Alerta

BIODEGRADABLE LIPIDS AND FORMULATIONS FOR DELIVERY OF MRNA

Absstract of: WO2025144929A2

Provided herein are compounds, such as compounds of Formulae (I), (I'), (XI), (XII), and (XIII), and pharmaceutically acceptable salts, solvates, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases). Also provided herein are methods of synthesis of compounds of Formulae (I'), (XI), (XII), (XIII), and (VIII).

肺サーファクタント生体模倣ナノ粒子に関する組成物および方法

Absstract of: WO2021071823A1

Compositions and methods comprising Pulmonary Surfactant (PS)-biomimetic nanoparticles are disclosed. Specifically, the disclosure is related to a composition comprising a nanoparticle with an average size of 200-400 nm, including a plurality of pulmonary surfactant biomimetic molecules, wherein the nanoparticle is negatively charged; and one or more cargo molecules that are enveloped by the nanoparticle, wherein the cargo molecule has a molecular weight up to 1200 Da.

治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法

Absstract of: WO2019204666A1

Provided nucleic acid- based expression construct for the target cell-specific production of a therapeutic protein, such as a pro-apoptotic protein, within a target cell, including a target cell that is associated with aging, disease, or other condition, in particular a target cell that is a senescent cell or a cancer cell. Also provided are formulations and systems, including fusogenic lipid nanoparticle (LNP) formulations and systems, for the delivery of nucleic acid-based expression constructs as well as methods for making and using such nucleic acid-based expression constructs, formulations, and systems for reducing, preventing, and/or eliminating the growth and/or survival of a cell, such as a senescent cell and/or a cancer cell, which is associated with aging, disease, or other condition as well as methods for the treatment of aging, disease, or other conditions by the in vivo administration of a formulation, such as a fusogenic LPN formulation, comprising an expression construct for the target cell-specific, production of a therapeutic protein, such as a pro-apoptotic protein, in a target cell that is associated with aging, disease, or other condition, in particular a target cell that is a senescent cell or a cancer cell.

TREATMENT OF PRIMARY CILIARY DYSKINESIA WITH SYNTHETIC MESSENGER RNA

Absstract of: EP4834921A2

Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.

白斑を治療するためのチロシナーゼに基づく経皮送達システム

Absstract of: CN119837821A

The invention provides a tyrosinase-based transdermal delivery system for treating leucoderma, which is of a core-shell structure, the core is tyrosinase, and the shell is a polymer shell layer. According to the invention, double bonds are formed on tyrosinase, and the surface of protein is coated with a polymer protection layer through an in-situ polymerization reaction, so that the activity of the tyrosinase can be effectively protected, and the transdermal delivery of the tyrosinase can be promoted; and the tyrosinase nanogel can be controlled to be delivered to melanocytes deep in skin and hair follicles in a targeted manner by regulating and controlling polymerization conditions of a polymer shell, so that in-situ synthesis of melanin is promoted. According to the transdermal delivery system, active small molecules with an anti-inflammatory function can be loaded at the center of the tyrosinase, and the small molecules are delivered to an inflammatory area of vitiligo in a targeted manner along with the tyrosinase nanogel and then are released, so that inflammation is relieved, and melanocytes are protected from being damaged.

USE OF IRON-LOADED CARBON NANOPARTICLE SUSPENSION INJECTION FOR COMBINED ADMINISTRATION

Absstract of: EP4834368A1

A carbon nanoparticle-Fe suspension injection for use in a method for inhibiting tumor growth, wherein the method comprises combined administration of the carbon nanoparticle-Fe suspension injection and an anticancer drug, characterised in that the anticancer drug is administered orally or intravenously, and the carbon nanoparticle-Fe suspension injection is administered through intratumoral injection. After the carbon nanoparticle-Fe suspension injection is injected into a tumor, the carbon nanoparticle-Fe suspension injection has the effect of enriching the anticancer drugs orally or intravenously administered into the tumor, improving concentrations of the anticancer drugs in the tumor, such that the anticancer drugs are enabled to act more accurately on a tumor site, doses and toxic and side effects on normal cells and tissues are relatively reduced.

シールドされていない脂質ナノ粒子を用いたＤＮＡまたはＲＮＡカーゴの送達方法

Absstract of: US20250114306A1

The present disclosure provides unshielded lipid nanoparticles and a process that enables the production of such unshielded lipid nanoparticles, thereby overcoming previous challenges of making particles without causing aggregation thereof. The lipid nanoparticles comprise a nucleic acid cargo molecule; a sterol or a derivative thereof present at a content of at least 12 mol %; a neutral lipid, such as a phospholipid having a choline head group present at a content of between 22 mol % and 65 mol %; and an ionizable cationic amino lipid present at a content of between 15 mol % and 45 mol %; wherein the lipid nanoparticle is non-sterically stabilized with a hydrophilic polymer-lipid conjugate, or otherwise unshielded and wherein each mol % content is relative to total lipid present in the lipid nanoparticle.

