Alerta

NUCLEIC ACID DELIVERY COMPOSITIONS

Absstract of: WO2025049606A1

Compositions and methods for delivering nucleic acids (e.g. DNA) to cells are described herein. In some embodiments, the compositions and methods involve the use of lipid nanoparticles comprising cationic lipids, helper lipids, conjugated (PEG) lipids and structural (sterol) lipids wherein the helper lipid has at least one acyl chain bearing an unsaturation which improves in vivo delivery of the nucleic acid pay load.

中空メソポーラスシリカロッド、その製造方法および活性物質を担持する方法

Absstract of: WO2024248269A1

The present invention provides a hollow mesoporous silica rod, a method for preparing same, and a method for supporting an active substance. The hollow mesoporous silica rod of the present invention has a wide inner hollow and a large aspect ratio, can stably support a large amount of active substance, and can penetrate the surface of the skin to easily reach a predetermined depth inside the skin, thereby effectively delivering the active substance.

ハイブリッドエクソソーム及びその用途

Absstract of: WO2025216375A1

The present invention relates to a hybrid exosome formed by the fusion of a human cell-derived exosome and an artificial exosome. When an active ingredient labeled with a fluorescent material was attached or encapsulated in the hybrid exosome of the present invention and applied to cells, the active ingredient was observed in the cells. In addition, when the exosome was applied to skin tissue, fluorescence expression could also be observed in the dermal layer. Therefore, the hybrid exosome according to the present invention can be used as a novel drug delivery system.

脂质纳米颗粒制剂的稳定化

Absstract of: WO2025128958A1

Aspects of the disclosure relate to compositions and methods for improving the stability of lipid nanoparticles (LNPs). In some embodiments, the LNPs comprise one or more active pharmaceutical ingredients (API) encapsulated therein. The disclosure is based, in part, on compositions that directly or indirectly reduce the degradation (e.g., oxidation, hydrolysis, etc.) of one or more lipids components of the lipid nanoparticle. In some embodiments, the compositions comprise a citrate drug product matrix, EDTA drug product matrix, methionine drug product matrix, and/or a tryptophan drug product matrix. The disclosure also provides methods for storing compositions contemplated herein as well as methods for improving the stability of the API.

核酸脂质纳米颗粒制剂、其制备方法及其应用

Absstract of: CN122342735A

本发明公开了一种核酸脂质纳米颗粒制剂、其制备方法及其应用，具体公开了一种脂质纳米颗粒制剂，其包括脂质纳米颗粒和冻干保护剂；所述脂质纳米颗粒包括脂质载体和核酸；所述脂质载体包括可电离脂质化合物或其药学上可接受的盐、磷脂、甾醇和PEG脂质；所述可电离脂质化合物为式I所示的化合物。本发明的核酸脂质纳米颗粒制剂冻干后脂质纳米颗粒完整性较好、理化性质较佳或冻干制剂复溶后生物活性较好。

一种共载PD1抗体和过氧化氢酶的仿生纳米载体及其制备和应用

Absstract of: CN122342737A

本发明公开了一种共载PD‑1抗体和过氧化氢酶的纳米载体。具体地，所述纳米载体含有：30~60 wt%中性磷脂、20~50 wt%阴离子磷脂、1~20 wt%载脂蛋白和/或其模拟肽、0.1~10 wt%鱼精蛋白、0.01~5 wt%透明质酸、1~10 wt% aPD‑1抗体和1~10 wt%过氧化氢酶，按所述纳米载体的总重量为100 wt%计。本发明的纳米载体共同包封PD‑1抗体与过氧化氢酶，可以协同增强对乳腺癌的治疗效果。

