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226
resultados
Última actualización
04/10/2026 [06:50:00]
Resultados 50 a 75 de 226
Resumen de: AU2025259439A1
Provided is a lipid mix composition for forming lipid nanoparticles (LNP) in association with a DNA, for use in transfecting a cell of hematopoietic lineage, the lipid mix composition comprising an ionizable lipid, a phospholipid, a stabilizer, and cholesterol. The LNP, a method of modifying cells of hematopoietic lineage using the LNP, a cell of hematopoietic lineage modified by the method, and a method of treatment for immune deficiency, cancer, autoimmune disease or genetic insufficiency are also provided.
Resumen de: EP4814036A1
0001 The present invention relates to a compound according to general formula (L<1>)(L<2>)(L<3>)
Resumen de: WO2025109567A1
Provided herein are emulsion composition comprising a medium chain triglyceride (MCT), preferably tricaprilin. Also, described here are additional ingredients that make these emulsion compositions more suitable for oral intake, such as one or more emulsifiers, buffers such as phosphate buffers, as well as optionally glycerol and a sweetener and/or a flavoring agent. Described herein are methods of making and using these emulsions. The emulsions described may be used for oral administration of tricaprilin to treat and/or prevent various diseases or disorders.
Resumen de: EP4813405A2
The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with an enzyme to afford efficient delivery of payload to a cell or tissue of interest via subcutaneous administration. In some embodiments, the present invention provides a process in which mRNA-loaded lipid nanoparticles are co-mixed with various amounts of hyaluronidase and administered via subcutaneous administration. The resulting payload can be efficiently delivered to the liver and other organs or tissues of a treated subject.
Resumen de: EP4813402A2
0001 Provided herein are compositions and methods of use thereof for the treatment of fungal infections and diseases.
Resumen de: EP4813398A2
The present disclosure provides a double-stranded RNAi agent for targeting and regulating HBV gene expression and a use thereof. The double-stranded RNAi agent comprises an antisense strand and a sense strand complementary to the antisense strand forming a duplex region. The nucleotide sequence of the antisense strand is as shown in SEQ ID NO: 11, or the nucleotide sequence of the antisense strand is a modified sequence of the sequence shown in SEQ ID NO: 11. Results from cell and animal experiments demonstrate that the double-stranded RNAi agent provided in the present disclosure can significantly reduce the expression of one or more HBV genes, block the viral life cycle, and can be used to develop drugs for treating diseases related to HBV gene expression.
Resumen de: WO2025111442A1
The present disclosure relates to lipids and compositions thereof. In various aspects of the invention, the compositions are lipid nanoparticle compositions used to deliver various DNA molecules and/or therapeutic agents to selected targets, such as cells for gene delivery, and/or to prevent or treat diseases or disorders in a subject in need thereof.
Resumen de: WO2025111297A1
This disclosure relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR). mRNA therapies of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects.
Resumen de: WO2025109111A1
The present disclosure relates to multimers capable of efficiently activating immune cells, such as T cells. In particular, it relates to multimers comprising at least two different ligands, such as a MHC-peptide and an anti-CD28 antibody, capable of binding to and together activate said immune cell. The disclosure further provides nucleic acid, vectors, and compositions encoding or comprising said multimers, and various methods for their use.
Resumen de: WO2025108545A1
The invention provides sustained delivery of nucleic acids from hydrophobic/organic media, formulations and biomaterial depots.
Resumen de: EP4813916A1
Problem To make it possible to provide a silicate mineral having an average particle diameter larger than several hundred nanometers without containing impurities such as crystalline silica.Solution The silicate mineral of the present invention has contents of each of crystalline silica and asbestos of 0.1 wt% or less. It is preferable that the mineral contains a carbonate, and it is preferable that it has an average particle diameter of 100 nm or more. Further, the mineral is suitably used in cosmetics, sanitary products, pharmaceuticals, and foods. The silicate mineral of the present invention is obtained by subjecting a natural silicate mineral to warm-water or hot-water treatment, or hydrothermal reaction treatment, at pH 9.4 or less.
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.
Resumen de: US20260258407A1
0000 Compositions and methods for reducing complement activation by introducing one or more alterations into a complement factor B (CFB) polynucleotide in a cell. In particular embodiments, the invention of the disclosure features a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying a CFB polynucleotide, where the modification is associated with reduced expression, and/or reduced activity of the CFB polypeptide encoded by the polynucleotide.
