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Benzaldehyde acetal acid-degradable amphiphilic lipid and self-assembling peptides

Resumen de: US20260053954A1

Compounds comprising a benzaldehyde acetal acid-degradable amphiphilic lipid and self-assembling peptides are incorporated in lipid nanoparticle (LNP) and used to transfect cells.

COMPOSITIONS AND METHODS FOR IMPROVING COGNITIVE FUNCTION

Resumen de: US20260053831A1

Disclosed herein are methods for managing disorders or conditions, or treating symptoms of disorders or conditions, comprising administering 5HT receptor agonists. The disorders may involve cognitive function and the methods of improving may comprise administering low doses of 5HT receptor agonists to subjects in need thereof. Improvement of the symptoms of a disorder involving cognitive function can be achieved where the disorders comprise mood, cognitive, anxiety, and depression disorders. Also disclosed herein are pharmaceutical compositions, formulations, and dosage forms of 5HT receptor agonists.

ORAL FORMULATIONS COMPRISING POROUS SILICA PARTICLES AND MEDICAL USES THEREOF

Resumen de: US20260053848A1

There is provided herein an oral formulation comprising: (a) water; (b) porous silica particles having pores in the mesoporous range; and (c) a thickening agent, wherein the average pore size of the pores in the mesoporous range is from about 7.0 to about 25.0 nm; and the thickening agent is selected from xanthan gum and microcrystalline cellulose or a mixture thereof. There are also provided uses of such formulations.

A DELIVERY SYSTEM COMPRISING SILICON-CONTAINING MATERIAL

Resumen de: US20260053753A1

A method for promoting the controlled binding and release of a bioactive or pharmaceutical agent from a composition comprising silicon nanoparticles, wherein the silicon nanoparticles comprise at least 50% by weight silicon, the method comprising treating the surface of the silicon nanoparticles with at least one lipid, and treating the surface of the silicon nanoparticles with at least one amino acid, wherein the ratio of lipid to silicon is from 1:1 to 15:1. Also related compositions and methods.

KAEMPFEROL BIOMIMETIC NANO-MATERIAL, METHOD FOR PREPARING SAME AND USE THEREOF

Resumen de: US20260053754A1

A kaempferol-mesenchymal stem cell membrane biomimetic nano-material allowing for more accurate targeting to injured issues is provided. The biomimetic nano-material is simple in preparation method, high in universality, and suitable for large-scale production, mesenchymal stem cell membranes are used to improve the biocompatibility and targeting ability of carriers and increase the in vivo circulation time of drugs; the biomimetic nano-material allows for targeted delivery of kaempferol to the liver, providing a novel treatment method for ALF.

DELIVERY OF NUCLEIC ACIDS USING A TARGETED PEPTIDE CARRIER SYSTEM

Resumen de: WO2024220878A1

A layer-by-layer ELP-nucleic acid (NA) nanoparticle (LENN) complex for targeted delivery of a therapeutic NA cargo to tumor cells; a method of formulating a LENN complex using a layer-by-layer deposition process (LbL); LENN complex formulations; pharmaceutical compositions comprising a LENN complex formulations; and methods of treating bladder cancer in a subject via LENN-mediated delivery of a therapeutic NA cargo.

VACCINE COMPOSITIONS AND USES THEREOF

Resumen de: WO2024220936A1

The present disclosure provides vaccine compositions comprising a plurality of distinct antigens. Also provided are nucleic acid vaccine composition comprising one or more nucleic acids encoding for a plurality of distinct antigens. The plurality of distinct antigens comprises a combination of influenza antigens. The vaccine composition can be formulated for delivery as a mRNA/LNP, a recombinant protein, a virus-like particle (VLP), or DNA. Methods of preventing an influenza infection and methods of inducing an immune response are also disclosed.

DELIVERY OF POLYNUCLEOTIDES FROM LIPID NANOPARTICLES COMPRISING RNA AND IONIZABLE LIPIDS

Resumen de: WO2024220625A1

Provided herein is a method for delivering an RNA composition to a subject for in vivo production of a protein or a peptide in the subject, comprising administering to the subject an RNA composition comprising a polynucleotide that encodes a protein or a peptide, formulated within (a) a plurality of lipid nanoparticles (LNP) comprising synthetic structural lipids and an ionizable lipid, or (b) a lipid reconstructed natural messenger packs (LNMPs) comprising natural lipids and an ionizable lipid. Also provided herein are methods for treating diseases or disorders associated with the gastrointestinal tract, stomach, small or large intestine, mesenteric lymph node, pancreas, colon or rectum, caecum, and/or spleen, comprising orally or enterally administering the RNA composition described herein.

FLUORINATED CATIONIC LIPIDS FOR USE IN LIPID NANOPARTICLES

Resumen de: EP4700017A2

Compounds are provided having the following structure:or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein R^2a, R^2b, R^3a, R^3b, R⁷, R⁸, R⁹, L¹, L², G¹, G², G³, b, and c are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

RECONSTITUTABLE DRY POWDER FORMULATIONS AND METHODS OF USE THEREOF

Resumen de: MX2025012337A

The present disclosure is directed to the use of reconstituted mRNA dry powder particles for parenteral administration. The present disclosure is also directed to a method of generating dry powder particles supplemented with appropriate excipients for optimal thermostability and in vivo expression.

