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POLYMERIC MICELLE COMPRISING SINGLE POLYMER CHAIN OF SELF-AGGREGATING TYPE

Resumen de: EP4755372A1

The present invention addresses the problem that many conventional nano-sized contrast agents are aggregates of a plurality of constituent molecules and, under diluted conditions resulting from prolonged blood circulation, dissociate by reaching a critical micelle concentration (CMC) or critical aggregate concentration (CAC) or lower, such that it is difficult to control in vivo kinetics thereof up to the discharge phase. The present invention provides a polymeric micelle comprising a single polymer chain of a self-aggregating type, said single polymer chain containing one or more gadolinium complexes and having a particle size of not more than 10 nm in water.

EXCIPIENT GRANULATIONS

Resumen de: MX2026001366A

Excipient granulations containing a viscosifying agent, a disintegrant, and one or more additional excipients are disclosed. An excipient granulation can be combined with a pharmaceutical granulation to provide a pharmaceutical composition. The excipient granulations can be used to increase the viscosity of an aqueous composition such as an oral pharmaceutical composition. When added to an aqueous solution, the excipient granulation can dissolve to provide a suspension of the pharmaceutical granules in a viscous solution. The excipient granulation can be used to improve the palatability of oral pharmaceutical compositions containing a pharmaceutical granulation.

LIPID-LIKE COPOLYMERS AND METHOD OF PRODUCING THESE AND THE USE THEREOF, PHARMACEUTICAL COMPOSITIONS COMPRISING SAID COPOLYMERS, AND THE USE OF SAID COMPOSITIONS

Resumen de: NL2035538B1

0001 The present invention relates to a lipid-like copolymer comprising hydrophilic and hydrophobic units. The present invention further relates to a pharmaceutical composition comprising the lipid-like copolymer and a therapeutic oligonucleotide. The present invention furthermore relates to the use of the pharmaceutical composition as a medicament, in a method of treating or preventing a disease, in a method of treating, preventing or ameliorating cancer, and in a method for inducing an immune response. The present invention moreover relates to producing the pharmaceutical composition. The present invention also relates to the use of the lipid-like copolymer for encapsulation of therapeutic oligonucleotides.

NUCLEIC ACID ENCODED RUNX3 TRANSCRIPTION FACTOR

Resumen de: WO2025027060A1

The present invention is inter alia directed to artificial nucleic acid constructs, preferably RNA, comprising at least one coding sequence encoding a RUNX3 transcription factor or a fragment or variant thereof, wherein the nucleic acid preferably comprises at least one heterologous untranslated region (UTR). Further provided are pharmaceutical compositions comprising the nucleic acid, preferably formulated in polyethylene glycol/peptide polymers, polymeric carriers, or lipid-based carriers. Also provided are methods of treating or preventing disorders, diseases, or conditions, and medical uses, in particular ocular disorders, diseases, or conditions such as proliferative vitreoretinopathy (PVR) or epiretinal membranes (EPR), using the RUNX3 encoding nucleic acids described herein.

AGENTS AND METHODS FOR TARGETED DELIVERY TO CELLS

Resumen de: WO2025026553A1

The invention relates to agents and methods for targeted delivery of nucleic acids to cells. In some embodiments, the invention involves a particle, said particle comprising a connector compound comprising (i) a moiety incorporating the connector compound into the particle through a charge in the moiety incorporating the connector compound into the particle interacting with an opposite charge in the particle, and (ii) a first interacting moiety, and said particle carrying a nucleic acid payload. A docking compound comprising (i) a second interacting moiety, and (ii) a moiety binding to a cell surface antigen interacts with the connector compound through the first interacting moiety and the second interacting moiety binding to each other, and thus targets the complex to a target cell of interest.

LYOPHILIZED FORMULATIONS AND LIQUID FORMULATIONS OF LIPID NANOPARTICLES

Resumen de: WO2025026304A1

Provided are lyophilized formulations of lipid nanoparticles comprising a cationic lipid and a cryoprotectant combination, which contains sucrose and a non-polar amino acid.

DIALYSATE COMPOSITION COMPRISING A SPECIFIC COMPOUND

Resumen de: EP4501332A1

The present invention relates to a dialysate composition having a composition having a composition allowing for a balance with blood comprising mineral salts and a pH buffer, and comprising at least one iron oxide nanoparticle (Fe²⁺ and/or Fe³⁺) coated with at least one polymeric compound.

