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一种负载CDNF/Rg3仿生脂质纳米颗粒的制备方法和应用

Resumen de: CN122643260A

本发明属于纳米药物递送技术领域，具体涉及一种负载CDNF/Rg3仿生脂质纳米颗粒的制备方法和应用。所述方法通过构建过表达ARC的神经干细胞并提取其细胞膜，采用微流控技术制备负载CDNF circRNA和人参皂苷Rg3的ROS响应性脂质纳米颗粒，再经薄膜挤出获得ARC‑RCD‑LNP。该纳米颗粒经鼻腔给药后可进入脑内，借助ARC相关神经元靶向特性提高受损神经元摄取，并在ROS微环境下释放Rg3和CDNF circRNA；CDNF circRNA翻译为CDNF蛋白，抑制α‑Syn聚集，并与Rg3协同发挥抗炎、抗氧化、缓解内质网应激和抗凋亡作用，从而修复受损神经元并改善运动功能障碍。

含冰片结构的可电离脂质化合物及其制备方法和应用

Resumen de: CN122647415A

本发明涉及含冰片结构的可电离脂质化合物及其制备方法和应用，属于生物医药技术领域。本发明通过对冰片分子C2位羟基进行修饰，引入多胺结构及疏水性尾链，制得一类新型含冰片结构的可电离脂质化合物。该化合物具有以下优势：（i）冰片结构可有效突破皮肤角质层屏障，显著提升经皮吸收效率；（ii）多胺结构在酸性条件下可电离，通过静电吸附作用高效携载核酸药物，有助于增强核酸稳定性并促进溶酶体逃逸；（iii）尾链修饰增强了化合物的脂溶性，赋予其脂质特性，可作为固体脂质纳米粒的制备原料。本发明还提供了所述化合物的制备方法，该类含冰片结构的可电离脂质化合物在核酸药物的经皮递送领域展现出良好的应用前景。

一种脂质纳米颗粒及其在胞内或体内蛋白质递送中的应用

Resumen de: CN122643257A

本发明公开了一种脂质纳米颗粒及其在胞内或体内蛋白质递送中的应用。本发明所述脂质纳米颗粒包括阳离子脂质、可电离脂质、辅脂脂质、胆固醇和聚乙二醇化脂质五种脂质组分；所述脂质纳米颗粒可以实现高效的活细胞内和体内蛋白质递送，且不影响被递送蛋白质的生物学活性；所述脂质纳米颗粒成分安全，具有很好的生物相容性。本发明具有广泛应用前景。

一种纳米晶氟苯尼考颗粒及其制备方法

Resumen de: CN122643262A

本发明公开了一种纳米晶氟苯尼考颗粒及其制备方法，属于兽药制剂及纳米药物技术领域。所述方法包括：将氟苯尼考溶解于食用级乙醇中制得有机相；将注射用水冷却后加入聚乙二醇‑1000维生素E琥珀酸酯和植酸，制得水相；将两相料液泵入高压微射流对撞反应器中进行连续化混合结晶；产物经减压脱醇、浓缩及干燥，即得类球形纳米晶氟苯尼考颗粒。本发明摒弃了传统有毒溶剂及超声破碎工艺，利用高压微射流的微湍流与植酸的氢键晶面调控作用，一步制备粒径为80～150nm的纳米晶。所得颗粒流动性好、无有毒溶剂残留，25℃下水相溶解度大幅提升，且悬浮稳定性超过120小时。

一种用于治疗肺部肿瘤的药物组合物及其应用

Resumen de: CN122643432A

本发明公开了一种用于治疗肺部肿瘤的药物组合物及其应用。本发明提供的用于治疗肺部肿瘤的药物组合物包括能够肺部递送CXCL10 mRNA的纳米颗粒和靶向基因修饰免疫效应细胞；所述靶向基因修饰免疫效应细胞为CAR‑T细胞、CAR‑NK细胞、CAR‑M细胞或TCR‑T细胞。本发明证实肺部递送CXCL10 mRNA的纳米颗粒能够增强靶向基因修饰免疫效应细胞（如CAR‑T细胞）的肺部肿瘤浸润及抗肿瘤疗效。本发明不仅为肺部肿瘤免疫治疗提供了具有明确机制支撑的可转化方案，也为拓展mRNA药物在肝外器官的递送与多疾病应用场景提供了重要的技术参考与产业化价值。

