Alerta

High-density lipoprotein mimetic nanoparticles using lipid conjugated core scaffolds

Resumen de: AU2025260005A1

Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. ov o v

POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS

Resumen de: WO2024229321A1

This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects.

COMPLEX

Resumen de: AU2024283556A1

The present invention addresses the problem of providing a technology for improving poor water solubility of a compound of general formula (1), and suppressing excessive release of cytokines and myelotoxicity of the compound of general formula (1). The present invention solves the problem by a complex comprising: a modified polysaccharide containing a hydrophobic group; and a compound represented by general formula (1).

RECOMBINANT TISSUE PLASMINOGEN ACTIVATOR (TPA) FRAGMENTS AND USES THEREOF

Resumen de: AU2024272346A1

The present technology relates to recombinant polypeptides, or nucleotides encoding the same, comprising tissue plasminogen activator (tPA) fragments and uses thereof for treating cardiovascular diseases. In some embodiments, the tPA fragments comprise the kringle 2 domain of tPA.

EXTRACELLULAR VESICLES FOR THE DELIVERY OF PAYLOADS TO EUKARYOTIC CELLS

Resumen de: AU2024267555A1

The invention relates to the production, modification, and use of extracellular vesicles (EVs), particularly for the delivery of payloads of nucleic acids. The EVs can contain retroviral capsids and can be coated with polymers. The EVs can be treated with a protease to remove proteins on the outer surface of the EV to create shaved EVs. The shaved EVs can be coated with a polymer and used for the delivery of payloads of nucleic acids to cells.

Compositions and methods for the treatment of anesthesia-induced neurotoxicity

Resumen de: AU2025259971A1

A formulation comprising an oligonucleotide selected from the group consisting of an oligonucleotide having one of SEQ ID No.:1 through SEQ ID No.:42 or a variant thereof. Also, a method of treating anesthesia-induced neurotoxicity. The method may comprise administering the formulation. The formulation may be administered prior to, concomitant with, subsequent to, or combinations thereof administration of a general anesthetic comprising a fluorinated compound. The oligonucleotide may be incorporated into a carrier system, for example, a liposome, a biodegradable polymer, a hydrogel, or a cyclodextrin, a nucleic acid complex, a virosome, or combinations thereof. Also, a method of treating anesthesia-induced neurotoxicity. A formulation comprising an oligonucleotide selected from the group consisting of an oligonucleotide having one of SEQ ID No.:1 through SEQ ID No.:42 or a variant thereof. Also, a method of treating anesthesia-induced neurotoxicity. The method may comprise administering the formulation. The formulation may be administered prior to, concomitant with, subsequent to, or combinations thereof administration of a general anesthetic comprising a fluorinated compound. The oligonucleotide may be incorporated into a carrier system, for example, a liposome, a biodegradable polymer, a hydrogel, or a cyclodextrin, a nucleic acid complex, a virosome, or combinations thereof. Also, a method of treating anesthesia-induced neurotoxicity. ct c t

NANOPARTICLES FOR USE IN THE TREATMENT OF INFECTIONS CAUSED BY BIOFILMS

Resumen de: US2025352486A1

The present invention is based on a nanodevice comprising a nanoparticle comprising a therapeutic agent, a molecular drill comprising a compound with catalytic activity, and a self-propulsion system. The molecular drill in combination with the self-propulsion system allows breaking the matrix of biofilms formed by infectious microorganisms with high efficiency and the molecular drill, in turn, allows the therapeutic agent to be released from the nanoparticle under specific conditions, preferably in the acidic environment of an infection. The invention relates to the nanodevice, to the nanodevice used in the treatment of an infection caused by biofilms, and to the use of the nanodevice to disinfect samples in vitro or inert materials ex vivo.

LIPOSOMAL POWDER FORMULATIONS OF LIPOPHILIC AND WATER-SOLUBLE SUBSTANCES

Resumen de: AU2024269428A1

The invention provides submicronic liposomal formulations in a dry powder form characterized in that they can incorporate various types of lipophilic and hydrophilic actives while maintaining their original structure and size under various production conditions, reconstitution mediums, varying pH, osmolarity and storage conditions.

