lipid, and a phospholipid. Also disclosed herein are compositions comprising the LEC-specific lipid nanoparticle and a therapeutic agent. The present disclosure further relates to methods of delivering an agent to a lymphatic system in a subject, comprising administering to the subject an effective amount of the composition of the LEC-specific lipid nanoparticle and therapeutic agent.
Resumen de: US20260166171A1
The present disclosure features a method of preparing a population of payload-associated cell complexes (PACCs), as well as related methods of use thereof.
Resumen de: AU2024389988A1
Described herein are methods, compositions, and systems derived from uncultivated microorganisms useful supplementing liver enzyme deficiencies.
Resumen de: AU2024383218A1
The present disclosure relates to multimers capable of efficiently activating immune cells, such as T cells. In particular, it relates to multimers comprising at least two different ligands, such as a MHC-peptide and an anti-CD28 antibody, capable of binding to and together activate said immune cell. The disclosure further provides nucleic acid, vectors, and compositions encoding or comprising said multimers, and various methods for their use.
Resumen de: AU2024378001A1
The disclosure relates to compositions and methods for improving pulmonary delivery of therapeutic nucleic acids, e.g., DNA, mRNA, by enhancing transfection efficiency and thus efficacy of the therapeutic nucleic acids, using inhaled, Intranasal, Intratracheal, bronchial instillation and/or topical administration Intratracheal, bronchial instillation and/or topical administration transfection efficiency enhancing agents, including but not limited to 2-mercaptoethane sulfonate or cysteamine.
Resumen de: AU2024382784A1
A pharmaceutical composition comprising a nucleic acid construct and the medical use thereof. Specifically, the present invention relates to a lipid nanoparticle comprising a nucleic acid construct represented by formula I, which can achieve highly efficient delivery of a exogenous target gene, allowing the exogenous target gene to be highly efficiently and rapidly expressed in the body. The present invention has the advantages of no gene integration risk and being easy to scale up to an industrial level, is a more ideal treatment approach than naked plasmids, and can be used as a gene therapy drug for a plurality of diseases.
Resumen de: WO2026124214A1
The present invention relates to the technical field of drug delivery and particularly to a freeze-dried formulation of lipid nanoparticles, a preparation method therefor, and use thereof. In the present invention, suitable freeze-dried formulation formulas, including freeze-drying protectant types and concentrations, buffer types and concentrations, and lipid nanoparticle formulas, are screened to obtain a freeze-dried formulation of lipid nanoparticles that comprises 10%-20% w/v sucrose as a single-component freeze-drying protectant, a 1-20 mM Tris or HEPES buffer as a buffer system, and a specific ionizable lipid. The freeze-dried formulation is simple to prepare and still retains excellent physicochemical properties and in vivo transfection efficiency after reconstitution, demonstrating good prospects in the development and application of nucleic acid drugs.
Resumen de: US20260166173A1
0000 A peptide targeting a BRCA2 protein and an application thereof, and a drug for treating prostate cancer are provided, which relate to the technical field of protein peptides. The amino acid sequence of the peptide is shown in SEQ ID NO: 1. The peptide has strong binding ability with BRCA2 protein, and can deliver the BRCA2 protein into prostate cancer cells, which can effectively inhibit cell proliferation and tumor growth, and has an ability of targeted degradation of BRCA2. Sensitivity of prostate cancer cells to PARP inhibitor can be significantly enhanced by combining the peptide with PARP inhibitor. Thus, the peptide can be used in preparing the drug for treating mCRPC or in preparing a reagent for degrading BRCA2 protein. BRCA2-targeted PROTAC drug can provide a new treatment strategy for prostate cancer, and PARP targeting inhibitor is combined to provide PARP inhibitor synthesis lethality treatment effect for mCRPC patients.
Resumen de: US20260166134A1
An mRNA, an mRNA vaccine formulation, and a preparation method and use thereof are provided. An amino acid sequence of the mRNA is set forth in SEQ ID NO: 2. Studies have demonstrated that the mRNA vaccine formulation prepared from the mRNA having the amino acid sequence set forth in SEQ ID NO: 2 can effectively stimulate dendritic cell maturation and secretion of pro-inflammatory cytokines, thereby activating effector T cells to kill prostate cancer cells. A preparation method of the mRNA vaccine formulation is simple and suitable for industrial-scale production. By using lipid nanoparticles (LNPs) as a carrier, the mRNA vaccine formulation achieves high encapsulation efficiency, enabling robust induction of immune responses in vivo with high immunogenicity.
Resumen de: WO2024254192A2
The present invention provides a method of treating neurodegenerative diseases. The method comprises the step of administering to a subject in need thereof an effective amount of a polymer-flavonoid conjugate, or a nanocomplex having an outer shell comprising one or more polymer-flavonoid conjugates and optionally an inner shell comprising one or more flavonoid oligomer and a drug such as anti-CD3 or anti-CD33 encapsulated within the shells. The present method brings therapeutic effective materials through blood-brain barrier to treat neurodegenerative diseases. The present method is effective to treat neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Lewy body dementia and Huntington's disease.
