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208
resultados
Última actualización
11/05/2025 [06:54:00]
Resumen de: WO2023205424A2
The present invention relates to ionizable lipids and lipid nanoparticle compositions thereof. The nanoparticle compositions are useful in the delivery of therapeutic agents such as nucleic acids.
Resumen de: CN119950733A
本发明属于药物载体技术领域，涉及一类可离子化脂质载体、其组合物与应用。该脂质载体为一种或多种氨基酸生育酚酯或其药学上可接受的盐，用于递送治疗剂或预防剂，其中，治疗剂或预防剂为siRNA、shRNA、miRNA及mRNA核酸分子，多肽或蛋白质中的一种或多种。在优选的实施例中，氨基酸结构部分为赖氨酸、组氨酸、精氨酸，或及其衍生物的残基。本发明还公开了包括一种或多种可离子化脂质载体与治疗剂或预防剂制成的组合物，以及该脂质载体或组合物在制备核酸药物、小分子药物、多肽或蛋白质药物中的应用，其中，所制备的负载RNA脂质纳米粒，可在体内表达或沉默蛋白，具有稳定性好、转染效率高、毒性低、安全有效等优点。
Resumen de: CN119301261A
The present disclosure relates to the field of molecular virology, including nucleic acid molecules comprising a modified viral genome or self-replicating RNA, pharmaceutical compositions containing said nucleic acid molecules, and the use of such nucleic acid molecules and compositions for producing a desired product in a cell culture or in vivo. Also provided are methods for eliciting a pharmacodynamic effect in a subject in need thereof, as well as methods for preventing and/or treating a variety of health conditions.
Resumen de: CN119950450A
本发明公开了一种巨噬细胞膜包裹的纳米复合体、其制备方法及其应用，巨噬细胞膜包裹的纳米复合体是由巨噬细胞膜包裹且由表面阳离子特性的层状双氢氧化物LDH纳米材料和microRNA‑182制备得到的，纳米复合体的粒径为20~150 nm。本发明的巨噬细胞膜包裹的纳米复合体，一方面体内靶向心肌缺血再灌注损伤区域，另一方面对抗心肌缺血再灌注损伤后的心肌细胞焦亡，并且达到保护远期心功能改善心肌纤维化的目的，能够有效对抗心肌缺血再灌注损伤，对急性心肌梗死患者具有重大意义，应用前景好。
Resumen de: CN119950448A
本发明公开了一种γ‑聚谷氨酸/玉米醇溶蛋白复合纳米颗粒及其制备方法和应用，包括，将玉米醇溶蛋白溶于乙醇溶液中，得到玉米醇溶蛋白溶液；将γ‑聚谷氨酸溶于去离子水中，得到γ‑聚谷氨酸溶液；将玉米醇溶蛋白溶液加入γ‑聚谷氨酸溶液中，蒸发除去溶液中的乙醇，离心除去不溶物，得到γ‑聚谷氨酸/玉米醇溶蛋白复合纳米颗粒分散液；经冷冻干燥得到γ‑聚谷氨酸/玉米醇溶蛋白复合纳米颗粒粉末。本发明的γ‑聚谷氨酸/玉米醇溶蛋白复合纳米颗粒制备方法简单、成本低、且绿色安全，得到的纳米颗粒可以在较宽的pH、离子强度和温度范围内保持稳定，有着良好的生物相容性，可以应用于对生物活性成分的高效包埋，并改善其活性与稳定性。
Resumen de: CN119950699A
本发明公开了一种用于原位捕获抗原和淋巴结靶向递送的纳米疫苗及其制备方法和应用。该纳米疫苗包括聚合物纳米组装体和药物，所述聚合物纳米组装体包括两亲性聚合物与阳离子脂质体共组装体；所述两亲性聚合物包括聚乙二醇‑聚磷酸酯嵌段共聚物；所述药物包括免疫佐剂。本发明提供的纳米疫苗不仅可以用于原位捕获肿瘤抗原，还可以利用其小尺寸优势携带抗原和免疫激动剂靶向递送至肿瘤引流淋巴结，促进树突状细胞成熟，成熟树突状细胞加工处理抗原呈递给T细胞，激活CD8+T细胞，诱导强大的抗肿瘤免疫反应。
Resumen de: CN119950714A
本发明属于生物医药技术领域，具体涉及一种多模态成像的纳米诊疗剂及其制备方法和应用。该制备方法包括以下步骤：S1、配置IR780的二甲亚砜溶液；S2、配置单宁酸水溶液；S3、配置六水合三氯化铁水溶液；S4、超声状态下向IR780的二甲亚砜溶液中逐滴滴加超纯水，滴加完毕继续超声；S5、超声状态下向S4混合液中逐滴滴加单宁酸水溶液，滴加完毕继续超声；S6、超声状态下向S5混合液中逐滴滴加三氯化铁水溶液，滴加完毕继续超声，8000～10000转速离心水洗。本发明克服了传统诊断剂及治疗剂治疗周期长等不足，具有生物相容性好、毒副作用小和光调控能力强的优点。
Resumen de: JP2024056845A
To provide a therapeutic nanoparticle comprising an oncologic drug and taurolidine, which harness the synergistic effect of taurolidine on these oncologic drugs so as to allow for greater efficiency and reduced toxicity associated with the oncologic drugs.SOLUTION: Provided is a therapeutic nanoparticle, comprising at least one oncologic drug and taurolidine, whereby to provide the simultaneous delivery of the at least one oncologic drug and taurolidine, thereby harnessing the synergistic effect of taurolidine on the at least one oncologic drug. Preferably, the at least one oncologic drug comprises TNF-related apoptosis-inducing ligand (TRAIL).SELECTED DRAWING: None
Resumen de: CN119949514A
本发明提供一种具有减肥功效的纳米微胶囊及其制备方法和应用。该纳米微胶囊包括芯材和包覆在芯材表面的壁材，其中，芯材包括椰子甘油二酯，壁材包括食源肽、药食同源物质、表没食子儿茶素没食子酸酯。本发明的纳米微胶囊可以通过多种途径促进脂肪分解、以多机制的方式达到减肥功效，促使每个组分发挥协同效应，从而增强整体的减肥功效，且对人群具有普适性；其能够控制体重及腰围，调理脂代谢平衡，遏制食欲，从而达到减肥的功效，还具有缓解高脂饮食造成的肝脏、肾脏的炎性病变的潜力，最终达到健康减肥的功效。
Resumen de: AU2023276484A1
Provided herein are compounds comprising an oligonucleotide molecule conjugated to a ligand for a glucose transporter (GLUT) for modulation of a target molecule, compositions comprising the same, and methods of using the compositions.
Resumen de: CN119950396A
本发明属于生物医药技术领域，具体涉及一种含有肿瘤源性总RNA纳米疫苗的可溶性微针及其制备方法和应用。本发明利用鱼精蛋白可以和RNA以正负电相结合的特点，将鱼精蛋白、TdRNA和CpG复合，形成纳米疫苗，用于治疗或预防肿瘤。富含肿瘤抗原广谱遗传编码的TdRNA和CpG的协同作用随后驱动树突状细胞的成熟和细胞毒性T淋巴细胞的激活，从而产生强大的抗肿瘤免疫，有效地治疗或预防肿瘤，并且无显著的生理毒性，安全性高。此外，这种微针装载RNA疫苗的方式可以有效保护RNA，防止RNA体外降解，降低了储存及运输难度。
Resumen de: MY202341A
Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
Resumen de: CN119950449A
本发明公开了一种用于猴痘预防的吸入式仿生纳米粒子及其制备方法与应用。所述吸入式仿生纳米粒子以模拟肺表面活性物质的脂质和巨噬细胞膜混合形成该纳米粒子的载体，包裹猴痘抗原和cGAMP，合成吸入式仿生纳米粒子，所述吸入式仿生纳米粒子可以有效增强抗猴痘病毒的体液免疫和细胞免疫。本发明还提供所述吸入式仿生纳米粒子在制备鼻吸入式疫苗制剂中的应用，所述鼻吸入式疫苗制剂能诱导在肺部和粘膜组织的粘膜免疫，有效地保护正痘病毒通过阴道和肠腔入侵，阻止猴痘病毒的性传播；以所述鼻吸入式疫苗免疫可以完全预防小鼠因正痘病毒属VACV的减毒株感染而死亡，且完全消除皮疹的产生，本发明对实现猴痘的有效预防具有意义。
Resumen de: CN119950538A
本发明提供了用于递送核酸药物的CXCR4抑制性聚合物及其应用。具体地，提供了一种活性成分组合在制备治疗肿瘤或抑制肿瘤细胞的药物组合物或药盒中的用途，其中，所述活性成分组合包括：(a)所述第一活性成分：靶向RRM2的核酸药物；和(b)第二活性成分：吉西他滨。本发明的含CXCR4抑制性聚合物的核酸药物纳米颗粒能够有效抑制胰腺癌的生长和转移。此外，本发明提供的核酸药物纳米颗粒与吉西他滨联用时，可协同地治疗胰腺癌，尤其是耐药性或难治性胰腺癌。
Resumen de: CN119950757A
本发明针对肿瘤内过氧化氢不足和一氧化氮超短寿命的肿瘤治疗障碍，提出了一种活性氧和活性氮级联抗肿瘤纳米药物，以植物醛和天然氨基酸的联合作用作为抗肿瘤治疗的核心内容。以纳米颗粒为载体，在所述载体外包覆生物相容性材料，负载或接枝植物醛，封装天然氨基酸。在肿瘤酸性环境中，植物醛释放羟基自由基实现活性氧疗法。在产生羟基自由基的同时，植物醛还会缓慢释放过氧化氢，进而引发天然氨基酸向一氧化氮的转化，实现活性氮疗法。植物醛的控制释放致使一氧化氮释放得以可控，时空释放活性氧和一氧化氮的实现，从而充分利用活性氧和一氧化氮在药物动力学上的互补性，产生强大的抗肿瘤效应。本发明制备易操作，原料易获得，制备的负载植物醛和天然氨基酸的纳米颗粒具有较好颗粒分散性、化学稳定性和生物相容性，是一种理想的化学‑气疗‑铁死亡级联癌症治疗试剂。
Resumen de: CN119278031A
The present disclosure relates generally to the field of nucleic acid (e.g., DNA or RNA, in particular mRNA or inhibitory RNA, e.g., siRNA) compositions comprising a multivalent anion (e.g., inorganic polyphosphate), methods for preparing and storing such compositions, and use of such compositions in therapy.
Resumen de: US2025144042A1
Described are coated particles containing a core and a coating, and pharmaceutical formulations containing these coated particles. The core contains a polymer or a hydrophobic drug. The coating contains a glycoprotein or a combination of a sugar and a protein. The coating surrounds the core. The coated particles are effectively absorbed by mucosa such as intestinal mucosa, GI tissue, and/or vaginal mucosa, and show increased systemic uptake following oral or mucosal administration. Optionally, the coated particles contain one or more active agents encapsulated in the core and/or embedded in the coating of the particles, for systemic or local delivery.