Resumen de: CN122827948A
本发明公开了一种仿生纳米铜离子药物的制备方法及其在奥希替尼耐药肺癌中的应用，制备包括：（1）H1975细胞膜囊泡的提取；（2）脂质体的制备；（3）脂质体杂化囊泡溶液的制备：将脂质体和H1975细胞膜囊泡按比例均匀混合，于挤出器中挤出一次或多次，得到仿生杂化囊泡；将所得仿生杂化囊泡进行透析处理，得脂质体杂化囊泡溶液；（4）将游离的奥希替尼和ES‑Cu按比例混合后加入到脂质体杂化囊泡溶液中，震荡孵育，经透析处理后，即可。本发明在H1975肿瘤细胞膜囊泡‑脂质体杂化囊泡中实现奥希替尼与ES‑Cu的高效主动共载，并通过同源靶向摄取，实现了“激酶抑制+铜死亡诱导”的协同作用。
Resumen de: CN122831857A
本发明涉及生物医药和药物制剂学领域，尤其涉及一种阳离子脂质化合物及其制备方法和应用。本发明通过在阳离子脂质结构中引入环酰亚胺结构单元，获得了一类新型阳离子脂质化合物，该类脂质可用于制备脂质纳米颗粒并递送mRNA等核酸分子，有利于调节脂质分子的疏水性、空间构型和降解特征，从而改善脂质纳米颗粒的形成能力、核酸包封能力和细胞递送效率。环酰亚胺作为可降解链接片段，具有良好的生物相容性。获得同时兼顾肝脏富集明显、递送效率与安全性的阳离子脂质化合物及包含阳离子脂质化合物的组合物。
Resumen de: CN122828012A
本发明属于生物医药与纳米递送技术领域，具体涉及一种脂质纳米颗粒及其制备方法与应用。本发明的胰腺靶向脂质纳米颗粒，包含脂质组分以及封装的核酸；其中脂质组分包含可离子化脂质、辅助磷脂、甾醇组分和PEG‑脂质。本发明的脂质纳米颗粒胰腺选择性高，胰腺辐射比例稳定在90%以上；且治疗用途广泛，在急性胰腺炎治疗、胰腺癌基因编辑和mRNA肿瘤疫苗三个应用场景中均验证了治疗效果，具有广阔的临床应用前景。
Resumen de: CN122828112A
本发明提供一种双靶点mRNA疫苗、制备方法及其应用，属于医用配制品技术领域。所述mRNA由目的基因转录得到，所述目的基因包括TROP2 scFv、NKG2D scFv，所述TROP2 scFv的核苷酸序列如序列表中SEQ ID NO.2所示，所述NKG2D scFv的核苷酸序列如序列表中SEQ ID NO.4所示。本发明的疫苗，通过靶向TROP2和NKG2DL两个抗原，增强抗原靶向性，可以提高对三阴性乳腺癌的治疗效果，本发明通过对TROP2 scFv和NKG2D scFv的核酸人工序列进行优化，提高了LNP/mRNA‑TROP2‑NKG2DL‑CAR疫苗对三阴性乳腺癌的治疗效果。
Resumen de: CN122828754A
本发明公开了一种复合纳米酶、负载其的水凝胶及其制备方法与应用，属于功能材料制备技术领域。针对现有铁基单原子纳米酶因平面四配位结构导致催化活性不足及单原子位点易团聚、稳定性差的问题，本发明复合纳米酶包括铁‑氮五配位单原子纳米酶和二氧化锰纳米颗粒，铁‑氮五配位单原子纳米酶具有中空多孔碳骨架，二氧化锰纳米颗粒沉积于其表面及孔道内，形成铁锰双活性位点协同催化结构。该材料通过轴向氮配位调控铁电子结构与自旋态提升本征活性，通过调控氧化应激微环境，利用双金属协同效应增强多种类酶活性，用于制备促进肩袖损伤后腱‑骨愈合的新型生物材料。
Resumen de: CN122827915A
本申请属于细胞外囊泡技术领域，涉及黄芩细胞外囊泡在制备具有抗炎、抗氧化功效的产品中的应用。本申请制备得到的黄芩细胞外囊泡具有较高的安全性，良好的细胞相容性，可以有效抑制炎症相关信号分子NO的过度生成，缓解细胞内氧化应激水平，调节巨噬细胞极化状态，抑制中性粒细胞中促炎性过氧化物酶活性，抑制中性粒细胞胞外捕获网的形成，且黄芩细胞外囊泡可以负载黄芩苷，黄芩苷和黄芩细胞外囊泡具有一定的相互配合，共同发挥抗炎和抗氧化的作用，可以应用于化妆品和药品中。
Resumen de: CN122832236A
本申请涉及医用高分子材料技术领域，具体涉及一种季铵型的可降解嵌段共聚物及其制备方法、纳米药物递送体系及应用。包括共聚物中间体和含芳香环结构单元，共聚物中间体包括聚乙二醇嵌段和聚氨酯嵌段。聚氨酯嵌段为双羟基单体、双羟基叔胺单体以及双异氰酸酯单体通过聚合反应形成的结构单元；聚乙二醇嵌段为通过一端具有氨基或羟基的聚乙二醇封端剂与所述聚氨酯嵌段的端异氰酸酯基反应形成的结构单元；含芳香环结构单元为含苯环单体通过共价键接枝于共聚物中间体上形成的结构单元。该可降解嵌段共聚物能够实现细胞摄取能力增强、肿瘤组织深层渗透、靶向富集效率提升以及微环境响应性降解的协同优化效果，显著提高抗肿瘤药物递送效率。
