Alerta

FUSOGENIC LIPID NANOPARTICLES AND METHODS FOR THE MANUFACTURE AND USE THEREOF FOR THE TARGET CELL-SPECIFIC PRODUCTION OF A THERAPEUTIC PROTEIN AND FOR THE TREATMENT OF A DISEASE

Resumen de: US2025346925A1

Provided nucleic acid-based expression construct for the target cell-specific production of a therapeutic protein, such as a pro-apoptotic protein, within a target cell, including a target cell that is associated with aging, disease, or other condition, in particular a target cell that is a senescent cell or a cancer cell. Also provided are formulations and systems, including fusogenic lipid nanoparticle (LNP) formulations and systems, for the delivery of nucleic acid-based expression constructs as well as methods for making and using such nucleic acid-based expression constructs, formulations, and systems for reducing, preventing, and/or eliminating the growth and/or survival of a cell, such as a senescent cell and/or a cancer cell, which is associated with aging, disease, or other condition as well as methods for the treatment of aging, disease, or other conditions by the in vivo administration of a formulation, such as a fusogenic LPN formulation, comprising an expression construct for the target cell-specific production of a therapeutic protein, such as a pro-apoptotic protein, in a target cell that is associated with aging, disease, or other condition, in particular a target cell that is a senescent cell or a cancer cell.

METHOD FOR PURIFYING COMPOSITION

Resumen de: US2025346554A1

A method for purifying a composition, the method including a step of dissolving a composition including a compound represented by Formula (1) in Formula (1), R1 represents —N(R2)—R2 (R2 represents a C1 to C4 alkyl group), each R3 and R4 represents a C3 to C8 alkanediyl group, R5 represents a hydroxyl group, R6 represents —R7—OH (R7 represents a C4 to C12 alkanediyl group) or a hydrogen atom, and n represents an integer of 0 or 1 in an aqueous layer to perform liquid-liquid extraction, in which an oil layer used for the liquid-liquid extraction includes one or more kinds of liquids selected from the group consisting of a ketone-based liquid, an ester-based liquid, and an ether-based liquid, all of which have a solubility parameter (SP value) of 14.8 to 20.5 (MPa1/2).

Ribose-modified cap analog and use thereof

Resumen de: AU2025201351A1

The present disclosure provides a ribose-modified cap analog and a use thereof, and belongs to the technical field of chemical and biological engineering. The ribose-modified cap analog has a structure of formula (I). The ribose-modified cap analog described herein can improve the stability of mRNA and/or the translation efficiency of mRNA. The present disclosure provides a ribose-modified cap analog and a use thereof, and belongs to the technical field of chemical and biological engineering. The ribose-modified cap analog has a structure of formula (I). The ribose-modified cap analog described herein can improve the stability of mRNA and/or the translation efficiency of mRNA. eb e b

LIPID COMPOUNDS, COMPOSITIONS, AND USES THEREOF

Resumen de: AU2024279964A1

The present disclosure provides lipid compounds and compositions (e.g., lipid nanoparticle (LNP) compositions) comprising lipid compounds of the present disclosure. The present disclosure provides methods of delivering an active agent (e.g., polynucleotide) to a cell or tissue in a subject, preferably an extrahepatic cell or tissue, comprising administering to the subject an effective amount of a lipid nanoparticle of the present disclosure, wherein the lipid nanoparticle comprises lipid compounds of the present disclosure and the active agent (e.g., polynucleotide).

CORTICOSTEROID NANOSUSPENSIONS AND USES THEREOF

Resumen de: AU2024265712A1

Suspension formulations of nanoparticles of clobetasol propionate are described. The suspensions can be used therapeutically to treat skin and ocular burns; to enhance wound healing; to prevent or reduce hypertrophic scarring/keloids; to treat allergic rhinitis/sinusitis, asthma, inner ear disorders including hearing loss, tinnitus, or vertigo, tenosynovitis, tendinitis, enthesitis or arthritis.

CROSSING THE BLOOD BRAIN BARRIER TO TREAT DISEASES OF THE BRAIN

Resumen de: WO2025233518A1

The invention relates to antibody-conjugated nanoparticles comprising an mRNA payload. The invention further relates to methods for using such antibody-conjugated nanoparticle and therapeutic uses thereof.