Resumen de: US2025144018A1
The bio-microbur therapeutic delivery platform is a three-dimensional (3D)-oriented nanoneedle platform shaped like a microscale version of a fruit bur. The bio-microbur may be used for drug delivery and other biological applications, including without limitation delivery of oral vaccines or other oral biologics. Similar to a fruit bur's ability to adhere to different surfaces, the bio-microbur therapeutic delivery platform adheres to biological tissue, cell membranes, and biological gels. The bio-microbur therapeutic delivery platform includes a core and a plurality of nanoneedles secured to the core and extending outwardly therefrom, adapted for carrying and delivering a therapeutic agent. The treating agent may be an SSRI, such as fluoxetine.
Resumen de: US2025145976A1
The present disclosure provides optimized mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system. Also provided herein are delivery systems for use of the CRISPR/Cas system in methods of in vivo and ex vivo genome editing.
Resumen de: US2025146016A1
The present disclosure relates to circular, non-viral DNA vectors, compositions including one or more of the disclosed vectors, and methods for delivering and/or expressing one or more therapeutic genes (e.g., proteins) in mammals, e.g., human patients. In some embodiments, the present disclosure is directed to circular, non-viral DNA vectors, such as circular non-viral DNA vectors including at least two inverted repeat sequences, where the at least two inverted repeat sequences are separated by a non-repeated nucleotide sequence which is not part of the at least two inverted repeat sequences. In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element.” In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element,” but include at least a portion of a bacterial origin of replication.
Resumen de: US2025144043A1
Among the various aspects of the present disclosure is the provision of compositions and methods for treating cancer and sensitizing cancer or tumors to treatments. An aspect of the present disclosure provides for a method of preventing treatment resistance in non-HPV-associated cancer comprising administering a pharmaceutical composition comprising HPV-associated DNA (e.g., HPV-associated exosomes). Another aspect of the present disclosure provides for a method of increasing response to cancer treatment, optionally, surgery, radiation therapy, and chemotherapy in a subject having, suspected of having, being treated for, having previously been treated for, or diagnosed with cancer comprising administering a pharmaceutical composition comprising HPV-associated DNA (e.g., HPV-associated exosomes).
Resumen de: WO2025097132A1
The present invention is directed to a capsid containing (a) an endogenous Gag polypeptide, (b) one or more effector-polypeptide conjugates, where each effector-polypeptide conjugate independently comprises an endogenous Gag polypeptide, and (c) optionally a heterologous cargo, such as an mRNA or siRNA.
Resumen de: WO2025096980A1
Disclosed are methods and compositions for functional genetic modifications at selected genomic sites such as KLKB1 gene. Also provided are cell populations, which comprise the functional genetic modification at one or more selected gene loci.
Resumen de: WO2025096878A1
This disclosure provides improved RNA molecules, including mRNA molecules that can be produced by in vitro transcription and are suitable for in vivo transfection using an appropriate delivery vehicle, such as a lipid nanoparticle (LNP) or targeted lipid nanoparticle (tLNP). The improved RNA include particular combinations of 5' untranslated region (UTR) and 3' UTR, particular 3' UTRs, or particular open reading frame sequences. Also provided herein are compositions of the LNP, or tLNP with an antibody as a targeting moiety, such as anti-CD8 antibodies that are used as targeting moiety.
Resumen de: WO2025096848A1
The disclosure relates to microparticles and nanoparticles comprising a porous polymer matrix comprising an uncapped polymer for sustained delivery of a net positively charged therapeutic agent. More particularly the disclosure relates to particles comprising PLGA or PLA which have a first state with relatively more interconnected pores at a first pH and a second state with relatively less interconnected pores at a second pH. Methods of making the particles and administering the particles are also provided.
Resumen de: AU2023369585A1
The present disclosure relates to RNA molecules encoding a respiratory syncytial virus (RSV). The present disclosure further relates to compositions comprising the RNA molecules formulated in a lipid nanoparticle (RNA-LNP). The present disclosure further relates to the use of the RNA molecules, RNA-LNPs and compositions for the treatment and/or prevention of RSV infection-induced acute respiratory tract illness, including pneumonia and bronchitis.
Resumen de: WO2025091119A1
A blood cell-based therapeutic agent delivery system is disclosed. A blood cell-based therapeutic agent delivery vehicle is useful for delivering a therapeutic agent into a recipient organ cell. The blood cell-based therapeutic agent delivery vehicle is made by a method comprising encapsulating the therapeutic agent into a lipid nanoparticle to produce a therapeutic agent-containing lipid nanoparticle, and transfecting the therapeutic agent- containing lipid nanoparticle into a blood cell by incubating the therapeutic agent-containing lipid nanoparticle with the blood cell to form a transfected blood cell. The administration of the transfected blood cell causes the transfected blood cell to be in close proximity to or direct contact with the recipient organ cell, thereby causing a transfer of the therapeutic agent into the recipient organ cell. A composition for a blood cell-based therapeutic agent delivery vehicle for use in delivering a therapeutic agent into a recipient organ cell is also disclosed.
Resumen de: WO2025092866A1
Provided is an isolated mRNA molecule, which contains a nucleotide sequence encoding a chimeric immunogenic polypeptide, wherein the chimeric immunogenic polypeptide contains an immunogenic fragment of hemagglutinin (HA) of influenza A H5N1, an immunogenic fragment of hemagglutinin (HA) of influenza A H1N1, and an immunogenic fragment of hemagglutinin (HA) of influenza B Victoria, which are linked to each other. Further provided are a composition and vaccine containing the mRNA, a fusion protein encoded thereby, and a method using same for inducing an immune response to influenza virus in a subject.
Resumen de: WO2025096329A1
The present disclosure is directed to methods of preparing peptide nanoparticle formulations, and in particular, peptide nanosuspensions, using low shear milling. More specifically, the disclosure is directed to methods of preparing peptide nanosuspensions by applying low frequency acoustic energy to an admixture comprising a peptide, an aqueous dispersion medium comprising a surface-active polymer and optionally a surfactant, and milling media, until the peptide has been milled to nanoparticle size. Also described are stable peptide nanosuspensions prepared by the methods.
Resumen de: WO2025093745A1
The present invention relates to glycoengineered extracellular vesicles, as well as methods of production of such glycoengineered extracellular vesicles, methods of glycoengineering extracellular vesicles, and uses of glycoengineered extracellular vesicles.
Resumen de: WO2025093665A1
The present disclosure relates generally to functionalized particles containing moieties capable of binding Immunoglobulin D (IgD), methods for producing them, and to pharmaceutical compositions containing them and their uses in medicine.
Resumen de: WO2025095526A1
The present invention relates to a synthetic nucleic acid molecule comprising 3'-UTR polynucleotide with improved translation efficiency and a vaccine composition comprising the same, and more particularly to a synthetic nucleic acid molecule comprising 3'-UTR with improved translation efficiency manufactured by inclusion of specific motif and a codon-optimized signal sequence and an antigen encoding sequence, and a vaccine composition comprising the same. The synthetic nucleic acid molecule according to the present invention comprises a 3'-UTR polynucleotide with improved translation efficiency, which can effectively induce the expression of an antigenic polypeptide, which is useful for vaccine development because it can be expected to increase immunogenicity as a vaccine.
Resumen de: WO2025093798A1
The present invention relates to nanoparticles for the controlled release of curcumin comprising: a core formed by aggregation of gold nanoparticles where said aggregate is in the form of a nanostar; a first coating consisting of a mesoporous silica layer where the curcumin is encapsulated or embedded; a second coating covering the porous silica layer, where said second coating consists of octadecyltrimethoxysilane; a third coating covering the layer of octadecyltrimethoxysilane, where said third coating consists of a heat-sensitive compound. The present invention further relates to the preparation method for said nanoparticles and to the use thereof.
Resumen de: WO2025092744A1
Disclosed are an aminocholesterol derivative and a composition comprising same. Nanoparticles containing the aminocholesterol derivative are used for delivering nucleic acid drugs to improve the delivery efficiency for the nucleic acid drugs, thereby enhancing the targeting and therapeutic effect of the nucleic acid drugs. Thus, the nanoparticles are of great significance for the development and application of nucleic acid prophylactic and therapeutic agents.
Resumen de: WO2025093041A1
An immunogenic fragment of African swine fever virus CD2v protein, a recombinant protein, an immunogenic composition, and a use thereof. The provided immunogenic fragment of African swine fever virus CD2v protein or a variant thereof with immunogenicity can greatly improve the expression quantity while retaining the strong immunocompetence.
Resumen de: WO2025093042A1
Provided are an immunogenic fragment, a recombinant protein, and an immunogenic composition of the African swine fever virus E248R protein, as well as the use thereof. The immunogenic fragment of the African swine fever virus E248R protein or an immunogenic variant thereof can greatly improve expression levels while retaining strong immunological activity.
Resumen de: WO2025097153A1
The present disclosure related to methods of treating various diseases that can benefit from modulating angiogenesis and/or endothelial-to-mesenchymal transition (EndMT) pathways, such as retinopathies and cancer, by modulating family with sequence similarity 222 member A (FAM222A) expression in the endothelium (e.g., endothelial cells) of a subject. For example, FAM222A can be modulated with FAM222A inhibitors described herein, thereby reducing angiogenesis, or FAM222A activators described herein, thereby increasing angiogenesis.
Resumen de: WO2025097049A1
Disclosed herein are compositions and methods for CMC production of RNA therapeutic complexes (nanostructures) that contain SN-38 (7-Ethyl-10-hydroxycamptothecin) and/or Irinotecan. In particular, disclosed herein is an RNA nanoparticle having at least three synthetic RNA nucleotides coupled to each other, wherein the at least three synthetic RNA oligonucleotides form a central ore domain and at least three double-stranded arms arranged around the core domain and extending away from the central core domain, wherein at least one of the three double-stranded arms is conjugated with Irinotecan and/or SN-38 with an esterbond that is cleavable by esterase in cancer tissue or cancer cells.
Resumen de: WO2025095618A1
The present invention relates to a cationic lipid and a method for preparing same and, more specifically, to: a cationic lipid which, due to the specific structure thereof, forms a complex with an anionic drug, and thus is useful for drug delivery; and a method for preparing same.
Resumen de: WO2025095659A1
The present invention relates to a cationic lipid and a method for preparing same and, more specifically, to: a cationic lipid which, due to the specific structure thereof, forms a complex with an anionic drug, and thus is useful for drug delivery; and a method for preparing same.
Resumen de: WO2025096824A1
Disclosed are lipid nanoparticle (LNP) delivery systems that specifically target T cells. The LNP delivery system comprises antibodies conjugated to the surface of the LNP, e.g., via maleimide chemistry, that target at least two T cell surface proteins, e.g., CD3 and CD28. The LNP delivery system can have a single population of LNP conjugated to either a bispecific antiCD3/antiCD28 antibody, or two monospecific antiCD3 and antiCD28 antibodies, or two populations of LNP wherein each population comprises a monospecific antibody. The payload of the LNP delivery system can be, e.g., mRNA encoding chimeric antigen receptors (CAR), a linear DNA fragment or a plasmid encoding chimeric antigen receptors (CAR) or therapeutic proteins such as antibodies, components of a gene editing systems (e.g., CRISPR-Cas), small molecules, antibody-drug conjugates (ADC), and any combination thereof, either encapsulated in the LNP or attached to its surface (e.g., conjugated). Also provided are lipids, pharmaceutical compositions, kits, and methods of treatment.