用于刺激T细胞的脂质纳米颗粒

Absstract of: WO2025210521A1

The present disclosure provides lipid nanoparticles that are particularly beneficial in inducing an immune response in a subject, particularly by stimulating CD8+ T cells associated with protein or protein fragments of interest. The lipid nanoparticles can have a negative zeta potential and/or particular configurations and/or relative amounts of a phospholipid, an ionizable lipid, and a conjugated lipid. The disclosure also provides vaccines, other pharmaceutical compositions, and methods including the provided lipid nanoparticles.

一种嘉义乳酪杆菌胞外多糖硒纳米粒及其制备方法与应用

Absstract of: CN123024093A

本发明涉及纳米生物医药技术领域，公开了一种嘉义乳酪杆菌胞外多糖硒纳米粒及其制备方法与应用，该纳米粒包括嘉义乳酪杆菌胞外多糖和分散于其中的硒纳米粒，且所述硒为无定形态的零价硒。其制备方法为将嘉义乳酪杆菌胞外多糖溶液与亚硒酸钠溶液混合后，加入抗坏血酸溶液作为还原剂，在避光加热条件下进行还原反应分离制得。本发明的胞外多糖作为基质有效抑制了纳米硒的聚集与结晶，不仅显著提升了纳米粒的结构稳定性与缓释性能，协同增强了巨噬细胞的吞噬能力，还能通过调控促炎与抗炎因子的分泌有效干预巨噬细胞极化方向，在制备免疫调节及抗炎药物中具有重要应用价值。

基于外泌体的配体递送系统及其应用

Absstract of: CN123026697A

本发明公开了一种基于外泌体的配体递送系统及其应用。该系统包含一条人工设计的多肽表达元件，从N端到C端依次为：N端融合肽、第一连接肽、改造的CD63、第二连接肽、C端融合肽。所述改造的CD63由天然CD63在其N端和C端分别延展出一个胞内区域、一个跨膜区域和一个胞外区域形成，使改造后的CD63成为6次跨膜蛋白，且其N端和C端均位于外泌体的膜外侧。本发明还提供了编码该系统的核酸、重组载体、工程化细胞株、工程化外泌体及其制备方法与应用。该系统可在细胞分泌外泌体的过程中同时完成靶向肽与功能配体在膜外侧的装载，实现了靶向识别与配体递送的一体化，在靶向毛囊干细胞并激活Wnt信号通路等方面具有重要应用价值。

用于斑马鱼胚胎的脂质纳米颗粒、mRNA递送复合纳米试剂及其制备方法

Absstract of: CN123024051A

本发明公开了一种用于斑马鱼胚胎的脂质纳米颗粒，包括以下摩尔比例的组分：4‑(N,N‑二甲基氨基)丁酸(二亚油基)甲酯：35~45mol%，1,2‑二油酰‑sn‑甘油‑3‑磷酰乙醇胺：10~20mol%，胆固醇：28~38mol%，1,2‑二油酰基‑3‑三甲基氯化铵丙烷：5~15mol%，穿膜肽TAT‑聚乙二醇‑磷脂：0.5~4.0mol%。该脂质纳米颗粒能够稳定包封核酸并通过非侵入式有效递送核酸进入胚胎。本发明还公开了包括上述脂质纳米颗粒的mRNA递送复合纳米试剂及其制备方法。

用于治疗ADSL基因缺陷相关疾病的外泌体及其制备方法和应用

Absstract of: CN123027182A

本发明提供了用于治疗ADSL基因缺陷相关疾病的外泌体及其制备方法和应用。本发明的外泌体通过如下方法获得：使用含有ADSL的慢病毒颗粒感染间充质干细胞，并从间充质干细胞中分离纯化得到了负载ADSL的外泌体。所获得的外泌体能够有效治疗ADSL基因缺陷相关疾病，克服了大分子无法穿透血脑屏障的缺点。本发明提供了首个针对ADSL基因缺陷相关疾病的非病毒基因治疗策略，填补了相关领域的空白。

用于脂质纳米颗粒中的阳离子脂质化合物

Absstract of: WO2025174858A1

The present disclosure provides cationic lipid compounds having the following Structure (I): where G1, G2, R1, R2, R3a, R3b, R3c, R3d, R4a, R4b, R4c, R4d, L1, L2, and L3 are as defined herein. The present disclosure also discloses pharmaceutically acceptable salts or stereoisomers thereof. Also provided by the present disclosure are uses of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