Resumen de: CN122827946A
本发明公开了一种负载姜酚的中空二氧化锰纳米颗粒及其制备方法和应用，包括以下步骤：制备纳米二氧化硅悬浮液；在超声条件下向二氧化硅悬浮液中加高锰酸钾溶液，搅拌后得核壳结构的SiO₂@MnO₂纳米颗粒；将SiO₂@MnO₂纳米颗粒分散于碳酸钠溶液并加热孵育，去除二氧化硅内核，得中空二氧化锰纳米颗粒；将中空二氧化锰纳米颗粒加入姜酚溶液，搅拌，制得负载姜酚的中空二氧化锰纳米颗粒。该纳米颗粒对椎间盘退变的病态微环境产生多重协同疗效。既能清除过量过氧化氢，还能生成氧气改善椎间盘内部的缺氧状态。其载体外壳能够在高氧化应激区域智能降解，实现姜酚的靶向、按需释放，协同发挥强效的抗炎和细胞保护作用，促进椎间盘修复。
Resumen de: CN122832013A
本发明涉及一种咪唑基二元非对称仿生脂质化合物及其制备方法和应用，咪唑基二元非对称仿生脂质结构仿天然磷脂与胆固醇设计，其疏水尾部由天然磷脂的二酰甘油酯与胆固醇的甾环共同组成，并且在咪唑环的2位上修饰有不同烷基链长的二甲氨基，形成具备非对称疏水尾部特征的咪唑基仿生脂质。咪唑基二元非对称仿生脂质具备良好的生物相容性，可替代脂质辅料（磷脂和胆固醇）与治疗性药物混合，能够制备得到治疗性药物被装载在内的载药脂质纳米颗粒，可作为药物递送系统用于脑靶向药物的制备；形成的药物递送系统能够有效穿越血脑屏障，较好地实现所装载治疗性药物的脑靶向递送。
Resumen de: CN122827924A
本发明属于生物技术领域，具体涉及一种纳米凝胶、纳米凝胶‑核酸复合物、其制备方法和应用。本发明的纳米凝胶具有可调控的核‑壳结构，或无核壳结构，形成核结构的物质包括式I化合物，所述式I化合物中各基团的定义如文中所述。纳米凝胶形成壳结构的材料包括中性聚合物和两性聚合物。本发明的纳米凝胶粒径均一，相较于在基因转染领域被广泛作为性能基准的bPEI25k及主流商业化试剂，该纳米凝胶在显著降低细胞毒性的同时，对多种核酸的转染效率实现了提升。
Resumen de: CN122828136A
一种载灯盏花素的系统，包括细胞外囊泡和灯盏花素，灯盏花素载于细胞外囊泡内。本发明提供的系统，能够靶向富集于受损的牙周微血管区域。灯盏花素的血管扩张活性与施万细胞外囊泡携带的促血管生成因子（如：VEGF、miR‑126）发挥协同作用，有效修复糖尿病所致的毛细血管基底膜增厚与内皮功能障碍，显著逆转牙周组织的退行性血管变化，恢复局部血供，能逆转血管退行性变，为牙周组织再生提供必要的营养与氧供基础。以该本发明的系统为活性成分制备的药物或医疗器械，可有效应用于牙周再生领域。
Nº publicación: CN122831824A 29/09/2026
Solicitante:
四川大学
Resumen de: CN122301710A
The invention provides a lipid compound as well as a preparation method and application thereof and a pharmaceutical composition, and belongs to the field of medicines. According to the present invention, the chemical structure of the lipid molecule is innovatively designed, and particularly the hydrophilic head group and/or the hydrophobic tail structure are/is specifically modified, such that the specific expression of mRNA in the liver can be achieved, the off-target effect of the non-target organ can be effectively avoided, and the important scientific significance and the wide application prospect can be provided.
BOPI
Sede Electrónica
