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50
resultados
Última actualización
02/08/2026 [06:45:00]
Resumen de: US20260218235A1
0000 A composition includes an adeno-associated virus (AAV) vector in a lipid nanoparticle (LNP). The AAV vector can include a heterologous nucleic acid sequence, optionally an inverted terminal repeat (ITR) positioned 5′ of the heterologous nucleic acid sequence and an ITR positioned 3′ of the heterologous nucleic acid sequence. The AAV vector can further include expression control elements (e.g., a promoter and/or enhancer), and intron, and/or or a polyadenylation signal.
Resumen de: EP4782459A1
The present invention relates to Vitamin B12 monomer having the structure represented by the formula 4 or 3, a method of synthesis of Vitamin B12 monomer having the structure represented by the formula 4 or 3, a method of synthesis of nanogels modified with Vitamin B12 using precipitation polymerization, a use of the nanogel modified with Vitamin B12 in drug delivery systems, a pharmaceutical composition comprising nanogel modified with Vitamin B12 and active ingredient, and a pharmaceutical composition comprising nanogel modified with Vitamin B12 and active ingredient for use in the treatment of cancer.
Resumen de: WO2025062440A1
The present invention relates to a topical nano-dispersion composition comprising one or more non-steroidal anti-inflammatory drugs (NSAIDs), muscle relaxants, local anesthetics, analgesics, gabapentinoids, alone or combinations thereof. The composition includes a dispersed phase containing drug(s), lipids, surfactants, amphiphilic penetration enhancers, and solvents, along with an aqueous phase. The nano-dispersion has particle sizes below 300 nm with unimodal particle size distribution, offering enhanced skin permeability and controlled drug release for up to 24 hours. The composition can be formulated into gels, creams, patches, lotions, liquid, or sprays, providing improved patient compliance through non-greasy, fast-absorbing formulations with extended shelf life.
Resumen de: EP4781980A1
The present invention provides a method for producing a ligand-modified lipid nanoparticle encapsulating a nucleic acid, including the following Steps a) to c) and the likeStep a) of mixing an alcohol solution comprising an ionic lipid, a sterol, and a PEG lipid with an acidic buffer having a pH of 1 to 6.5 to obtain a suspension of a lipid nanoparticle free of nucleic acids,Step b) of mixing the lipid nanoparticle free of nucleic acids and a nucleic acid solution to obtain a suspension of a lipid nanoparticle encapsulating the nucleic acid, andStep c) of mixing the suspension of the lipid nanoparticle encapsulating the nucleic acid and a ligand-bound lipid to obtain a suspension of a ligand-modified lipid nanoparticle encapsulating the nucleic acid.
Resumen de: WO2025064475A2
Disclosed are lipidoid compounds having the structure of formula (X) or formula (I): wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.
Resumen de: WO2025062311A2
This disclosure relates to methods of manufacturing cannabinoids (including cannabinoid precursors) and/or derivatives thereof. 5
Resumen de: WO2025061835A1
The present invention relates to oligoglycerol-containing lipids and in particular to cationic or cationizable oligoglycerol-containing lipids and to a method for providing these oligoglycerol-containing lipids. Further, the present invention relates to liposomes, in particular thermosensitive liposomes comprising the novel oligoglycerol-containing lipids. Further the invention relates to liposomes comprising the novel oligoglycerol-containing lipids, which liposomes have an adjustable surface charge.
Resumen de: WO2025059599A1
The disclosure provides ionizable lipids for constructing lipid nanoparticles. The disclosure further provides conjugates comprising a targeting moiety and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (e.g., payload) for delivery to immune cells, hematopoietic stem cells (HSCs), or liver cells. The conjugates can be delivered to cells ex vivo or formulated in a pharmaceutical composition to be directly administered to a subject in need thereof (e.g., via in vivo administration).
