Alerta

肿瘤微环境响应型级联生成活性氧的纳米平台及其制备方法、应用

Resumen de: CN119857110A

本发明提供一种肿瘤微环境响应型级联生成活性氧的纳米平台及其制备方法，该纳米平台包括FeOOH纳米纺锤体，所述FeOOH纳米纺锤体的外层包覆有四硫键掺入的树枝状介孔有机硅纳米粒子(DMOS NPs)，形成纳米颗粒DMOS@FeOOH。本发明的纳米平台能够在肿瘤组织中通过级联反应高效生成活性氧(ROS)，并实现对肿瘤细胞的选择性杀伤，具有较高的特异性、良好的治疗效果和较低的对正常组织的副作用，是一种具有广阔应用前景的肿瘤治疗策略。

ｍＲＮＡ送達方法およびその組成物

Resumen de: AU2023244571A1

The present disclosure provides a lipid nanoparticle for extrahepatic delivery of mRNA, the lipid nanoparticle comprising: (i) mRNA cargo; (ii) a phosphatidylcholine lipid content of from 30 mol% to 70 mol%; (iii) a ionizable, cationic lipid content of from 5 mol% to 50 mol%; (iv) a sterol selected from cholesterol or a derivative thereof; and (v) a hydrophilic polymer-lipid conjugate that is present at a lipid content of 0.5 mol% to 5 mol. Further provided is a lipid nanoparticle comprising encapsulated mRNA and 20 to 70 mol% of a phosphatidylcholine lipid, an ionizable lipid; and at least one of a sterol and a hydrophilic polymer-lipid conjugate, the lipid nanoparticle exhibiting at least a 10% increase in gene expression of the mRNA in vivo as measured in one or more extrahepatic organs or tissues. Further provided are methods of administration of such lipid nanoparticles.

肝臓へのプラスミドＤＮＡデリバリーのための脂質ナノ粒子の組成物およびその調製方法

Resumen de: WO2023192503A1

Lipid nanoparticle formulations with cell type specific transfection activity and capable of producing Th1 and/or Th2 response in vivo and their use for plasmid DNA or mRNA delivery is disclosed.

高ステロール含有脂質ナノ粒子

Resumen de: CN118922178A

The present disclosure provides a lipid nanoparticle comprising: a nucleic acid load molecule; sterols or derivatives thereof present in high levels; a neutral lipid; lipid can be ionized; and a hydrophilic polymer-lipid conjugate present in a content of between 0.5 and 3 mol%, wherein each mol% content is relative to the total lipid present in the lipid nanoparticles.

一种靶向治疗黄褐斑的siRNA脂质纳米颗粒及其制备方法和应用

Resumen de: CN119857085A

本发明提供了一种靶向治疗黄褐斑的siRNA脂质纳米颗粒及其制备方法和应用，属于生物医药技术领域。本发明提供了一种治疗黄褐斑的LNP‑siRNAs，包括靶向抑制黑色素形成关键分子的siRNAs。本发明筛选出有效的siRNA序列，在细胞中进行了验证，而后将有明显抑制作用的siRNAs包装成LNP‑siRNAs，评估LNP‑siRNAs的包装质量，以及将LNP‑siRNAs用于细胞和动物，评估其对黑色素的抑制效应。本发明包装成的LNP‑siRNAs后用于黑色素细胞和动物皮肤，发现其能有效抑制黑色素的形成，可作为筛选并制作临床治疗黄褐斑的潜在药物。

一种石萝藦提取物纳米粒组合物、制备方法及应用

Resumen de: CN119857120A

本发明提供了一种石萝藦提取物纳米粒、制备方法及应用，属于生物医药技术领域。纳米粒由囊心填充物、载体材料、表面活性剂、乳化剂和水组成；所述囊心填充物为石萝藦提取物；所述载体材料为聚乙二醇‑聚乳酸。配比如下：L‑LA、PEG和辛酸亚锡的摩尔比为100:1:1制得聚乙二醇‑聚乳酸(PEG‑PLLA)，PEG‑PLLA与PC重量比为1:3制得PC@PEG‑PLLA纳米颗粒。PC@PEG‑PLLA NPs的平均粒径为(113.1±2.5)nm，PDI为(0.173±0.020)，电势为(‑46.7±1.9)mV，同时具有良好的稳定性。作为治疗自身免疫性肝炎(AIH)的药物具有较高的价值。

编码流感病毒抗原的自扩增RNA

Resumen de: MX2025000397A

Self-amplifying RNA (saRNA) molecules encoding an influenza virus antigen and methods of use thereof are disclosed herein.