PROCESS FOR PREPARING DISPENSABLE TESTOSTERONE CREAM

Resumen de: AU2024276494A1

This invention relates to a process of preparing an oil-in-water emulsion comprising testosterone for use in a pump-action dispensing device. It further relates to the oil-in-water emulsion produced by the process of the invention, and the combination of the emulsion with a pump-action dispensing device.

METAL AQUO-COMPLEX MEDIATED SYNTHESIS OF ZINC OXIDE-BASED STRUCTURES

Resumen de: AU2024250977A1

Provided herein are dispersible powder compositions comprising metal oxide particles and water, as well as methods of preparing dispersible powder compositions.

Novel lipid nanoparticles for delivery of nucleic acids

Resumen de: AU2025256172A1

The invention relates to novel polymer conjugated lipids and to novel compositions comprising said novel polymer conjugated lipids useful for the delivery of nucleic acids into living cells. 5 The invention relates to novel polymer conjugated lipids and to novel compositions comprising said novel polymer 5 conjugated lipids useful for the delivery of nucleic acids into living cells. ct c t

LPA RECEPTOR ANTAGONIST-DERIVED LIPID COMPOUNDS AND USES THEREOF

Resumen de: WO2025235487A1

Disclosed herein are lipid compounds and compositions comprising lipid compounds and methods of making and use thereof.

INTERTUMORAL AND INTRATUMORAL DELIVERY OF CYTOKINES USING MESOPOROUS SILICA RODS AS AN IMMUNOMODULATING SYSTEM

Resumen de: WO2024148364A1

Embodiments of the invention include methods of treating conditions that are ameliorated by stimulating an immune response. In aspects, the methods include injection of mesoporous silica rods (MSRs) at or near an affected tissue. The MSRs can include a cytokine (e.g., IL-2 or IL-12) and/or an adjuvant. The MSRs can induce an innate immune response to treat cancer, infection and other ailments. The methods can include administering an additional medicament such as an immune checkpoint inhibitor.

抗ＣＯＶＩＤ－１９剤としての自己組織化両親媒性ポリマー

Resumen de: MX2024000185A

There are provided improved amphiphilic comb polymers, comprising a hydrophilic backbone with regularly-spaced pendant hydrophobic moieties, having well-controlled molecular weights, structures, and end groups. The polymers self-assemble into core-corona nanoparticles in aqueous environments, which are capable of disrupting viral coat proteins, and which are capable of encapsulating antiviral drugs and prodrugs. Regularly-spaced targeting moieties optionally mediate the adherence of the nanoparticles to the viral coat. The compositions of the invention are useful as treatments for viral infection, including infections with SARS-CoV-2.

ポリマーソームによる標的化送達のための方法及び組成物

Resumen de: NZ819515A

The present disclosure relates to a copolymer and a polymersome for targeted delivery of biomolecules to a living organism. The exemplary copolymer comprises an initiator block, a propagator block, and a linkage connecting the initiator block and the propagator block. The initiator block comprises a glycan head configured to provide a targeted delivery, and the propagator block comprises a functional moiety selected from a guanidine group, zwitterion group, a diethylene triamine, or a combination thereof; configured to provide desired properties for the polymersome. Exemplary initiator blocks are shown in the abstract drawing.

IONIZABLE CATIONIC LIPIDS INCORPORATING SILICON: SYNTHESIS AND LNP FORMULATION

Resumen de: WO2025052296A1

The present invention belongs to the field of biomedicine and drug delivery as well as pest and vector controls. The invention relates to a novel ionizable cationic lipid family incorporating silicon, which belongs to the trademark LipexSil® second generation lipids, wherein the tail is connected to the headgroup with biodegradable silyl acetal linker. Lipids containing silyl acetal linker(s) are state-of-the-art and are effective as ionizable cationic lipids in the formulation of empty or loaded lipid nanoparticles (LNPs). The novel linkers according to the invention are designed by means of proprietary borane catalysts WO2022129966. The invention describes the synthesis of the lipids of formula (I), formation and characterization of nanoparticles and biological experiments demonstrating that the lipid nanoparticles prepared with these novel lipids can efficiently deliver their cargo (e.g. RNA, DNA, mRNA, siRNA, dsRNA, pDNA, micro RNA, circular DNA, small biologically active molecules) into the cells.