AMINO LIPIDS FOR LIPID-BASED DELIVERY SYSTEMS

Resumen de: EP4755873A1

The invention relates to ionizable cationic amino lipids and lipid-based delivery systems, such as lipid particles comprising ionizable cationic amino lipids of the invention and their use for delivering a payload into a cell.

METHOD AND SYSTEM

Resumen de: WO2025032322A1

A method of generating a plurality of different surface-decorated nanoparticles, decorated with different surface decoration, comprising: forming a plurality of microdroplets in a microfluidics device, each microdroplet comprising a nanoparticle and a respectively different macromolecule encoding a different surface decoration molecule; synthesising the surface decoration molecule, within each microdroplet, based on the macromolecule encoding the surface decoration molecule; conjugating the nanoparticle and the surface decoration molecule, within each microdroplet, to form surface decorated nanoparticles.

STEALTH-STABILIZED SULFUR-CONTAINING LIPO/POLYAMINO ACIDS

Resumen de: WO2025031991A1

It relates to sulfur-containing lipo-polyamino acid conjugates of formula (I), and acceptable salts, stereoisomers and mixtures thereof. It also relates to self-assembled particles comprising these lipo/polyamino acid conjugates and optionally active agents, and to compositions comprising the lipo-polyamino acid conjugates or the self-assembled particles comprising them. It relates as well to the use of the lipo-polyamino acid conjugates, self-assembled particles, or compositions comprising them in medicine, cosmetics and diagnostics, and to the use of the lipo-polyamino acid conjugates of formula (I) as carriers.

TUMOR AGNOSTIC DRUG DELIVERY WITH SELF-AGGLOMERATING NANOHYDROGELS

Resumen de: WO2025030109A1

Delivery of oligonucleotides and other agents to tumor microenvironments is challenging due to their rapid clearance, nuclease susceptibility, and inability to traverse plasma membranes, impeding their effective use for the treatment of a variety of disorders. Disclosed herein is a self-agglomerating nanohydrogel and methods of delivering an agent as well as methods of treating cancer with the nanohydrogel.

A METHOD OF PREPARING AN AQUEOUS DISPERSION OF NANOPARTICLES

Resumen de: CN121729223A

The present invention relates to a method for preparing an aqueous nanoparticle dispersion of a hydrophobic active pharmaceutical ingredient (API). The invention also relates to a variety of nanoparticles produced by such a method. In addition, the invention also relates to application of the nanoparticles.

吸入用医薬組成物

Resumen de: WO2024246304A1

Disclosed is a pharmaceutical composition for inhalation, comprising lipid carriers comprising a pharmaceutical agent, the therapeutic uses thereof, and a method of making same.

一种编码烟曲霉嵌合抗原的环状RNA及其应用

Resumen de: CN122145650A

本发明涉及一种编码烟曲霉嵌合抗原的环状RNA及其应用，属于生物医学与疫苗技术领域。本发明首次将环状RNA技术应用于抗烟曲霉感染，为真菌疫苗开发提供了新策略。选取烟曲霉细胞膜蛋白FtrA的非跨膜区构建抗原，避免了跨膜区表达难题和免疫识别不确定性；融合IgK信号肽和Fc结构域，显著提高抗原分泌效率、稳定性和免疫呈递效果。LNP递送系统成熟、高效，能有效将疫苗递送至宿主细胞并表达抗原，本发明提供的LNP@CircRNA，特别是LNP@CircRNAFtrA2，在不使用佐剂的情况下，能在小鼠体内诱导产生高水平的IgG抗体和T细胞免疫应答，实现体液免疫和细胞免疫的双重激活。

新規の上皮透過促進剤

Resumen de: WO2024245589A1

The invention relates to a composition comprising a cold-water insoluble crosslinked dextrin and a fatty acid having 8 to 17 carbon atoms. This invention also relates to a method for making such composition. The invention also relates to the use of a combination of a cold-water insoluble crosslinked dextrin and of a fatty acid having 8 to 17 carbon atoms for increasing the epithelial permeation of an active ingredient, or for the epithelial delivery of an active ingredient.