Resumen de: WO2025064908A1
Compositions and methods are provided for ultrasound-triggered drug release that enables high drug loading potential for varied drugs of interest, has minimal drug release without ultrasound, can be activated following intravenous administration, and utilizes an ultrasound-activation mechanism that is mechanically-based, without requiring heating above body temperature or cavitation, and is activated with a low duty cycle and shorter ultrasound protocol than is used for heat or sonodynamic activation. These parameters are accomplished by tuning the acoustic impedance and osmolarity of the liquid core of an acoustically activatable particle, e.g. liposomes or emulsions, to maximize ultrasound responsiveness while maintaining overall stability of the structure in the absence of ultrasound.
Resumen de: WO2025061842A1
The invention relates to an oligonucleotide, comprising a sequence that targets a c.877-2 A>G mutation of the transglutaminase-1 (TGM1) gene. In embodiments, the oligonucleotide hybridizes to a target region of the TGM1 gene comprising the c.877-2 A>G mutation (mutation site), and the oligonucleotide is 10 to 35 nucleotides in length. The invention further relates to a gene editing system targeting a c.877-2 A>G mutation of the TGM1 gene, comprising said oligonucleotide, and a base editor, prime editor or RNA-guided endonuclease. In further aspects, the invention relates to a pharmaceutical composition, preferably for use in the treatment of a condition associated with a c.877-2 A>G mutation of the TGM1 gene, comprising said oligonucleotide or said gene editing system with a pharmaceutically acceptable carrier. The invention further relates to an in vitro method for modifying a c.877-2 A>G mutation of the TGM1 gene.
Resumen de: WO2025061694A1
The present invention relates to hollow graphitic carbon nitride nanospheres (hg- C3N4) for use as medicaments. In particular, the present invention relates to hollow graphitic carbon nitride nanospheres (hg-C3N4) as biocompatible and injectable photovoltaic retinal prosthesis for use in the treatment or alleviation of visual deficit and/or cardiac disease. The invention also relates to pharmaceutical compositions comprising the hollow graphitic carbon nitride nanospheres (hg- C3N4).
