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基于脂质体、外泌体与金属有机框架协同靶向治疗脂肪肝的纳米药物递送系统及其制备方法

Resumen de: CN120713866A

本发明提供基于脂质体、外泌体与金属有机框架协同靶向治疗脂肪肝的纳米药物递送系统及其制备方法，涉及新型纳米材料制备技术领域，纳米药物递送系统包括：脂质体、间充质干细胞外泌体和金属有机框架纳米颗粒；脂质体为载体，包载具有缓释特性的金属有机框架纳米颗粒、间充质干细胞外泌体和miR‑204‑3p。通过整合脂质体、外泌体和金属有机框架的优势，实现对MASLD的协同治疗。该系统采用脂质体作为智能载体，包载具有缓释特性的MOF纳米颗粒和间充质干细胞外泌体，同时负载治疗性miR‑204‑3p。MOF材料兼具药物控释和活性氧清除功能，与外泌体及miRNA协同发挥抗氧化、抗炎和抗纤维化作用。该系统具有pH响应特性，能在MASLD病灶的酸性微环境中智能释放药物，实现肝脏靶向治疗。

腫瘍病変の光音響画像化及び光熱療法のための生体材料被覆ナノ構造

Resumen de: WO2024068100A1

Metal based nanoparticles coated with a polymer functionalized with thiol groups and 5 -NH groups, said polymer preferably being selected among thiolated chitosan, thiolated and aminated alginic acid, thiolated and aminated hyaluronic acid, or a protein preferably selected from the group consisting of albumin and gelatin, or a synthetic thiolated and aminated polymer, preferably α-thio-ω-amino polyethylene glycols; wherein said groups are linked to a ligand of the integrin family receptors, 10 preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a heterobifunctional crosslinker, the crosslinker bearing functional groups able to bind to amino groups, preferably selected from the group consisting of N-hydroxysuccinimidyl ester group (NHS ester), an isocyanate group (-NCO), an isothiocyanate group (-NCS), a Sulfo-N-15 hydroxysuccinimidyl ester group (sulfo-NHS ester), or a carboxylic acid group which is connected by activation with carbodiimide coupling agents; and/or a functional group able to bind thiol groups, preferably selected from the group consisting of: a maleimide group, a terminal vinyl group or a terminal alkyne group; and/or functional groups able to bind alkynes such as azides; and/or functional groups able 20 to bind azides, such as alkynes.

神経筋障害の治療のための組成物及び方法

Resumen de: TW202424194A

The present disclosure relates to adeno-associated virus (AAV)-mediated delivery of nucleic acids for treating neuromuscular disorders, e.g., X-linked myotubular myopathy (XLMTM), in patients in need thereof. The AAV vectors of the disclosure may include, e.g., a transgene encoding a myotubularin protein operably linked to one or more transcription regulatory elements. In some embodiments, the one or more transcription regulatory elements are specifically active in muscle and/or liver tissue.

核酸複合体及びその使用

Resumen de: WO2024068674A1

The present disclosure provides complexes and methods of use. In some embodiments, a complex described herein is a complex comprising a cationic polymer, an anionic polymer, and a monomeric RNA molecule, wherein the cationic polymer and the monomeric RNA molecule form a core complex encapsulated by the anionic polymer. In some embodiments, a complex comprises a linear cationic polymer, an anionic polymer, and a monomeric RNA molecule, wherein the cationic polymer and the monomeric RNA molecule form a core complex encapsulated by the anionic polymer.

면역치료를 위한 항원-포획 나노입자들 및 제형들

Resumen de: AU2024219072A1

The present invention provides novel antigen-capturing nano-particles (ACNPs) and pharmaceutical formulations containing such ACNPs. Also provided herein are methods for preparing the ACNP formulations, and methods for the treatment of a disease (such as cancer) in a subject with the ACPNs or a pharmaceutical formulation containing the ACPNs.

화합물 또는 그 염, 지질 조성물, 의약 조성물 및 송달 캐리어

Nº publicación: KR20250141799A 29/09/2025

Solicitante:

후지필름가부시키가이샤

Resumen de: WO2024190817A1

The present invention addresses the problem of providing: a compound, or a salt thereof, which constitutes a lipid composition that can realize a high nucleic acid encapsulation rate and excellent nucleic acid delivery when mRNA is used; and a lipid composition, a pharmaceutical composition, and a delivery carrier that can realize a high nucleic acid encapsulation rate and excellent nucleic acid delivery when mRNA is used. The present invention provides a compound represented by formula (1), or a salt thereof. In the formula, R1 represents a hydrocarbon group having 1-24 carbon atoms; R2 represents a hydrocarbon group having 1-6 carbon atoms, R3 represents a hydrocarbon group having 6-24 carbon atoms and having a branched chain; R4 and R5 each represents a hydrocarbon group having 1-6 carbon atoms which are optionally substituted; R6 represents a hydrogen atom or a hydrocarbon group having 1-18 carbon atoms; L represents *-O-C(O)- or *-C(O)O -; a represents an integer of 1-12; and b, c, and d each represents an integer of 1-4.