Resumen de: WO2025095328A1
The present invention relates to nanoparticles, a preparation method therefor, a pharmaceutical composition containing the nanoparticles, and an anticoagulant containing the composition, the nanoparticles comprising: a low-molecular-weight heparin (LMWH); and a lipid bound to the reducing end of the LMWH, wherein the lipid consists of 4-24 carbon atoms and is self-assembled in an aqueous solution. Specifically, it has been identified that when the nanoparticles are administered in vivo, the anticoagulant effect is higher than that of a conventional LMWH and the effect is sustained over time. Therefore, the nanoparticles, the composition containing the nanoparticles, and the anticoagulant containing the composition can be widely used in various medical fields.
Resumen de: WO2025095229A1
The present invention relates to messenger RNA (mRNA)-encapsulated double nanoparticles, a method for preparing same, and a pharmaceutical use thereof and, more specifically to: mRNA-encapsulated double nanoparticles comprising a first nanoparticles core in which mRNA, which is an active ingredient, is bonded to an anionic polymer surface or is inserted into the anionic polymer surface, and a second nanoparticle shell in which a biocompatible polymer or a polymer conjugate is formed to surround the outside of the core; a composition for mRNA delivery comprising the double nanoparticles; a composition for a vaccine comprising the double nanoparticles, and the like. The double nanoparticles according to the present invention can be used to safely and efficiently deliver mRNA to a target site.
Resumen de: US2025144252A1
Carbon nanoparticle suspension injection-Fe, a preparation method, an application and a use method. The carbon nanoparticle suspension injection-Fe includes carbon nanoparticle suspension injection and ferrous sulfate, where a concentration of the carbon nanoparticle suspension injection is 20-100 mg/mL, a concentration of ferrous ions in the carbon nanoparticle suspension injection-Fe is 0.5-60 mg/mL, and a particle size of carbon nanoparticles in the carbon nanoparticle suspension injection-Fe is 90-250 nm, and a pH value is 2.8-6.0. The carbon nanoparticle suspension injection-Fe serves as a developing agent after being injected into a tumor to show distribution of the carbon nanoparticle suspension injection-Fe and retention in the tumor on a magnetic resonance image.
Resumen de: US2025144041A1
A pharmaceutical composition for treating retinitis pigmentosa that includes statin-loaded nanoparticles. The pharmaceutical composition is used in a method of treating retinitis pigmentosa involving administering to a subject in need thereof the pharmaceutical composition that includes statin-loaded nanoparticles.
Resumen de: US2025144039A1
There are disclosed compositions comprising core-shell nano-elements composed in their core of a water-insoluble thermoplastic compound having a molecular weight of more than 0.6 kDa and in their shell of at least one fatty amine, or a part thereof, the core-shell nano-elements having a viscosity of 107 mPa·s or less and being dispersed in a polar carrier as nano-elements having an average diameter of 200 nm or less. The water-insoluble thermoplastic compound can be capable of stimulating the synthesis of skin proteins and/or enabling the delivery of active agents upon application of the core-shell nano-elements to a surface to be treated therewith. Methods for preparing the composition and uses thereof are also provided.
Resumen de: US2025144088A1
The present invention concerns a nanocarrier formulation for use in the treatment of bacterial infections of the lower airways by inhalation therapy.
Resumen de: US2025144183A1
The present disclosure relates to methods, compositions and kits for modulating the expression of LPA gene and for treating lipoprotein-related diseases, for example cardiovascular diseases, in a subject by gene editing.
Resumen de: US2025144169A1
Provided herein are methods and compositions for treating solid tumor cancers.
Resumen de: US2025144040A1
Composite particle including the following: (1) a nanocrystal of an organic compound, and (2) a block copolymer associated with one or more surfaces of the nanocrystal of an organic compound. The organic compound, in some embodiments, is a pharmaceutical compound, a therapeutic compound, and/or a bioactive compound. The block copolymer may be a block copolymer with at least one hydrophilic poly(2-oxazoline) block. Pharmaceutical compositions may include these composite particles. The pharmaceutical compositions may include a dispersion that has an aqueous or aqueous-based continuous phase and a dispersed phase that includes the composite particles. The dispersion may be administered to sick patients, including those diagnosed with or exhibiting symptoms of COVID-19. For example, the dispersion may be aerosolized, and a patient may inhale the aerosol.
Resumen de: US2025144232A1
Disclosed are compositions comprising a lipid nanoparticle and a modified biomolecular corona. In some embodiments, the modified biomolecular corona comprises a fused cell-specific binding domain. In some embodiments, the modified biomolecular corona protein has been additionally modified such that it does not bind substantially to its natural receptor. In some embodiments, the fused cell-specific binding domain binds to a target cell.
Resumen de: US2025144228A1
Provided herein are conjugate molecules comprising an antibody or antibody fragment capable of binding polyclonal immnunoglobulins within a subject. Such conjugates are useful for recruiting immune cells of the subject to one or more cell types that are targeted by the conjugate. Also provided herein are compositions comprising the conjugates, including pharmaceutical compositions that may be administered to a subject, for such a purpose as to treat or prevent a disease.
Resumen de: US2025144204A1
An adjuvant composition, comprising a complex comprising an adjuvant and microparticles of a biodegradable polymer and/or cyclodextrin, wherein the adjuvant is incorporated into the microparticles of the biodegradable polymer and/or encapsulated in the cyclodextrin.
Resumen de: US2025144200A1
Disclosed herein are intranasal immunogenic compositions comprising viral glycoprotein nanoparticles, which are suitable for use as vaccines. The nanoparticles present antigens from pathogens surrounded to and associated with a detergent core resulting in enhanced stability and good immunogenicity. Dosages, formulations, and methods for preparing the vaccines and nanoparticles are also disclosed. Methods for using the intranasal immunogenic compositions to stimulate an immune response in a subject against a virus are also disclosed.
Resumen de: US2025144236A1
A nitrogen-branched non-linear PEGylated lipid of Formula (1), wherein, X is —CRa< or(Ra is H or a C1-12 alkyl group); B1 and B2 are linking bonds or C1-20 alkylene groups; L1 and L2 are linking bonds or divalent linking groups; R1 and R2 are C1-50 aliphatic hydrocarbon groups or C1-50 residues of aliphatic hydrocarbon derivative, each containing 0-10 heteroatoms; Ld is a linking bond or a divalent linking group; Ncore is a multivalent group of valence y+1, and contains a trivalent nitrogen-atom branching core connected to Ld; y is 2, 3, 4, 5, 6, 7, 8, 9, or ≥10; Lx is a linking bond or a divalent linking group; XPEG is a polyethylene glycol component. The non-linear PEGylated lipid herein can realize better surface modification of LNP. The lipid nanoparticle pharmaceutical composition and its formulation exhibit higher drug efficacy, especially for nucleic acid drugs.
Resumen de: US2025144233A1
Nanoparticle compositions for delivery of nucleic acids to subjects including aminoalkyl branched lipid-like molecules as carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the nanoparticle containing are described. Also provided are methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects.
Resumen de: US2025144237A1
The disclosure relates to compositions and methods for improving pulmonary delivery of therapeutic nucleic acids, e.g., DNA, mRNA, by enhancing transfection efficiency and thus efficacy of the therapeutic nucleic acids, using inhaled, Intranasal, Intratracheal, bronchial instillation and/or topical administration Intratracheal, bronchial instillation and/or topical administration transfection efficiency enhancing agents, including but not limited to 2-mercaptoethane sulfonate or cysteamine.
Resumen de: US2025144234A1
This disclosure provides improved RNA molecules, including mRNA molecules that can be produced by in vitro transcription and are suitable for in vivo transfection using an appropriate delivery vehicle, such as a lipid nanoparticle (LNP) or targeted lipid nanoparticle (tLNP). The improved RNA include particular combinations of 5′ untranslated region (UTR) and 3′ UTR, particular 3′ UTRs, or particular open reading frame sequences. Also provided herein are compositions of the LNP, or tLNP with an antibody as a targeting moiety, such as anti-CD8 antibodies that are used as targeting moiety.
Resumen de: US2025144028A1
The present disclosure relates generally to lipids, lipid nanoparticle formulations, and methods of using the same for delivering nucleic acids, such as mRNA.
Resumen de: US2025144216A1
The present invention is directed to second harmonic generation particle comprising a photosensitizer spaced by a non-ionic water-soluble spacer, method of manufacturing thereof and use thereof such as for photodynamic therapy.
Resumen de: US2025144038A1
Nanoparticles and formulations for preventing or reducing peri-/post-menopausal bone loss and/or obesity in a subject are disclosed. The nanoparticles contain a cage, such as a zeolitic imidazolate framework (“ZIF”), a surface modifying agent, a targeting ligand, and an active agent. The surface modifying agent is attached to the outer surface of the cage and the targeting ligand is exposed to the surrounding environment. The active agent is encapsulated in the cage. The targeting ligand binds to a reproductive hormone or a receptor of a reproductive hormone. The active agent can be a ribosome inactivating protein, an apoptosis inducer, a hormone, a receptor ligand, or a nucleic acid, or a combination thereof, that inactivate ribosomes of gonadotroph cells and/or prevent or reduce secretion of follicle-stimulating hormones in the subject. Uses for formulations incorporating the nanoparticles for preventing or reducing peri-/post-menopausal bone loss and/or obesity in a subject are also disclosed.
Resumen de: US2025144036A1
The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel cationic lipid as well as additional lipids such as ionizable lipids, phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
Resumen de: US2025144037A1
Provided are a mRNA vaccine composition with improved storage stability and a method of improving storage stability of the vaccine composition.
Resumen de: US2025144035A1
Provided herein are devices and dosage forms useful in delivering macromolecular active ingredients or drugs, such as proteins, peptides and nucleic acids, through epithelial membranes, such as intestinal epithelium. Also provided are trans-epithelial drug delivery methods and methods of treatment of diabetes or insulin resistance, or to induce weight loss.
Resumen de: US2025145975A1
Methods which make use of baculovirus vector (BV)-magnetic nanoparticle (BV-MNP) complexes to facilitate in vivo delivery of clustered regularly interspaced palindromic repeat (CRISPR) systems are provided. BV-MNP complexes carrying a CRISPR nuclease and a guide RNA having homology to an immune checkpoint gene can be administered to a subject to inhibit the immune checkpoint gene. Inhibition of the immune checkpoint gene can promote a desired immune response in the subject. Methods which leverage the contrast provided by MNPs in BV-MNP complexes in combination with magnetic resonance imaging (MRI) to detect in vivo delivery of CRISPR systems are also provided.
Resumen de: WO2024006410A1
This document relates to methods and materials for treating vascular stenosis. For example, nanoparticles (e.g., poly lactic-co-glycolic acid (PLGA) nanoparticles) including one or more inhibitors of a monocyte chemoattractant protein (MCP) polypeptide (e.g., bindarit) are provided. In some cases, a composition (e.g., a hydrogel composition) including one or more nanoparticles including one or more inhibitors of a MCP polypeptide (e.g., bindarit) can be placed in direct contact with an adventitia of one or more blood vessels (e.g., one or more blood vessels at risk of stenosis formation) within a mammal (e.g., a mammal such as a human that underwent an angioplasty) to reduce or eliminate stenosis formation in the blood vessel(s).
