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AN ANALYTICAL METHOD USING LC-MS/MS PROTEOMICS TO CHARACTERIZE PROTEINS TRANSLATED FROM MRNA

Resumen de: WO2024072929A1

The present disclosure provides a method of assessing translation efficacy of an mRNA using liquid chromatography-tandem mass spectrometry (LC-MS/MS). The mRNA is first translated into protein either in a cell lysate (cell-free translation; CFT) or inside a cell (cell-based translation; CBT) and analyzed using LC-MS/MS. The method provides advantages such as speed and convenience over traditional immunoassay-based methods of detecting translated proteins. Translation using CBT may be necessary in certain formulations of the mRNA, such as when the mRNA or a mixture of mRNAs is encapsulated inside a lipid nanoparticle.

NANOCOMPOSITES FOR ENHANCED CELLULAR PAYLOAD DELIVERY

Resumen de: WO2024077034A2

Generally, the present disclosure is directed to compositions and methods of using the same. In some embodiments, a composition described herein comprises a nanoparticle, a plurality of nanofibers disposed on an exterior surface of the nanoparticle, and a payload disposed within an interior of the nanoparticle. The nanoparticle has an average size in three dimensions, and the plurality of nanofibers has an average length in a long dimension. In some cases, a ratio of the average size of the nanoparticle to the average length of the nanofibers is between 2 and 250.

IONIZABLE LIPID, PREPARATION METHOD THEREOF, AND COMPOSITION FOR PREPARING LIPID NANOPARTICLE

Resumen de: EP4596531A1

An ionizable lipid having a structure represented by the following formula (I):wherein Y is independently selected from a group consisting of -NH-, -O-, -S-, and a single bond; X is independently selected from -NR¹R² or a nitrogen-containing heteroaryl group; L¹ and L² are each independently selected from a group consisting of a C₁-C₁₀ alkylene group, a C₂-C₁₀ alkenylene group,; R¹ and R² are each independently selected from a group consisting of H, a substituted or unsubstituted C₁-C₁₀ hydrocarbyl group, a substituted or unsubstituted C₁-C₁₀ heterohydrocarbyl group, a substituted or unsubstituted C₆-C₂₀ aryl group, and a substituted or unsubstituted C₁-C₂₀ heteroaryl group; R³ is a C₅-C₃₀ alkyl group; R⁴ is a C₅-C₃₀ alkyl group; n is an integer selected from 1 to 10; and m and p are each independently an integer selected from 1 to 20.

一种治疗银屑病及复发性银屑病的新型mRNA核酸药物

Resumen de: CN120420339A

本发明属于生物医药领域，具体涉及一种治疗银屑病及复发性银屑病的新型mRNA核酸药物。本发明提供一种治疗银屑病及银屑病相关疾病的mRNA，所述mRNA核苷酸序列如SEQ ID NO.1或SEQ ID NO.2所示。本发明还提供包括前述的mRNA的核酸药物，所述药物采用脂质体纳米颗粒包裹mRNA。本发明还提供前述的mRNA或前述的核酸药物在制备预防和/或治疗银屑病及银屑病相关疾病的药物中的应用，所述银屑病相关疾病包括银屑病关节炎、儿童性银屑病、复发性银屑病。以及，本发明提供前述的mRNA或前述的核酸药物在制备抗炎症药物中的应用。本发明的mRNA药物能够起到治疗银屑病的效果，实现银屑病症状缓解；控制银屑病复发，克服目前银屑病生物制剂对耐药性以及停药无法的难题。

用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物

Resumen de: MX2022003269A

The application relates to lipids of Formula (A), Formula (B) and Formula (1-1), and to lipid nanoparticles (empty or loaded LNPs) including such a lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

一种新型脂质化合物以及组合物和核酸脂质纳米颗粒制剂

Resumen de: CN120423968A

本申请提供阳离子脂质化合物、包含其的组合物及应用。为了给基因药物、核酸疫苗、小分子药物等制剂的递送提供更多的选择，本申请提出通式#imgabs0#所示的阳离子脂质化合物，或其药物可用的盐、前药或立体异构体。本申请的阳离子脂质化合物可用于递送核酸药物或小分子药物，丰富了阳离子脂质化合物种类，对核酸预防剂及治疗剂的发展和应用具有重要的意义。

一种用于核酸递送的新型可电离脂质及其LNP组合物和疫苗

Resumen de: WO2023143601A1

Provided in the present invention are a novel cationic lipid, a lipid nanoparticle and a nucleic acid vaccine. In the present invention, a specific cationic lipid is selected for preparing a lipid nanoparticle mRNA vaccine, which is found to have better in-vitro stability and can stimulate a stronger immunoreaction compared with LNPs prepared from cationic lipids in the prior art.