MUCOSAL FORM FOR DRUG DELIVERY THROUGH THE MUCOSA AND METHODS OF TREATMENT WITH SAME

Resumen de: AU2024243584A1

Mucosal patches are disclosed that have a support layer supporting an active layer that has a collagen carbon dot nanocomposite carrying an active agent. The collagen carbon dot nanocomposite is absorbable through a mucosa. The mucosal patches are part of various methods of treatment.

MICRORNA-BASED PARTICLE FOR THE TREATMENT OF DYSREGULATED IMMUNE RESPONSE

Resumen de: AU2024207086A1

A miRNA-mimic based therapeutic particle is disclosed herein. The particles comprise a synthetic miRNA or mimic of miR-187-3p encapsulated in a lipid nanoparticle (LNP) carrier or synthetic miR-193b-5p inhibitor encapsulated in a lipid carrier or their combination encapsulated in a lipid carrier. The lipid nanoparticle carrier is made up of at least four (4) types of lipids, in which the four (4) types of lipids include a) an ionizable cationic lipid selected to be positively charged in a formulation buffer (pH 4), which binds and protects the negatively charged miRNA, and facilitates endosomal escape, and is neutral in a storage buffer, b) a sterol in the structure of the lipid nanoparticle (LNP)., c) a structural helper lipid selected to contribute to lipid nanoparticle stability and/or enhances endosomal release, and d) a PEGylated-lipid selected such that it stabilizes the therapeutic particle and protects it from opsonization.

METHOD FOR EVADING PREEXISTING ANTI-PEG ANTIBODY IN HUMAN BODY

Resumen de: EP4649962A1

The present invention discloses a method for circumventing pre-existing anti-PEG antibodies in the human body and the use of a terminal hydroxyl-modified PEGylated nanocarrier in the preparation of a drug that circumvents pre-existing anti-PEG antibodies in the human body. The present invention also discloses a terminal hydroxyl-modified PEGylated nanocarrier and a nanoformulation comprising the same. The terminal hydroxyl-modified PEGylated nanocarrier and nanoformulation according to the present invention have a low binding to pre-existing anti-PEG antibodies in the human body, so that they can circumvent being rapidly cleared in the human blood and better exert the therapeutic effect. In addition, the terminal hydroxyl-modified PEGylated nanocarrier and nanoformulation can reduce complement activation by circumventing a binding to pre-existing anti-PEG antibodies in human blood, thereby alleviating side effects such as clinical injection reactions.

COMPOUND, PREPARATION METHOD, AND LIPID NANOPARTICLES AND PHARMACEUTICAL COMPOSITION THEREOF

Resumen de: EP4650349A1

The invention provides a compound, its preparation method, and lipid nanoparticles, as well as a pharmaceutical composition. The compound includes at least one structural formula (I-1) and may be provided as a pharmaceutically acceptable salt, prodrug, or isomer. It increases structural diversity among lipid molecules and is biodegradable. The L1a, L1b, and L1c groups in the compound can be interchanged with L2, enabling the integration of other molecules like steroids or peptides. This interchangeability expands the functionality of cationic lipid compounds, allowing for the direct coupling of functional molecules to the lipid compound to enhance its functional scope. The replaceable L groups offer greater flexibility in selecting L1a, L1b, L1c, and L2.

IONIZABLE LIPIDS AND NANOPARTICLES COMPRISING SAME

Resumen de: WO2024150222A1

One or more ionizable lipid(s) and lipid nanoparticles comprising same and further encapsulating a polynucleic acid are provided. Pharmaceutical compositions comprising a therapeutically effective amount of the lipid nanoparticles encapsulating a therapeutically active polynucleic acid are also provided. Pharmaceutical compositions are used in delivering of the polynucleic acid to a lung tissue of a subject are also provided.