Resumen de: CN121532174A
The present disclosure describes a lipid-polymer hybrid nanoparticle and a method of synthesizing such a nanoparticle or a composition containing such a nanoparticle. These nanoparticles are made from a biodegradable polymer-based micellar core surrounded by a lipid-based shell, wherein a majority of the agent is present on the inner periphery of such nanoparticles due to physical adhesion to lipid molecules. Only a small amount of medicament is encapsulated in the micelle core. Thus, the lipid-based shell becomes the primary excipient part of the nanoparticle, and the core containing the biodegradable polymer becomes the secondary excipient part of the nanoparticle.
Resumen de: WO2024254459A2
Disclosed are methods of treating complex regional pain syndrome in a subject by administering a therapeutic MSC secretome product made by a method comprising culturing bone marrow-derived MSCs under conditions that include oxygen tension below 5% and a culture media with a pH below 7.
Resumen de: WO2026127735A1
The present invention relates to a PD-L1-targeting PROTAC peptide, PROTAC nanoparticles comprising same, and a composition for cancer prevention or treatment containing same as an active ingredient, wherein in cancer tissues, the PD-L1 receptor is subjected to a direct lysosomal degradation pathway and a PROTAC-mediated degradation pathway, thereby not only inducing PD-L1 protein degradation but also fundamentally blocking PD-L1 protein regeneration, thus inhibiting the transmission of immunosuppressive signals in the interaction between cancer cells and T cells to activate immune responses, so that the effect of inducing the death of cancer cells can be continuously and effectively achieved.
Resumen de: WO2026127822A1
There is provided compound comprising a structure represented by general formula (1): wherein R1, R3, R4, R5, and R6 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; R7 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; R10 is optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; k is 0 or 1; n and l are each independently ≥ 1; A comprises galactose and/or a derivative(s) thereof; and B comprises a lipid and/or a derivative(s) thereof
Resumen de: WO2026127823A1
There is provided a compound comprising a structure represented by general formula (1) wherein A comprises a sterol and/or a derivative(s) thereof; B comprises: (i) a carbohydrate and/or a derivative(s) thereof; or (ii) an oligopeptide or a polypeptide comprising carbohydrate and/or a derivative(s) thereof; R1 and R3 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; R2 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; R4 is –H or –C(=O)R, where R is optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; p = 0 or 1; and q = 0 or 1.
Resumen de: WO2026123105A1
An AI-based automated self-driving laboratory system comprises a machine learning module (MLM) operatively coupled to a laboratory module (LM). Pretrained with a set of molecules to learn their structures and properties, the MLM is configured to produce an initial set of molecules for the LM to synthesize and analyze based on the learned structures and properties, and receive feedback of structures and properties of the initial set for further training and produce a next iteration for the LM to synthesize and analyze. The LM is configured to synthesize and analyze the initial set, relay the initial set's structures and properties for the iterative training, receive from the MLM, and synthesize and analyze the next iteration. The MLM is iteratively retrained to produce the next iteration for iterative synthesis and analysis by the LM until at least one termination criterion is met. An ionizable lipid of Formula I is disclosed.
Resumen de: US20260166075A1
0000 The disclosure relates to pharmaceutical compositions including Ir/Cu nanoenzymes, methods of treatment for neurodegenerative disease or disorder, and kits including the compositions. The Ir/Cu nanoenzymes can suppress oxidative stress in neuronal cells and inhibit aggregation of alpha-synuclein.
Resumen de: US20260167972A1
A nanoparticle complex contains short-interfering RNA (siRNA) and one or more amino acid derivatives. The nanoparticles may be spherical or micelle-like. The complex may have a mixture of spherical/micelle-like and fibril-like nanoparticles, or a mixture of spherical/micelle-like, sheet-like and/or fibril-like nanoparticles. The complexes may be used as part of a method of delivering RNA or DNA into a cell, where the RNA or DNA is mixed with one or more amino acid derivatives, the mixture is diluted into a cell culture medium, and a cell is dosed with the diluted mixture. The RNA may be small interfering RNA (siRNA). The method may reduce messenger RNA (mRNA) levels and/or protein levels related to a specific gene or genes by at least 50% and exhibit cell viabilities equal to or exceeding 40% after 48 hours.
Resumen de: WO2026125660A1
The present invention refers to Particles comprising circular RNA and compositions thereof as well as methods of their use.
Resumen de: WO2026128814A1
Disclosed are solid compositions, comprising a lipid nanoparticle, a nucleic acid, and an antioxidant; wherein the nucleic acid and the antioxidant are encapsulated by the lipid nanoparticle.
Resumen de: WO2026128792A1
Disclosed is a thermally stable, single-dose mRNA-LNP (lipid nanoparticle) platform.
Resumen de: WO2026127586A1
The present invention relates to a novel lipid and a preparation method therefor and, more particularly, to a lipid and a preparation method therefor, in which the lipid can be ionized to form a complex with an anionic drug, allowing various molecular designs according to a target tissue or organ, and thus is useful for tissue- or organ-specific drug delivery.
Resumen de: WO2026128506A1
The present disclosure provides an ionizable lipid of Formula (I) or Formula (II).
Resumen de: WO2026127599A1
The present invention relates to a novel lipid and a method for preparing same and, more particularly, to a lipid and a method for preparing same, wherein the lipid is ionizable to form a complex with an anionic drug and allows for various molecular designs depending on a target tissue or organ, thereby being useful for tissue- or organ-specific drug delivery.