一种生物杂交纳米酶机器人药丸及其制备方法和在调节肠道炎症与菌群失调的应用

Absstract of: CN122342733A

本发明公开了一种生物杂交纳米酶机器人药丸，具备多模式调节炎症性肠病中的肠道氧化还原稳态和菌群失调，步骤如下：步骤一、使用硝酸锌、乙酰丙酮铁和甲醇等药物合成金属有机框架前体，之后煅烧得到铁单原子纳米酶；步骤二、培养富集长双歧杆菌和人诱导多能干细胞，并分别提取二者的外泌体或外囊泡膜；步骤三、将纳米酶与益生菌囊泡膜与干细胞外泌体共挤出，制备生物膜‑纳米酶复合机器人；步骤四、将制得的生物膜‑纳米酶复合粒子封装于磁响应药丸中，形成工程化生物杂交纳米酶机器人药丸。工程化生物杂交纳米酶机器人药丸能够在进入生物体肠道后，在旋转磁场控制下靶向滞留于肠道炎症部位，之后在脉冲磁场作用下促进纳米酶释放并增强酶活性，从而提升治疗效果。工程化生物杂交纳米酶机器人药丸具备生物相容性和治疗安全性，在治疗消化道炎症领域具有广阔应用前景。

miR-215-5p/3p激动剂在制备治疗肠纤维化的药物中的应用

Absstract of: CN122342761A

本发明公开了miR‑215‑5p/3p激动剂在制备治疗肠纤维化的药物中的应用。miR‑215‑5p/3p进入肠上皮细胞后，能恢复其在疾病中的生理表达水平，进而抑制ZEB2和STC1介导的上皮细胞EMT信号通路，显著降低成纤维细胞活化、胶原沉积和ECM过度生成，延缓甚至逆转肠纤维化进展，为肠道纤维化提供了一种安全、有效、可推广的新型治疗方案。

一种治疗小儿便秘中药组合物及其制备方法

Absstract of: CN122342778A

本发明涉及治疗小儿便秘中药技术领域，且公开了一种治疗小儿便秘中药组合物及其制备方法，包括以下制备步骤：将生地黄、熟地黄、生白术、火麻仁、桑椹、白芍、莱菔子、枳壳、炙甘草混合得到中药原料，中药原料加水煎煮，收集煎煮液后，进行浓缩，得到浓缩浸膏；将浓缩浸膏、复合辅料、玉米淀粉和乙醇混合均匀，制粒，干燥，得到治疗小儿便秘中药组合物复合辅料复配生地黄、熟地黄、生白术、火麻仁、桑椹、白芍、莱菔子、枳壳、炙甘草中药材，具有治疗小儿便秘的功效，且复合辅料中的益生菌具有较高的活性，能够显著增加治疗效果。

一种靶向Siglec15肿瘤增强免疫治疗疗效的纳米粒及其制备方法与应用

Absstract of: CN122342734A

本发明公开了一种靶向Siglec15肿瘤增强免疫治疗疗效的纳米粒及其制备方法与应用，利用生物矿化法制备了Cu2+缀合的磷酸钙纳米粒(CuCaP)，并在纳米粒表面包覆了DTC和Si‑Siglec15RNA。CuCaP@DTC‑Si‑Siglec15在肿瘤酸性微环境发生降解，释放出Ca2+、Cu2+，Cu2+与DTC配位形成毒性CuET，通过通路诱导细胞凋亡，诱导肿瘤细胞ICD。另外，纳米粒释放的Ca2+，导致细胞Ca2+过载，诱导线粒体功能失常，也促进了肿瘤细胞凋亡和ICD效应。并借助Si‑Siglec15RNA以到达高表达Siglec15的肿瘤部位，避免了对正常细胞的毒性。通过联用aPD‑L1可激活强烈的抗肿瘤免疫反应，增强对肿瘤的杀伤，抑制肿瘤转移和复发。

基于嗜碱性粒细胞膜构建的仿生纳米颗粒及其制备方法、在制备治疗过敏性疾病药物中的应用

Absstract of: CN122342736A

本发明公开了一种基于嗜碱性粒细胞膜构建的仿生纳米颗粒及其制备方法、在制备治疗过敏性疾病药物中的应用，主要包括支撑体、包裹在支撑体外的大鼠嗜碱性白血病细胞系的细胞膜，该细胞膜表面具有FcεRI 蛋白。通过支撑体对细胞膜进行支撑，使其具有相对稳定的形状，形成仿生纳米颗粒。通过FcεRI 蛋白与IgE的高亲和力结合作用，降低IgE浓度，缓解IgE介导的过敏级联反应。

生体触媒組成物

Absstract of: WO2025008442A1

The present invention relates to a composition comprising a solid carrier, a lipase or a fragment thereof immobilized on the surface of the solid carrier, an agent which interacts with the lid domain of the lipase or a fragment thereof, and a protective layer to protect the lipase or the fragment thereof by embedding the lipase or the fragment thereof, wherein the lipase or a fragment thereof is in the open conformation. The present invention also relates to methods of producing said composition.