一种乏氧-活性氧双响应白蛋白纳米递送系统的制备及其在制备结直肠癌药物中的应用

Absstract of: CN123024175A

本发明属于生物医药技术领域，具体涉及一种乏氧‑活性氧双响应白蛋白纳米递送系统的制备及其在制备结直肠癌药物中的应用。所述系统以MC4A修饰的BSA介导肿瘤细胞高效摄取，BSA‑C4A依托MC4A的主客体作用包载前药，在常氧环境中稳定，富集于肿瘤乏氧微环境时，MC4A内部的偶氮键被还原酶裂解，释放前药，并靶向到线粒体。前药以硫缩酮为活性氧敏感连接子，将光敏剂尼罗蓝衍生物与化疗药物喜树碱偶联（NB‑TK‑CPT）。在特定波长激光照射下，凭借NB特有的I型光动力效应，通过非氧依赖的电子转移途径在乏氧区域产生杀伤性活性氧，同时促使TK连接子断裂实现CPT定向释放，放大光动力治疗与化疗协同抗肿瘤效应。

一种基于抗氧化疏水骨架的可电离脂质、脂质纳米颗粒及其在核酸递送中的应用

Absstract of: CN123026645A

本发明公开了一种基于抗氧化疏水骨架的可电离脂质、脂质纳米颗粒及其在核酸递送中的应用，可电离脂质分子结构由具有对称的抗氧化疏水尾部、环境敏感型连接臂和多功能可电离氨基头部三部分组成。本发明提供的可电离脂质能够在胞内原位清除ROS，重塑胞内氧化微环境，缓解细胞氧化应激，显著提升核酸的翻译效率；由其构建的核酸递送载体兼具高核酸包封率、优良的靶细胞摄取能力与突出的体内蛋白表达水平。

天麻素鼻腔-脑靶向双通道协同递送制剂及其制备方法

Absstract of: CN123024035A

本发明公开了一种天麻素鼻腔‑脑靶向双通道协同递送制剂及其制备方法，属于药物制剂领域。针对现有天麻素鼻腔给药仅依赖单一嗅觉通路、脑内覆盖有限、滞留时间短等问题，本发明制剂包含壳聚糖修饰PLGA纳米粒（80‑120nm）、TAT肽修饰脂质纳米粒（200‑300nm）和泊洛沙姆温敏凝胶。两种纳米粒分别靶向鼻腔嗅区和呼吸区，经嗅觉通路和三叉神经通路协同入脑，温敏凝胶延长鼻腔滞留。实验表明，双通道制剂脑干天麻苷元AUC为单嗅区制剂的2.49倍，未修饰TAT肽时脑干AUC仅为其31.6%，鼻腔滞留半衰期为普通溶液的8.3倍。本发明用于制备治疗神经衰弱、头痛、偏头痛、失眠症等药物。

一种咪唑降解型可电离脂质及其制备方法与应用

Absstract of: CN123026559A

本发明公开了一种咪唑降解型可电离脂质及其制备方法与应用，属于生物医药技术领域，可电离脂质以咪唑基团为亲水头部，以含有酯键和/或二硫键的2‑4条支化链为疏水尾部，通过迈克尔加成反应合成。将该脂质与辅助脂质组装成脂质纳米颗粒后，可用于体内外高效递送mRNA、saRNA和circRNA等多种核酸药物。本发明采用上述的一种咪唑降解型可电离脂质及其制备方法与应用，通过采用2‑4条支化疏水尾部并引入酯键或二硫键等多重可降解结构，显著提升了内涵体逃逸效率和体内生物安全性，同时实现了对免疫细胞的高效转染以及对mRNA、saRNA、circRNA等多种RNA的普适性递送。

一种靶向复合纳米载药颗粒的制备方法及其应用

Absstract of: CN123024052A

本发明涉及医药技术领域，具体公开了一种靶向复合纳米载药颗粒的制备方法及其应用，该靶向复合纳米载药颗粒由淀粉与经过透明质酸修饰的PEG ‑ b ‑ PMAA胶束复配形成。由于CD44受体在炎症巨噬细胞或滑膜细胞中高表达，通过引入透明质酸对CD44受体进行特异性识别，解决了纳米载药颗粒被动靶向、富集效率低的问题。通过淀粉对聚合物胶束进行物理复配，实现高载药量和高稳定性。本发明的胶束稳定性好、生物利用度高、缓释性能优良，能提高防治痛风性关节炎的效率，并降低药物系统毒性，在制备抗痛风性关节炎的药物中具有广阔应用前景。