Resumen de: WO2020163705A1
Provided herein are compositions of lipid-based nanoparticles, such as exosomes, that comprise a therapeutic anti-aging agent. Also provided are methods of using such compositions to treat a patient having an age-associated disorder. In particular, exosomes comprising a telomerase-encoding RNA are provided along with methods of their use in treating age-associated disorders.
Resumen de: WO2019210078A1
Aspects of the invention described herein concern targeting of a FOXP3 cDNA, e.g., full-length human-codon optimized, into a FOXP3 locus or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human lymphocyte. The compositions and materials described herein provide specificity for CRISPR/Cas-mediated gene regulation of murine, non-human primates or human FOXP3. Guide RNA sequences are used to target the FOXP3, AAVS1, and other candidate loci for CRISPR/Cas-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided. The alternative compositions described herein can be delivered in the form of Ribonucleoprotein (RNP) and may be used to target human and/or non-human primate FOXP3. Reagents are comprised of novel guide RNA sequences and can generate high frequency of on-target cleavage in combination with a Cas protein and novel gene delivery cassettes including FOXP3 cDNA +/-other cis linked gene products.
Resumen de: US20260166063A1
0000 The present disclosure relates, in general, to compositions comprising phosphatidylcholine and phosphatidylcholine derivatives, e.g., DLPC (1,2-dilauroyl-sn-glycero-3-phosphocholine) or DPPC, for the treatment of fibrosis, including liver fibrosis and associated conditions such as fatty liver disease, non-alcoholic steatohepatitis (NASH) and cirrhosis, or lung fibrosis and conditions associated with lung fibrosis.
Resumen de: US20260165979A1
0000 Candidate molecules may be constituent components of various lipid nanoparticles. A C-glycoside glycolipid compound of formula (I):
0000
0000 or formula (II):
0000
0000 a lipid nanoparticle comprising the same, or a pharmaceutical composition, particularly a vaccine, which comprises the lipid nanoparticle.
Resumen de: US20260167727A1
0000 The present invention relates to anti-DDR2 antibodies. More specifically, the present invention relates to nano-antibodies binding to DDR2 and various derivatives of the nano-antibodies. The present invention also relates to pharmaceutical compositions comprising these antibodies and derivatives and pharmaceutical uses thereof. The anti-DDR2 nano-antibodies of the present invention have strong binding activity and can be used for diagnosis, prevention and/or treatment of diseases mediated by abnormal DDR2 expression (such as cancer and inflammatory diseases), as well as imaging of cells expressing DDR2, such as biopsy navigation and intraoperative navigation.
Resumen de: US20260165982A1
0000 Provided herein are conjugated lipids and lipid-based particles, such as liposomes and lipid nanoparticles, containing the conjugated lipids. Methods of making the conjugated lipids and the lipid-based particles are described. Conjugated lipids include a lipid conjugated to a cell penetrating peptide. The lipid-based particles may include a payload. Compositions including the lipid-based particles may be administered to a subject.
Resumen de: US20260166162A1
A water-soluble carbon chain substance for regulating transmembrane transport and fluidity of cell membranes, and the preparation and use thereof. The water-soluble carbon chain substance comprises a carbon chain which originally has a certain water solubility, and the structure thereof comprises at least one or more hydrophobic carbon chain parts and one or more hydrophilic groups or residues; and also comprises a high-hydrophilicity complex formed by coupling a hydrophobic carbon chain part to a water-soluble molecule; the water-soluble carbon chain can be used for preventing and treating infection including viruses, bacteria and fungi, anti-ageing, and preventing and reducing the occurrence of neurodegenerative diseases such as Alzheimer's disease; by reducing the non-specific phagocytosis of the nano-drug by cells, the effect of the nano-drug on target lesions is improved.
Resumen de: US20260166149A1
0000 A near-infrared responsive bilirubin composite nanoparticle-loaded periodontal microneedle and a preparation method thereof are provided. A near-infrared responsive bilirubin composite nanoparticle includes a bilirubin-gelatin composite nanoparticle, wherein a surface of the bilirubin-gelatin composite nanoparticle is coated with a macrophage cell membrane and an organic-metal coordination supramolecular network coating in sequence; an outer surface of the near-infrared responsive bilirubin composite nanoparticle is modified with 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N-folate(polyethylene glycol) (DSPE-PEG-FA); in the bilirubin-gelatin composite nanoparticle, bilirubin and gelatin are covalently bonded via an amide bond; and in the organic-metal coordination supramolecular network coating, an organic ligand is anthocyanin, and a coordinating metal ion is ferric ion.
Resumen de: US20260166148A1
0000 A nanoparticle assembly is disclosed for the treatment and visualization of cancers. The assembly includes a core and two surrounding copolymer layers. The surrounding layers include hydrogel polymers, dextran-iron oxide nanoparticles, and quantum dots having a tail with a photosensitizer and an aptamer targeting cancer cells. Exposure of the assembly to an AC magnetic field creates heat and vibration from magnetostrictive nanobeads in the core. Piezoelectric elements generate electric charges from the vibration that activate fluorescence in the quantum dots, which in turn activate the photosensitizer to generate reactive oxygen species that induce apoptosis in cancer cells. Alternative anticancer mechanisms include thermolytic activation of oxygen independent cytotoxic free radicals from heat caused by magnetostrictive vibration. Thermolysis can also release immunological factors embedded in hydrogel polymers. 2-<18>fluoro-2-deoxyglucose (<18>F-FDG) may be incorporated as a diagnostic tool that can visualize cancer sites with PET-CT. Methods of preparing the nanoparticle assembly are disclosed.