利用固相固定的微生物转谷氨酰胺酶MTG和溶液中的MTG对抗体赖氨酸残基的位点特异性缀合

Resumen de: US2022333093A1

Site-specific modification of proteins with microbial transglutaminase (MTG) is a powerful and versatile strategy for a controlled modification of proteins under physiological conditions. We present evidence that solid-phase microbead-immobilization can be used to site-specifically and efficiently attach different functional molecules important for further downstream applications to proteins of therapeutic relevance including scFV, Fab-fragment and antibodies. We demonstrate that MTG remained firmly immobilized with no detectable column bleeding and that enzyme activity was sustained during continuous operation, which allowed for a convenient recycling of the enzyme, thus going beyond solution-phase MTG conjugation. In addition it is showed that immobilized MTG shows enhanced selectivity towards a certain residue in the presence of several reactive residues which are all targeted if the conjugation was carried out in solution. It is also reported on the site-specific lysine conjugation of antibodies using potent glutamine containing peptides with immobilized and MTG in solution. In addition, the generation of dual site-specifically conjugated IgG1 with immobilized and MTG in solution is reported, i.e. site-specific conjugation to glutamine and lysine residues of IgG1 antibody. Site-specific glutamine conjugation with small peptides containing a lysine residue and a functional moiety is also described.

新規な上皮透過促進剤

Resumen de: WO2024245590A1

The invention relates to a composition comprising a cold-water insoluble crosslinked dextrin and a compound selected from 1-decanoyl-rac-glycerol and carboxymethyl cellulose. This invention also relates to a method for making such composition. The invention also relates to the use of a combination of a cold-water insoluble crosslinked dextrin and of a compound selected from 1-decanoyl-rac-glycerol and carboxymethyl cellulose, for increasing the epithelial permeation of an active ingredient, or for the epithelial delivery of an active ingredient.

一种可离子化脂质化合物及其应用

Resumen de: CN122145411A

本发明提供如通式I所示的可离子化脂质化合物，其中，R1和R2各自独立地为C8~C20的直链或支链烃基；R3为被至少一个R4基团取代的、包含至少一个氮原子作为环成员的5至8元杂环基，其中所述R3基团通过一个叔胺氮原子与丙二酸二酯骨架相连接；R4为C1~C6烷基或C1~C6烷酰基，且所述C1~C6烷基或C1~C6烷酰基任选地被1至3个独立地选自羟基、氨基、卤素、氰基和4~8元脂肪环基或芳香环基的取代基所取代，其中所述羟基、氨基任选地被4~8元脂肪环基或芳香环基所取代。本发明还提供了包含所述脂质化合物的脂质纳米颗粒、药物组合物及其用途。本发明的可离子化脂质化合物相对于传统阳离子脂质化合物安全性高、包封效果好，为药物的高效、安全递送提供更优解决方案。I。

黄芪外泌体负载毛蕊异黄酮制得的工程外泌体及制备方法和应用

Resumen de: CN122140652A

本发明提供一种黄芪外泌体负载毛蕊异黄酮制得的工程外泌体及制备方法和应用。以黄芪外泌体为载体，通过共孵育方式负载毛蕊异黄酮，得到工程外泌体。该体系粒径约 100～200 nm，保持典型双层膜结构，同时实现较高药物负载率和良好稳定性。体外实验表明，AS‑Exos‑CA 可被 PC12 等神经元样细胞高效摄取；体内 DiR 标记及活体成像结果证实 AS‑Exos‑CA 能跨越小鼠血脑屏障并在脑组织中富集。MPP⁺ 损伤 PC12 细胞模型及MPTP 诱导的帕金森病小鼠模型研究显示，AS‑Exos‑CA 能显著提高细胞存活率和小鼠运动功能，减少黑质多巴胺能神经元丢失，激活 Nrf2/System Xc⁻/GPX4 抗氧化通路，降低 Fe²⁺、MDA、ROS 水平并提高 GSH，减轻线粒体损伤，抑制铁死亡。与单用 CA 或 AS‑Exos 相比，本发明系统具有更优的神经保护和抗帕金森治疗效果。