一种基于肿瘤抗原表位肽的多靶点DC疫苗及其应用

Resumen de: CN122643427A

一种基于肿瘤抗原表位肽的多靶点DC疫苗及其应用，属于肿瘤免疫治疗技术领域，该疫苗包含hr‑8偶联肽、免疫佐剂和载体，以及免疫记忆增强因子，其中hr‑8偶联肽为hr‑8靶向肽通过化学偶联方式与至少两种不同肿瘤抗原CTL表位肽连接形成的偶联产物，免疫佐剂为TLR9激动剂CpG ODN与TLR3激动剂poly(I:C)的联合佐剂体系，载体为PLGA纳米颗粒，免疫记忆增强因子为IL‑7和/或IL‑15。本发明利用hr‑8肽特异性结合树突状细胞表面DEC‑205受体，实现多靶点抗原的高效协同递送和交叉呈递，联合佐剂与记忆因子显著增强CTL激活并促进长效免疫记忆形成，使得多靶点DC疫苗具有靶向性强、抗原覆盖广、延长无复发生存期的优点。

ヒンジドメインを有するヒト化ＮＯＴＣＨ受容体

Resumen de: WO2021207526A1

The present disclosure generally relates to, inter alia, a new class of chimeric Notch receptors containing a fully humanized transcriptional effector, engineered to modulate gene expression and cellular activities in a ligand-dependent manner. The new chimeric Notch receptors surprisingly retain the ability to transduce signals in response to ligand binding despite that the Notch extracellular subunit (NEC), which includes the negative regulatory region (NRR) previously believed to be essential for the functioning of Notch receptors is completely absent. In addition, the new receptors described herein incorporate an extracellular oligomerization domain to promote oligomer formation of the chimeric receptors. Also provided are compositions and methods useful for producing such receptors, nucleic acids encoding same, engineered cells genetically modified with the nucleic acids, as well as methods for modulating an activity of a cell and/or for the treatment of various diseases such as cancers.

ビエッティ結晶性ジストロフィーの治療用医薬製剤

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).

一种小反刍兽疫RNA疫苗及其制备方法与应用

Resumen de: CN120550105A

The invention discloses a DNA vaccine, and the DNA vaccine prepared by taking an H gene and an F gene of a peste des petits ruminants virus strain with a microbial preservation number of CCTCC NO: V202487 as coding genes of immunogens has good immunogenicity and immune protection capability, and has an application prospect.

硼中子俘获治疗用白蛋白纳米平台以及包含该平台的硼中子俘获治疗用组合物

Resumen de: WO2025165163A1

The present invention relates to an albumin nanoplatform for boron neutron capture therapy and a composition for boron neutron capture therapy comprising same. The albumin-based nanoplatform according to the present disclosure can effectively deliver boron to specific tumor tissues by simultaneously conjugating a boron compound containing an excessive amount of boron and a targeting molecule as a carrier for specifically targeting tumor tissues via click chemistry functional groups introduced into the albumin surface. In particular, the nanoplatform enables sufficient delivery of boron molecules to target tumor tissues even at doses less than one-tenth of those required by conventional boron neutron capture therapy (BNCT) drugs, and thus can be applied as a composition for boron neutron capture therapy and as an anticancer therapeutic agent.

ナノ・イン・マイクロカプセル化治療用核酸乾燥粉末および当該乾燥粉末を含有する医薬品

Resumen de: EP4497433A1

The invention relates to Nano-in-micro (NIM) encapsulated bioactive siRNA dry powder comprising lipid nanoparticles, the lipid nanoparticles comprising from at least an ionizable cationic lipid, a helper lipid, a stealth lipid, and siRNA, wherein the lipid nanoparticles are encapsulated in sugar and/or sugar alcohol, wherein the mass ratio of siRNA to a sugar and/or sugar alcohol is more than 0.10%, especially between 0,90% and 1,30%. The invention also relates to a respective pharmaceutical product containing the dry powder in a sealed volume.