Resumen de: US20260166071A1
0000 Provided herein are compositions and methods of reducing adduct formation.
Resumen de: WO2022081426A1
This disclosure provides peptides which can therefore be used for various therapeutic purposes, such as inhibiting the progression of a hyperproliferative disorder, including cancer; treating infectious diseases; enhancing a response to vaccination; treating sepsis; and promoting hair re-pigmentation or lightening of pigmented skin lesions.
Resumen de: US20260165981A1
The present invention features new lipid-polymer composite particles that are useful for the formulation of bioactive agents for administration to a subject. The nanoparticles include a block copolymer, a lipid, e.g., phospholipid, and a sterol. The formulations of a bioactive agent (e.g., a therapeutic agent, a nutraceutical agent, or a recreational agent) described herein provide for easier loading of lipid-polymer composite particles with higher drug loading capacity, increased stability of the formulations, and lower surface tension of water, which allows for lipid coating and entrapment.
Resumen de: CN115461082A
Provided are gene therapeutic compositions and methods for targeting ATP-binding cassette subfamily A member 4 (ABCA4) or functional fragments thereof in a patient, thereby treating or reducing genetic macular degeneration, including Stargardt's disease or other diseases involving retinal degeneration.
Resumen de: US20260166174A1
The present invention provides a generic platform for delivering molecules with low blood-brain barrier (BBB) penetration into the brain. The nano-delivery system is based on a core nanoparticle which is conjugated through a first polymeric linker to a brain-internalizing transporter moiety, and is further conjugated to a second polymeric linker bound to an active agent selected from a biologically active molecule or a labeling molecule. Further provided is a process for preparation of the nano-delivery system. The present invention further provides pharmaceutical compositions comprising the nano-delivery system and its use in therapeutic and/or diagnostic methods.
Resumen de: AU2025344951A1
The present disclosure relates to ionisable lipids of Formula I and related structures that comprise a headgroup, core, and hydrophobic substituents. The lipids are capable of forming lipid particles, including lipid nanoparticles (LNPs), in combination with additional lipids such as neutral lipids, charged lipids, structural lipids, or PEGylated lipids. The lipid particles are suitable for delivery of therapeutic agents, including polynucleotides, peptides, antibodies, and small molecules. Compositions comprising the ionisable lipids and methods of forming and using the lipid particles are also provided.
Resumen de: AU2024373408A1
The application relates to a lipid compound in Formula (I), a lipid nanoparticle containing it, a preparation method thereof, and a use in drug delivery.
Resumen de: WO2025080649A1
Disclosed herein are POEGMA-based block copolymers that have phase transition and self-assembly properties. The disclosed block copolymers can take advantage of these properties to form particles that can effectively encapsulate and deliver drugs. An example block copolymer includes a first block that includes POEGMA with ethylene glycol side chains of 2 monomers, 3 monomers, or combinations of both; and a second block that includes POEGMA with ethylene glycol side chains of 1 monomer, 2 monomers, or combinations of both. Also disclosed herein are compositions that include the block copolymers, methods of treating a disease or disorder, and methods of delivering a drug.
Resumen de: WO2025038371A1
A solid lipid nanoparticle (SLN) comprising an anionic form of a drug, which comprises a phosphate, a phenolate, or a carboxylate, entrapped in (a) a cationic form of the lipid SM-102 or a cationic form of the lipid ALC-0315, (b) distearoylphosphatidylcholine, (c) cholesterol, and (d) 1,2-dimyristoyl-rac-glycero-3-methoxypolyethylene glycol-2000 or ALC-0159, wherein the anionic form of the drug and the cationic form of the lipid of (a) form an ionic complex or a salt; a SLN comprising an anionic form of a drug, which comprises a phosphate, a phenolate, or a carboxylate, entrapped in dimethyldidodecylammonium bromide or 1,2-dioleoyl-3-trimethylammonium-propane; pharmaceutical compositions comprising the SLNs; and methods of making the SLNs.
Resumen de: WO2025038450A1
Disclosed herein are compositions comprising engineered immune cells and immunomodulatory nanoparticles for localized targeted therapy in the treatment of cancer and other diseases, injuries, or conditions. The engineered immune cells comprise chimeric antigen receptors (CARs) that secrete bispecific immune cell engager (e.g., bispecific T cell Engagers ("BiTEs"), e.g., a bispecific macrophage engager, e.g., a bispecific neutrophil engager). The CAR.bispecific immune cell engager engineered immune cells are combined with silica nanoparticles (with or without the immune cell engager targeting ligands attached thereto) for combinatorial treatment of the disease or condition.
Resumen de: EP4759305A1
Provided is a particle in which a core particle is coated with a coating layer and strength of which is ensured. Further provided are an orally disintegrating tablet and the like having improved physical properties (particularly, compactibility, disintegrability, tableting failures, tablet hardness after humidification, and friability after humidification). In a particle according to the present disclosure, a core particle is coated with a coating layer and the coating layer contains cellulose nanofibers. An orally disintegrating tablet according to the present disclosure includes particles containing cellulose nanofibers having an average particle diameter of less than 10 pm.
