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NANOSYSTEM FOR USE IN CAPTURING, DIRECTING AND/OR TRANSPORTING BIOLOGICALLY ACTIVE MOLECULES TO ADIPOSE TISSUE

Resumen de: EP4789683A1

The present invention relates to a nanosystem for use in capturing, directing and/or transporting biologically active molecules to adipose tissue, therefore serving to treat illnesses such as obesity and/or type 2 diabetes.

METHOD OF DELIVERY OF DNA OR RNA CARGO USING UNSHIELDED LIPID NANOPARTICLES

Resumen de: US2025114306A1

0000 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.

PEPTIDE NANOSPONGES FOR DRUG DELIVERY

Resumen de: WO2025076082A1

The present disclosure describes peptide nanosponges comprising a plurality of self-assembled peptide building blocks. The plurality of self-assembled peptide building blocks comprises a first peptide building block comprising: a branched polymeric core comprising at least three arms; a first block co-peptide covalently linked to one of said arms, wherein said first block co-peptide comprises a first peptide block and a second peptide block which is different from the first peptide block; a lipid-based capping moiety covalently attached to the first block co-peptide; and a therapeutically active compound covalently attached via a cleavable linkage to the first block co-peptide. Advantageously, the block co-peptide is covalently attached to the core such that it is resistant to enzymatic cleavage from the core.

IMMUNOGENIC COMPOSITIONS

Resumen de: WO2025074292A2

The invention relates to lipid nanoparticles, immunogenic compositions and methods for use thereof.

HAEMATOPOIETIC STEM AND/OR PROGENITOR CELL DELIVERY METHOD AND COMPOSITIONS FOR USE THEREOF

Resumen de: AU2024354577A1

The present disclosure provides a method for delivery of cargo to a haematopoietic stem or progenitor cell of a subject, the method comprising contacting a lipid nanoparticle encapsulating the cargo with the haematopoietic stem or progenitor cell ex vivo or in vivo, thereby causing cellular uptake of the cargo, the lipid nanoparticle having between 30 mol% and 70 mol% of a neutral or zwitterionic amphipathic lipid having a net-neutral charge at physiological pH, an ionizable cationic lipid, and a sterol, wherein the lipid nanoparticle is substantially uncharged at physiological pH and has an apparent pKa of between 6.0 and 7.5. Further provided are lipid nanoparticle compositions and uses thereof.

IONIZABLE CATIONIC LIPIDS WITH CONSERVED SPACING AND LIPID NANOPARTICLES

Resumen de: WO2025076113A1

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 delivering 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 a 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.

遺伝子送達における非ウイルスベクターとしての樹状構造

Resumen de: WO2022212838A1

Disclosed herein are novel dendritic structures that can be used in combination with other lipid components to form nanoparticles with nucleic acids, to facilitate the intracellular delivery of nucleic acids both in vitro and in vivo. Nanoparticle compositions comprising the compounds and methods for their use for treating or preventing diseases or conditions are also provided.

規定のサイズのナノ粒子を製造する方法

Resumen de: EP4501320A1

0001 The present invention relates to a method of preparing an aqueous dispersion of nanoparticles of a hydrophobic active pharmaceutical ingredient (API). The present invention also relates to a plurality of nanoparticles produced by such method. Furthermore, the present invention relates to uses of such nanoparticles.

イオン化可能な脂質

Resumen de: WO2022136641A1

The present invention generally relates to the field of ionizable (also termed cationic) lipids, and in particular provides a novel type of such lipids as represented by formula (I). The present invention further provides methods for making such lipids as well as uses thereof, in particular in the preparation of nanoparticle compositions, more in particular nanoparticle compositions comprising nucleic acids. It further provides vaccine formulations comprising nanoparticle compositions based on the ionizable lipids disclosed herein.

イオン化可能なカチオン性脂質および脂質ナノ粒子ならびにその合成および使用の方法

Resumen de: AU2021393593A1

Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., immune cells), and methods of making and using such lipids and targeted lipid nanoparticles.