用于脂质纳米颗粒的可电离脂质

Resumen de: WO2025157978A1

The present invention relates to ionizable lipids for use in lipid nanoparticles, lipid nanoparticle formulations comprising these ionizable lipids, alone or in combination with other lipids and/or polymers. The lipid nanoparticles formulations may be formulated with nucleic acids for their delivery to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous or intratumoral administration.

지질 나노입자에 적합한 이온화성 지질

Resumen de: 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.

一种具有液晶性能的嵌段共聚物纳米粒子及其制备方法和应用

Resumen de: CN122647680A

本发明公开了一种具有液晶性能的嵌段共聚物纳米粒子及其制备方法和应用。所述嵌段共聚物纳米粒子结构式如(1)式所示。本发明通过使用聚合诱导自组装方法中动力学陷阱的特点，获得高固含量且尺寸均一的液晶嵌段共聚物纳米粒子。本发明不仅可获得传统蠕虫状液晶嵌段共聚物胶束，并可获得可调节双折射程度的球形液晶嵌段共聚物胶束。本发明不仅提高了液晶嵌段共聚物纳米粒子的可加工性能，亦通过固含量提供显著提升其大规模制造的可能，在高分子材料和生物医药方面有显著的应用价值。

一种选择性包埋人参皂苷Rd的玉米蛋白粒子制备方法及应用

Resumen de: CN122643261A

本发明涉及活性物质包埋递送技术领域，具体涉及一种选择性包埋人参皂苷Rd的玉米蛋白粒子制备方法及应用。制备方法包括以下步骤：（1）将玉米醇溶蛋白溶解于乙醇溶液中；（2）向玉米醇溶蛋白溶液中加入人参总皂苷；（3）配制酪蛋白缓冲液，将酪蛋白缓冲液快速加入上述混合液；（4）采用旋转蒸发方式去除乙醇；（5）过滤纳米粒子分散液；（6）冷冻干燥。现有技术需使用高纯度人参皂苷Rd单体作为包埋原料，粗提物直接包埋又无选择性，产物成分混杂、质量不可控。本发明利用玉米醇溶蛋白对人参皂苷Rd的特异性结合能力，从含多种皂苷混合物中选择性富集人参皂苷Rd，包埋总皂苷中Rd占比可达90%以上。显著降低整体制备成本，适配规模化工业化生产。

RNA及包含其的四价流感疫苗

Resumen de: CN122643425A

本发明公开了RNA及包含其的四价流感疫苗。具体地公开了免疫原性组合物，其包括编码流感病毒H1N1、H3N2、H5N1和H7N9的HA蛋白的四种RNA分子。将该免疫原性组合物包裹在可离子化脂质纳米颗粒中得到mRNA疫苗。本发明的mRNA疫苗具有良好的免疫原性，能够高效表达，可在体内实现稳定安全的表达和有效激活免疫反应，免疫的小鼠可以在体内引起良好的体液免疫，并且有很好的保护作用。本发明的mRNA疫苗作为一种通用型流感疫苗，可提供针对流感病毒的更广泛的保护，具有良好的实际应用价值。

一种具有GSH/ROS双重响应机制的聚合物及其制备方法和应用、脂质纳米颗粒

Resumen de: CN122647726A

本发明属于药物递送载体及化疗类药物领域，具体涉及一种具有GSH/ROS双重响应机制的聚合物及其制备方法和应用、脂质纳米颗粒。该聚合物具有GSH/ROS双重响应机制，其化学结构通式如下所示：A‑R‑B；所述A为具有胆固醇或其衍生物的第一化学结构；所述R为具有GSH响应的第二化学结构；所述B为具有如式I所示的第三化学结构。由此，本发明通过化学结构设计赋予其GSH/ROS双重响应机制，并将其进一步应用于药物递送系统及其在肿瘤协同治疗中，实现了基于氧化还原双响应脂质纳米颗粒的多模态协同治疗策略，为恶性肿瘤治疗提供了具有显著协同增强效应的新技术方案。