用于特异性识别HIV gp120蛋白的多肽分子及其应用

Resumen de: CN122145575A

本发明提供了用于特异性识别HIV gp120蛋白的多肽分子及其应用，属于生物医药技术领域。本发明提供了一种特异性靶向并结合HIV gp120的先导多肽衍生物，所述先导多肽包含SEQ ID No.1所示的氨基酸序列。本发明还提供了基于所述先导多肽制备得到的先导多肽衍生物，以及先导多肽衍生物构建得到的多肽纳米粒，均具备gp120蛋白结合能力。本发明所述衍生物和纳米粒，具有良好的病毒中和抑制效果。本发明所述系列多肽分子能够与游离HIV病毒、HIV感染细胞以及游离gp120特异性结合，从而阻断HIV感染细胞、延缓AIDS疾病进程、实现AIDS治疗药物靶向递送、检测以及诊断。

一种基于内源性启动子劫持的肝靶向基因编辑系统及应用

Resumen de: CN122146793A

本发明公开了一种基于内源性启动子劫持的肝靶向基因编辑系统及应用，属于生物医药领域。该系统由LNP包裹的修饰化Cas核酸酶mRNA（第一组分）与携带治疗性转基因供体的无启动子病毒载体（第二组分）组成。其利用LNP实现Cas核酸酶在肝脏的瞬时爆发表达，介导内源性高表达基因位点产生双链断裂，诱导无外源启动子的治疗性转基因定点整合，并利用剪接受体（SA）机制劫持内源启动子驱动表达。本发明通过“双重安全锁”设计，解决了外源强启动子随机整合致癌风险及核酸酶长期表达的免疫毒性。实验证明，该系统编辑效率高、长效稳定且无脱靶，可用于血友病、高胆固醇血症等多种肝源性代谢疾病。

制造用于制造大麻素的前体化合物的方法和/或制造大麻素的方法

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).

一种偶联GO-203并负载DMAMCL的脂质纳米载体、制备方法及其应用

Resumen de: CN122140654A

本发明涉及一种偶联GO‑203并负载DMAMCL的脂质纳米载体及其制备方法和应用，属于生物医药与纳米制剂领域。所述脂质纳米载体由内至外依次包括水相内核、脂质双分子层以及表面修饰层，其中水相内核中负载水溶性二甲胺甲基氯化物（DMAMCL）；脂质双分子层包裹所述水相内核，并嵌入香豆素‑6作为荧光标记物用于示踪与检测；所述脂质双分子层外表面设置有功能化修饰层，包括DSPE‑PEG2000‑COOH和DSPE‑PEG2000‑GO‑203，其中GO‑203为细胞膜穿透肽。本发明提供的脂质纳米载体可实现GO‑203与DMAMCL的协同递送，用于调控肿瘤免疫微环境并用于三阴性乳腺癌的治疗。

一种靶向治疗炎症性疾病的药物递送系统及其制备方法和应用

Resumen de: CN122140655A

本发明提供了一种靶向治疗炎症性疾病的药物递送系统及其制备方法和应用，属于生物药物技术领域。一种靶向治疗炎症性疾病的药物递送系统，为吞噬有载药纳米颗粒的M2型巨噬细胞，所述载药纳米颗粒包括用于包覆药物的生物可降解高分子材料。本发明制备的药物递送系统通过M2型巨噬细胞的天然炎症趋化特性实现肺部靶向递送，结合载药纳米颗粒的药物缓释作用，显著提升了药物靶向性和利用度，从而提高药效，具有良好的生物安全性，为包括碳青霉烯耐药肺炎克雷伯菌（CRKP）肺炎在内的多种炎症性疾病的临床治疗提供了新的有效手段。

一种虾青素和二氢槲皮素共载脂质体及其在制备降尿酸制品中的应用

Nº publicación: CN122140629A 05/06/2026

Solicitante:

中国海洋大学

Resumen de: CN122140629A

本发明涉及生物技术领域，具体涉及一种虾青素和二氢槲皮素共载脂质体及其在制备降尿酸制品中的应用。按照“体外功能筛选—优选比例确定—共载脂质体构建—表面包覆稳态化处理—降尿酸应用验证”的步骤展开。通过理化表征、稳定性试验和动物实验对所得体系进行验证，结果表明，该共载脂质体能够降低高尿酸模型小鼠的血清尿酸水平、抑制肝脏黄嘌呤氧化酶活性，同时改善肾功能相关指标及组织病理损伤。