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LIPID-BASED NANOPARTICLES COMPRISING IL-35

Resumen de: WO2025133115A1

The invention relates to lipid-based nanoparticles comprising mRNA molecule(s) encoding interleukin 35 (IL-35), and their use to treat inflammatory and/or auto-immune diseases.

LIPID NANOPARTICLES HAVING NON-POLAR LIPIDS FOR NUCLEIC ACID DELIVERY TO THE LIVER

Resumen de: WO2025129333A1

The present disclosure provides a method for delivering a nucleic acid cargo to the liver of a subject, the method comprising administering to the subject a lipid nanoparticle encapsulating the nucleic acid and comprising a non-polar lipid. The lipid nanoparticle may exhibit a blebbed morphology, despite the absence or low levels of phospholipid. The disclosure further provides compositions for delivering nucleic acid cargo to the liver in order to treat various diseases, disorders and conditions in the subject.

LIPID DELIVERY VEHICLE

Resumen de: WO2025129271A1

The claimed invention is to a lipid delivery vehicle for eliciting a tolerogenic immune response in a subject, comprising nuclear factor-KB (NF-KB) inhibitor and a peptide antigen, wherein the lipid delivery vehicle comprises 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine (POPC) and l-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol (POPG).

COMPOSITIONS AND METHODS

Resumen de: WO2025133105A1

A composition comprising: (a) a cationically ionizable lipid; (b) cholesterol; (c) a cholesterol ester; and (d) a neutral lipid; wherein the cholesterol ester (c) is present in an amount of up to about 75 mol% of the total of cholesterol (b) and cholesterol ester (c) present in the composition, is disclosed. Methods of preparing the composition, nucleic acid-lipid particle compositions and methods of preparing them using the composition, and their use in medicine, are also disclosed.

MALIC AND GLUTARIC ACID BASED IONIZABLE LIPIDS

Resumen de: WO2025134071A1

Provided herein are ionizable or cationic lipids of Formula (I): (I), or pharmaceutically acceptable salts thereof. The ionizable or cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

LIPID NANOPARTICLES

Resumen de: AU2024403484A1

The present invention relates to the field of lipid nanoparticles (LNP); more specifically comprising an ionizable lipid, a phospholipid, a sterol, a PEG lipid and one or more nucleic acids. The LNP's of the present invention are characterized in having an ionizable lipid content of above 55 mol% and a ratio of cholesterol to phospholipid of 1 : 1 or less, such as about 1 : 2 or less. The present invention provides use of the LNP's for extrahepatic deliveryof nucleic acid molecules, specifically mRNA; thereby making them highly suitable for use in the treatment of cancer, autoimmune diseases, genetic disorders, hematological disorders, aging, fibrosis, neurological disorders, cardiovascular diseases or infectious diseases.

USES OF LIPID NANOPARTICLE COMPOSITIONS

Resumen de: WO2025130965A1

A method of preventing, treating or managing a disease in an organ or a tissue in a subject, comprising administration of a lipid nanoparticle comprising a messenger RNA (mRNA) encoding a protein to the subject having the disease in an organ or tissue via a systemic administration route, wherein the half-life of the protein is less than certain value as determined in mouse serum, and wherein the comparative accumulation level of the protein in the organ or tissue when the lipid nanoparticle comprising the mRNA encoding the protein is administered via the systemic administrative route is higher than the comparative accumulation level of the protein in the organ or tissue when the protein is administered via the same systemic administration route.

COMPOSITIONS AND METHODS FOR ENHANCED GENOME EDITING USING CAS9 FUSION PROTEINS

Resumen de: WO2025137069A1

The present invention relates to nucleic acids comprising a sequence encoding a fusion protein comprising Cas9 or a variant or fragment thereof, Rad18 or Rad52 or a variant or fragment thereof, and CtIP or a variant or fragment thereof, vectors encoding the same, pharmaceutical compositions thereof and methods of modifying a genomic sequence in cells by administering the compositions.