機能性のイオン化可能なリン脂質

Resumen de: WO2022040641A2

Ionizable phospholipids and compositions and methods relating thereof are provided herein. In some aspects, the ionizable phospholipids provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

核酸ベースの混合ワクチン

Resumen de: MX2022015132A

The present invention is inter alia directed to pharmaceutical compositions comprising at least one nucleic acid encoding at least one antigenic peptide or protein from a Coronavirus, preferably a pandemic Coronavirus, and at least one nucleic acid encoding at least one antigenic peptide or protein from a further virus, e.g. an Influenza virus or an RSV virus. Pharmaceutical compositions provided herein are suitable for use in treatment or prophylaxis of an infection with at least one Coronavirus and at least one further virus infection, and may therefore be comprised in a combination vaccine. The nucleic acid sequences of the pharmaceutical compositions and combination vaccines are preferably in association with a polymeric carrier, a polycationic protein or peptide, or a lipid nanoparticle (LNP). The invention is also directed to first and second and further medical uses of the pharmaceutical compositions and combination vaccines, and to methods of treating or preventing a Coronavirus infection and a further virus infection.

治療、イメージング、およびセラノスティック用途向けの小型超均一ナノ医薬組成物

Resumen de: WO2020056333A1

A targetable nanoconstruct capable of simultaneously serving as a therapeutic platform for photodynamic therapy as well as an MR molecular imaging agent, free of heavy metal atoms. F3-cys targeting agent nanoconstructs, including 8PEGA-Ce6 NCs. A label-free 8PEGA nanoconstruct that can be directly and selectively imaged by MRI, using standard spin-echo imaging sequences with large diffusion magnetic field gradients to suppress the water signal.

NANOBUBBLES FOR ULTRASOUND MEDIATED NUCLEIC ACID DELIVERY

Resumen de: AU2024356988A1

Gas-filled nanobubbles for negatively charged genetic material delivery each includes a lipid membrane defining a gas containing internal void, wherein the lipid membrane includes a plurality of cationic lipids for complexing the negatively charged genetic material, an edge-activator incorporated between lipids of the membrane that enhances the flexibility of the membrane, and a membrane stiffener incorporated on an outer surface of the membrane that enhances the membrane's resistance to tearing.

一种肿瘤微环境响应型沸石咪唑骨架-67复合纳米递送体系及其制备方法与应用

Resumen de: CN122537557A

本发明公开了一种肿瘤微环境响应型沸石咪唑骨架‑67复合纳米递送体系及其制备方法与应用，属于肿瘤纳米药物与基因治疗技术领域。所述纳米递送体系为细胞膜包覆的 Cas9 RNP@ZIF‑67@NaCl 复合纳米颗粒，其以 NaCl 纳米颗粒为晶种原位生长 ZIF‑67 骨架，负载 Cas9 核糖核蛋白复合物后进一步包覆肿瘤细胞膜形成。所述纳米递送体系在肿瘤酸性微环境中降解释放 Co2+、NaCl 及 Cas9 RNP，其中 Co2+ 催化细胞内 H2O2 产生活性氧，实现化学动力学治疗；Cas9 RNP 靶向敲除 UXS1 基因，阻断 UDP‑木糖合成通路，从而抑制肿瘤细胞生长；NaCl 提高胞内渗透压并诱导肿瘤细胞裂解。所述纳米递送体系具有肿瘤靶向递送及微环境响应释放特性，可实现基因编辑治疗、化学动力学治疗及渗透压裂解的协同抗肿瘤作用。本发明为肿瘤协同治疗提供了一种新的纳米递送平台。

一种靶向超声引导射频消融诊疗纳米系统及其制备方法和应用

Resumen de: CN122537552A

本发明属于生物医药技术领域，具体涉及一种靶向超声引导射频消融诊疗纳米系统及其制备方法和应用。本发明所述靶向超声引导射频消融诊疗纳米系统为多层复合纳米气泡结构，由外至内包括靶向修饰的脂质双分子层外壳、载药内核以及核心气体。本发明所述靶向超声引导射频消融诊疗纳米系统不仅实现了高灵敏度的超声精准影像引导与主动靶向，同时，其原位氧化修饰显著增强肿瘤抗原免疫原性，激发强效的系统性抗肿瘤免疫反应以抑制射频消融术后肿瘤的转移与复发，形成一种全新的诊疗一体化系统，可以作为靶向超声造影剂使用，为肿瘤的诊断和治疗提供更为有效的途径。