目的遺伝子ＲＮＡ製剤

Resumen de: CN120548193A

The present disclosure relates to RNA molecules having multivalent capabilities against a variety of viral strains or antigens and methods of making the same.

ＴＧＦ－ベータ阻害剤およびＩＬ－１２をコードする核酸ならびにその使用

Resumen de: MX2025004540A

The present disclosure provides for a nucleic acids encoding for a transforming growth factor inhibitor and IL-12. Further provided herein are nucleic acids encoding for chemokine receptor CXCR3. Additionally described herein are oncolytic viruses comprising the nucleic acids described herein. Compositions described herein are further described for use in the treatment of cancer.

COMPOUND FOR DELIVERING DRUG, AND LIPOSOME AND DRUG CARRIER

Resumen de: EP4647421A1

The present invention provides a compound, which is a compound represented by formula (I) or a stereoisomer, tautomer, solvate, or pharmaceutically acceptable salt thereof represented by formula (I):X1, X2, and X3 are each independently a C1-C15 alkyl group optionally substituted; R1 and R2 are each independently a C1-C40 alkyl group optionally substituted, a C1-C40 heteroalkyl group optionally substituted, a C2-C40 alkenyl group optionally substituted, a C2-C40 heteroalkenyl group optionally substituted, a C2-C40 alkynyl group optionally substituted, or a C2-C40 heteroalkynyl group optionally substituted; R3, R4, R5, and R6 are each independently H, halogen, or a C1-C3 alkyl group optionally substituted; the substituent group is independently selected from halogen, -OH, -SH, -NH2, -NO2, cyano, or C1-C3 alkyl. This compound has the advantages of low cytotoxicity, strong delivery capability, and good immunostimulatory effect.

タンパク質発現量を増加させるＵＴＲ分子

Resumen de: US2025333728A1

The present disclosure relates to a UTR molecule and use thereof. Specifically, the present disclosure relates to an mRNA molecule comprising 5′ UTR and 3′ UTR. The UTR molecule can significantly increase mRNA expression level.

一种用于逆转肿瘤耐药的载药纳米复合材料

Resumen de: CN120919071A

本发明公开了一种用于逆转肿瘤耐药的载药纳米复合材料，涉及医药化学应用技术领域；该载药纳米复合材料包括纳米级载体组分和铁死亡促进剂；纳米级载体组分为金、银、铂、钯、锡、钽、镱、锆、铪、铽、铥、铈、镝、铒、铕、钬、铁、镧、钕、镨和镥的纳米材料/复合纳米材料中的至少一种；铁死亡促进剂为靶向胱氨酸/谷氨酸反向转运体、靶向GSH、靶向GPX4、靶向溶酶体激活铁离子和靶向ROS中的至少一种。本发明的载药纳米复合材料Fe3O4@SAS在不损害正常细胞前提下，有效地诱导并促发耐药胃癌肿瘤细胞的铁死亡，达到逆转胃癌肿瘤细胞耐药的目的，为目前肿瘤细胞耐药治疗提供新的思路。

一种蛋白N-肉豆蔻酰化诱导的小细胞外囊泡装载系统及其应用

Resumen de: CN120924603A

本申请涉及生物医药技术领域，具体公开了一种蛋白N‑肉豆蔻酰化诱导的小细胞外囊泡装载系统(PMEVL平台)及其应用，本申请的PMEVL平台具有很强的特异性，有效性及广泛适用性；本申请明确了PMEVL平台可高效装载外源性化学合成的miRNAs或siRNAs，同时，也证明PMEVL平台有助于将内源合成的miRNAs或siRNAs，高效和特异的装载到小细胞外囊泡(sEVs)，并且依赖于N‑肉豆蔻酰基修饰发挥的作用；经过实验可知，PMEVL平台可装载不同siRNAs，或者同时将多个不同siRNAs装载到sEVs，从而在细胞间通讯和药物递送等领域展现出巨大的应用前景。