Resumen de: WO2025035015A1
Compositions and methods for delivery of nucleic acid therapeutics are disclosed herein. In some embodiments, a composition for treating a subject includes a dynamic hydrogel, and a nucleic acid therapeutic encapsulated by the dynamic hydrogel. The dynamic hydrogel can include a polymer and a plurality of nanoparticles. The polymer can be non-covalently crosslinked with the plurality of nanoparticles.
Resumen de: EP4759299A1
The present application relates to zinc-magnesium layered hydroxide nanoparticles as a drug delivery system for the treatment of periodontitis and/or peri-implantitis. The nanoparticles herein described have a layered hydroxide structure, have enhanced cell interaction and uptake, are biodegradable, efficient at drug loading, have an improved, pH-responsive drug release profile, and enhanced biological efficacy. The application also discloses a method to obtain the zinc-magnesium layered hydroxide nanoparticles.
Resumen de: WO2025034782A1
The disclosure provides nucleic acid transporters in the form of copolymers derived from lipid carbonate monomers and cationic aminoester monomers prepared using various amino alcohol initiators, including linear and branched amino alcohol initiators, cyclic amino alcohol initiators and imidazole initiators. The compounds are useful for the delivery of nucleic acids to cells in vitro, ex vivo, or in vivo.
Resumen de: WO2025034780A1
Provided are statistical copolymers derived from lipid carbonate monomers and cationic aminoester monomers useful in the delivery of nucleic acids across cell membranes, both in vitro and in vivo. The compounds described herein are shown to be particularly effective in delivery of mRNA into cells with resultant high protein expression, both in vitro and in vivo.
Resumen de: WO2025030426A1
Provided is a pharmaceutical preparation for treating Bietti's crystalline dystrophy (BCD), comprising a recombinant AAV expressing CYP4V2, sodium chloride, poloxamer, phosphate, and water for injection, and having a pH between 7.0 to 7.6. The pharmaceutical preparation has an excellent virus titer stability, very few AAV aggregates, a high mRNA expression of the target gene after multiple times of freezing and thawing, and/or an excellent high temperature stability. Moreover, the animal experiments in vivo demonstrate that the preparations with the formulations have an excellent property in reducing local inflammatory response of the retina, and further clinical trials demonstrate that the pharmaceutical preparations conform to the relevant technical specifications for safety and effectiveness in the Chinese Pharmacopoeia and the United States Pharmacopoeia (USP).
Resumen de: WO2024249559A2
Provided herein are compounds of Formula (I), or a pharmaceutically acceptable salt thereof. Further disclosed are nanoparticles comprising one of the compounds and methods of using the same for treating drug addiction, drug dependence, drug overdose, opioid use disorder, pain, chronic pain, fibromyalgia, arthritis, or obesity.
Resumen de: WO2025034764A1
Ionizable lipids are provided that are useful for delivering macromolecules, such as nucleic acids, into eukaryotic cells. The lipids can be used alone, in combination with other lipids and/or in combination with other transfection enhancing reagents to prepare transfection complexes.
Resumen de: WO2025034870A1
The present application, in certain aspects, pertains to methods and compositions for the treatment of a hormone-dependent cancer (such as an endometrial cancer (e.g., endometrioid endometrial cancer) or hormone-receptor positive breast cancer), using a composition comprising nanoparticles comprising sirolimus and an albumin in combination with an estrogen suppressor (such as letrozole or fulvestrant).
Resumen de: CN122208549A
本发明提供一种视网膜光感受器细胞亲和肽‑LNP‑基因药物复合物及其应用,本发明通过在脂质纳米颗粒表面嵌合来源于层粘连蛋白或玻连蛋白等细胞外基质的亲和肽,实现了对光感受器细胞的高效靶向递送,显著提高基因药物在视网膜光感受器细胞中的递送效率和表达效率,并在视网膜退行性病变模型中表现出明确的视功能修复效果。该技术具有递送效率高、生物相容性好、靶向性强及可扩展性强等优点,为视网膜退行性疾病及相关眼科疾病的基因药物治疗提供了一种新的技术路径。
Resumen de: CN114340733A
The present invention provides a method for improving or accelerating wound healing in a subject, the method comprising administering to a wound in a subject in need thereof an agent that inhibits expression of a clock gene wherein the clock gene is neuronal PAS domain protein 2 (Npas2). The invention also relates to methods for regenerating alveolar bone, regenerating connective tissue at a wound site and for reducing wound area size, comprising administering to a subject a bone loss site or a wound site, in particular an open wound site, an agent that inhibits the expression of Npas2.