一种新型两亲性胆红素纳米颗粒及其制备方法

Resumen de: CN120420298A

本发明涉及纳米技术领域，提供了一种新型两亲性胆红素纳米颗粒及其制备方法，所述纳米颗粒由胆红素和NH2‑PEG‑b‑PCL构成，亲水性PEG链位于表面提高水溶性并防止免疫清除，疏水性PCL核心包裹胆红素形成稳定结构。制备方法包括：将NH2‑PEG‑b‑PCL溶于二甲亚砜搅拌溶解；加入胆红素搅拌过夜；将混合液加入超纯水中搅拌形成均一液体；置于透析袋中避光透析去除溶剂。本发明解决了胆红素水溶性差和光稳定性低的问题，具有优异生物相容性。

一种诊疗一体化靶向超声爆破纳米泡及其制备方法和应用

Resumen de: CN118873695A

The invention relates to the technical field of biomedical nano materials, in particular to a diagnosis and treatment integrated targeted ultrasonic blasting nano bubble as well as a preparation method and application thereof. The nanobubble is a targeted drug-gene nanobubble miRNA/FTY720/PFP-coated PEI-T7NBs, and can be used for preparing a thyroid ultrasound diagnosis medicament or a thyroid cancer treatment medicament.

用于治疗癌症的IL-12基因疗法和抗VEGF联合

Resumen de: WO2024054855A1

The present disclosure relates to a combination therapy comprising an anti-VEGF antibody, a nanoparticle formulated plasmid comprising an IL-12 coding nucleic acid, and, optionally, at least one adjunctive chemotherapeutic drug, and methods of treatment using such combination therapies and/or compositions.

一种甘草次酸白蛋白纳米粒、制备方法及应用

Resumen de: CN120420299A

本发明公开了一种甘草次酸白蛋白纳米粒，所述纳米粒呈均一的球形；所述纳米粒包括甘草次酸、白蛋白；所述纳米粒粒径为≦200nm，包封率90％～95.10％，载药量为30.56％～63.40％，所述纳米粒24小时内释放的甘草次酸为60％～70％；本发明还公开了一种甘草次酸白蛋白纳米粒的制备方法，该方法得到的纳米粒粒径小且分布均一，其稳定性和安全性随白蛋白占比增加而增加；其中，富含白蛋白的纳米粒在预防和治疗急性肝损伤实验中展现双模式药理活性。

一种活性氧激活型近红外区光热试剂及其制备方法和应用

Resumen de: CN120424104A

本发明公开了一种活性氧激活型近红外区光热试剂及其制备方法和应用，通过将酚羟基功能化的氮杂氟硼荧类染料与对溴苯硼酸频哪醇酯通过成醚反应，构建出具有活性氧响应的近红外区光热试剂，并进一步将其制备成纳米粒子，该纳米粒子与活性氧反应后，在808nm激光光照下，其光热效率显著提升，具有优异的光热成像效果和光热治疗功能。

包含新的纳米粒支架的疫苗

Resumen de: AU2023399881A1

The present invention provides novel engineered nanoparticle scaffold sequences that are derived from the 13-01 protein. Relative to the known 13-01 protein or variants thereof, the novel 13-01 derived scaffold sequences of the invention contain an extended N-terminal helix. Also provided in the invention are vaccine constructs that contain various immunogenic proteins displayed on the novel nanoparticle scaffold sequences described herein. The vaccine constructs of the invention include, e.g., nanoparticles displaying tandem repeats of influenza M2e proteins or HCV E2 core proteins.

一种靶向响应性纳米材料的制备方法、产品及应用

Resumen de: NL2040524A

The present invention relates to the field of nanomaterial technology, and more particularly, to a preparation method, product, and application of a targeted responsive nanomaterial. The preparation method comprises the following steps: Step 1: Dissolve a surfactant in a buffer solution, then add perfluoro-lS-crown-S-ether, an anticancer drug, and an oxidant, followed by vortexing and sonication to obtain a suspension; Step 2: Add a dopamine hydrochloride solution to the suspension and sonicate to obtain solution A; Step 3: Stir solution A and then centrifuge it; remove the supernatant and perform ultrafiltration to obtain a concentrate; Step 4: Mix the concentrate With cRGD- PEGSK-NHS, stir, and ultrafilter to concentrate, thereby obtaining the targeted responsive nanomaterial. The preparation method of the present invention is simple. The nanomaterials prepared using the method of the invention are not only expected to become a safer and more effective approach to tumor treatment but also lay a solid foundation for the future development of similar multifunctional composite nanopharmaceutical products.