一种治疗肝细胞癌的核酸药物及其制备方法和应用

Resumen de: CN119857150A

本发明提供了一种治疗肝细胞癌的核酸药物制剂及其制备方法和应用，属于核酸药物技术领域。本发明提供了一种核酸递送载体，包括中性胞苷脂材DNCA、胱氨酸骨架阳离子脂材CLD和辅助脂材1,2‑二硬脂酰基‑sn‑甘油‑3‑磷酸乙醇胺‑聚乙二醇DSPE‑PEG按特定比例复配得到。本发明还提供了一种治疗肝细胞癌的核酸药物，包括核酸递送载体、包裹在所述核酸递送载体内靶向IGF1RmRNA的siRNA和细胞转染液。所述核酸药物可高效靶向小鼠肝细胞皮下瘤部位，显著抑制肿瘤生长，且无肝肾毒性。本发明技术方案为抗该肝细胞癌的siRNA药物在临床的广泛应用奠定了基础。

ＣＩＤＥＢを標的にするｓｉＲＮＡを用いて肝疾患を処置するための組成物および方法

Resumen de: MX2024012237A

Disclosed herein are compositions comprising siRNAs capable of downregulating Cell Death-Inducing DFF45-like Effector Protein B (<i>CIDEB</i>) gene expression or a variant thereof. Also disclosed herein are methods of using such compositions in the treatment of a liver disease or injury, such as fatty liver disease (FLD), non-alcoholic fatty liver disease (NAFLD) or non-alcoholic steatohepatitis (NASH).

細胞変換

Resumen de: WO2023199036A1

The present application relates to a nucleic acid molecule comprising a promoter operably linked to a nucleic acid sequence encoding Myocyte Enhancer Factor 2C (MEF2C), or a functional variant thereof, wherein the promoter is for expression of MEF2C in macroglia. Also provided are vectors, compositions, products, methods, cells, medical uses and methods of treatment.

一种用于治疗阿尔兹海默症的双药纳米颗粒及其制备方法和应用

Resumen de: CN119837843A

本发明属于生物医药技术领域，公开一种用于治疗阿尔兹海默症的双药纳米颗粒及其制备方法和应用，将Aβ抑制剂通过化学键连接在两亲性聚合物上，连有Aβ抑制剂的两亲性聚合物和STING抑制剂混合，在超声下缓慢的滴加到水中，超声、过滤取上层，得到双药纳米颗粒，本发明制备的双药纳米颗粒具有较好的生物相容性、稳定性，能穿过血脑屏障并同时对Aβ和小胶质细胞STING通路作用，解聚Aβ并促进Aβ清除、抑制小胶质细胞STING通路激活，减少释放炎症因子损伤神经元；能明显减少脑部沉积的Aβ和显著抑制脑内小胶质细胞STING通路的激活，明显的改善了小鼠记忆能力、空间识别能力，改善了脑部炎性微环境。

活性氧响应的紫杉醇前药及其高载药纳米制剂及制备方法

Resumen de: CN119841854A

本发明涉及药物技术领域，具体提供了一种活性氧响应的紫杉醇前药，具有式Ⅰ或式Ⅱ所示结构。本发明针对紫杉醇疏水聚集问题，对紫杉醇药物进行了改性，改性后的紫杉醇药物水溶性大大增强，并获得了自组装性能，在水溶液中可以进行自组装得到纳米粒子，所述纳米粒子具有超高的载药量和载药效率，并且可以特异性响应肿瘤微环境中高浓度的活性氧，既能改善传统紫杉醇纳米制剂载药量的不足，又能在肿瘤位置响应释放紫杉醇，有利于减少药物用量，降低毒副作用，对提升患者预后，延长患者生存期具有重要意义。