一种可电离脂质化合物、包含其的脂质纳米颗粒及其制备方法和应用

Resumen de: CN122647348A

本发明提供了一种可电离脂质化合物、包含其的脂质纳米颗粒及其制备方法和应用。其选自如式（I）所示的可电离脂质或其消旋体、立体异构体、几何异构体、互变异构体、氮氧化物、水合物、同位素标记物、溶剂化物、多晶型物、代谢产物、酯、药学上可接受的盐、前药或其混合物形式；其中，R1和R6各自独立地选自C1‑C20的直链或支链烷基，R2、R5、R7和R8各自独立地为‑(CH2)m‑，m为2至10的整数，R3和R4各自独立地选自H或C1‑C5的烷基，X选自‑O‑、‑S‑或‑Se‑。该化合物化学结构明确、可生物降解，基于其制备的脂质纳米颗粒具有肺部优先富集或肺部靶向递送能力。（I）

一种二聚体轮状病毒疫苗及其应用

Resumen de: CN122647621A

本发明提供一种基于VP8‑Fc的融合蛋白，以及以该融合蛋白为核心构建的轮状病毒蛋白亚单位疫苗和mRNA疫苗。动物实验结果显示，无论是蛋白亚单位疫苗还是mRNA疫苗，免疫后均能诱导小鼠产生强烈的体液免疫应答，表现出优异的免疫原性及潜在的交叉保护能力。本发明通过轮状病毒VP8结构域与Fc片段的融合设计，提供了一种能诱导广谱、强效免疫应答的新型轮状病毒疫苗。

NANOPARTICLE FORMULATIONS

Resumen de: WO2026178127A1

The present invention relates to lipid nanoparticle compositions that include a polypeptide, a cation, a compound capable of coordinating cations, and a plurality of encapsulating lipids, as well as methods of using the lipid nanoparticle compositions in the treatment of disease, and methods for preparing the lipid nanoparticle compositions. The lipid nanoparticle compositions may be used for treating diseases in a subject, such as autoimmune diseases or cancer.

COMPOSITIONS FOR TREATING CANCER WITH KRAS MUTATIONS AND USES THEREOF

Resumen de: US20260248963A1

0000 The present application provides methods of treating a cancer in subjects who have been subjected to a KRAS inhibitor and/or harbor a KRAS secondary mutation by administering complexes (e.g., genome-editing complexes or complexes having RNAi) or nanoparticles specifically targeting a mutated KRAS. Exemplary genome-editing complexes or nanoparticles comprise cell-penetrating peptides and a guide RNA, and optionally a DNA nuclease (such as Cas9) or a polynucleotide encoding the DNA nuclease.

IONIZABLE LIPIDS COMPRISING THIOAMIDES, PREPARATION THEREOF, USES THEREOF, AND COMPOSITIONS THEREOF

Resumen de: WO2026175979A1

The present invention relates to the field of ionizable lipids and compositions thereof. More particularly, the present invention relates to ionizable lipids or pharmaceutically acceptable salts thereof comprising a moiety, the moiety comprising an ionizable nitrogen attached adjacent to a thioamide group. The present invention further relates to a method for preparing said ionizable lipids, lipid nanoparticles comprising said ionizable lipids, and medical uses thereof (e.g, to deliver biologically active agents, such as nucleic acids, to cells and tissues).

METHODS FOR PREPARING NANOPARTICLE FORMULATIONS

Resumen de: WO2026178134A1

The present invention relates to methods of preparing lipid nanoparticle compositions that include a polypeptide, a cation, compound capable of coordinating cations, and a population of encapsulating lipids. The methods afford lipid nanoparticles after a single operation and have favorable properties for the delivery of therapeutic agents.

LIPID NANOPARTICLE

Resumen de: WO2026175904A1

The present invention relates to a lipid nanoparticle comprising: (a) an ionizable lipid; (b) a helper lipid; (c) a sterol; and (d) a PEGylated lipid, wherein: (i) the PEGylated lipid comprises a hydrocarbon chain comprising more than 14 C atoms; and/or (ii) the PEGylated lipid is at a concentration of at least 3 %mol.

METHODS FOR PREPARING NANOPARTICLE COMPOSITIONS

Nº publicación: WO2026178144A1 27/08/2026

Solicitante:

SAROS THERAPEUTICS INC [US]
SAROS THERAPEUTICS, INC.

Resumen de: WO2026178144A1

The present invention relates to methods of preparing lipid nanoparticle compositions that include a polypeptide, a cation, compound capable of coordinating cations, and a population of encapsulating lipids. The methods afford lipid nanoparticles after a single operation and have favorable properties for the delivery of therapeutic agents.