Resumen de: US2024042070A1
Described herein are compositions and methods for treating cancer using a RIG-I agonist precursor comprising single-stranded 5′ uncapped triphosphate or biphosphate antisense oligonucleotide having a sequence complementary to an endogenous miRNA; optionally wherein the compositions and methods comprise a nanoparticle for targeted delivery of the RIG-I agonist precursor and a radiolabel to a tumor micro environment.
Resumen de: EP4548757A1
The present invention relates to a method for producing a nonhuman primate animal model of cerebral infarction, comprising administering endothelin to basal ganglia and thalamic region of a nonhuman primate, and thereby inducing basal ganglia damage, thalamus damage, and internal capsule damage; and a pharmaceutical composition for the treatment of cerebral infarction at a subacute to chronic stage, penetrating branch infarction, or cerebral infarction having brain damage in a penetrating branch territory, comprising a NeuroD1 protein or a polynucleotide encoding the NeuroD1 protein.
Resumen de: EP4549464A1
Provided are a new nanobody targeting human HER2/ErbB2 (HER2-Nanobody, HER2-Nb), a method for preparing same, and use thereof. The monoclonal nanobody efficiently and specifically binds to a purified HER2 protein and HER2 on the surface of a tumor cell, blocks a signal pathway of tumor HER2, and can be used for preparing a therapeutic antibody, an antibody-fusion protein or an antibody-drug conjugate targeting HER2-positive tumors.
Resumen de: WO2024006960A1
The present disclosure relates to compositions comprising lipid nanoparticles for delivering nucleic acid molecules into cells. Also included are methods for producing and using such compositions.
Resumen de: EP4548973A2
A pharmaceutical composition including insulin, Docosahexaenoic acid (DHA) and coenzyme Q10 and methods of manufacturing and using the composition are provided.
Resumen de: WO2024003363A1
The present invention relates to a method and compositions for optimized cytosolic delivery of active agents, in particular nucleic acids, using a specific class of cationic amphiphilic compounds. The method and compositions of the invention enhance intracellular release of the agents and can be used for the treatment of various disorders.
Resumen de: AU2023300349A1
Provided is a lipid formulation capable of forming a lipid-based nanoparticle comprising an ionizable lipid to phospholipid molar ratio of 0.1 - 1.30 of in association with a nucleic acid payload, and in some embodiments, a stabilizing agent. In embodiments, the nucleic acid payload is a vaccine genetic element.
Resumen de: MX2024000185A
There are provided improved amphiphilic comb polymers, comprising a hydrophilic backbone with regularly-spaced pendant hydrophobic moieties, having well-controlled molecular weights, structures, and end groups. The polymers self-assemble into core-corona nanoparticles in aqueous environments, which are capable of disrupting viral coat proteins, and which are capable of encapsulating antiviral drugs and prodrugs. Regularly-spaced targeting moieties optionally mediate the adherence of the nanoparticles to the viral coat. The compositions of the invention are useful as treatments for viral infection, including infections with SARS-CoV-2.
Resumen de: US2025099394A1
The present invention provides a lipid nanoparticle (LNP) formulation comprising at least one sialic acid (SA)-containing lipid. The LNP formulation is capable of effectively binding cell surface Siglecs, transfecting certain cells in vitro and targeting certain cells in vivo. The present invention also provides methods of using the LNP compositions described herein for pharmaceutical applications. For example, the LNPs provided herein are useful for the intracellular delivery of a nucleic acid therapeutic to a subject.
Resumen de: CN119925300A
本发明属于抗膀胱癌药物技术领域，公开了一种IL 12mRNA脂质纳米粒，包括IL 12mRNA和用于递送IL 12mRNA的载体，所述的载体包括一种或多种氨基酸生育酚氧烷酯或其药学上可接受的盐。以及公开了该脂质纳米粒的制备方法和在制备抗膀胱癌药物上的应用。该脂质纳米粒以氨基酸生育酚氧烷酯作为递送IL 12mRNA的可离子化脂质分子载体，有效的将IL 12mRNA递送到肿瘤细胞内，避免了mRNA被酶降解的风险，同时，在小鼠膀胱癌治疗组中发挥抗肿瘤作用，抑制了癌细胞的增长，并且具有较好的生物安全性。
Resumen de: CN119925299A
本发明涉及医用材料技术领域，尤其涉及一种抗肿瘤疫苗及其应用。所述抗肿瘤疫苗包括抗肿瘤纳米颗粒；所述抗肿瘤纳米颗粒包括：用于编码质膜膜泡关联蛋白的核酸、阳离子脂质和非阳离子脂质。本发明经过研究发现，基于肿瘤血管内皮细胞上特异性表达的抗原分子（质膜膜泡关联蛋白）制备的抗肿瘤疫苗可以高效激活肿瘤患者的免疫抑制微环境，诱导生成大量杀伤性T细胞，其能够靶向识别并杀伤具有PLVAP蛋白表达的肿瘤血管内皮细胞，高效破坏肿瘤血管，在抗肿瘤生物材料领域具有重要价值。
Resumen de: CN119930762A
本公开涉及非洲猪瘟病毒EP153R蛋白的免疫原性片段、重组蛋白、免疫原性组合物及其用途。本公开提供的非洲猪瘟病毒EP153R蛋白的免疫原性片段或其具有免疫原性的变体能够大大提高表达量，同时保留了强免疫活性。
Resumen de: CN119925303A
本发明提供一种纳米材料及其制备方法，所述所述纳米材料是以白蛋白(BSA)为模板，触发BSA与多酚(TA)的氧化偶联，形成以铜硫化物为核心，白蛋白与多酚为外壳的有机无机杂化纳米材料(TA‑BSA@CuS)。并包裹过表达Gas6的鼻咽癌细胞膜，形成Gas6细胞膜/TA‑BSA@CuS纳米材料，该材料能够有效的抑制肿瘤的肺转移。
Resumen de: CN119925305A
本发明涉及药物制剂技术领域，具体涉及一种积雪草苷纳米结构脂质载体及其制备方法。该纳米结构脂质载体是由水相和油相混匀后均质而成，其中水相是含有乳化剂的溶液，该油相是含有积雪草苷、固体脂质和液体脂质的混合液，通过熔融乳化‑高压均质法制成。本发明制备的积雪草苷纳米结构脂质载体具有粒径小、包封率高和稳定性好的优势，且制备方法简单可控，重复性好，解决了积雪草苷不易溶于水和生物利用度低的难题，为积雪草苷提供了一种可供选择的递送系统。
Resumen de: CN119925644A
本发明涉及超声分子影像学技术领域，涉及一种仿生抗炎纳米超声造影剂及其制备方法和应用。所述仿生抗炎纳米超声造影剂以负载雷帕霉素的红细胞膜为壳膜材料，内部包裹有负载TAT肽、siRNA的微小介孔二氧化硅及全氟戊烷或全氟己烷。制得的仿生抗炎纳米超声造影剂生物安全性高，能够在体内高效转运siRNA和雷帕霉素，实现无创靶向抗炎治疗。
Resumen de: CN119925302A
本发明公开了一种基于褐藻多酚纳米营养载体的制备及应用研究，属于纳米材料的制备技术领域。具体包括：将精氨酸溶于水中，接着加入褐藻多酚，得到混合液；将PEG溶于PBS中，得到PEG溶液；将PEG溶液加入到混合液中进行搅拌，并加入过氧化氢反应，加入乙酸继续反应，经过流水透析、真空干燥得到褐藻多酚纳米粒子。本发明实现了褐藻多酚这类天然多酚在溶解度、稳定性方面的限制，为天然多酚的实际应用提供了有力的技术支撑；所合成的纳米粒子在以脂多糖诱导的巨噬细胞炎症反应中表现出优异的抗炎特性，在DSS诱导的小鼠溃疡性结肠炎展示出优异的抗氧化与抗炎能力，为有效预防溃疡性结肠炎提供了一定的理论基础。
Resumen de: CN119925584A
本发明涉及动物疫苗制备技术领域，公开了一种鸡支原体病灭活疫苗的制备方法，包括以下步骤：支原体菌株的培养：将鸡支原体菌株接种至SP4培养基中，培养温度为35～39℃，培养时间为48～72小时，收集支原体菌体；灭活与抗原保护：在支原体悬液中加入灭活剂和抗原保护剂进行灭活与抗原保护反应，反应温度为25～37℃，反应时间为3～6小时；纳米递送系统的制备：通过壳聚糖与海藻酸钠的复合结构制备纳米颗粒递送系统。通过动态灭活与抗原保护协同技术方案，通过在灭活过程中同步引入聚赖氨酸、谷氨酰胺和精氨酸的抗原保护体系，显著避免了灭活剂对支原体抗原表位的破坏。
Resumen de: CN119924515A
本发明涉及纳米载体技术领域，公开了负载姜黄素的核壳型纳米颗粒及其构建方法，包括以下步骤：将玉米醇溶蛋白溶于乙醇溶液中，并添加还原性糖进行反应，然后进行压差震荡解聚处理，得到玉米醇溶蛋白溶液；向玉米醇溶蛋白溶液中加入姜黄素，得到负载姜黄素的玉米醇溶蛋白溶液；然后加入CaCl2溶液和果胶溶液混合，滴入磷酸盐溶液，进行反应，形成沉淀；经水洗、离心、冷冻干燥，得到负载姜黄素的复合纳米颗粒。本发明采用上述的负载姜黄素的核壳型纳米颗粒及其构建方法，以玉米醇溶蛋白为核，磷酸钙为壳，并通过配位络合作用在外部包裹果胶，使纳米颗粒提高稳定性的同时，具备pH响应特性。
Resumen de: CN119925298A
本发明公开了一种线粒体功能障碍仿生纳米药物及其制备和应用，所述仿生纳米药物包含作为载体的RGD工程化外泌体，以及修饰在所述载体的表面的地喹氯铵，以及封装在所述载体内部的奥沙利铂。本发明仿生纳米药物(OXA@Exo‑RD)可在血液循环过程中保护线粒体障碍货物，通过顺序靶向增加货物的线粒体蓄积，货物1(DQA)诱导氧化应激，引发线粒体介导的细胞凋亡和线粒体功能障碍；货物2(OXA)破坏线粒体DNA，阻止DNA修复启动，从而克服化疗耐药。两种货物协同克服CRC耐药，并抑制转移。
Resumen de: CN119930716A
本公开提供了一类抗原提呈细胞靶向脂质以及包含其的组合物。具体的涉及包含所述脂质的纳米微粒用于递送核酸类药物，其能够提高核酸类药物的巨噬细胞等抗原提呈细胞的转染效率，降低其对其他非靶细胞的转染效率，对核酸类药物的发展和应用具有重要的意义。
Resumen de: CN119925301A
本发明涉及一种ROS响应型过硫化物生物大分子纳米前药、纳米制剂及其制备方法和应用，属于纳米制剂技术领域。本发明利用ROS响应小分子基团对氢过硫化物进行保护，克服现有的氢过硫化物不稳定的缺点；同时，利用生物大分子作为氢过硫化物的载体，即增加了氢过硫化物的肝靶向性、水溶性及生物安全性，又实现多位点修饰和载药量的可调控；此外本发明纳米制剂反应流程简单，条件温和，有利于临床转化。
Resumen de: CN119925588A