MULTI-ANTIGENIC CANCER VACCINES, DENDRITIC CELL TARGETING AGENTS AND ASSOCIATED METHODS

Resumen de: WO2025137170A1

The present invention provides compositions including nanoparticles and at least two polynucleotide or protein antigens separated by a linker. The fusion polynucleotides or fusion polypeptides may encode or may be cancer antigens. The nanoparticles are engineered to target dendritic cells. Methods of using the compositions and cells treated with the compositions are also provided.

BASE EDITORS WITH MODIFIED EDITING WINDOWS

Resumen de: WO2025137465A1

Base editors with modified editing windows, such as base editors containing a deaminase domain and a nucleic acid programmable DNA binding protein domain (napDNAbp), where the napDNAbp is modified relative to a reference napDNAbp to alter the base editing window of the base editors relative to a reference base editor containing the deaminase domain and the reference napDNAbp. In some embodiments, the base editing window is shifted and/or expanded relative to the reference base editor.

METHOD FOR MANUFACTURING NANOBUBBLE-BASED DRUG CARRIER USING FOCUSED ULTRASOUND TECHNOLOGY AND NANOBUBBLE-BASED DRUG CARRIER MANUFACTURED THEREBY

Resumen de: EP4830722A1

The present disclosure relates to a method for manufacturing a nanobubble-based drug delivery vehicle using a focused ultrasonic technology, and a nanobubble-based drug delivery vehicle manufactured thereby, and more specifically, to a method for manufacturing a drug delivery vehicle using a focused ultrasonic technology, the drug delivery vehicle including a shell, and a drug and nanobubbles contained inside the shell, and a nanobubble-based drug delivery vehicle manufactured thereby.

FORMULATIONS

Resumen de: EP4830709A2

0001 The invention provides lipid nanoparticle-based compositions with improved properties for delivery of biologically active agents, engineered cells, and methods for delivery of the agents.

特定の化合物を含む透析液組成物

Resumen de: EP4501332A1

The present invention relates to a dialysate composition having a composition having a composition allowing for a balance with blood comprising mineral salts and a pH buffer, and comprising at least one iron oxide nanoparticle (Fe²⁺ and/or Fe³⁺) coated with at least one polymeric compound.

一种高效穿透血脑屏障的负载多肽的黄精外泌体及其制备方法和应用

Resumen de: CN122624620A

The invention discloses a preparation method of polypeptide-loaded polygonatum sibiricum exosome capable of efficiently penetrating a blood brain barrier, which comprises the following steps: (1) extracting polygonatum sibiricum exosome from fresh polygonatum sibiricum to obtain purified polygonatum sibiricum exosome; and (2) co-incubating the polypeptide and the purified polygonatum sibiricum exosome to obtain the polypeptide-loaded polygonatum sibiricum exosome capable of efficiently penetrating the blood brain barrier. The prepared polygonatum kingianum exosome is complete in vesicle structure, polypeptide is loaded on the polygonatum kingianum exosome, and the encapsulation efficiency is high and reaches 91.23%. The polypeptide-loaded polygonatum kingianum exosome prepared by the preparation method disclosed by the invention has higher oxidation resistance and is easier to penetrate through a blood brain barrier. On one hand, the polygonatum kingianum exosome efficiently delivers the polypeptide, so that the polypeptide can better play a role in improving learning and memory, and on the other hand, the polygonatum kingianum exosome and the polypeptide have a synergistic effect, so that the effect of improving the learning and memory is jointly enhanced. The invention provides a new thought for the application of the functional factor delivery system in central nervous system diseases, and has a wide application prospect.