新型共聚物

Resumen de: TW202412846A

The present invention addresses the problem of providing a novel copolymer that can be used for a drug delivery technique. The present invention relates to a copolymer obtained by binding a target-affinity molecule to a copolymer X comprising structural units represented by formula (A), (B), and (C). In the formulae, R1, R2, and R3 are the same or different and each represent a hydrogen atom or a C1-3 alkyl group; R4 represents a C1-3 alkyl group; R5 represents a hydrogen atom, a C1-18 alkyl group, an optionally substituted, 3- to 8-membered cycloalkyl group, an adamantyl group, an optionally substituted C6-18 aryl group, or an optionally substituted, 5- to 10-membered heteroaryl group; X1, X2, and X3 are the same or different and each represent an oxygen atom, a sulfur atom, or N-R7; R6 represents a hydrogen atom, a leaving group, or a linker; R7 represents a hydrogen atom or a C1-3 alkyl group; m is an integer of 1-100; and n is an integer of 0-3..

一种新型AIE光热剂及其制备方法与应用

Resumen de: CN119841848A

本发明公开了一种新型AIE光热剂及其制备方法与应用。本发明的AIE光热剂的结构式如下所示，其发光性能和光热转化性能优越，可用于制备肿瘤诊断试剂或肿瘤治疗药物。本发明制备的负载AIE光热剂和STING激动剂的仿生纳米粒子能够实现药物至肿瘤组织高效递送，基于AIE分子的多模态成像能力对肿瘤进行靶向示踪，其光热效应能够增强肿瘤细胞ICD和DC细胞的STING通路激活，增增强抗肿瘤免疫治疗，最终抑制远端肿瘤生长，肿瘤复发和再转移。本发明材料制备工艺简单，成本较低，适用于规模生产和临床医疗使用。#imgabs0#

一种基于超氧化物歧化酶的纳米透皮递送系统及其制备方法

Resumen de: CN119837842A

本发明提供一种基于超氧化物歧化酶的纳米透皮递送系统，为小分子活性成分‑超氧化物歧化酶‑聚合物壳层的胶囊复合结构，小分子活性成分负载在超氧化物歧化酶中，超氧化物歧化酶表面被聚合物壳层包覆，所述基于超氧化物歧化酶的活性成分透皮递送系统直径为20‑100nm，聚合物壳层厚度为7.5‑52.5nm。本发明递送系统中聚合物壳层为超氧化物歧化酶和具有抗氧化作用的小分子功能物质提供保护作用，所述胶囊复合结构为纳米凝胶的形式，纳米凝胶不仅促进其渗透深度，而且减轻了其对皮肤的刺激作用。本发明原料生物友好，制备方法简单，收率高，可以工业化大规模生产。

一种肿瘤免疫微环境调控型光激活铁死亡纳米粒及其制备方法和应用

Resumen de: CN119838009A

本发明公开了一种肿瘤免疫微环境调控型光激活铁死亡纳米粒及其制备方法和应用，涉及生物医药技术领域。该制备方法包括以下步骤：将白蛋白和铁蛋白溶于水中，调节pH为碱性后加入光敏剂和硫辛酸，混合后再加入溶剂，进行混合反应，之后加入交联剂，继续反应后，经过离心和干燥处理，得到所述肿瘤免疫微环境调控型光激活铁死亡纳米粒。该肿瘤免疫微环境调控型光激活铁死亡纳米粒通过诱导肿瘤细胞铁死亡、激活树突状细胞和调控M2型肿瘤相关巨噬细胞的M1型极化来逆转肿瘤免疫抑制微环境，进而增强肿瘤免疫治疗效果。

一种用于皮肤抗炎的蓝萼甲素纳米透皮递送系统及其制备方法

Resumen de: CN119837841A

本发明提供一种用于皮肤抗炎的蓝萼甲素纳米透皮递送系统，为蛋白‑蓝萼甲素‑聚合物壳层的胶囊复合结构，所述蛋白对皮肤有亲和作用，蓝萼甲素负载于蛋白中，聚合物层包覆在蛋白的表面，所述蓝萼甲素纳米透皮递送系统尺寸为30‑80nm，聚合物壳层厚度为10‑35nm。聚合物外壳表面性质可以根据透皮递送的深度需求进行调控，使得蛋白质突破皮肤屏障被高效递送至皮肤深处再将蓝萼甲素缓慢释放，靶向缓解皮肤炎症，解决蓝萼甲素治疗皮肤炎症因透皮效果不理想而治疗效果低下的缺陷。