本发明涉及生物医药技术领域，公开了基于gE/pORF7双抗原纳米晶与智能相变佐剂的重组带状疱疹疫苗，包括由gE蛋白和pORF7蛋白通过化学键连接形成的双抗原纳米晶，其中gE蛋白构成内核，pORF7蛋白通过动态共价键偶联于所述内核表面；以及包裹于所述双抗原纳米晶外层的智能相变佐剂，所述佐剂由温敏性聚合物载体和包封于其中的免疫刺激分子组成，所述温敏性聚合物载体在25～40℃范围内发生相变。本发明疫苗可同步诱导高滴度中和抗体和长效T细胞免疫，显著提升对潜伏病毒库的清除能力，同时降低接种不良反应风险，适用于老年及免疫缺陷人群的带状疱疹预防。
Resumen de: CN119925563A
本发明涉及生物医药技术领域，本发明涉及本发明涉及一种基于干细胞靶向修复脑卒中脑细胞的小分子肽组合物。本发明设计了具有靶向归巢和分化调控功能的小分子肽，并将其合理组合，还构建了药物递送系统。该组合物能引导干细胞精准归巢到受损脑区，调控其分化为功能性脑细胞，且能突破血脑屏障。经测试，显著提高了干细胞治疗效果，增强了治疗的安全性和有效性。
Resumen de: CN119925348A
本发明提供一种鞣花酸纳米颗粒，无需添加大量辅料和复杂制备工艺，即能解决鞣花酸溶解度、透皮吸收性能、口服生物利用度极低的问题。将其用于制备具预防或治疗脱发功效的食品、保健食品、药品、化妆品、日化用品中，可使脱发模型小鼠的毛发生长改善，外用治疗效果最佳，小鼠毛发浓密油亮，毛发重量与长度相较于模型组都得到极显著改善；口服组也具较优的治疗效果；同时预灌胃后造模显示出相较于模型组的优良毛发生成情况，可知其同时具有预防作用。同时，鞣花酸纳米颗粒泡沫剂具有优异的分散性和渗透性，其轻盈的泡沫结构更易在皮内横向与纵向扩散。更进一步，鞣花酸纳米颗粒显示出向皮肤深层渗透的作用，最深可直达及靶向皮肤筋膜层。
Resumen de: CN119931037A
本发明属于生物医药领域，尤其涉及一种脂质‑亲水聚合物及其制备方法和应用。本发明提供的脂质‑亲水聚合物具有式（I）所示结构，式（I）中，R1为聚合度在20~50的亲水聚合物基团，R2为C2~C4烷基，R3为C2~C4烷基，R4为含有C10~C22单尾或C10~C22双尾的疏水性脂质基团，L1为酯键，L2为酰胺键或酯键。本发明通过分子结构设计，在亲水聚合物与脂质之间引入缩硫酮敏感化学键，获得了可断裂型脂质‑亲水聚合物。在此基础上，通过提升脂质‑亲水聚合物在脂质纳米颗粒中的摩尔百分占比，能够在增强脂质纳米颗粒稳定性的同时实现高效转染。#imgabs0#式（I）。
Resumen de: CN119925304A
本发明公开了一种抗炎的脂质Phoyunbene C纳米材料、制备方法及应用。该纳米材料主要由Phoyunbene C纳米点核、DSPE‑PEG2000、大豆卵磷脂和胆固醇构成；制备过程中，将Phoyunbene C溶于磷酸盐缓冲液中，加热、搅拌，制得水相；将DSPE‑PEG2000、大豆卵磷脂、胆固醇溶于乙醇中，加热、搅拌、超声分散，得到有机相；将有机相和水相溶液分别装载入注射器，通过双相微流道混合工艺，得到脂质Phoyunbene C纳米点溶液。本发明所提供的脂质Phoyunbene C纳米材料的制备方法简便，制备条件温和，实现了在体内外更强的抗炎功效，有效改善骨关节炎症状。
Resumen de: CN119925291A
本发明涉及细胞膜仿生递药技术领域，具体涉及靶向动脉粥样硬化病灶的细胞膜仿生递药系统的制备方法及其产品和应用，步骤为先培养巨噬细胞，提取和纯化巨噬细胞膜，并加入酶抑制复合物并储存；将储存的巨噬细胞膜利用带有纳米膜的微型挤压机反复挤出获得巨噬细胞膜微囊；利用溶剂挥发法制备基于PEG‑PLGA的载药纳米颗粒；最后将巨噬细胞膜微囊和载药纳米颗粒分别从微流控电穿孔芯片的两个入口注入，再施加两段式交变电场，处理后得到分散均匀的混合液，再将该混合液采用带有纳米膜的微型挤压机共挤出，即可。本发明制备的细胞膜仿生递药系统具有生物可降解、药物缓释、长效循环、结构稳定、主被动双重高效靶向的优势，能有效抑制动脉粥样斑块的发展。
Resumen de: WO2024028492A1
The present invention relates to methods of determining the efficiency of RNA encapsulation in lipid nanoparticles (LNPs). In some embodiments, methods according to the present invention comprise a step a) of contacting a sample comprising RNA encapsulated in LNPs with a first fluorophore and a second fluorophore, thereby forming fluorophore-RNA complexes, and a step b) of detecting the fluorescence signals of the complexed first and second fluorophore, wherein the first fluorophore permeates the LNPs and wherein the second fluorophore does not permeate the LNPs.
Resumen de: CN119931074A
本发明公开了一种纳米金属有机框架化合物及其制备方法和应用。所述纳米金属有机框架化合物包括从内向外依次设置的内核、包覆于内核表面的第一壳层，以及化学修饰并包覆于所述第一壳层表面的第二壳层，所述内核包括铜‑半胱胺纳米颗粒，所述第一壳层包括2‑甲基咪唑锌盐，所述第二壳层包括透明质酸。本发明提供的纳米金属有机框架化合物材料具有光敏特性及酶催化活性，并且可以有效靶向肿瘤细胞。并且整个制备过程简单、环保，利于规模化生产，为癌症的治疗提供了新的思路和方法。
Resumen de: AU2023347022A1
Disclosed is a lipid nanoparticle (LNP) encapsulating a nucleic acid cargo preferably comprising messenger ribonucleic acid (mRNA). The LNP comprises at least a cationic lipid fraction, and a stabilizer fraction. The stabilizer fraction preferably comprises at least one polyethylenglycol (PEG) lipid. Furthermore, the LNP comprises at least one glycerol dialkyl glycerol tetraether (GDGT) lipid, as obtained e.g. from archaea of the genus Sulfolobus, optionally among other ether lipids. Also disclosed is a pharmaceutical composition comprising the LNP, such as an mRNA vaccine.
Resumen de: CN119925306A
本发明公开了一种环黄芪醇‑花型乳糖在制备治疗急性病毒性心肌炎药物中的用途。本发明公开了黄芪有效成分环黄芪醇治疗病毒性心肌炎，以纳米载体运载环黄芪醇治疗病毒性心肌炎，除了能够通过调节免疫系统抑制机体免疫风暴外，还可能通过激发靶器官细胞内在防御机制活力发挥抗病毒作用，对实验性柯萨奇B3病毒(CVB3)小鼠心肌炎模型具有显著的改善作用。
Resumen de: AU2023317842A1
The present invention relates to compositions that can deliver therapeutic molecules such as nucleic acids to mammalian cells, and to the human and animal body and methods of preparing and using the same. The compositions comprise nanoparticles comprising a peptide dendrimer, a nucleic acid and a lipid. The compositions of the invention find utility in the field of medicine, such as for treating cancer and autoimmune diseases.
Resumen de: CN119212744A
Compositions for topical delivery of a drug to an intracranial region, such as brain tissue or brain tumor. Methods for local delivery of drugs or therapies to an intracranial region. The composition may include a hydrogel in which a chemotherapeutic drug or an immunotherapeutic drug is dispersed. Kits comprising a composition or solutions that can be combined to form a composition.
Resumen de: CN119909061A
本发明提供一种鞣花酸纳米颗粒，无需添加大量辅料与复杂制备工艺，即能解决其溶解度、透皮吸收性能、皮肤深层递送性能极低的问题。将其用于制备具美白、祛斑、抗氧化、抗衰老、清除自由基、祛皱、防晒、增强皮肤的弹性、减少色素沉积、保湿、抗菌、抗病毒、治疗脱发、治疗白发、皮肤去油脂、治疗黑色素瘤、疤痕修复、创面愈合功能中的一种或多种功效的药品、化妆品、日化用品中，显示出向皮肤深层渗透药物的作用，最深可直达及靶向皮肤筋膜层。深层递送性能的提升与溶解度显著增强相关，也与纳米化性质相关，显示鞣花酸纳米颗粒在外用制剂，尤其是皮内深层药物递送的外用制剂的开发前景，这种深层递送不依赖于微针等装置，是完全无创递送的方法。
Resumen de: MX2024013106A
The present disclosure provides dual functioning compounds, compositions, formulations, and methods for inducing or modulating an immune or inflammatory response and treating diseases or disorders (e.g., cancer, autoimmune diseases, inflammatory diseases, and infectious diseases) with the compounds or compositions thereof. In particular disclosed herein are dual functioning compounds comprising a stimulator of interferon (IFN) genes (STING) agonist moiety and a second active moiety selected from an indoleamine 2,3-dioxygenase (IDO) inhibitor and phosphatidylinositol 3â¿`kinase (PI3K) inhibitor, and compositions and formulations thereof.
Resumen de: AU2023260204A1
An instant nanoparticle composition and a preparation method therefor. The composition contains an active ingredient, albumin and a particle stabilizer, and optionally contains a lyoprotectant. The active ingredient has the following characteristics: insoluble or slightly soluble in water, and soluble or easily soluble in specific organic solvents, preferably paclitaxel or docetaxel.
Resumen de: CN119909081A
本发明属于纳米医学技术领域，具体涉及一种抑制卵巢癌的组合物、纳米颗粒、制备方法和应用，包括蟾毒它灵和尼拉帕利，所述蟾毒它灵和尼拉帕利的摩尔比为6.25‑12.5:5000‑10000，本发明能够提高卵巢癌的抑制效果。
Resumen de: CN119909039A
本发明提供一种装载NLRP3‑sgRNA核酸序列靶向巨噬细胞的纳米颗粒及防治葡萄膜炎的应用。本发明负载具有特异启动子的NLRP3‑sgRNA质粒，制备纳米颗粒PLGA‑sgNLRP3，然后将巨噬细胞膜包被于PLGA‑sgNLRP3纳米颗粒表面，制备得到巨噬细胞膜包被的纳米颗粒MM/PLGA‑sgNLRP3。本发明能将PLGA‑sgNLRP3和MM/PLGA‑sgNLRP3招募至视网膜病变部位，从而增加药物在病变部位的积蓄，增强基因干预作用，实现高效内毒素诱导的葡萄膜炎病变部位靶向功能，不仅能够减轻视网膜损伤、炎症反应，防止视网膜屏障受损，且具有良好的生物安全性，副作用小，疗效高。
Resumen de: AU2023342576A1
Generally, a polymer nanomaterial encapsulation system useful in the production of polymer encapsulated nanoparticles comprised of a hydrophobic nanoparticle encapsulated in the hydrophobic region of the polymer with the external hydrophilic region of the polymer ensuring water-solubility and affording a functional group which can be utilized for the production of nanoparticle conjugates. Specifically, particular embodiments include a polymer nanoparticle structure including one or more of: a quantum dot and/or a superparamagnetic iron oxide nanoparticle and/or an upconverting nanoparticle, encapsulated in polystyrene-b-polyethylene glycol amine for the production of antibody conjugates useful in the capture of cellular targets.