一种具有双酶活性的纳米诊疗制剂及其制备方法和应用

Resumen de: CN122973653A

本发明公开了一种具有双酶活性的纳米诊疗制剂及其制备方法和应用，属于纳米材料技术领域。该纳米制剂以锰四羧基苯基卟啉（Mn‑TCPP）为配体、锆氧簇为金属节点构建具有类超氧化物歧化酶（SOD）活性的金属有机框架Mn‑PCN，并在其表面负载铈纳米酶，利用Ce3+/Ce4+可逆转化赋予其类SOD和类过氧化氢酶（CAT）双活性，进一步用透明质酸（HA）修饰最外层以实现CD44受体靶向。该制剂通过Mn‑PCN与铈纳米酶的级联催化协同清除超氧阴离子和过氧化氢，缓解氧化应激与缺氧，并诱导巨噬细胞从促炎M1表型向抗炎M2表型极化，从而重塑类风湿关节炎免疫微环境。本发明提供的纳米制剂制备方法可控，具有良好的生物相容性和靶向性，在类风湿关节炎等氧化应激相关疾病的治疗中具有应用前景。

一种促炎症消退介质的稳定化仿生纳米制剂及其制备方法和应用

Resumen de: CN122973386A

本发明涉及生物医用材料与纳米药物递送技术领域，具体涉及一种高稳定性的促炎症消退介质仿生纳米制剂及其制备方法和应用。组成为PM‑H‑ZIF‑8@SPM。其中SPM选自RvE1、RvD1或RvE2中的任意一种。H‑ZIF‑8为组氨酸修饰的沸石咪唑酯骨架材料，PM为天然血小板膜。本发明利用H‑ZIF‑8的多孔结构及组氨酸修饰带来的高亲和力，将SPM稳定地封装于内核，有效隔离其与体内降解酶的接触，显著提高其稳定性和体内半衰期。利用天然血小板膜外层进行表面“伪装”，借助血小板膜表面的“自我识别”蛋白（如CD47）逃避免疫系统清除实现长循环；同时，利用血小板膜与受损血管内皮特异性结合的天然属性，实现对肺部炎症部位的主动靶向富集。利用H‑ZIF‑8在酸性条件下结构解体的特性，使载体在到达pH值较低的肺部炎症微环境后，实现药物在病灶区域的“按需”精准释放。

负载小干扰RNA的肿瘤基因药物及其制法和应用

Resumen de: CN122973467A

本发明提供了负载小干扰RNA的肿瘤基因药物及其制法和应用。具体地，本发明提供了负载小干扰RNA(siRNA)的肿瘤基因药物及其肿瘤基因药物，所述负载小干扰RNA的全细胞肿瘤基因药物包括(a)阳离子脂质纳米粒；和(b)抑制PDE4D基因表达的小干扰RNA，其中，所述阳离子脂质纳米粒的Zeta电位为8‑20mv；所述阳离子脂质纳米粒的平均粒径为80‑160nm。本发明的肿瘤基因药物具有高细胞摄取效率和高载药量，通过特异性下调PDE4d表达，从而提高抗原提呈细胞的抗原呈递能力。此外，本发明的肿瘤基因药物与肿瘤抗原联用，产生强烈的适应性免疫应答水平，从而协同且高效地激发免疫反应，显著抑制肿瘤生长。

一种双硒类脂质化合物、核酸药物递送系统及其制备方法

Resumen de: CN122978020A

本发明涉及一种双硒类脂质化合物、核酸药物递送系统及其制备方法。所述双硒类脂质化合物是一种新型的双硒类可电离脂质化合物，其制备方法简单，具有高效脾脏靶向性，且生物相容性良好，能够将核酸安全高效地递送入脾脏。

用于递送系统的可电离阳离子脂质化合物及其用途

Resumen de: WO2025194409A1

The present disclosure relates to novel lipids for delivering one or more biologically active molecules to a subject. The present application also relates to a composition or a nanoparticle comprising said lipid, methods of preparing said lipid, use of said lipid and method of using said lipid.