一种铁死亡激动剂、缺氧响应型纳米共递送系统及其应用

Resumen de: CN120923499A

本发明属于药物制备技术领域，具体涉及一种铁死亡激动剂、缺氧响应型纳米共递送系统及其应用。本发明通过在RSL3分子中引入氯乙酰基片段，合成了新型铁死亡激动剂RSL3‑ClAc，显著增强了其与GPX4的结合能力，进而提高了铁死亡诱导效率。同时，将RSL3‑ClAc与缺氧响应型光敏剂TCy5‑NO2进行自组装，构建了纳米递药系统。该纳米递药系统利用纳米载体的EPR效应和缺氧响应特性，能够有效突破肿瘤屏障，精准定位病灶。在肿瘤缺氧微环境中，通过硝基还原酶（NTR）触发TCy5‑NO2光敏剂激活，并同步释放RSL3‑ClAc以增强铁死亡诱导效果。

一种具有骨靶向的核酸脂质纳米颗粒的制备方法与应用

Resumen de: CN120923571A

本发明公开了一种具有骨靶向的脂质纳米颗粒的制备方法和用途，具有骨靶向的脂质化合物或其药学上可接受的盐或立体异构体中所述骨靶向的脂质化合物具有式(I)所示结构。本发明的脂质化合物或其药学上可接受的盐或立体异构体组成的脂质纳米颗粒具有骨靶向，并且在体内循环的过程中表现出良好的稳定性，可以在常温条件下储存、运输和使用，不良反应的发生率较低。该类脂质纳米颗粒在作为核酸药物载体时，展现出良好的骨骼组织靶向能力，优异的生物活性和较高的蛋白表达水平。

一种基于喹啉可电离脂质的脂质纳米组合物及其应用

Resumen de: CN120919332A

本发明涉及生物技术领域，具体涉及一种基于喹啉可电离脂质的脂质纳米组合物及其应用。本申请公开了一种纳米组合物，其包含可电离脂质、中性磷脂、胆固醇、PEG脂质，所述可电离脂质的结构如式Ⅰ所示。经试验验证，本申请公开的纳米组合物，具有良好的安全性和核酸递送效果。

一种红色抗菌纳米硒粒子及其制备方法

Resumen de: CN120919076A

本发明提供了一种红色抗菌纳米硒的制备方法，将羧甲基纤维素粉末与高碘酸盐溶液混合，进行羧甲基纤维素的氧化反应，获得氧化改性羧甲基纤维素粉末，再将氧化改性羧甲基纤维素粉末与多胺基聚合物溶液进行接枝反应，得到阳离子型胺基化改性羧甲基纤维素，然后将硒酸根物质溶解于去离子水中，待完全溶解后添加所得阳离子型胺基化改性羧甲基纤维素，加热搅拌，生成红砖色沉淀，最后离心干燥后得到所述纳米硒粒子。该方法解决了纳米硒粒子容易团聚的问题，制备的纳米硒粒子具有很高的生物活性和生物利用率，并且无额外还原剂使用。

一种用于癫痫治疗的超声响应仿生压电纳米药物及其制备方法和应用

Nº publicación: CN120919312A 11/11/2025

Solicitante:

国家纳米科学中心

Resumen de: CN120919312A

本发明涉及一种用于癫痫治疗的超声响应仿生压电纳米药物及其制备方法和应用，包括：经硫酸刻蚀后的铪基压电纳米颗粒UIO‑66‑NH2、抗癫痫药物、小胶质细胞膜、转铁蛋白受体靶向肽；其中，所述抗癫痫药物负载于经硫酸刻蚀后的铪基压电纳米颗粒中形成载药核心；所述小胶质细胞膜包覆于载药核心的外表面；所述转铁蛋白受体靶向肽连接于小胶质细胞膜上。本发明的超声响应仿生压电纳米药物入脑效率高，释药量稳定，制备方法路径清晰，适用于多种抗癫痫药物的靶向递送及癫痫等神经系统疾病的治疗与研究，其可采用静脉注射的给药方式，无创给药，能够避免手术带来的损伤，电刺激在体内产生，无需经过电极的植入与取出，避免了带来的二次损伤和感染。