Resumen de: CN122208552A
本发明涉及一种仿生基质囊泡及其制备方法及应用,具体涉及一种仿生基质囊泡及其制备方法及在制备治疗骨质疏松或骨老化药物中的应用。为解决现有技术存在靶向单一细胞内信号通路的干预策略,难以有效重塑整体细胞外稳态,导致骨再生能力下降治疗效果有限的不足之处,本发明一种仿生基质囊泡包括ALP‑nHA复合纳米颗粒以及包封在ALP‑nHA复合纳米颗粒外部的膜结构,所述膜结构为hMSCs膜;所述膜结构的表面设置有黏附或靶向特征,避免了现有技术中人工合成囊泡通常只携带单一药物或酶,或者只模拟膜结构,无法同时实现催化、成核、锚定的问题,同时,还提供了其制备方法及应用。
Resumen de: CN122208550A
本发明公开了一种仿生矿化乳酸氧化酶LOD@CeZol纳米粒的制备方法及应用;属于纳米材料和骨衰老疾病治疗技术领域,其制备步骤:按比例先将一定量的乳酸氧化酶(LOD)在超纯水中溶解,然后滴加入含有唑来膦酸的10% Tris‑HCl溶液,之后将硝酸锌溶液滴加到上述混合溶液中进行原位矿化反应,经过离心、冷冻干燥后得LOD@CeZol纳米粒。本发明中LOD@CeZol纳米粒的乳酸氧化酶活性,过氧化氢酶活性构成级联反应,加上其本身的超氧化物歧化酶活性(SOD)和谷胱甘肽过氧化物酶活性(GPx),能够将退变髓核细胞中的超氧阴离子转化为无毒的氧气和水,从而缓解髓核细胞退变进程,更重要的是,LOD催化乳酸底物产生丙酮酸,能够显著降低髓核细胞内部堆积的乳酸含量,继而降低髓核细胞的乳酸化修饰水平,逆转髓核细胞的衰老退变。
Resumen de: WO2025146027A1
The present disclosure relates to a PEG lipid and the use thereof. The PEG lipid can be used for preparing a lipid nanoparticle that delivers an active ingredient. The present disclosure further relates to a pharmaceutical composition and a lipid nanoparticle comprising the PEG lipid, as well as the use thereof in preparing a pharmaceutical.
Resumen de: WO2025102261A1
Lipid compounds for delivering a therapeutic agent, a preparation method therefor and the use thereof. The lipid compounds are compounds with structural formula (I) or pharmaceutically acceptable forms thereof. The lipid compounds may be used in combination with other lipid components, such as neutral lipids, cholesterol, and polymer-bound lipids, so as to form lipid nanoparticles used for delivery of therapeutic agents (e.g. nucleic acid molecules) to achieve therapeutic or prophylactic purposes (e.g. vaccination), thus enriching the types of ionizable lipid compounds.
Resumen de: WO2025098237A1
A lyophilized RNA-LNP (e.g., mRNA-lipid nanoparticle), a method of making or using the same, such as for vaccination using an mRNA encoding an antigenic vaccine (e.g., SARS-CoV-2).
Resumen de: WO2025080209A1
There is provided a compound represented by general formula (1) for preparing lipid nanoparticles encapsulating a therapeutic, prophylactic and/or biological agent: wherein AR comprises a unit from a poly(amino acid); R1 and R2 are each independently a hydrophobic group; R3, R4, and R5 are each independently H, optionally substituted alkyl, optionally substituted alkenyl or optionally substituted alkynyl; R7 is –H or –C(=O)R8, wherein R8 is optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl or optionally substituted alkoxy; m ≥ 1; and n ≥ 1.
Resumen de: US2025127728A1
Ionizable cationic lipids, methods for synthesizing the same, intermediates useful in synthesis of the ionizable cationic lipids and methods of synthesizing the intermediates are disclosed. The ionizable cationic lipids are useful as a component of lipid nanoparticles (LNP), which in turn can be used for the delivery of nucleic acids into cells in vivo or ex vivo. LNP compositions are also disclosed, including LNP comprising a functionalized lipid to enable conjugation of a binding moiety, and targeted LNP (tLNP), that is an LNP in which a binding moiety has been conjugated to the functionalized lipid and can serve as a targeting moiety to direct the tLNP to a desired tissue or cell type.
Resumen de: WO2025059107A1
The present disclosure relates, in one aspect, to ionizable lipid compounds of formula (I). In another aspect, the present disclosure relates to the preparation of ionizable lipids of formula (I). In another aspect, the present disclosure relates to lipid nanoparticles (LNPs) comprising at least one ionizable lipid of formula (I), and methods of use thereof.
Resumen de: WO2025057230A1
The present disclosure discloses particle including at least one bi-layer outer membrane, a plurality of vacuoles and at least one payload. The bi-layer outer membrane defining a lumen including an outer layer and an inner layer. Each of the outer layer and the inner layer define a hydrophobic portion and a hydrophilic portion. The outer layer including at least a plurality of cationic lipids, a plurality of neutral lipids, a plurality of helper lipid, and a plurality of polyethers. The vacuoles are disposed within the lumen defined by the bi-layer outer membrane. Each of the vacuole includes a membrane layer defining a lumen encapsulating the payload. Each of the membrane layer and the inner layer including at least the plurality of cationic lipids, the plurality of neutral lipids, and the plurality of helper lipids.
Resumen de: WO2024249419A2
An antibacterial nanoparticle comprising an inner silica core; a gold (Au) nanosphere surrounding the inner silica core; a surface layer comprising silver (Ag) surrounding the gold nanosphere or the silver is alloyed into the gold nanosphere; and an antimicrobial peptide conjugated to the surface layer, wherein the antimicrobial peptide is selected from the group consisting of a cysteine terminated Syn71 peptide, a cysteine terminated MC1-2 peptide, and a cysteine terminated Syn20 peptide.
Resumen de: WO2024243410A1
The present disclosure relates to a process for the preparation of tolerizing immune modifying nanoparticles encapsulating antigens associated with primary biliary cholangitis (PBC), compositions comprising the particles and use thereof for the treatment of PBC.