CAPSID VARIANTS AND USES THEREOF

Resumen de: US2025319207A1

Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell (e.g., an ocular cell). The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants and methods of using same for delivery of a transgene.

TIMESCALE-GUIDED MICROFLUIDIC SYNTHESIS OF POLYPHENOL-IRON III NETWORK NANOCAPSULES OF HYDROPHOBIC DRUGS

Resumen de: WO2024220674A1

A scalable method of continuous microfluidic synthesis of tannic acid-iron III (TA-FeIII) network (TFN) nanocapsules of a hydrophobic drug; a scalable method of continuous microfluidic synthesis of epigallocatechin-3-gallate iron III (EGCG-FeIII) network, (EFN) nanocapsules; hydrophobic drug nanocapsules synthesized in accordance with the method; and a method of administering a hydrophobic drug to a patient in need thereof by administering to the patient the hydrophobic drug nanocapsules.

CONJUGATED POLYETHYLENEIMINE COMPOUNDS AND COMPOSITIONS, AND USES THEREOF FOR THE TREATMENT OF ATHEROSCLEROSIS, CARDIOVASCULAR, AND LUNG DISEASE

Resumen de: WO2024220742A1

Provided herein are lipid conjugated polyethyleneimine (PEI) compounds of Formula (I), which are useful to form delivery systems, such as lipid nanoparticles, for delivery of agents (e.g., nucleic acid molecules) to cells (e.g., endothelial cells). Also provided herein are methods for treating atherosclerosis, a cardiovascular disease, a pulmonary hypertension (e.g, pulmonary arterial hypertension or secondary pulmonary hypertension), a lung cancer, a lung metastasis, chronic obstructive pulmonary disease (COPD), bronchiolitis obliterans, bronchiolitis obliterans organizing pneumonia (BOOP), pulmonary fibrosis, or a fibrotic lung disease, comprising administering to a subject a pharmaceutical composition comprising lipid conjugated polyethyleneimine (PEI) compounds of Formula (I), an agent, and a pharmaceutically acceptable excipient.

LIPID NANOPARTICLE (LNP) DELIVERY SYSTEMS AND FORMULATIONS

Resumen de: MX2025012462A

The present disclosure describes compositions, nanoparticles (such as lipid nanoparticles), and/or lipid nanoparticle compositions and methods of their use.

プロタンパク質転換酵素サブチリシンケキシン９（ＰＣＳＫ９）編集のための組成物及び方法

Resumen de: US2025313845A1

The present disclosure provides compositions and methods for modifying a PCSK9 gene. In some aspects, the present disclosure provides a guide RNA, compositions thereof, and pharmaceutical compositions comprising a guide RNA or a composition as described herein. In some aspects, the present disclosure also provides uses and methods of using a guide RNA, a composition thereof, or a pharmaceutical composition as described herein, for inducing a double-strand break or a single-strand break in a PCSK9 gene, for reducing expression of a PCSK9 gene in a cell or subject, and for treating a patient having or at risk of having a PCSK9-related disease or condition.

核酸カーゴのデリバリーのためのナノ粒子

Resumen de: GB2641968A

Nanoparticles suitable for delivery of a linear DNA molecule are provided. Nanoparticles suitable for delivery of mRNA or DNA are provided. Further provided are uses of the nanoparticles including the use of the nanoparticles for treating disease and the use of the nanoparticles in vaccines.

パーキンソン病の治療用組成物および方法

Resumen de: MX2025007688A

Compounds, compositions, uses, and methods for increasing cell viability of a dopaminergic neuron, or for preventing or treating dopaminergic neuronal death, are provided herein. In certain examples, methods for reducing symptoms and/or for preventing or treating Parkinson's disease in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a^GDP), one or more expressible nucleic acids encoding Rab1a^GDP, or a combination thereof.

がんを治療するための治療薬としての腫瘍溶解性ウイルス

Resumen de: CN120603935A

The present invention relates to compositions and methods for treating cancer using oncolytic viruses, components thereof, and/or derivatives thereof. In particular, the present invention relates to modified picornaviruses comprising changes in one or more of its capsid proteins that confer enhanced binding capacity to cancer cells.

脂质纳米颗粒的冻干制剂和液体制剂

Resumen de: WO2025026304A1

Provided are lyophilized formulations of lipid nanoparticles comprising a cationic lipid and a cryoprotectant combination, which contains sucrose and a non-polar amino acid.

包含胰酶的功能化生物催化组合物

Resumen de: AU2024288835A1

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 wherein the lipase or a fragment thereof is in the open conformation, a protease or a fragment thereof immobilized on the surface of the solid carrier, an amylase 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, a protective layer to protect the lipase or a fragment thereof, the protease or a fragment thereof and the amylase or a fragment thereof by embedding the lipase or a fragment thereof, the protease or a fragment thereof and the amylase 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.

