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229
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Última actualización
14/07/2026 [07:45:00]
Resultados 75 a 100 de 229
Resumen de: US20260193172A1
0000 A cycloalkane-based lipid compound of Formula 1 and a pharmaceutically acceptable salt thereof are disclosed. The cycloalkane-based lipid compound is a compound having a form in which carbonyl-based substituents are bonded to the central structure of a cycloalkane. The cycloalkane-based lipid compound is used as an ionizable lipid, which is a component of a lipid nanoparticle for delivering a nucleic acid.
0000
Resumen de: US20260191798A1
A gene delivery system and applications thereof in the technical field of biological medicines that is particularly useful in the preparation of drugs for treatment of tumors are disclosed. The gene delivery system and applications relate to technology for inducing the differentiation of malignant tumor cells into mature cells, in which regulating the expression of HNF4 alpha protein in the malignant tumor cells using messenger ribonucleic acid, the malignant phenotype of the malignant solid tumor cells is inhibited, and the effective treatment of the malignant solid tumors is achieved. The gene delivery system and applications are therefore applicable to a method of preparing a drug for treating malignant solid tumors and to a method of treating a patient having a malignant solid tumor.
Resumen de: US20260191929A1
The present invention relates to CD117 targeted LNP molecules for delivery of pro-apoptotic mRNA to hematopoietic stem cells (HSCs) and methods of use thereof for preconditioning a subject for hematopoietic stem cell therapy or to deplete the subject's HSC population for the treatment of a disease or disorder.
Resumen de: US20260191938A1
One aspect according to the present invention relates to: a gold nanozyme including glycol chitosan and gold particles; and a pharmaceutical composition for preventing or treating inflammatory bowel disease, the composition comprising the gold nanozyme. The gold nanozyme and the pharmaceutical composition comprising same according to one aspect of the present invention was found to inhibit the expression of inflammatory factors, inhibit the generation of reactive oxygen species (ROS) and reactive nitrogen species (RNS) inside cells, and reduce the production of nitric oxide (NO) inside cells. In addition, the gold nanozyme was found to inhibit the secretion of Hight Mobility Group Box 1 (HMGB1) when further including glycyrrhizin. In addition, the gold nanozyme and the composition comprising same were found to enable the recovery of the length, weight, etc., of damaged intestines, and thus can be used in the inflammatory bowel disease prevention and/or treatment industry/market.
Resumen de: WO2025046121A1
Disclosed is a lipid nanoparticle (LNP) encapsulating a nucleic acid cargo preferably comprising messenger ribonucleic acid (mRNA). The LNP comprises at least an ionizable lipid fraction, and a stabilizer fraction. The stabilizer fraction preferably comprises at least one polyethylenglycol (PEG) lipid. Furthermore, the ionizable lipid fraction comprises at least one ionizable glycerol dialkyl glycerol tetraether (GDGT) lipid. Also disclosed is a pharmaceutical composition comprising the LNP, such as an mRNA vaccine. In a further aspect, the invention relates to the ionizable GDGT lipids and methods for producing them.
Resumen de: WO2025045142A1
Disclosed herein are immunogenic compositions having circular RNAs encoding VEGF polypeptides. Related methods for manufacture and therapeutic uses thereof are also provided herein.
Resumen de: WO2025049925A2
The present disclosure provides compositions comprising lipidoid compounds, methods of preparing such compositions, and the use of these compositions in gene delivery applications.
Resumen de: EP4772512A1
The present disclosure discloses drugs for preventing and/or treating Alzheimer's disease (AD). A CF3CN derivative provided by present disclosure has any one of structural formulas 1-4 shown below. All four CF3CN derivatives have TrkB agonist activities; and specifically, the CF3CN derivative shown in formula 2 serves as an optimal derivative. In vivo PK studies reveal that the CF3CN derivative shown in the formula 2 is capable of improving a B/P Ratio of CF3CN, and overcoming the limitations of CF3CN. Nanoparticles are prepared by encapsulating the CF3CN derivatives with zein and lactoferrin, which may further enhance an oral bioavailability and a brain drug concentration, thereby improving AD treatment effects. By further improving the formulation and administration route, a liposome is employed to encapsulate the CF3CN derivative for both oral and intranasal administration, which effectively solves the problems of low bioavailability and low brain drug concentration of the derivative.
Resumen de: WO2025049579A1
The invention provides intranasal formulation for pulmonary delivery of nanoparticles, compositions, uses, and manufacturing methods thereof. In one aspect, an intranasal formulation for intranasal or intrapulmonary administration includes a viscosity agent, an epithelial adherence agent, and a foaming agent. The viscosity agent may include carboxymethylcellulose, the epithelial adherence agent may include poly-lysine, and the foaming agent may include gelatin hydrolysate.