一种高危亚型HPV肿瘤疫苗及其应用

Resumen de: CN120420424A

本专利提供一种高危亚型HPV肿瘤疫苗及其应用，其包括纳米脂质颗粒和表达HPV病毒抗原的RNA，表达HPV病毒抗原的RNA包括编码HPV病毒肽表位的RNA，HPV病毒肽表位包括蛋白E6、E7重组蛋白，所述纳米脂质颗粒包裹RNA。该疫苗的优势在于自主研发的纳米脂质颗粒可以同时包裹多个RNA，有效表达多种高危型HPV的E6、E7抗原。RNA分子设计上通过E6、E7串联的形式进行融合表达，并通过密码子优化实现核酸序列的高效翻译。该疫苗能够有效激活体内肿瘤特异性免疫反应的同时保证安全性，在临床应用中兼顾多种高危型HPV相关癌症的治疗。

一种具有缩阴紧润功能的女性生殖护理制剂及其制备方法

Resumen de: CN120420415A

本发明公开了一种具有缩阴紧润功能的女性生殖护理制剂及其制备方法，涉及女性生殖护理技术领域。本发明通过制备金缕梅提取物纳米颗粒，其中的活性成分单宁酸能促进阴道中弹性蛋白的合成，减少经皮水分流失，增强皮肤紧实度，达到缩阴紧润的效果；本发明通过京尼平、马齿苋提取物与重组人源Ⅲ型胶原蛋白三者之间的相互交联作用制备了改性重组人源Ⅲ型胶原蛋白，重组人源Ⅲ型胶原蛋白可以激活人成纤维细胞增殖和分化，增加真皮层胶原紧密度；还有大量亲水基团，具有锁水保湿的能力，使皮肤保持充盈状态；马齿苋提取物中含有多糖，能与重组人源Ⅲ型胶原蛋白协同作用，修复断裂的胶原纤维网络，改善阴道松弛和干涩的问题。

神経障害性疼痛を治療するためのナノ粒子の組成物

Resumen de: MX2025001088A

The invention concerns a novel and innovative composition for the treatment of neuropathic pain (NP). Specifically, the invention concerns HfO₂ nanoparticles and/or aggregates of HfO₂ nanoparticles, a composition comprising said nanoparticles and/or aggregates of nanoparticles, and their use in the treatment of NP.

脂质化合物、其脂质纳米颗粒、其组合物及其制备方法与用途

Resumen de: CN120398793A

本公开属于生物医药技术领域，具体涉及脂质化合物、其脂质纳米颗粒、其组合物及其制备方法与用途。本公开具有如下优点：本公开的脂质化合物制备的脂质纳米颗粒对核酸具有良好的体内递送效率。

一种基于中空介孔氮化碳的纳米探针及其制备方法和应用

Resumen de: CN120392701A

本发明公开了一种基于中空介孔氮化碳的纳米探针及其制备方法和应用，属于医药技术领域。制备方法包括如下步骤：S1、利用硬模板法合成中空介孔氮化碳HCNS；S2、HCNS与KSCN进行水热反应合成改性中空介孔氮化碳HCNxS；S3、将HCNxS分散于DOX/PBS溶液中，DOX充分负载于HCNxS上得到DOX@HCNxS，利用活化的HA封闭HCNxS的孔道，得到DOX@HCNxS‑HA。DOX@HCNxS‑HA的中空结构赋予了其DOX 19.05％的负载率，表面两性特性与HA修饰实现CD44受体介导的肿瘤靶向递送，并在酸性微环境中触发pH响应药物释放；DOX@HCNxS‑HA可通过ROS介导来破坏溶酶体膜与质子海绵效应，显著降低溶酶体共定位率(皮尔逊相关系数从0.73降至0.18)，促进DOX在肿瘤细胞内富集，使MDA‑MB‑231细胞存活率降至13.8％，具备对乳腺癌细胞的杀伤作用。