COMPOSITE LIPID NANOPARTICLE AND PREPARATION METHOD THEREOF, RNA VACCINE, DRUG AND USE

Resumen de: EP4649964A1

The present disclosure belongs to the technical field of lipid nanoparticle biological application, and discloses a composite lipid nanoparticle and a preparation method thereof, an ribose nucleic acid (RNA) vaccine, a drug and use. The composite lipid nanoparticle provided by the present disclosure includes a lipid composition and a recombinant antibody protein containing a streptavidin tag; and the lipid composition comprises an ionizable lipid, an auxiliary lipid, cholesterol, a polyethylene glycol lipid and a biotinylated polyethylene glycol lipid in a mass ratio of (40-90):(0.1-20):(20-50):(0.5-10):(0.5-10). Through strong interaction between the recombinant antibody protein with the streptavidin tag and the biotinylated polyethylene glycol lipid in the composite lipid nanoparticle, the composite lipid nanoparticle achieves safe, convenient, rapid, efficient, and stable linkage between a lipid nanoparticle (LNP) and a recombinant antibody protein, and the recombinant antibody protein can be adaptively replaced according to target cells and/or tissues, with high flexibility and applicability. Moreover, the recombinant antibody protein on the obtained composite lipid nanoparticle still has a good ability to specifically recognize and bind a corresponding protein.

AMINO LIPID COMPOUND, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: EP4650343A1

An amino lipid compound, and a preparation method therefor and the use thereof. The present invention further relates to a lipid nanoparticle and a pharmaceutical composition containing the amino lipid compound, and the use thereof.

LIPID NANOPARTICLE COMPOSITIONS AND USES THEREOF

Resumen de: US2025090472A1

The present application relates to lipid nanoparticles having a diameter above certain value, as well as the preparation and uses of such compositions. Lipid nanoparticles presented in the application generally have a diameter of at least 160 nm. Such lipid nanoparticles are useful in the delivery of therapeutic agents, such as nucleic acids, in vivo to none-hepatic organs (e.g., lung) for the treatment or prevention of certain diseases or disorders.

一种脑靶向递送系统及其制备方法和应用

Resumen de: CN120960172A

本申请提供一种脑靶向递送系统，包括：作为药物载体的介孔普鲁士蓝纳米酶、包裹于所述药物载体表面的红细胞膜；所述红细胞膜经过狂犬病毒糖蛋白多肽修饰。本申请提供一种还提供一种药物，还包括负载于所述药物载体上的活性物质，所述活性物质包括L‑精氨酸和丁苯酞中的至少一种。该脑靶向递送系统用于协同治疗缺血性脑卒中时，免疫逃逸能力显著增强、脑靶向效率较未修饰纳米粒提高10倍以上、脑内滞留时间≥24h。

一种利丙双卡因纳米乳膏及其制备方法

Resumen de: CN120960136A

本发明提供了一种利丙双卡因纳米乳膏及其制备方法，属于药物制剂技术领域。本发明提供的利丙双卡因纳米乳膏中，包载利丙双卡因纳米粒分散在乳膏基质中，包载利丙双卡因纳米粒能够控制利多卡因和丙胺卡因具有稳定的比例，并且能够使利丙双卡因纳米乳膏具有良好的缓释效果；本发明利用乳膏基质的作用提高利多卡因和丙胺卡因的透皮渗透性。实施例结果显示，本发明提供的利丙双卡因纳米乳膏具有良好的稳定性、渗透性和缓释效果。

稳定的核酸组合物以及用于制备、储存和使用该组合物的方法

Resumen de: WO2024194454A1

The present disclosure relates generally to the field of stabilized compositions comprising particles dispersed in an aqueous phase, wherein the aqueous phase comprises a buffer system and has a pH of about 4.0-5.5 and wherein the particles contain (i) nucleic acid (such as DNA or RNA, in particular mRNA or inhibitory RNA, e.g., siRNA); and (ii) a cationic or cationically ionizable lipid, methods for preparing and storing such compositions, and the use of such compositions in therapy.