一种抗原MNA及其应用和制备的疫苗、药物

Resumen de: CN119841930A

本发明公开了一种抗原MNA及其应用和制备的疫苗、药物，属于生物医药技术领域。本发明发现并设计的新型肿瘤新抗原（MNA）具有强免疫原性，能够有效激活T细胞免疫反应；且仅在肿瘤细胞中表达，不受中枢免疫耐受的影响，能够精准靶向肿瘤细胞，减少对正常组织的损伤。且利用circRNA作为新抗原MNA的表达载体，稳定性高，高效表达，诱导强烈的新抗原特异性T细胞反应，无需佐剂。本发明为肿瘤新抗原疫苗的开发和个性化免疫治疗提供了新的思路，有助于推广至更多的高突变负荷肿瘤类型，具有广阔的临床应用前景。

SUBEROYLANILIDE HYDROXAMIC ACID (SAHA) DRUGS, CONJUGATES, AND NANOPARTICLES, AND METHODS OF USE THEREOF

Resumen de: US2025120928A1

The present disclosure provides polymer-drug conjugates comprising suberoylanilide hydroxamic acid (SAHA), derivatives thereof, and salts thereof. The disclosure further provides nanoparticles comprising the polymer-drug conjugate. The disclosure provides methods of treating a disease or disorder in a subject by administering the conjugate or a plurality of nanoparticles comprising the conjugate to the subject. In some embodiments, the disease or disorder is sepsis, septic shock, hemorrhagic shock, or poly-trauma.

新規高分子化合物及びそれを含む核酸送達用組成物

Resumen de: US2025115715A1

Provided are a polymer compound and a composition for nucleic acid delivery comprising the same, wherein the polymer compound has a high binding affinity to nucleic acids and effectively protects nucleic acids from nucleases, thereby improving the nucleic acid delivery effect and stability.

LIPID NANOPARTICLES

Resumen de: US2025120914A1

The present disclosure relates to lipid nanoparticles and methods of delivering active agents to target organs, tissues, or cells by utilizing the lipid nanoparticles.

Method and Apparatus for Drug Delivery to Surgical Margins

Resumen de: US2025120904A1

A method and apparatus for the targeted delivery of chemotherapy to a surgical cavity, consisting of a triggered nanoparticle encapsulating a therapeutic agent and an energy delivery device that applied trigger energy to the surgical cavity. Following surgical removal of a cancerous tumor, the nanoparticle is administered, and the energy delivery device applies trigger energy to the surgical cavity and proximal tissue. The goal is the delivery of a therapeutic drug dose to cancerous and precancerous cells remaining after surgery, to prevent local tumor recurrence.

COMPOSITIONS AND METHODS RELATED TO HIV-1 IMMUNOGENS

Resumen de: US2025122247A1

The present invention provides HIV-1 vaccine immunogens. Some of the immunogens contain a soluble gp140-derived protein that harbors a modified N-terminus of the HR1 region in gp41. Some of the immunogens contain an HIV-1 Env-derived trimer protein that is presented on a nanoparticle platform. The invention also provides methods of using the HIV-1 vaccine immunogens for eliciting an immune response or treating HIV infections.

SARS-COV-2 VACCINES

Resumen de: US2025121052A1

Disclosed herein are vaccine compositions that include SARS-CoV-2 MHC epitope-encoding cassettes and/or full-length SARS-CoV-2 proteins. Also disclosed are nucleotides, cells, and methods associated with the compositions including their use as vaccines.

BRAIN TARGETED NANOPARTICLES OR CONJUGATES AND METHODS OF USE THEREOF

Nº publicación: US2025121098A1 17/04/2025

Solicitante:

ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIV [US]
Arizona Board of Regents on behalf of Arizona State University

Resumen de: US2025121098A1

The present invention provides nanoparticles or conjugates comprising at least one ligand that selectively targets major facilitator superfamily domain-containing protein-2a (MFSD2A). In various embodiments, the nanoparticles or conjugates of the invention target at least one cell comprising MFSD2A (e.g., endothelial cells of blood brain barrier). In some embodiments, the nanoparticles or conjugates of the invention cross the blood brain barrier and/or blood retinal barrier. In other aspects, the present invention relates to methods for in vivo delivery of diagnostic and/or therapeutic agents to a brain. In other aspects, the present invention relates to methods of preventing or treating a neurological or cognitive disease or disorder using the nanoparticles or conjugates of the invention.