用于肿瘤治疗的仿生光活性纳米制剂及其制备方法与应用

Resumen de: CN121550425A

本发明属于生物医药技术领域，尤其涉及一种用于肿瘤治疗的仿生光活性纳米制剂及其制备方法与应用，包括纳米过氧化钙与聚多巴胺、碘化物IR780合成CaO2@PDA‑IR780，再通过细胞膜涂层技术合成CaO2@PDA‑IR780@M的方法。本发明所得CaO2@PDA‑IR780@M是一种仿生光活性纳米制剂，可以利用纳米过氧化钙原位产氧增强IR780的PDT效果来改善对于肿瘤的疗效、利用PDA酸响应实现制剂在肿瘤内的可控释放和利用细胞膜对纳米颗粒的功能化修饰来实现体内长循环与肿瘤靶向递送；且光动力和光热活性高，具有良好肿瘤靶向特性和安全性，在治疗肿瘤的应用上前景广泛。

一种基于铁皮石斛纳米囊泡的线粒体靶向纳米酶及其制备方法与应用

Resumen de: CN121550181A

本发明公开了一种基于铁皮石斛纳米囊泡的线粒体靶向纳米酶及其制备方法与应用，属于生物医学材料与组织工程技术领域。本发明提供的线粒体靶向纳米酶兼具增强ROS清除能力与线粒体靶向功能。该线粒体靶向纳米酶结合铁皮石斛纳米囊泡形成具有核壳结构的复合体，从而显著提升其生物相容性。形成的复合体进一步融合于水凝胶中，可以获得用于糖尿病难治性伤口促愈合的复合水凝胶敷料。

一种基于米糠蛋白的纳米马达及其在制备抗动脉粥样硬化的药物中的应用

Resumen de: CN121550177A

本发明提供一种基于米糠蛋白的纳米马达及其在制备抗动脉粥样硬化的药物中的应用，属于生物医药技术领域。该基于米糠蛋白的纳米马达，采用包括如下步骤的方法制备：（1）将二氢槲皮素的乙醇溶液滴加至米糠蛋白溶液中，均质后超声，离心，取沉淀，冷冻干燥后，得到RBP‑DHQ核心纳米颗粒；（2）RBP‑DHQ核心纳米颗粒的表面依次偶联L‑精氨酸和D‑甘露糖，得到功能化纳米颗粒；（3）将功能化纳米颗粒混悬液与螺旋藻分散液混合，搅拌，得到基于米糠蛋白的纳米马达RDHQ‑Arg@Man/SP。本发明纳米马达兼具ROS响应自驱动能力和巨噬细胞靶向性，免受胃肠道环境破坏，口服生物利用度高，抗动脉粥样硬化疗效突出。

一种磷脂酰丝氨酸修饰麦醇溶蛋白纳米粒及其制备方法和应用

Resumen de: CN121550180A

本发明公开了一种磷脂酰丝氨酸修饰麦醇溶蛋白纳米粒及其制备方法和应用。磷脂酰丝氨酸修饰麦醇溶蛋白纳米粒，所述纳米粒为核‑壳结构，以聚乳酸‑羟基乙酸共聚物包载麦醇溶蛋白作为核心，外部包覆有磷脂酰丝氨酸与 DSPE‑PEG2000 形成的脂质复合膜。磷脂酰丝氨酸修饰麦醇溶蛋白负载纳米颗粒，模拟细胞凋亡小体的膜信号特征，增强巨噬细胞对纳米颗粒的识别与摄取，诱导其耐受性表型转化，从而恢复乳糜泻患者对麦醇溶蛋白的免疫耐受，在简化制备工艺的基础上进一步解决现有纳米免疫治疗耐受效应不稳定及缺乏代谢调控的问题。通过上述技术路线制得的纳米颗粒具有良好的粒径分布和稳定性，能够有效递送抗原并诱导免疫耐受，是一种新型可转化的乳糜泻治疗纳米制剂。

一种抗DNMT3A抗体及其在骨关节炎中的应用

Nº publicación: CN121554589A 24/02/2026

Solicitante:

南华大学附属南华医院

Resumen de: CN121554589A

本发明涉及生物医药技术领域，具体公开了一种抗DNA甲基转移酶3A（DNMT3A）的单克隆抗体及其在骨关节炎治疗中的应用。该抗体为单链抗体scFv‑A7，通过噬菌体展示技术筛选获得，其重链可变区（VH）互补决定区（CDR）包含SEQ ID NO:1、SEQ ID NO:2和SEQ ID NO:3所示序列，轻链可变区（VL）CDR包含SEQ ID NO:4、SEQ ID NO:5和SEQ ID NO:6所示序列，全长序列为SEQ ID NO:7。该抗体具有高亲和力，与DNMT3A蛋白的结合EC50值为5.2 nM。在骨关节炎小鼠模型中，该抗体与鱼精蛋白形成纳米颗粒后，可有效递送至细胞核内，抑制DNMT3A活性和表达，降低血清炎症因子IL‑1β和IL‑6水平，改善关节功能评分。本发明为骨关节炎提供了靶向表观遗传调控的新型治疗策略，克服了现有小分子抑制剂选择性差的问题。