Resumen de: WO2024243480A1
The application relates to ionizable lipids. The formulations of ionizable lipids can be used for the delivery of one or more nucleic acid therapeutic agents, for example mRNA, siRNA or miRNA. The compositions can include additional lipids, such as non-cationic lipids, PEG-modified lipids and optionally cholesterol.
Resumen de: CN122208553A
本发明属于生物医药技术领域,具体涉及靶向递送circAMY2B的试剂在制备抗食管癌药物中的应用。本发明的靶向递送circAMY2B的试剂在制备抗食管癌药物中的应用中,所述的试剂为靶向递送circAMY2B的脂质纳米颗粒。本发明具有如下技术效果:本发明首次将靶向递送 circAMY2B 的脂质纳米颗粒应用于抗食管癌药物的制备,开拓了circAMY2B 的临床应用新领域,为食管癌的治疗提供新的药物选择,具有以下显著优点:1、靶向性强,疗效显著;2、稳定性高,生物利用度好;3、生物安全性好,毒副作用低。
Resumen de: CN122208536A
本发明公开了一种胆酸修饰型姜黄素聚合物脂质纳米粒及其制备工艺,属于药物制剂技术领域。该纳米粒以姜黄素为模型药物,胆酸为修饰剂,聚己内酯为聚合物载体,大豆卵磷脂为脂质成分,泊洛沙姆188为表面活性剂,采用乳化溶剂挥发法制备而成。本发明通过单因素实验结合Box‑Behnken响应面设计优化制备工艺,确定最优处方参数,所制备的胆酸修饰型姜黄素聚合物脂质纳米粒(CUR@CA‑PLNs)包封率高、粒径分布均匀、稳定性良好,且具有pH响应释药特性,能显著改善姜黄素水溶性差、化学稳定性低等问题,同时保留姜黄素的抗氧化活性,为姜黄素的临床应用提供了一种高效、稳定的药物递送系统。
Resumen de: CN122213231A
本发明公开了一种基于抗人ICAM‑1单域抗体的靶向协同治疗体系及其应用。首先,本发明提供了一种新型抗人ICAM‑1单域抗体,该单域抗体对衰老细胞表面ICAM‑1有高亲和力和高选择性。其次,本发明利用该单域抗体对经过“低氧+自噬诱导”预处理的年轻化间充质干细胞进行表面工程化修饰,获得能特异性靶向衰老微环境的靶向性年轻化MSCs。最后,本发明将所述靶向性年轻化MSCs与包载SIRT1激活剂SRT1720的缓释纳米微球共同整合于温敏水凝胶中,形成一种组合物。该组合物能够高效、特异性地富集于衰老组织,并通过MSCs的旁分泌作用与药物的缓释释放产生协同效应,显著清除衰老细胞、逆转衰老相关分泌表型、改善线粒体功能并恢复组织活力,在抗衰老治疗领域具有广阔的应用前景。
Resumen de: CN122208591A
本发明公开了一种蔓荆子黄素纳米制剂的制备方法,属于中药制剂领域。该方法将丝素蛋白与泊洛沙姆F127溶于水中作为水相,将蔓荆子黄素溶于丙酮中作为有机相,在搅拌条件下将有机相逐渐滴加于水相中,挥除有机溶剂,即得纳米制剂。本发明制备工艺简便,所得纳米制剂粒径均一、分散性好,显著提高了蔓荆子黄素的水溶性和口服生物利用度,同时利用丝素蛋白的黏膜黏附性和泊洛沙姆的稳定作用,实现了肠道靶向蓄积,可用于溃疡性结肠炎等炎症性肠病的口服治疗。
Resumen de: CN122212960A
本发明提供一种可电离阳离子脂质化合物及其应用,所述可电离阳离子脂质化合物具有式I所示结构。所述可电离阳离子脂质化合物通过特定的结构设计,由其制备得到的脂质纳米颗粒(LNP)对核酸分子具有优异的负载能力和更高的递送效率,且可以显著提高核酸分子在体内的翻译表达水平。
Resumen de: CN122208755A
本发明公开了一种协同声动力‑免疫治疗的纳米系统及其制备方法和应用,所述制备方法包括采用Shewanella细菌膜包覆PEG修饰的BaTiO3纳米颗粒,得到B@BM;将高表达TIM‑3 scFv的工程化巨噬细胞膜与R848进行混合并进行超声处理,得到R@CM,再进行PAH修饰;将B@BM和PAH修饰的R@CM进行混合共挤出;该纳米系统能够高效产生ROS,以及诱导肿瘤细胞发生ICD,同时释放新抗原;并能够激活树突细胞/巨噬细胞,阻断TIM‑3/Galectin‑9通路,通过SDT、免疫检查点阻断以及TLR激活“三者协同”的策略,实现对结直肠癌的协调性清除,并建立长期免疫记忆。
Resumen de: CN122208785A
本发明涉及生物医药领域,具体涉及心肌损伤治疗药物及其制备方法和应用,心肌损伤治疗药物包括一种纳米粒,纳米粒包括具有中空介孔结构的普鲁士蓝,普鲁士蓝内封装有相变造影剂和β‑烟酰胺单核苷酸;普鲁士蓝的外侧包有脂质膜,脂质膜上修饰有缺血心肌靶向肽。通过本申请,构建了一种集特异性递送、时空控释与可视化诊疗于一体的多功能纳米载体系统,实现了精准治疗。
Resumen de: CN122208775A