操作されたフェニルアラニンアンモニアリアーゼ（ＰＡＬ）を含む官能基化組成物

Absstract of: WO2025008450A1

The present invention relates to a composition comprising a solid carrier, an engineered phenylalanine ammonia lyase or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the engineered phenylalanine ammonia lyase or a fragment thereof by embedding the engineered phenylalanine ammonia lyase or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses thereof.

一种用于基因递送和内体逃逸的包裹了治疗性核酸的纳米虫

Absstract of: WO2025077898A1

An effective and safe composition comprises an anionic gold-polydopamine core-shell nanoworm as an alternative gene carrier for bypassing the bottleneck of endosomal entrapment. The nanoworm can be used in methods of delivering therapeutic oligonucleotides to a subject. A polydopamine shell supports the surface adsorption of nucleic acids. The anionic nucleic acid-encased nanoworm can then enter cells without transfection agents and activate the ClC3 H +/Cl - exchanger in late endosomes to mediate vesicular accumulation of H + and Cl -, which causes membrane rupture, and finally escape to cytosol without cell-penetrating peptides or mechanical stimuli. The nanoworm can be further used for programming cellular responses, i. e., primary macrophage polarization and stem cell differentiation, for the treatment of diseases, such as kidney fibrosis and acute liver injury.

应用于保护胰岛β细胞的功能和数量的胰岛素原mRNA疫苗

Absstract of: CN122321112A

本发明涉及mRNA疫苗的技术领域，具体公开了一种应用于保护胰岛β细胞的功能和数量的胰岛素原mRNA疫苗。所述疫苗由脂质纳米颗粒（LNP）和被所述LNP包裹的mRNA组成；所述mRNA为编码胰岛素原（proinsulin）的mRNA序列，且所述mRNA序列中所有的尿苷均被N1‑甲基假尿苷取代。本发明的mRNA疫苗实现了对胰岛β细胞数量与功能的双重保护作用，相较于现有仅关注血糖控制或免疫调节的干预手段，本发明从根源上延缓了1型糖尿病的发生进程，为1型糖尿病的预防和早期干预提供了全新的技术方案和药物选择。

造血干细胞和/或祖细胞递送方法及用于该方法的组合物

Absstract of: WO2025073056A1

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.

结合编码ELAVL3隐蔽外显子的核酸的试剂、药物组合物以及在管理神经疾病中的用途

Absstract of: WO2025076126A1

This disclosure relates to agents such as antisense oligonucleotides which bind nucleic acids encoding cryptic exons suppressing or preventing cryptic splicing of ELAVL3 and uses in treating or preventing TDP-43 related neurodegenerative or neurological diseases or conditions relates thereto. In certain embodiments, the antisense oligonucleotide is a nucleobase polymer capable of decreasing cellular levels of or expression of ELAVL3 cryptic exon RNA and increasing or restoring functional ELAVL3 protein expression substantially excluding cryptic exon RNA peptide sequence incorporation.

用于肺靶向递送核酸治疗剂的脂质复合物纳米颗粒

Absstract of: WO2025067389A1

A drug delivery system composing nanoparticles, in particular lipid nanoparticles ( LNPs ), and messenger RNA ( mRNA ), as well as methods for lung-targeted delivery of the nanoparticles.

脂质纳米颗粒制剂及其使用方法

Absstract of: WO2025065716A1

It relates, in part, to lipid nanoparticles (LNPs) comprising a payload molecules (i.e., nucleic acid molecules and/or therapeutic agents) and methods of use thereof for in vivo delivery of the payload molecules to a target cell or tissue. In certain embodiments, the payload molecules comprise polypeptides, nucleic acids molecules, enzymes, or any combination thereof. In certain embodiments, the target cell is a hematopoietic stem cells (HSCs). In certain embodiments, it relates to the use of the LNPs described herein for the treatment, prevention, and/or amelioration of diseases and/or disorders in a subject, including but not limited to blood disorders.

肝酶表达的补充

Absstract of: WO2025117858A1

Described herein are methods, compositions, and systems derived from uncultivated microorganisms useful supplementing liver enzyme deficiencies.