Resumen de: CN119909184A
本发明属于生物医药技术领域，具体涉及一种双穿膜肽修饰的含STING抑制剂特异靶向血管内皮的纳米粒子及其制备方法与应用。本发明将STING抑制剂包裹于脂质体中得到脂质体纳米药物，以脂质体作为药物载体能提高其水溶性及生物利用度；同时，利用细胞穿膜肽和靶向穿膜肽共同修饰脂质体纳米药物，两者协同提高穿膜性能，帮助药物穿透眼部屏障，靶向视网膜血管内皮细胞，增加药物的生物利用度。并且，所得纳米粒子是以液态的形式存在，能够直接将其作为滴眼液用于治疗视网膜病变的眼睛，这样便捷、无创的眼部疾病治疗方式，能够更好地造福视网膜疾病患者，减少患者视网膜血管新生，有利于疾病的治疗和预后。
Resumen de: CN119912537A
本发明公开了一种RSV抗原、核酸、表达元件、药物组合物及其应用。所述RSV抗原为在SEQ ID NO:1的基础上包含突变A102C和S362C。所述RSV抗原具有比现有技术RSV更强的免疫原性，可以取得较高的中和抗体滴度，具有更高的保护效力。
Resumen de: CN119909036A
一种基于不饱和脂肪酸的铁死亡调节纳米粒的制备方法，该脂质纳米粒由模型药、单不饱和脂肪酸、不同PEG分子量的磷脂聚乙二醇偶联物复合制成，方法是：将模型药、单不饱和脂肪酸与不同PEG分子量的磷脂聚乙二醇偶联物混合，然后溶解于乙醇中，超声混匀，滴入去离子水中，搅拌、旋蒸，除去甲醇和乙醇，离心，除上清液，干燥，即得；原料丰富，制备方法简单，易生产，产品质量好，性质稳定，能通过将多酚类药物有效递送至胰腺炎症部位，极大提高药物生物利用度以及胰腺炎症靶向能力，抑制胰岛β细胞的铁死亡来发挥治疗糖尿病的作用，是治疗糖尿病和胰腺炎药物上的一大创新，社会和经济效益巨大。
Resumen de: CN119909163A
本发明涉及生物医药技术领域，公开了一种基于糖链修饰与相变材料的长效稳定重组RSV疫苗，所述疫苗包括以下质量份数的组分：动态糖链修饰的RSV F蛋白：1份；多级相变材料：3～5份，所述多级相变材料由聚乙二醇‑硬脂酸酯和月桂酸‑胆固醇复合物组成，其中聚乙二醇‑硬脂酸酯与月桂酸‑胆固醇的质量比为2～3:1；TLR9激动剂：0.05～0.15份；STING激动剂：0.02～0.08份；两亲性嵌段共聚物PLGA‑PEG‑PLL：0.5～1.5份。本发明解决了传统疫苗易失活、靶向性差及免疫原性不足的问题，适用于病毒性感染疾病的预防与治疗，具备规模化生产的应用潜力。
Resumen de: CN119909021A
本发明公开了一种共负载黑色素茶多酚‑镁的Janus微球及其制备方法与应用，利用茶多酚和镁离子在碱性条件下络合形成纳米颗粒以及利用强碱溶解头发中的角蛋白获得黑色素纳米颗粒，将二者分别与甲基丙烯酰胶原混合配制成溶液作为流动相，以植物油作为分散相溶液，在微流控装置中，双通道流动相同轴共流，分散相流体利用流动剪切力将流动相分散为单分散液滴，液滴在紫外激光照射下固化形成共负载黑色素/茶多酚‑镁的Janus微球。本发明借助微流控技术，实现共负载黑色素/茶多酚‑镁的Janus微球的高均一性制备，可以实现协同抗氧化和抗菌治疗，可应用于各种具有抗氧化和抗菌需求的生物医学应用，如促进糖尿病创面、细菌感染性创面等的愈合。
Resumen de: CN119909035A
本发明公开了一种用于黑色素瘤靶向成像和光热治疗的自组装纳米颗粒，旨在解决现有光热治疗黑色素瘤方法中靶向性不强、光热效率低以及生物相容性差的问题。本发明设计了一种基于特定多肽(SA‑7)、芳香氨基酸(FF)和卟啉(TPP)的自组装纳米颗粒。通过自组装形成的纳米颗粒能够高效靶向黑色素瘤细胞，在638nm激光照射下产生强效的光热效应。同时，该纳米颗粒具有良好的生物相容性和代谢性能，可用于黑色素瘤的精准成像和治疗。
Resumen de: CN119909206A
本发明公开一种用于ECT成像和放射增敏治疗的诊疗一体双模态纳米颗粒及其制备方法和用途，属于肿瘤诊疗药物技术领域。所述纳米颗粒由小尺寸铪‑没食子酸纳米颗粒标记可检测标记物得到；所述小尺寸铪‑没食子酸纳米颗粒由聚乙烯吡咯烷酮、四氯化铪与没食子酸通过配位组装形成。本发明提供的双模态纳米颗粒可同时用于ECT成像和放射增敏治疗，具有良好的生物安全性和优异的放疗增敏性能，在体内具有很好的代谢特性，克服常规纳米颗粒会在体内滞留的缺陷。
Resumen de: CN119909037A
本发明公开了一种负载胰岛素的复合纳米颗粒及其制备方法和应用，复合纳米颗粒具有核壳结构，核为L‑缬氨酸修饰的壳聚糖与胰岛素自组装形成的聚电解质络合物，壳为阴离子多糖；或者复合纳米颗粒具有多层核壳结构，核为苯硼酸修饰的明胶与胰岛素自组装形成的聚电解质络合物，外面形成两层壳：L‑缬氨酸修饰的壳聚糖、褐藻糖胶。本发明可作糖尿病治疗的口服给药和注射给药。
Resumen de: US2022143136A1
The present disclosure provides compositions and methods for increasing the activity of an annexin protein to treat a cellular membrane injury in a patient in need thereof.
Resumen de: CN119912352A
本发明提供了一种氟代脂质体及其制备方法和应用。这一系列结构新颖的高度氟化的可离子化的脂质分子制备方法具有合成简单、产率高的优点。本发明还提供了包含氟代可离子化脂质或复合离子化脂质的LNP，所述LNP粒径分散性好，对于核酸的包封率高，生物体内毒性较低，可以高效的递送核酸药物使其具有更好的进细胞效率、核酸的表达效率，并具有脾脏靶向性，在免疫治疗中具有重大的应用前景。
Resumen de: CN119912351A
本发明涉及一种脂质化合物及其组合物。具体而言，本发明涉及式(I)的脂质化合物，包含其的脂质纳米颗粒，及其制备方法和在药物递送中的用途。#imgabs0#
Resumen de: JP2024094323A
To provide innovative drugs, medical products and imaging agents.SOLUTION: Provided is a method of imaging a subject, comprising: (i) administering to the subject an imaging agent having a plurality of nanostructures, each of the nanostructures comprising a multi-armed PEG and one or more additives that interact with the multi-armed PEG, and the multi-armed PEG of each of the plurality of nanostructures is sufficient to provide a nuclear magnetic resonance image when scanned in step (ii); (ii) following step (i), performing an imaging scan of the subject, the imaging scan being a nuclear magnetic resonance scan; and (iii) providing a nuclear magnetic resonance image of the multi-armed PEG of the plurality of nanostructures.
Resumen de: CN119909038A
本发明属于药品技术领域，具体涉及一种蜂皇浆冻干粉的包衣方法及其产品、应用、养肝胶囊及其制备方法。所述蜂皇浆冻干粉的包衣方法，包括以下步骤：S1、将包衣材料溶解于溶剂中，得包衣液：S2、采用包衣液对蜂皇浆冻干粉进行包衣，得包衣蜂皇浆冻干粉；所述包衣材料包括羟丙甲纤维素、改性麦芽糊精和异麦芽酮糖醇。采用本发明提供的包衣蜂皇浆冻干粉制备得到的养肝胶囊在阴凉、常温和加速条件下的水分含量均合格，而且避免了蜂皇浆冻干粉含量的严重下降。
Resumen de: CN119912435A
本发明公开了一种用于肺部疾病治疗的纳米颗粒及其制备方法与应用。本发明所提供的用于肺部疾病治疗的纳米颗粒包括纳米颗粒载体和核酸分子；所述纳米颗粒载体，其原料包括所述TM2和DOPE，两者的摩尔比为1:2。本发明提供的包载核酸的纳米颗粒能够高效地将核酸分子递送至肺部，下调致病蛋白表达；能够显著增加药物在肺部的沉积，有效保证了给药以后的效果，同时具有给药方式简便，无创伤，安全性高等优点，是一种具有高创新性的高效肺部沉积的一种方式，在肺部相关疾病治疗方面具有很好的应用前景。
Resumen de: US2022125899A1
The present application is related to a method of treating a cancer by administering to a human subject multiple doses of a mRNA cancer vaccine formulated as a lipid nanoparticle wherein the cancer vaccine comprises one or more mRNAs each having one or more open reading frames encoding 3-50 peptide epitopes, and wherein each of the peptide epitopes are portions of personalized cancer antigens or portions of cancer hotspot antigens. The present application further relates to a method of treating cancer by combining anti-cancer immunotherapy with the administration of the aforementioned mRNA cancer vaccine.
Resumen de: CN119424347A
The invention discloses an application of naringenin nanoparticles in preparation of a ceftiofur long-acting preparation. The invention discloses naringenin (5, 7-dihydroxy-2 (4-hydroxyphenyl) chroman-4-ketone), which is a flavonoid substance. The naringenin nanoparticles are prepared, the naringenin nanoparticles are mixed with the ceftiofur for combined use, and it is found that the naringenin can effectively intervene in the pharmacokinetic process of the ceftiofur and significantly prolong the in-vivo half-life period of the ceftiofur, so that the in-vivo action time of the ceftiofur is prolonged, and the curative effect of the ceftiofur is improved. Moreover, the application method is simple, easy to operate, low in cost and suitable for large-scale popularization.
Resumen de: CN119909158A
本发明涉及蜂毒肽，特别涉及一种抗肿瘤的蜂毒肽组合物及其应用；本发明所述抗肿瘤的蜂毒肽组合物由蜂毒肽和TPGS‑CA通过自组装和静电作用所构成。蜂毒肽与TPGS‑CA形成纳米药物后，首先能够降低蜂毒肽的溶血性，减少其副作用；其次，TPGS‑CA具有较强的负电性，可以逃避网状内皮系统的吞噬延长体内循环时间；最后纳米药物通过EPR效应到达治疗部位后，TPGS‑CA在微酸性环境中能够水解成TPGS和CA，能够进一步加强蜂毒肽的抗肿瘤效果，从而达到高效抗肿瘤的目的。
Resumen de: AU2023260654A1
The present invention relates to nanoparticle bioconjugates comprising binding proteins for binding to a cancer cell and an antigen of an immune cell, and uses and methods of treatment comprising administration thereof.