一种靶向MMP1和MMP3的皮肤抗衰老siRNA组合及其应用

Absstract of: CN123027226A

本发明属于核酸药物、皮肤科学、功能性化妆品与医美制剂技术领域，具体而言，涉及一种靶向MMP1和MMP3的皮肤抗衰老siRNA组合及其应用。本发明提供了一种靶向MMP1和MMP3的皮肤抗衰老siRNA组合，所述siRNA组合包括：靶向人MMP1 mRNA的第一siRNA和靶向人MMP3 mRNA的第二siRNA。本发明的siRNA组合及其组合物既可以用于功能性化妆品实现日常抗衰老护理，也可应用于医美术后修复或作为皮肤外用药物用于治疗光线性皮肤病，具有极大的商业价值和市场潜力。

用于跨越血睾屏障靶向家禽睾丸组织的组合物及其制备方法和应用

Absstract of: CN123024053A

本申请公开一种用于跨越血睾屏障靶向家禽睾丸组织的组合物及其制备方法和应用。本申请的组合物包括平均粒径为10‑1000 nm的负电性纳米颗粒，其包含外壳和包裹于外壳或其内部的活性成分，外壳包含质量比为3:1‑6:1的阴离子脂质和胆固醇。本发明通过负电性脂质体包覆纳米硒，促进了纳米硒跨越血睾屏障靶向富集于睾丸组织，改善了种公鸡产精性能，延长了种用寿命。

カチオン性脂質及びその製造方法

Absstract of: WO2025084582A1

The present invention relates to a cationic lipid and a method for preparing same, and more specifically to: a cationic lipid which, due to the specific structure thereof, forms (or is capable of forming) a complex with an anionic drug, and thus is useful for drug delivery; and a method for preparing same.

适用于脂质纳米颗粒的可电离脂质

Absstract of: WO2025133951A1

The present disclosure provides nitrogen-containing silicon ether ionizable lipid compounds and lipid nanoparticles including the ionizable lipid. The disclosure further relates to lipid nanoparticles including a provided ionizable lipid compound together with a phospholipid, e.g., a phospholipid that includes at least one unsaturated tail and a head group having a positively charged nitrogen. The provided materials are particularly beneficial in applications involving delivery of a nucleic acid. The disclosure also provides pharmaceutical compositions and methods including the provided ionizable lipids and/or lipid nanoparticles.

一种仿生载多粘菌素B纳米粒及其制备方法和应用

Absstract of: CN122998097A

本发明涉及生物医药技术领域，具体为一种仿生载多粘菌素B纳米粒及其制备方法和应用，仿生载多粘菌素B纳米粒包括聚合物纳米核心和包覆于所述聚合物纳米核心表面的巨噬细胞膜仿生层；所述聚合物纳米核心载有多粘菌素B和柠康酸酐修饰聚赖氨酸；所述巨噬细胞膜仿生层表面负载抗生物膜环肽。该纳米粒可用于制备治疗铜绿假单胞菌肺部感染的药物，并可降低游离多粘菌素B的细胞毒性，兼顾抗菌效果与用药安全性，更好的满足实际应用需求。

用于递送治疗剂的脂质化合物及含其的组合物

Absstract of: WO2026092704A1

Disclosed in the present invention are a lipid compound for delivering a therapeutic agent and a composition containing same. The present invention provides a lipid compound represented by formula I or a pharmaceutically acceptable salt thereof. The lipid compound provided by the present invention, as a drug carrier, exhibits high encapsulation efficiency, high expression level, and good targeting property.

一种基于TIPE2表达调控的抗炎和抗新生血管双靶点纳米颗粒及其制备方法和应用

Nº publicación: CN122998100A 30/10/2026

Applicant:

山东大学齐鲁第二医院

Absstract of: CN120837687A

The invention discloses a preparation method of a nano gene vector based on PBAE-TAT-AT7, and mainly relates to the field of biological medicines. Comprising the following steps: S1, synthesizing PBAE; s2, washing the PBAE; s3, adding the TAT-AT7 peptide and the PBAE into the dimethyl sulfoxide, and synthesizing the PBAE-TAT-AT7 peptide, so as to obtain the PBAE-TAT-AT7 peptide; s4, carrying out freeze drying on the PBAE-TAT-AT7 synthesized in the step S3; and S5, carrying out vortex mixing on the PBAE-TAT-AT7 prepared in the step S4 and the pDNA, so as to obtain the PBAE-TAT-AT7/pDNA nano gene compound. The PBAE-TAT-AT7/pDNA nano-gene compound has the beneficial effects that the PBAE-TAT-AT7 is connected with the TAT-AT7 peptide to enhance the cell penetrating power of the gene vector, and the pDNA is wrapped by electrostatic adsorption, so that the PBAE-TAT-AT7/pDNA nano-gene compound not only can be used for in-vitro cell transfection, but also can be used for realizing intraocular gene therapy through intravitreal injection, and has the advantages of low toxicity and high efficiency.