同源膜纳米靶向抗脓毒性急性肾损伤药物递送系统

Absstract of: CN122320904A

本发明涉及生物医学工程与纳米药物递送技术领域，具体公开了同源膜纳米靶向抗脓毒性急性肾损伤药物递送系统，包括：实心硅球制备；介孔化及刻蚀；药物负载；同源细胞膜制备；包覆与融合；表征与质量控制；本发明采用KTP 修饰RBCM 囊泡能提供同源表面蛋白/受体，实现对肾小管细胞的高选择性识别与内吞，显著提高药物在病变肾组织的累积与生物利用度，从而增强治疗效果并降低系统性暴露；中空、介孔结构提供高载药量与可控释放位点，外覆同源膜既抑制生理条件下的早期泄漏，又在炎症、酸性微环境或细胞内环境中促进药物释放，兼顾缓释与靶向激活两方面优势。

一种基于磷酸钙脂质纳米粒的双靶向DNA疫苗递送系统

Absstract of: CN122321115A

本发明公开一种基于磷酸钙脂质纳米粒的双靶向DNA疫苗递送系统，属于生物医药领域。该递送系统包括组蛋白 H1 与 HPV E6/E7 融合基因质粒的磷酸钙以及 DSPE‑PEG‑2000‑Mannose 的脂质双层膜，该系统通过甘露糖修饰实现树突状细胞靶向摄取，依托组蛋白 H1 介导 DNA 核转运，双重靶向显著提升抗原表达效率，体外与体内实验证实，该递送系统生物相容性好、稳定性高，可有效激活抗原特异性 T 细胞免疫应答，兼具 HPV 感染预防与宫颈癌治疗作用，为宫颈癌防治提供了新策略。

一种克服癌症TRAIL耐药性的复合载药外泌体及其制备方法

Absstract of: CN122320907A

本发明公开了一种克服癌症TRAIL耐药性的复合载药外泌体及其制备方法，涉及复合纳米药物制备技术领域，包括，工程化外泌体，所述工程化外泌体来源于能够表达肿瘤坏死因子相关凋亡诱导配体TRAIL的细胞，使得所述外泌体携带有所述TRAIL蛋白；以及靶向利钠肽受体A基因的干扰RNA，所述干扰RNA被包载于所述工程化外泌体内部；其中，所述工程化外泌体与所述干扰RNA协同作用，通过所述干扰RNA沉默NPRA基因以克服癌细胞对TRAIL的耐药性。本发明通过沉默NPRA基因，多通路重塑肿瘤细胞凋亡敏感性，从而高效克服TRAIL耐药性。

一种基于微流控芯片制备的二氢槲皮素油溶微囊及其制备方法与应用

Absstract of: CN122320903A

本发明公开了一种基于微流控芯片制备的二氢槲皮素油溶微囊及其制备方法与应用。本发明的制备方法：将二氢槲皮素和助溶剂在加热条件下溶解均匀，得到分散相；将表面活性剂和油脂在加热条件下溶解均匀，之后加入乳酸杆菌发酵溶胞产物得到混合液，将所得混合液进行高压均质处理，得到连续相；采用微流控芯片，将分散相和连续相通过注射泵分别泵入芯片的对应通道；在芯片的交叉口，连续相对分散相进行剪切、挤压并包裹，形成单分散的二氢槲皮素油溶微囊。本发明所使用的方法利用微流控芯片的精准流体控制能力，实现微囊粒径、结构的均一可控，工艺连续、重复性好，包封率高，并能有效提高二氢槲皮素的溶解度和稳定性。

一种EV71病毒mRNA疫苗及其应用

Nº publicación: CN122321116A 03/07/2026

Applicant:

盐城师范学院

Absstract of: CN122321116A

本发明涉及的是一种EV71病毒mRNA疫苗及其应用，这种EV71病毒mRNA疫苗包含编码EV71病毒vp1基因的mRNA分子，mRNA分子由脂质纳米颗粒包裹；mRNA分子的核酸序列如SEQ ID NO.1所示；mRNA分子的编码氨基酸的序列如SEQ ID NO.2所示。本发明提供的mRNA疫苗，显著提高小鼠体内产生的抗EV71病毒vp1的中和抗体滴度和特异性细胞免疫反应。