Resumen de: WO2025091046A1
Provided are compositions that include compositions of galactosyl -conjugated lipid nanoparticles (LNPs) encapsulating one or more active agents. In some embodiments, the galactosyl- conjugated LNPs have a lipid component having D-Lin~MC3-DMA, ALC-0315 and. SM-102, cholesterol, DSPC and DOPE, and DMG-2000-PEG. In some embodiments, the GalNAc-conjugated LNP has one or more galactosyl moieties bioconjugated to cholesterol present with a lipid component of the GalNAc -conjugated LNP. Also provided are methods for treating and/or preventing diseases, disorders, and/or conditions associated, with undesirable PCSK9 gene expression, optionally a cardiovascular disease, disorder, or condition, including but not limited, to atherosclerosis and/or thrombosis, and sepsis, septic shock, cytokine storm, or sequelae thereof.
Resumen de: AU2023347284A1
Provided here are novel engineered and isolated signal peptide sequences and compositions comprising these. Also provided are compositions and methods of using these signal peptides to enable secretion of heterologous polypeptides for therapeutic, diagnostic, and commercial value.
Resumen de: WO2025088346A1
Provided is lipid nanoparticle functionalised with a glycopolymer. The glycopolymer comprises at least four carbohydrate units, wherein each carbohydrate unit is independently selected from a monosaccharide, a disaccharide and an oligosaccharide. The lipid nanoparticle can be used for inducing an immune response in a subject.
Resumen de: WO2025088192A1
The present invention provides for inventive means and methods for the coupling of di(alkyl)amines to polypeptides or peptides capable of forming a random coil conformation. The present invention further provides inventive compounds produced by the herein detailed methods, wherein said compounds are characterized by formula (I): A-B-bC-D. Said compounds comprise a polypeptide/peptide capable of forming a random coil conformation, an N-terminal protecting group, a di(alkyl)amino group, and optionally a linker. Furthermore, the present invention provides lipid nanoparticles comprising said compounds characterized by formula (I) as well as means and methods for the production of said lipid nanoparticles. Formulations comprising the inventive compounds and/or the lipid nanoparticles of the present invention are also provided. Further, the present invention also relates to uses of the inventive compounds, the lipid nanoparticles, and/or the formulations disclosed herein.
Resumen de: WO2025090023A1
There is provided a compound comprising a structure represented by general formula (1) or an ionized form thereof for preparing lipid nanoparticles encapsulating a therapeutic, prophylactic and/or biological agent: wherein R5 and each R6 are independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; R3, and each R7 are independently optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; n is from 0 to 100; l and m are each independently 0 or 1; each A is independently selected from H, aliphatic alcohol, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, an epoxy ring- opening product and/or derivatives thereof; and B is R1R2N- or R23O- where R1 and R2 are each independently H, or a hydrophobic tail with the proviso that both of R1 and R2 are not H at the same 116 time; and where R23 is optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl.
Resumen de: WO2025090968A1
Bead-in-particles formats and their use in cytometric applications are described. In one aspect, the disclosure provides a bead-in-particle comprising an outer hydrogel body and an inner hydrogel bead. As described herein, the bead-in-particle is selectively tuned to have at least one morphological property substantially similar to subcellular macrostructures of interest present in a target cell. In one aspect of the disclosure, the bead-in-particle are used as a calibration reagent for the detection of a target cell in a sample, such as by image-based flow cytometry.
Resumen de: WO2025090417A1
The present disclosure provides bispecific stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., hematopoietic stem cells, to modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Resumen de: WO2025090663A1
The present disclosure provides novel polymer-conjugated lipids conjugated to a polyglycerol or a polyglycerol derivative. The present disclosure also provides lipid nanoparticles (LNPs) formulation using the polymer-conjugated lipids and methods of treating a disease by administering the LNP formulations, including multiple doses of the LNP formulations.
Resumen de: WO2025090756A2
Embodiments of the invention include methods of treating conditions that are ameliorated by stimulating an immune response. In aspects, the methods include subcutaneous injection of mesoporous silica rods into a subject or patient. The mesoporous silica rods can include a cytokine (e.g., IL-2 or IL-12) and/or an adjuvant. The mesoporous silica structures can induce an innate systemic immune response to treat cancer, infection and other ailments. Embodiments also include methods of producing mesoporous silica rods.
Resumen de: WO2025090612A1
The present technology provides a compound of Formula I, wherein PAO, R and n are defined herein. Also provided are lipid nanoparticles incorporating compounds of Formula I and methods of using same to deliver nucleotide, polynucleic acid or RNP to a cell.
Resumen de: WO2025090525A1
The disclosure provides conjugates comprising a targeting moiety a lipid nanoparticle (LNP) encapsulating a therapeutic agent (e.g., payload) for delivery to immune cells. The conjugates can be delivered to immune cells ex vivo or formulated in a pharmaceutical composition and can be directly administered to a subject in need of (i.e., via in vivo administration).
Resumen de: WO2025090891A1
In some embodiments, the invention relates to a composition for targeted delivery of intact mitochondrial DNA (mtDNA) to a target cell, the composition comprising a mtDNA molecule that is complexed to cell-penetrating peptides (CPPs) which promote cellular entry, wherein the complex forms a nanoparticle with the CPPs bound to the mtDNA molecule. In some embodiments, the invention relates to a method of treating a disease or disorder associated with mitochondrial dysfunction in a subject in need thereof.
Resumen de: WO2025090766A1
This invention relates to the utilization of superpararmagnetic polycrystalline iron oxide nanoparticles for magnetic hperthermia applications and methods. The present invention provides a magnetic hyperthermia method for treating a condition in a subject, the method comprising: obtaining at least one superparamagnetic nanoparticle, wherein the at least one superparamagnetic nanoparticle comprises a plurality of iron oxide primary crystals, wherein the plurality of iron oxide primary crystals comprise a plurality of crystallites.
Resumen de: WO2025090634A1
Compositions comprising a mesoporous silica nanoparticle (MSN) loaded with metal cations and oligonucleotides and methods of use.
Resumen de: WO2025090565A1
Methods and compositions for rejuvenating and reprogramming stem cells are disclosed. The methods involve administering to a subject an ABCB5 targeted composition comprising an anti-ABCB5 antibody conjugated to a therapeutic payload comprised of an epigenetic reprogramming factor or a nucleic acid encoding an epigenetic reprogramming factor in an effective amount to reprogram and rejuvenate ABCB5+ stem cells in the subject. The compositions include anti-ABCB5 antibody conjugated to a therapeutic payload comprising an epigenetic reprogramming factor or a nucleic acid encoding an epigenetic reprogramming factor.
Resumen de: WO2025090422A1
The present disclosure provides bispecific stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., immune effector cells such as T cells, B cells, natural killer cells, and dendritic cells, to modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Resumen de: WO2025090138A1
The present disclosure provides stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., T cells, B cells, natural killer cells, hematopoietic stem cells, to genetically modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Resumen de: WO2025089792A1
The present invention relates to a composition for drug delivery and a preparation method therefor and, more specifically, to: a composition for drug delivery which is formed such that a drug is encapsulated inside a nanoparticle structure formed by a polymer and a cationic lipid having a specific structure; and a preparation method therefor.
Resumen de: WO2025089791A1
The present invention relates to a novel ionizable lipid compound represented by Formula (I) or a salt thereof, and a lipid nanoparticle containing the same. The lipid nanoparticle containing the novel ionizable lipid compound according to the present invention has an excellent nucleic acid encapsulation efficiency and has a high efficiency of cellular delivery of nucleic acid.
Resumen de: WO2025089790A1
The present invention relates to a novel ionizable lipid compound represented by formula (I), or a salt thereof, and lipid nanoparticles comprising same. The lipid nanoparticles comprising the novel ionizable lipid compound, according to the present invention, have excellent nucleic acid encapsulation efficiency and high nucleic acid cell delivery efficiency.
Resumen de: WO2025089475A1
The present invention relates to a nucleic acid-encapsulated stabilized nanoliposome carrier and a method for producing same.
Resumen de: WO2025088609A1
Disclosed are biocompatible, biodegradable, and low immunogenic alga(e) Nanoparticles (aNPs) that can adhere and deliver active ingredient(s) to mucosal epithelium tissue, as well as compositions comprising the aNPs, methods of preparing the aNPs, and uses thereof in methods of treatment.
Resumen de: WO2025088597A1
A method is provided for treating solid tumors in a human body, the method including intravenously administering nanoparticles (32) having a negative zeta potential (ZP) at neutral pH between -5 and -40 mV; and administering chemotherapy drug molecules (34) having a positive ZP at neutral pH or a ZP at neutral pH of between -10 mV and 0 mV. The nanoparticles (32) and the chemotherapy drug molecules (34) are separate from each other until in the human body. The nanoparticles (32) and the chemotherapy drug molecules (34) are administered in respective amounts that are therapeutically effective in combination. The nanoparticles (32) localize on cancer cells of the tumors. The chemotherapy drug molecules (34) are attracted to the nanoparticles (32) in the human body by the opposite charges of the chemotherapy drug molecules (34) and the nanoparticles (32), thereby facilitating localization of the chemotherapy drug molecules (34) on the cancer cells of the tumors. Other embodiments are also described.
Resumen de: WO2025088242A1
A first aspect of the present invention relates to a nanoparticle aggregate for the treatment of venous thrombosis characterised in that it comprises a combination of negatively charged nanoparticles comprising rtPA and positively charged nanoparticles comprising DNase per nanoparticle. A second aspect of the present invention relates to a method for producing the nanoparticle aggregate. A third aspect of the invention relates to a pharmaceutical composition comprising the nanoparticle aggregate according to the first aspect of the invention. Lastly, the present invention discloses the nanoparticle aggregate for use as a drug and for the treatment of thromboembolic diseases.
Resumen de: WO2025087303A1
A nanoparticle complex comprises a bifunctional binder capable of non-covalently binding to the lipid nanoparticle in the nanoparticle complex.
Resumen de: WO2025086482A1
Disclosed in the present invention is an ionizable lipid compound and a use thereof. The ionizable lipid compound has a structure as represented by formula (I). In the ionizable lipid compound of the present invention, a polyoxa-structure is introduced in combination with a hydroxyl group at the head, so that the lipid compound has good biocompatibility and excellent in vivo mRNA delivery efficiency. The structural design is novel, the proportion of each component of a matched LNP dosage form is appropriate, and animal experiments have demonstrated that the delivery effect reaches an international advanced level of commercialized ionizable lipids and the safety is good.
Resumen de: WO2025086003A1
A Janus particle, comprising a particle core, the particle core comprising at least a first surface functionalized with a functional group and a second surface functionalized with a metal particle.
Resumen de: WO2025085950A1
The present invention is directed to lipid-based nanoparticles (e.g. lipid nanoparticles), formulations containing lipid-based nanoparticles and methods of treating diseases or conditions with said lipid-based nanoparticles and formulations thereof. The present invention provides a lipid-based nanoparticle comprising (a) an active agent, and (b) a plurality of capture binding domains displayed on the outer surface of the nanoparticle, wherein each capture binding domain is linked to the lipid-based nanoparticle through a site-specific linkage such that each capture binding domain is displayed in substantially the same orientation and capable of capturing a targeting moiety in an orientation that allows the targeting moiety to interact with its target. The present invention also provides a lipid-based nanoparticle comprising (a) an active agent, and (b) a plurality of targeting molecules displayed on the outer surface of the nanoparticle, wherein each targeting molecule is linked to the lipid-based nanoparticle through a site-specific linkage such that each targeting molecule is displayed in substantially the same orientation and capable of binding to a target on a cell surface.