Resumen de: WO2025048714A1
There is provided a compound represented by general formula (1) for preparing lipid nanoparticles encapsulating a therapeutic, prophylactic and/or biological agent: wherein A comprises a carbohydrate and/or a derivative thereof; R1 and R2 are each independently a hydrophobic group; R3, R4, R5, and R6 are each independently H, optionally substituted alkyl, optionally substituted alkenyl or optionally substituted alkynyl; R7, R9 and R10 are each independently optionally substituted alkyl, optionally substituted alkenyl or optionally substituted alkynyl; R8 is –O–, –S–, or –NRa–, where Ra is selected from the group consisting of H, optionally substituted alkyl, optionally substituted alkenyl or optionally substituted alkynyl; m is 0 or 1; n ≥ 1; and w ≥ 1.
Resumen de: WO2025049606A1
Compositions and methods for delivering nucleic acids (e.g. DNA) to cells are described herein. In some embodiments, the compositions and methods involve the use of lipid nanoparticles comprising cationic lipids, helper lipids, conjugated (PEG) lipids and structural (sterol) lipids wherein the helper lipid has at least one acyl chain bearing an unsaturation which improves in vivo delivery of the nucleic acid pay load.
Resumen de: WO2025049816A1
A method for inserting a polynucleotide exogenous transgene sequence at a pre- determined endogenous genetic locus in a hose cell genome includes: (i) a donor DNA template including a polynucleotide insert; a 5 '-homology arm; and a 3 '-homology arm. In some embodiments, the 5' homology arm and the 3' homology arm are complementary to the DNA in a target region; and (ii) a ribonucleoprotein complex (RNP) including (1) a Cas nuclease, and at least one small guide RNAs (sgRNA) that is complementary to at least one selected nucleic acid sequence within the pre-determined genetic locus in the host cell genome.
Resumen de: WO2025050106A1
The present disclosure relates to the field of ocular implants suitable for sustained release of drugs (e.g., water-soluble drugs) and use of the ocular implants for treatment of ocular conditions, including glaucoma.
Resumen de: WO2025003756A2
The present disclosure provides multivalent influenza vaccine compositions comprising at least three messenger RNAs (mRNAs) encoding a combination of influenza A and influenza B hemagglutinin (HA) antigens, wherein the mRNA encoding the HA antigen of the influenza A virus is present in a different ratio (w/w) than the mRNA encoding the influenza B virus, and methods of eliciting an immune response by administering said compositions. In particular, the disclosures relate to mRNA encoding these antigens formulated in a lipid nanoparticle (LNP).
Resumen de: CN122342761A
本发明公开了miR‑215‑5p/3p激动剂在制备治疗肠纤维化的药物中的应用。miR‑215‑5p/3p进入肠上皮细胞后，能恢复其在疾病中的生理表达水平，进而抑制ZEB2和STC1介导的上皮细胞EMT信号通路，显著降低成纤维细胞活化、胶原沉积和ECM过度生成，延缓甚至逆转肠纤维化进展，为肠道纤维化提供了一种安全、有效、可推广的新型治疗方案。
Resumen de: CN122342733A
本发明公开了一种生物杂交纳米酶机器人药丸，具备多模式调节炎症性肠病中的肠道氧化还原稳态和菌群失调，步骤如下：步骤一、使用硝酸锌、乙酰丙酮铁和甲醇等药物合成金属有机框架前体，之后煅烧得到铁单原子纳米酶；步骤二、培养富集长双歧杆菌和人诱导多能干细胞，并分别提取二者的外泌体或外囊泡膜；步骤三、将纳米酶与益生菌囊泡膜与干细胞外泌体共挤出，制备生物膜‑纳米酶复合机器人；步骤四、将制得的生物膜‑纳米酶复合粒子封装于磁响应药丸中，形成工程化生物杂交纳米酶机器人药丸。工程化生物杂交纳米酶机器人药丸能够在进入生物体肠道后，在旋转磁场控制下靶向滞留于肠道炎症部位，之后在脉冲磁场作用下促进纳米酶释放并增强酶活性，从而提升治疗效果。工程化生物杂交纳米酶机器人药丸具备生物相容性和治疗安全性，在治疗消化道炎症领域具有广阔应用前景。
Resumen de: CN122342736A
本发明公开了一种基于嗜碱性粒细胞膜构建的仿生纳米颗粒及其制备方法、在制备治疗过敏性疾病药物中的应用，主要包括支撑体、包裹在支撑体外的大鼠嗜碱性白血病细胞系的细胞膜，该细胞膜表面具有FcεRI 蛋白。通过支撑体对细胞膜进行支撑，使其具有相对稳定的形状，形成仿生纳米颗粒。通过FcεRI 蛋白与IgE的高亲和力结合作用，降低IgE浓度，缓解IgE介导的过敏级联反应。
Resumen de: CN122342735A
本发明公开了一种核酸脂质纳米颗粒制剂、其制备方法及其应用，具体公开了一种脂质纳米颗粒制剂，其包括脂质纳米颗粒和冻干保护剂；所述脂质纳米颗粒包括脂质载体和核酸；所述脂质载体包括可电离脂质化合物或其药学上可接受的盐、磷脂、甾醇和PEG脂质；所述可电离脂质化合物为式I所示的化合物。本发明的核酸脂质纳米颗粒制剂冻干后脂质纳米颗粒完整性较好、理化性质较佳或冻干制剂复溶后生物活性较好。
Resumen de: WO2025216375A1
The present invention relates to a hybrid exosome formed by the fusion of a human cell-derived exosome and an artificial exosome. When an active ingredient labeled with a fluorescent material was attached or encapsulated in the hybrid exosome of the present invention and applied to cells, the active ingredient was observed in the cells. In addition, when the exosome was applied to skin tissue, fluorescence expression could also be observed in the dermal layer. Therefore, the hybrid exosome according to the present invention can be used as a novel drug delivery system.