4-氨基哌啶化合物、其脂质纳米粒子和含有其的药物组合物

Resumen de: AU2023419278A1

The present invention addresses the problem of providing: a compound that is a 4-aminopiperidine lipid; lipid nanoparticles containing the lipid; and a pharmaceutical composition useful for nucleic acid medicines and the like.　The present inventors have discovered a compound that is a 4-aminopiperidine lipid or a salt thereof, examined lipid nanoparticles that have the potential of being formed into various pharmaceutical compositions, and revealed that the lipid that is the compound according to the present invention or a salt thereof can form lipid nanoparticles, and moreover, lipid nanoparticles in which nucleic acid is encapsulated (i.e., nucleic acid lipid nanoparticles). In addition, the nucleic acid lipid nanoparticles containing the lipid according to the present invention are expected to serve as a component of pharmaceutical compositions useful for prevention and/or treatment of astrocyte-related diseases.

加速糖尿病创面愈合的聚集诱导发光金属有机框架纳米粒子、微针贴片及制备方法与应用

Resumen de: CN120398858A

本发明公开了加速糖尿病创面愈合的聚集诱导发光金属有机框架纳米粒子、微针贴片及制备方法与应用，纳米粒子包括由聚集诱导发光化合物与金属锌离子构成的聚集诱导发光金属有机框架，装载在所述聚集诱导发光金属有机框架内部的一氧化碳释放前体、AIE光敏剂TTI‑COOH，包裹在所述聚集诱导发光金属有机框架外部的两亲性聚合物；微针贴片包括基底层、针体层，本发明优点包括：具有良好生物相容性、抗菌、抗炎、抗氧化、促细胞迁移和促血管生成的多种生物学功能特点，具有良好的吸湿性能和力学性能。

用于体内生成CAR-T细胞的脂质纳米粒及其构建方法和应用

Resumen de: CN120392694A

本发明属于生物工程技术领域，具体公开用于体内生成CAR‑T细胞的脂质纳米粒及其构建方法和应用。本公开一方面涉及脂质纳米粒LNP的制备方法及应用，另一方面涉及基于脂质纳米粒LNP制备Ab‑LNP的方法及Ab‑LNP的应用。本公开中，脂质纳米粒经微流控混合技术分别包封编码mRNA分子，随后经点击化学技术在获得的LNP表面修饰靶向T细胞的抗体，制得工程化mRNA‑LNP体内靶向递送载体，可以有效治疗多发性骨髓瘤或B淋巴白血病。

用于软骨再生的肽及其用途

Resumen de: WO2024122719A1

The present application relates to a peptide having cartilage regeneration effects, and uses thereof, and provides: a peptide comprising an amino acid sequence represented by SEQ ID NO: 1 or Arg(R)-Thr(T)-Leu(L)-Glu(E); a composition for cartilage regeneration comprising the peptide; and a pharmaceutical composition for preventing or treating cartilage diseases, comprising the composition for cartilage regeneration as an active ingredient.

用于延长血液和淋巴组织中的药物水平的多药脂质纳米颗粒组合物和相关方法

Resumen de: US2019350853A1

Provided are multi-drug lipid nanoparticles that stably incorporate multiple small molecule drugs with divergent hydrophobic and water solubility characteristics and related methods of making and using the same, The disclosed compositions and methods provide for enhanced stability of lipid nanoparticle drug formulations that can reliably provide drugs addressing different mechanistic targets with prolonged presence in the body for more efficacious treatment and avoidance of single drug resistance.

一种包载亲水性膜脂质纳米粒的眼用缓释凝胶递药体系及其制备方法

Nº publicación: CN120392642A 01/08/2025

Solicitante:

兰州大学

Resumen de: CN120392642A

本发明公开了一种包载亲水性膜脂质纳米粒的眼用缓释凝胶递药体系及其制备方法，眼用缓释凝胶递药体系包括亲水性膜脂质纳米粒和眼用凝胶，亲水性膜脂质纳米粒的原料包括卵磷脂、胆固醇、维生素E聚乙二醇琥珀酸酯、二硬酯酰乙醇胺‑聚乙二醇2000、羟丙基‑β‑环糊精和难溶于水的药物，眼用凝胶的原料包括泊洛沙姆407、泊洛沙姆188和透明质酸钠。本发明制备的凝胶在眼部环境下由于温度和渗透压双重作用形成膜剂，使得其在眼部实现药物的长效释放，提高了药物生物利用度。