一种用于核酸递送的可电离脂质、脂质纳米粒组合物与应用

Resumen de: CN122541505A

本发明涉及一种用于核酸递送的可电离脂质、脂质纳米粒组合物与应用。所述可电离脂质具有以下结构通式：。与现有技术相比，本发明制得的可电离脂质及脂质纳米粒组合物可广泛应用于核酸递送载体、基因治疗药物及mRNA疫苗的制备。

一种可离子化二茂铁衍生脂质分子及其应用

Resumen de: CN122541487A

本发明公开了一种可离子化二茂铁衍生脂质分子及其应用。本发明脂质分子的结构式如下，其中，X1为氧原子或者氮原子；X2为碳原子或氮原子；R1和R2各自独立地选自C4‑22烷基或C4‑22烯基；R3为H、C1‑4烷基或C2‑4烯基；m+n≥1。本发明脂质分子可实现在体内靶向递送至淋巴结，递送效率高。式(I)

一种用于治疗自身免疫性疾病的mRNA脂质纳米颗粒药物组合物

Resumen de: CN122537517A

本发明公开了一种用于治疗自身免疫性疾病的mRNA脂质纳米颗粒药物组合物，核心为编码自身免疫性疾病相关抗原表位的功能化 mRNA。所述 mRNA 的编码区包含经筛选与验证的 1 型糖尿病、系统性红斑狼疮、原发性胆管炎相关特异性抗原表位编码序列，可通过柔性连接子串联多表位组合；同时 mRNA 搭载优化的 5’UTR、3’UTR、5’帽结构与 polyA 尾，经密码子优化及核苷酸修饰后，兼具高稳定性与高效翻译表达能力。所述 mRNA 由靶向肝窦内皮细胞的脂质纳米颗粒包载递送，可高效诱导抗原特异性免疫耐受，抑制异常自身免疫反应，显著改善模型动物疾病指标，为自身免疫性疾病提供了新型靶向治疗方案。

Acmsd mRNA-LNP的制备与在结直肠癌治疗中的应用

Resumen de: CN122537563A

本发明属于生物制药领域，涉及Acmsd mRNA‑LNP的制备与在结直肠癌治疗中的应用。本发明发现犬尿氨酸下游代谢物吡啶甲酸能显著提升CD8+ T细胞数量、增殖（Ki67）和效应功能（IFNγ、TNFα、GzmB），并增强IFNγ对结直肠癌细胞的杀伤作用。进而构建Acmsd mRNA‑LNP递送体系（脂质摩尔比50% SM‑102、10% DSPC、38.5%胆固醇、1.5% DMG‑PEG2000，包封率≥60%，粒径130‑150 nm），瘤内注射可重塑肿瘤局部犬尿氨酸代谢途径，提高吡啶甲酸含量，改善抗肿瘤免疫，并与抗PD‑1抗体联合使用显著抑制MC38荷瘤小鼠肿瘤生长。本发明为结直肠癌免疫治疗提供了新型代谢重编程策略，具有广阔临床应用价值。

一种巨噬细胞靶向miRNA递送仿生体系及其制备方法和应用

Resumen de: CN122537559A

本发明属于生物医药与纳米递送技术领域，具体涉及一种巨噬细胞靶向miRNA递送仿生体系及其制备方法和应用。为解决现有miRNA核酸递送体系中存在的核酸稳定性差、负载效率低、靶向性不足、生物相容性有待提高以及难以在巨噬细胞内有效富集等问题，本发明以中空介孔二氧化硅纳米颗粒作为核心递送载体，经氨基化修饰后形成氨基化中空介孔二氧化硅纳米颗粒，负载miRNA调控核酸，并以巨噬细胞来源细胞膜囊泡进行仿生包覆，形成具有核‑膜结构的巨噬细胞靶向miRNA递送仿生体系。该体系能够实现miRNA调控核酸的高效负载，并兼具良好的生物相容性、体内安全性及巨噬细胞靶向递送能力，可作为巨噬细胞靶向核酸递送平台。