新颖的基于同型半胱氨酸的脂质及其用于递送核酸的用途

Resumen de: WO2025196065A1

Disclosed are lipids which can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

一种含PLGA-PEG的核酸脂质纳米颗粒及其制备方法和应用

Resumen de: CN122973387A

本发明属于核酸递送与纳米药物制剂技术领域，涉及一种含PLGA‑PEG的核酸脂质纳米颗粒、制备方法及应用。该颗粒包括SM‑102、辅助磷脂、胆固醇、PLGA‑PEG和被包载核酸，PLGA‑PEG作为表面稳定组分部分或全部替代DMG‑PEG；将含脂质的有机相与含核酸的酸性水相快速混合，使核酸结合并自组装，随后去除乙醇并置换缓冲液。在一实施方式中，PLGA‑LNP粒径约108nm、PDI为0.18、核酸包封率为99.5%。所述颗粒可在保持核酸表达的同时降低给药后TNF‑α和/或IL‑6释放，并可负载VEGF mRNA用于口腔黏膜损伤、口腔溃疡或创面修复。

基于生源性肌酸骨架的可电离脂质、脂质纳米颗粒及其核酸递送应用

Resumen de: CN122978008A

本发明属于生物医药技术领域，具体涉及基于生源性肌酸骨架的可电离脂质、脂质纳米颗粒及其核酸递送应用。基于肌酸的可电离脂质，具有通式(I)所示的化学结构：；其中，n为1~6的整数，R1和R2各自独立的选自碳原子数为6~20的烷基、碳原子数为6~20的含醚键的烷基、碳原子数为6~20的烯基、碳原子数为6~20的含醚键的烯基、碳原子数为6~20的炔基、碳原子数为6~20的含醚键的炔基。本发明基于肌酸母核的新型可电离脂质，有望在保持良好安全性基础的同时，提高核酸药物的包载效率、细胞摄取能力和内涵体逃逸效率，为构建安全高效的脂质纳米颗粒递送系统提供新的技术路径。

mRNA分子及其在制备预防或治疗经典猪瘟的药物中的应用

Resumen de: CN122979321A

本申请提出了mRNA分子及其在制备预防或治疗经典猪瘟的药物中的应用，所述mRNA分子编码第一蛋白和/或第二蛋白；所述第一蛋白选自猪瘟病毒E2蛋白的胞外区；所述第二蛋白包含所述第一蛋白自N端起第N1个至第N2个氨基酸。将本申请的mRNA分子免疫机体后，能够产生持久且高滴度的抗体，并诱导强烈的细胞免疫反应，从而有效抵御猪瘟病毒感染。并且，经一次免疫即可发挥较佳效果，这不仅减少了免疫操作的次数和工作量，降低了因多次免疫可能引发的应激反应和交叉感染风险，还提高了免疫效率，节省了时间和成本，尤其适合大规模养殖。

一种山药外泌体-铂纳米簇复合纳米材料及其制备方法和应用

Resumen de: CN122973538A

本发明属于纳米医学与生物医药材料技术领域，尤其涉及一种山药外泌体‑铂纳米簇复合纳米材料及其制备方法和应用。材料包括山药源纳米囊泡以及包覆于所述山药源纳米囊泡内部的介孔铂纳米簇。其制备方法包括：将介孔铂纳米簇与山药源纳米囊泡按照质量比100‑140:1的比例混合均匀，于2‑8℃条件下避光孵育过夜，使所述介孔铂纳米簇自组装包覆于所述山药源纳米囊泡内部，即得所述复合纳米材料。综上，本发明提供了一种结构明确、制备工艺温和、兼具抗氧化与免疫调控功能的山药外泌体‑铂纳米簇复合纳米材料及其制备方法和应用。

一种阳离子脂质、脂质纳米颗粒及应用

Nº publicación: CN122977985A 27/10/2026

Solicitante:

深圳大学

Resumen de: CN122977985A

本发明涉及医药技术领域，尤其涉及一种阳离子脂质、脂质纳米颗粒及应用。所述阳离子脂质的结构通式为：其中，R1为氨基苯基或氨基烷基，R2为饱和烷基链或不饱和烷基链，所述n为正整数。本发明提供的阳离子脂质通过嵌入大体积的苯环并巧妙构建三条尾链，其被塑造成更优化的圆锥形结构。这种独特设计不仅便于脂质纳米颗粒在细胞内的内体逃逸过程，还显著提高了其内部药物的递送效率。