Resumen de: WO2025128958A1
Aspects of the disclosure relate to compositions and methods for improving the stability of lipid nanoparticles (LNPs). In some embodiments, the LNPs comprise one or more active pharmaceutical ingredients (API) encapsulated therein. The disclosure is based, in part, on compositions that directly or indirectly reduce the degradation (e.g., oxidation, hydrolysis, etc.) of one or more lipids components of the lipid nanoparticle. In some embodiments, the compositions comprise a citrate drug product matrix, EDTA drug product matrix, methionine drug product matrix, and/or a tryptophan drug product matrix. The disclosure also provides methods for storing compositions contemplated herein as well as methods for improving the stability of the API.
Resumen de: WO2024248269A1
The present invention provides a hollow mesoporous silica rod, a method for preparing same, and a method for supporting an active substance. The hollow mesoporous silica rod of the present invention has a wide inner hollow and a large aspect ratio, can stably support a large amount of active substance, and can penetrate the surface of the skin to easily reach a predetermined depth inside the skin, thereby effectively delivering the active substance.
Resumen de: CN122342737A
本发明公开了一种共载PD‑1抗体和过氧化氢酶的纳米载体。具体地，所述纳米载体含有：30~60 wt%中性磷脂、20~50 wt%阴离子磷脂、1~20 wt%载脂蛋白和/或其模拟肽、0.1~10 wt%鱼精蛋白、0.01~5 wt%透明质酸、1~10 wt% aPD‑1抗体和1~10 wt%过氧化氢酶，按所述纳米载体的总重量为100 wt%计。本发明的纳米载体共同包封PD‑1抗体与过氧化氢酶，可以协同增强对乳腺癌的治疗效果。
Resumen de: CN122342778A
本发明涉及治疗小儿便秘中药技术领域，且公开了一种治疗小儿便秘中药组合物及其制备方法，包括以下制备步骤：将生地黄、熟地黄、生白术、火麻仁、桑椹、白芍、莱菔子、枳壳、炙甘草混合得到中药原料，中药原料加水煎煮，收集煎煮液后，进行浓缩，得到浓缩浸膏；将浓缩浸膏、复合辅料、玉米淀粉和乙醇混合均匀，制粒，干燥，得到治疗小儿便秘中药组合物复合辅料复配生地黄、熟地黄、生白术、火麻仁、桑椹、白芍、莱菔子、枳壳、炙甘草中药材，具有治疗小儿便秘的功效，且复合辅料中的益生菌具有较高的活性，能够显著增加治疗效果。
Resumen de: CN122342734A
本发明公开了一种靶向Siglec15肿瘤增强免疫治疗疗效的纳米粒及其制备方法与应用，利用生物矿化法制备了Cu2+缀合的磷酸钙纳米粒(CuCaP)，并在纳米粒表面包覆了DTC和Si‑Siglec15RNA。CuCaP@DTC‑Si‑Siglec15在肿瘤酸性微环境发生降解，释放出Ca2+、Cu2+，Cu2+与DTC配位形成毒性CuET，通过通路诱导细胞凋亡，诱导肿瘤细胞ICD。另外，纳米粒释放的Ca2+，导致细胞Ca2+过载，诱导线粒体功能失常，也促进了肿瘤细胞凋亡和ICD效应。并借助Si‑Siglec15RNA以到达高表达Siglec15的肿瘤部位，避免了对正常细胞的毒性。通过联用aPD‑L1可激活强烈的抗肿瘤免疫反应，增强对肿瘤的杀伤，抑制肿瘤转移和复发。
Nº publicación: JP2026522139A 06/07/2026
Solicitante:
ペルセオファーマアーゲー
Resumen de: WO2025008442A1
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, an agent which interacts with the lid domain of the lipase or a fragment thereof, and a protective layer to protect the lipase or the fragment thereof by embedding the lipase or the fragment thereof, wherein the lipase or a fragment thereof is in the open conformation. The present invention also relates to methods of producing said composition.
BOPI
Sede Electrónica