一种用于糖尿病性骨缺损的时序免疫代谢调控水凝胶系统及其制备方法

Resumen de: CN122537293A

本发明涉及生物医药技术领域，公开了一种用于糖尿病性骨缺损的时序免疫代谢调控水凝胶系统及其制备方法，用于糖尿病性骨缺损修复。其以氧化海藻酸钠‑明胶‑壳聚糖为自交联基底，集成免疫调节、二甲双胍/α‑酮戊二酸代谢调控、BMP‑2成骨诱导及谷胱甘肽抗氧化模块，通过活性氧靶向响应与三阶段时序释放，纠正免疫‑代谢‑氧化应激失衡，促进骨再生。制备工艺参数明确，生物相容性优，临床适用性强，可拓展至糖尿病相关软组织缺损修复。

一种核壳结构的金包覆磁性纳米颗粒及其制备方法和应用

Resumen de: CN122538781A

本发明公开了一种核壳结构的金包覆磁性纳米颗粒及其制备方法和应用。该纳米颗粒包括Fe3O4磁性纳米颗粒和包覆在其表面的金壳层，金壳层通过金种子介导生长形成，连续且均匀。制备方法包括：制备Fe3O4磁性纳米颗粒；对Fe3O4磁性纳米颗粒进行表面修饰；制备金种子；将金种子吸附在经表面修饰的Fe3O4磁性纳米颗粒表面；在金种子表面还原生长金壳层；经磁分离、洗涤得到产物。本发明制备的金壳层连续均匀、厚度精确可控，制备方法简便、重复性好，所得纳米颗粒化学稳定性强、磁响应性保持良好、易于表面功能化，可广泛应用于生物传感、免疫分析、磁分离、SERS检测、药物递送及癌症治疗等领域。

一种铈掺杂氧化锌复合纳米材料及其制备方法与应用

Resumen de: CN122537399A

本发明提供了一种铈掺杂氧化锌复合纳米材料的制备方法，包括以下步骤：（1）将壳聚糖加入到醋酸水溶液中，得到壳聚糖溶液；（2）将醋酸锌水溶液和醋酸铈水溶液依次滴加入到壳聚糖溶液中，50℃~70℃水浴反应20~40 min；（3）调节上述反应溶液的pH为8.0~10.0，50℃~70℃水浴反应1.5~2.5 h,离心干燥后，得到所述铈掺杂氧化锌复合纳米材料。本发明还提供了所述铈掺杂氧化锌复合纳米材料作为抗菌材料的应用，本发明所获得的铈掺杂氧化锌复合纳米材料，壳聚糖均匀包覆，材料结晶度高、分散性好；兼具高效、安全与稳定，为医用抗菌材料提供了支撑。

一种多抗原猴痘病毒RNA疫苗及其制备方法

Nº publicación: CN122542568A 11/08/2026

Solicitante:

中国人民解放军军事科学院军事医学研究院

Resumen de: CN119570817A

The invention relates to a multi-antigen monkey pox RNA vaccine and a preparation method thereof, and relates to the technical field of nucleic acid vaccines. The multi-antigen mRNA comprises a sequence encoding an antigenic polypeptide of the monkey pox virus or an antigenic fragment, variant or derivative thereof, the antigenic polypeptide or the antigenic fragment thereof comprising at least viral proteins of an intracellular mature virus and an extracellular envelope virus of the monkey pox virus. The invention provides a multi-antigen mRNA vaccine aiming at monkey pox viruses and a sequence, mRNA can express antigen protein in in-vitro cells, high-titer anti-monkey pox virus A29L, A33L, B6R, M1R and E8L protein IgG can be detected after a mouse is immunized, a high-titer anti-vaccinia Tian Tan strain live virus neutralizing antibody is generated, good immunogenicity is achieved, and the vaccine can be used for preparing the multi-antigen mRNA vaccine aiming at the monkey pox viruses. The important significance is realized on the prevention of the monkey pox virus.