Resumen de: WO2025065012A1
Disclosed herein are Clostridium neurotoxin (BoNT) or SNARE cleaving homologue variants, constructs, and methods of use. The BoNT or SNARE cleaving homologue variants can include one or more amino acid or domain substitutions, deletions, or insertions in the light chain.
Resumen de: WO2025061967A1
The present invention provides, in part, asymmetric "good" buffer-based cationic lipids of Formula (I'), and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those caused by viral or bacterial infection.
Resumen de: WO2025064507A1
The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV donor polynucleotide vectors and LNP compositions comprising a targeting gRNA pair and a nucleic acid encoding Cas-CLOVER fusion protein, and methods of using the compositions for treating PKU.
Resumen de: CN122461254A
本申请公开了聚碳酸酯‑脂质组合物、复合载体及其在mRNA脾脏靶向递送中的应用，属于生物医药技术领域。按照摩尔百分比计量，聚碳酸酯‑脂质组合物包括聚乙二醇‑聚碳酸酯嵌段共聚物0.1%~1%，可电离阳离子脂质95%~99%，永久电荷阳离子脂质1%~4%。通过对聚碳酸酯结构及脂质比例的系统优化，实现mRNA表达水平的提高；并提高树突状细胞、巨噬细胞及B细胞等脾脏免疫细胞中的mRNA递送与表达效率，同时降低肝脏等非靶器官的非特异性摄取，在mRNA疫苗、肿瘤免疫治疗及脾脏靶向递送领域具有良好的应用前景。
Resumen de: CN122461256A
本发明属于纳米药物制剂与肿瘤光动力治疗技术领域，具体涉及一种肿瘤双重靶向光敏剂递送纳米粒及其制备方法。该纳米粒以DSPE‑PEG2000、DSPE‑PEG2000‑cRGD、低分子量透明质酸钠为载体，包载疏水性光敏剂，形成粒径 60‑100 nm，具有cRGD/HA双重主动靶向作用的纳米粒。本发明制备工艺温和、绿色、可工业化放大，有效解决了传统光敏剂递送系统靶向性不足、制备工艺复杂等问题，显著提升了光动力治疗效果与临床转化潜力。
Resumen de: CN122461257A
本发明涉及药物制剂技术领域，特别涉及一种5‑氟尿嘧啶的靶向递送纳米粒制剂及制备和应用。制备为将水相加入至有机相中并加入聚乙烯醇溶液获得5‑氟尿嘧啶的初乳（W/O），而后将获得初乳注入外水相中，搅拌混匀使体系内有机相溶剂完全挥发，获得胶体，真空冻干即得纳米粒。本发明纳米粒具有生物降解性、缓释作用等特性，为乳腺癌等多种肿瘤的治疗提供了新的思路，为提升抗肿瘤化疗药物的治疗能力从新型药物制剂的角度拓展了思路和方法。
Resumen de: CN122461262A
本发明公开了一种磷酸铈和氧化铈协同矿化菌的方法及高耐酸抗氧化益生菌与应用，该方法通过在细菌表面原位矿化生成具有致密的、具有多尺度纳米结构的含铈纳米壳层，所示纳米壳层同时含有铈的氧化物与磷酸盐复合物，利用含铈纳米壳层的物理屏障作用和铈的价态转变特性，赋予益生菌高耐酸性和抗氧化能力。本发明构建的磷酸铈和氧化铈协同矿化菌在酸性环境和氧化应激条件下存活率显著提升，解决了益生菌生产、储存及体内转运过程中易失活的技术难题，实现了益生菌存活率的有效提升，进而使得益生菌在施用过程中能够有尽可能多的活菌到达目标位置并发挥作用，有效提高了益生菌的使用率。
Resumen de: CN122464906A