脂质组合物

Resumen de: CN120960443A

本发明涉及一种脂质组合物，其中包含聚合物脂质，所述聚合物脂质为式(I)所示结构的化合物，或其药学上可接受的盐、同位素变体、互变异构体或立体异构体。本发明还提供了包含所述脂质组合物的纳米颗粒组合物和药物组合物，以及所述脂质组合物在递送荷载中的应用。本发明脂质组合物中的聚合物脂质化合物可替代PEG脂质，具有生物相容性、易于在体内分解、无毒的优点，从而得到了一种新的脂质递送系统。

细胞中的基因组插入

Resumen de: US2025230471A1

The present disclosure provides compositions and methods for inserting heterologous payload sequences into a target-site in a host cell genome. The compositions and methods use non-LTR retrotransposon reverse transcriptase proteins that bind template RNAs comprising a payload sequence that encodes a protein or regulatory RNA. The template RNA can comprise modified uridines that are not cleavable by a ribozyme. The incorporation of modified uridines increases the efficiency of integration and expression of the payload sequence and decreases cellular toxicity.

一种双金属基纳米复合材料及其制备方法和应用

Resumen de: CN120960170A

本发明公开了一种双金属基纳米复合材料及其制备方法和应用，制备方法包括：将硝酸铁和硝酸铋分散于去离子中，搅拌至溶解，调节溶液pH至碱性，超声分散均匀后倒入聚四氟乙烯釜中进行水热反应，获得双金属基纳米材料FBO，分散到Tris缓冲液中，并加入盐酸多巴胺，避光条件下搅拌，离心、洗涤、干燥，获得黑色的纳米复合材料FBO@PDA，即得。本发明的双金属基纳米复合材料具有抗炎、促成骨和促血管化性能，制备方法简单，安全环保，成本低，易于操作，可作为骨修复材料用于颌骨坏死的再生修复领域，具有广阔的应用前景。

一种具有光热产生能力的近红外二区聚集诱导发光型光敏剂及其制备方法和应用

Resumen de: CN120965668A

本发明提供了一种具有光热产生能力的近红外二区聚集诱导发光型光敏剂及其制备方法和应用，属于生物化学材料领域。本发明提供的有机荧光化合物的分子结构中存在电子给体‑电子受体相互作用，其中受体部分中R基团的引入，有利于促进D‑A相互作用，促使吸收波长发生红移，增加对生物组织的穿透深度，提供具有更高分辨率的荧光成像结果；此外，本发明提供的有机荧光化合物给体部分为噻吩基乙烯偶联的久洛尼定以及共轭二烯偶联的4,4'‑二(N,N‑二甲氨基)二苯甲酮，其中4,4'‑二(N,N‑二甲氨基)二苯甲酮具有高度扭曲的空间结构，更有利于非辐射跃迁过程的发生，从而促进了光热性能的提升。

一种可离子化脂质以及由其制备的脂质载体及用途

Resumen de: CN120965753A

本发明公开了一种可离子化脂质以及由其制备的脂质载体及用途。具体地，本发明公开了一种如式(I)所示的可离子化脂质化合物、其立体异构体、互变异构体或药学上可接受的盐。本发明的化合物是一类兼具磷酸基团与阳离子头基的新型脂质分子，具有生产工艺简单、转染效率高、细胞毒性低和适用范围广等优点。

聚合物脂质化合物

Nº publicación: CN120965996A 18/11/2025

Solicitante:

广州市锐博生物科技有限公司

Resumen de: CN120965996A

本发明涉及一种聚合物脂质化合物，具体涉及一类式(I)的化合物，或其药学上可接受的盐、同位素变体、互变异构体或立体异构体。本发明还提供了包含所述化合物的纳米颗粒药物组合物，以及所述化合物及其组合物在递送荷载中的应用。本发明的聚合物脂质化合物可替代PEG脂质，具有生物相容性、易于在体内分解、无毒的优点，从而得到了一种新的脂质递送系统。