本发明涉及功能性纳米药物技术领域,尤其是涉及一种羟基功能化聚酰胺‑胺树状大分子共负载纤连蛋白/依达拉奉的纳米药物及其制备与应用。该纳米药物为羟基功能化树状大分子与纤连蛋白通过物理间相互作用力形成纳米复合物,并负载依达拉奉得到的纳米药物;羟基功能化树状大分子为将第五代聚酰胺‑胺树状大分子与4‑(溴甲基)苯硼酸通过酰胺键连接,并通过缩水甘油羟基化修饰树状大分子表面得到的羟基功能化树状大分子。该纳米药物能够穿透血脑屏障,精准靶向病变部位,协同调控小胶质细胞、神经细胞和血管内皮细胞,通过调节脑卒中损伤区域氧化应激和免疫激活状态,创造利于神经保护和血管再生的脑免疫微环境,实现缺血性脑卒中的高效协同治疗。
Resumen de: CN122208551A
本发明公开了一种肠上皮仿生纳米颗粒的构建方法及应用,属于纳米医学技术领域。所述肠上皮仿生纳米颗粒的制备方法为:将丁酸、丁酸盐或其衍生物负载于医用高分子材料的交联网络中;然后在其表面进一步包覆提取自肠上皮细胞的细胞膜,得到该纳米颗粒。其表面的膜受体能够吸附引起肠道屏障损伤的主要肠腔内诱发因素,同时负载的丁酸药物成分能够增强肠道屏障的功能。本发明肠上皮仿生纳米颗粒可以抑制典型消化系统肿瘤的发生、生长和转移。通过口服所述仿生纳米颗粒可通过减轻肠道屏障的破坏,有效抑制肝脏免疫微环境的紊乱,从而实现对肝细胞癌和结肠癌肝转移的强效抑制。强化肠道屏障为预防和治疗消化系统肿瘤指明了一种独特的策略。
Resumen de: CN122213245A
本发明公开了预防经典猪瘟的mRNA‑LNPs疫苗及其制备方法。本发明将猪瘟病毒E2蛋白分别与XCL1和泛素进行融合得到两种嵌合抗原,将编码两种嵌合抗原和单独E2的mRNA分别包封在脂质纳米颗粒中构建得到mRNA‑LNPs疫苗;在家兔和仔猪模型中系统评估了三种mRNA‑LNPs和E2亚单位疫苗的免疫原性及保护效果,结果显示,与未修饰的E2‑mRNA‑LNP和E2亚单位疫苗相比,经N端泛素修饰的Ub‑E2‑mRNA‑LNP和靶向树突状细胞的XCL1‑E2 mRNA‑LNP均可诱导强烈的体液和细胞免疫,且均可为感染猪瘟病毒强毒石门株的猪只提供完全保护,本发明提供的疫苗在预防经典猪瘟上具有应用前景。
Resumen de: WO2022168085A1
The present invention provides lipids and lipid nanoparticle formulations comprising these lipids, alone or in combination with other lipids. These lipid nanoparticles may be formulated with nucleic acids to facilitate their intracellular delivery both in vitro and for therapeutic applications. The present invention also provides methods of chemical synthesis of these lipids, lipid nanoparticle preparation and formulation with nucleic acids.
Resumen de: CN122213253A
本发明公开了具有软骨靶向特性的重组腺相关病毒、其包装系统、制备方法及应用,属于生物医药与基因治疗技术领域。本发明通过在rAAV衣壳的表面特定位点插入软骨组织靶向肽,使rAAV具备针对软骨的主动靶向能力。与常规rAAV 2/9相比,利用本发明制备的软骨靶向rAAV,可显著提高外源基因在软骨中的转导效率和表达水平,同时有效降低rAAV在肝脏等非靶向内脏组织中的富集程度及潜在毒副作用,为治疗骨关节炎等软骨相关疾病的基因治疗,提供了更安全、高效的AAV递送工具。
Resumen de: WO2025080565A1
The present disclosure relates to a low glycosylated spike protein and a vaccine designed to express the spike protein in vivo. The present disclosure also teaches a method for generating an immune response by utilizing the low glycosylated spike protein, which provides a broader protection across different variants. A method for identifying a glycan-shielded conserved peptide of a glycoprotein is also disclosed.
Nº publicación: KR20260089062A 15/06/2026
Solicitante:
영남대학교산학협력단
Resumen de: KR20260089062A
본 발명은 에어로졸 기반 시스템을 이용한 나노입자 제조방법 및 이에 따라 제조된 나노입자의 의학적 용도에 관한 것으로, 단일 패스(single-pass)의 에어로졸 기반 나노입자 제조 시스템을 이용하여 간단하고 단순화된 공정으로 나노입자를 제조할 수 있다. 상기에 따라 제조된 나노입자는 매트릭스 표면이 지질로 코팅되고 내부는 약물과 광감작제가 봉입된 복합체 형태로, 체내 약물 전달과 효능을 향상시켜 적은 약물 용량으로도 우수한 치료 효과를 달성하면서 약물에 따른 부작용을 감소시킬 수 있다.