本发明公开了一种BODIPY类化合物及其应用。本发明以BODIPY二聚体为功能母核，在分子特定α活性位点处共价偶联不同链长及不同极性功能端基的PEG侧链，构建了如式（Ⅰ）所示的BODIPY类化合物。构效关系研究表明，该类化合物可调控吸收波长，具有I/II型混合光敏化机制，摆脱了单一II 型对氧气的依赖，适配肿瘤乏氧环境，且可调控活性氧产率，远超吲哚菁绿；PEG 侧链改性提升水溶性，可在水相自组装成纳米颗粒，光热稳定性优异，近红外吸收发射性能好。体外细胞实验表明，该类化合物在激光照射下对肿瘤细胞具有显著光毒性，为肿瘤高效光动力治疗开辟了新思路。
Resumen de: CN122461261A
本发明公开了一种金属酚配位型厚朴酚‑顺铂纳米制剂及其制备方法与应用，属于医药纳米制剂技术领域。本发明区别于现有单纯和厚朴酚脂质体物理包埋制剂，采用厚朴酚双酚羟基与活化顺铂离子金属‑酚配位自组装构建金属多酚网络纳米体系；以厚朴酚为配位配体兼顺铂递送载体，复配DSPE‑PEG2000、DSPE‑PEG2000‑叶酸靶向辅料，精准调控组分摩尔比，将制剂粒径控制在100～200 nm。本发明制备工艺条件温和、粒径均一、载药量稳定，可通过腹腔注射实现全身抗非小细胞肺癌作用，相比现有和厚朴酚脂质体制剂具有显著创造性与临床应用价值。
Resumen de: CN122465159A
本发明属于抗菌抗氧化材料的技术领域，公开了一种高抗菌抗氧化的基于铁锌双金属配位聚合物纳米粒子及其制备与应用。方法：1）将聚乙烯吡咯烷酮溶于乙醇溶液，随后加入铁盐的乙醇溶液混合，调节pH为8‑9，提高搅拌的速度，滴加单宁酸的乙醇溶液，滴加完后继续搅拌，获得铁基单宁酸金属酚醛网络；2）向铁基单宁酸金属酚醛网络中加入氯化锌的乙醇溶液，搅拌反应8~12h，获得高抗菌抗氧化基于铁锌金属配位聚合物纳米粒子。本发明的方法简单，本发明的纳米粒子具有较好的抗氧化、抗菌作用。所述纳米粒子用于制备抗菌抗氧化材料，特别是制备治疗角膜溃疡的药物。
Resumen de: CN122461255A
一种改性水蛭素靶向递送系统及其构建方法和应用，属于生物制剂技术领域。所述系统先利用单宁酸对水蛭素表面改性，在单宁酸改性水蛭素的外层引入多糖壳体后获得，所述多糖为磷酸化β‑多糖PG、磷酸化右旋糖酐PD、硫酸化右旋糖酐SD、岩藻多糖FUS中的至少一种，所述水蛭素和单宁酸的摩尔比为1:10~100。通过在水蛭素表面引入单宁酸物理沉积，调控蛋白包封机制由静电力驱动向范德华力驱动，从而显著提高包封效率，并结合多糖构建模组化外壳，不仅能通过改变多糖选择，灵活实现对不同细胞的靶向，还能有效减缓降解速度，从而获得一种工艺简单、包封率高、靶向性强且稳定性优良的水蛭素递送系统。
Resumen de: CN122461499A
本发明属于生物医药技术和纳米医学技术领域，具体涉及一种双层纳米壳层修饰的工程化减毒沙门氏菌及其制备方法和应用。工程化减毒沙门氏菌为壳核结构，以改造的减毒鼠伤寒沙门氏菌VNP20009工程菌为核，内核工程菌株表面修饰纳米外壳；其中，改造的减毒鼠伤寒沙门氏菌VNP20009获得工程菌为通过基因工程手段构建表达Gasdermin E蛋白N端活性结构域（GSDME‑N）的工程菌VNP20009‑GSDME‑N（VNP‑G）；以及通过基因工程手段构建表达肿瘤坏死因子超家族成员TNFSF14（LIGHT）蛋白胞外功能区的工程菌VNP20009‑LIGHT（VNP‑L），将其和VNP‑G联用。工程菌在肿瘤微环境中能够稳定表达功能蛋白，显著增强肿瘤局部免疫应答并诱导肿瘤细胞发生焦亡，从而实现对实体瘤生长和转移的有效抑制。
Resumen de: CN122461252A
本发明提供了一种疏水药物纳米粒包封制剂、制备方法及应用，方法包括以下步骤：将酪蛋白酸钠溶于超纯水中，搅拌，得到酪蛋白酸钠原液；将果胶溶于超纯水中，配制成浓度为的果胶原液；将玉米蛋白溶解于乙醇中，配制成玉米蛋白溶液，搅拌，然后加入药物，继续搅拌；将酪蛋白酸钠原液和果胶原液加入到溶液中，然后调节pH；将得到的溶液加热保温，然后降至室温，诱导组分络合，得到药物‑玉米蛋白‑酪蛋白酸钠‑果胶纳米颗粒。本发明采用上述的一种疏水药物纳米粒包封制剂、制备方法及应用，赋予纳米颗粒优良的肠溶特性，粒径均一、结构可控，优异的抗胃聚集能力，制备工艺简单、绿色环保，显著提高槲皮素的肠道靶向性和口服生物利用度。
Nº publicación: CN122461259A 28/07/2026
Solicitante:
中山大学附属第一医院
Resumen de: CN122461259A
本发明属于纳米药物领域，公开了一种肿瘤靶向共递送纳米系统，递送纳米系统的核心为包载疏水性化疗药物的mPEG‑PLA共聚物，表层为吸附有抗肿瘤siRNA的DOTAP，同时表层还包含偶联抗体的功能化脂质。本发明构建了“抗体主动靶向+顺铂化疗+基因治疗协同递送”三位一体的纳米递送系统，能够显著增强疗效，实现“逆转耐药+杀伤肿瘤”的协同效应，使耐药细胞的IC50值显著下降，集落数目明显减少，同时在体内实验中也得到验证，并且该递送系统具有良好的胶体稳定性、生物安全性和相容性。
BOPI
Sede Electrónica