Resumen de: CN119055798A
Provided herein are methods comprising introducing a therapeutically effective amount of an adeno-associated virus (AAV) vector into the inner ear of a mammal, the AAV vector comprises a nucleotide sequence encoding the following polypeptides: (a) a polypeptide comprising an antibody heavy chain variable domain operably linked to a signal peptide and a polypeptide comprising an antibody light chain variable domain operably linked to a signal peptide; (b) a polypeptide comprising an antigen binding antibody fragment operably linked to a signal peptide; or (c) a soluble vascular endothelial growth factor receptor operably linked to the signal peptide.
Resumen de: MX2024012513A
This application disclose a polynucleic acid molecule conjugate comprising an antibody or binding fragment thereof conjugated to a polynucleic acid molecule that hybridizes to a target sequence of an atrogene; wherein the polynucleic acid molecule comprises at least one 2' modified nucleotide, at least one modified intemucleotide linkage, or at least one inverted abasic moiety; and wherein the polynucleic acid molecule conjugate mediates RNA interference against the atrogene, thereby treating muscle atrophy or myotonic dystrophy in a subject. In a certain embodiment, atrogene comprises atrogin-1 gene (FBXO32), MuRF1 gene (TRIA63), FOXO1, FOXO3, or MSTN.
Resumen de: AU2023357782A1
Micelle compositions comprising a hedgehog pathway inhibitor and their use in treating skin diseases, conditions, or disorders, such as skin cancers, including basal cell carcinoma, are disclosed.
Resumen de: US2025135007A1
Disclosed herein are compositions comprising sterol-amino-phosphate compounds and methods of making and use thereof. Also disclosed herein are methods of making and methods of use of the compounds, compositions, and/or lipid particles disclosed herein. For example, the compounds and compositions are useful for treating a disease or disorder in humans and in animals. In some examples, the subject is a human male and the disease or disorder comprises male infertility.
Resumen de: US2025134925A1
According to an example aspect of the present invention, there is provided isolated bioactive mitochondria coated with a layer of Metal Organic Framework (MOF) and a method for intracellular delivery and release of said coated bioactive mitochondria in cells. The invention also provides a method for maintaining bioactivity and for increasing storage time of bioactive mitochondria by using MOF encapsulation.
Resumen de: AU2023357320A1
The present disclosure provides RNA technologies for targeting Claudin-18.2 polypeptides. In some embodiments, such RNA technologies can be useful for treatment of diseases associated with positive expression of Claudin-18.2. For example, in some embodiments, such RNA technologies can be useful for treatment of Claudin-18.2 positive cancer, including, e.g., but not limited to biliary cancers, ovarian cancers, gastric cancers, gastro-esophageal cancers, pancreatic cancers. In some embodiments, such RNA technologies can be used in combination therapy (e.g., in combination with a chemotherapeutic agent). The present disclosure further provides RNA backbones containing specific sequences upstream and/or downstream from the coding sequence.
Resumen de: AU2023347022A1
Disclosed is a lipid nanoparticle (LNP) encapsulating a nucleic acid cargo preferably comprising messenger ribonucleic acid (mRNA). The LNP comprises at least a cationic lipid fraction, and a stabilizer fraction. The stabilizer fraction preferably comprises at least one polyethylenglycol (PEG) lipid. Furthermore, the LNP comprises at least one glycerol dialkyl glycerol tetraether (GDGT) lipid, as obtained e.g. from archaea of the genus Sulfolobus, optionally among other ether lipids. Also disclosed is a pharmaceutical composition comprising the LNP, such as an mRNA vaccine.
Resumen de: AU2023343598A1
Compounds, compositions, uses, and methods for reducing cell viability of a cancer cell, or for preventing or treating cancer, are provided herein. In certain examples, methods for reducing cell viability of a cancer cell and/or for preventing or treating cancer in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a
Resumen de: AU2023336224A1
Provided herein are lipid-based nanoparticle compositions, and methods of making and using thereof. The compositions include nanostructured lipid carriers (NLC), liposomes, lipid nanoparticles (LNPs), solid lipid nanoparticles (SLNs), oil-in-water emulsions, cationic lipid-nucleic acid complexes, cationic nanoemulsions (CNE), charge-altering releasable transporters (CARTs), or polymeric nanoparticles, and further comprise a saponin adjuvant, and optionally a sterol and/or a bioactive agent. The bioactive agent can be self-amplifying RNA. The compositions are capable of delivery of a biomolecule to a cell for the generation of an immune response, for example, for vaccine, therapeutic, allergy desensitization, or diagnostic uses. Compositions and methods related to making the compositions and using the compositions for stimulating an immune response are also provided.
Resumen de: TW202502301A
The present disclosure relates to a copolymer and a polymersome for targeted delivery of biomolecules to a living organism. The exemplary copolymer comprises an initiator block, a propagator block, and a linkage connecting the initiator block and the propagator block. The initiator block comprises a glycan head configured to provide a targeted delivery, and the propagator block comprises a functional moiety configured to provide desired properties for the polymersome.
Resumen de: US2025136985A1
Disclosed herein is a pharmaceutically effective composition comprising at least one of a phosphorothioate (PS)-based anti-miR-141-3p oligonucleotide; a peptide nucleic acid (PNA)-based anti-miR-141-3p oligonucleotide; and/or a gamma-peptide nucleic acid (γ-PNA)-based anti-miR-141-3p oligonucleotide; where at least one of the foregoing oligonucleotides is encapsulated in a biocompatible nanoparticle.
Resumen de: US2025135050A1
Iodide nanoparticles are provided. The nanoparticles can be potassium iodide (KI) nanoparticles, and can be formed of, a radioisotope, preferably 131I or 125I. The nanoparticles can include a coating that can increase the half-life of the particles, serve as a platform for attachment or entrapment of targeting moieties and/or additional active agents, or a combination thereof. Methods of use are also provided. For example, a method of treating a subject for cancer can including sensitizing the subject to radiation therapy by administrating the iodide nanoparticles to the subject in combination with one or more doses of radiation therapy. Methods of treating or imaging a subject can also include administering the subject an effective amount iodide nanoparticles, without the nanoparticles serving as radiosensitizer. In such methods, the nanoparticles typically include a radioisotope or an anticancer active agent, for example 131I, 125I, 124I, or 123I, or a chemotherapeutic agent.
Resumen de: US2025134989A1
The present invention relates to a novel vaccine composition for coronavirus infection-19 comprising a polymer-based mRNA carrier. Specifically, the present invention provides a vaccine composition comprising an mRNA carrier using a novel polymer capable of delivering a negatively charged genetic material such as mRNA, and the vaccine composition has excellent effects of enhanced in vivo immune activity such as high antibody formation ability and increased interferon-gamma production.
Resumen de: US2025134986A1
Provided herein are, inter alia, compositions or formulations comprising a nucleic acid (e.g. a RNA molecule) encapsulated in a lipid nanoparticle (LNP) and their uses.
Resumen de: US2025134994A1
The present disclosure relates to lipid-based delivery vehicles for mRNA vaccines, which include a lysophosphatidylcholine (LPC) compound for enhancing vaccine immunogenicity. The present disclosure also relates to methods for use of the mRNA vaccines.
Resumen de: US2025134969A1
A biosynthetic hemostat includes a plurality of biocompatible flexible nanoparticles wherein each nanoparticle includes a shell that defines an outer surface of the nanoparticle and a core, which is loaded with thrombin, and a plurality of von Willebrand factor-binding peptides (VBPs) and collagen-binding peptides (CBPs) that are linked to the shell and extend from the outer surface.
Resumen de: AU2023337954A1
The present disclosure provides delivery vehicle compositions comprising hydroxyalkyl-capped cationic peptoids, such as 2-aminopropane-1,3-diol-capped cationic peptoids, and complexes of the delivery vehicles with polyanionic compounds, such as nucleic acids. The disclosure further provides methods of making and using the delivery vehicle compositions and complexes, such as for the delivery polyanionic compounds (e.g., nucleic acids) to cells. The disclosure also provides methods of eliciting an immune response with the delivery vehicle complexes of the disclosure.
Resumen de: AU2023347284A1
Provided here are novel engineered and isolated signal peptide sequences and compositions comprising these. Also provided are compositions and methods of using these signal peptides to enable secretion of heterologous polypeptides for therapeutic, diagnostic, and commercial value.
Resumen de: TW202428269A
It is provided an ionizable lipid of formula (I) or a pharmaceutically acceptable salt thereof, or a stereoisomer of any one of them; a lipid nanoparticle comprising the ionizable lipid, particularly, as an encapsulation agent, optionally comprising a pharmaceutically active agent; and a pharmaceutical composition comprising the lipid nanoparticle. It is also provided a lipid nanoparticle or a pharmaceutical composition comprising thereof for use in medicine, and the use of the lipid nanoparticles as an encapsulating agent.
Resumen de: AU2023333536A1
Compositions and methods for making and using engineered NK cells, T cells and B cells that express a chimeric antigen receptor.
Resumen de: US2025135028A1
The present disclosure provides copolymers and glycopeptide antibiotic-loaded polymeric nanoparticles (PNPs) comprising the copolymers as well as charge neutral polymers for targeted and sustained delivery of glycopeptide antibiotics to treat bacterial infections, including but not limited to biofilm bacterial infections. The copolymers are block, alternate, or random copolymers comprising x units of the formulaand y units of the formulawherein Z is an organic moiety, R and R′ are each independently selected from the group consisting of hydrogen and C1-C18 alkyl, each of x and y is an integer of 1 or greater, the sum of x and y is an integer from about 40 to about 714, and y is from about 10% to about 90% of the sum of x and y.
Resumen de: US2025135017A1
Linker-drug compounds and antibody-drug conjugates that bind to human oncology targets are disclosed. The linker-drug compounds and antibody-drug conjugates comprise a splicing modulator drug moiety. The disclosure further relates to methods and compositions for use in the treatment of neoplastic disorders by administering the antibody-drug conjugates provided herein.
Resumen de: US2025135027A1
Disclosed herein are cell-targeting complexes that are coated on the surface with target specific antibodies for induction of biological stimulus in target cells/tissue/organs. In some embodiments, the cell-targeting complex involves nonnucleated (e.g. platelets, red blood cells (RBC)) or enucleated cells that have been thiolated, streptavidinylated, and then coated with biotinylated antibodies. In some embodiments, the cell-targeting complex involves multilayer alginate hydrogel beads that have been coated with polyanionic proteins using a polycation, which is then thiolated, streptavidinylated, and then coated with biotinylated antibodies.
Resumen de: US2025134827A1
It relates to a beaded nonwoven membrane comprising polymeric nanofibers and at least one active agent, wherein the active agent has a water solubility equal to or lower than 33 mg/mL. It also relates to a process for the preparation of the beaded nonwoven membrane, and to its use in medical, and veterinary applications.
Resumen de: US2025134825A1
The present invention relates to a method for producing a preferably lipid-based carrier system as well as to a corresponding carrier system, in addition to a composition or corresponding medicament based thereon, as well as to corresponding uses of the composition or medicant.
Resumen de: US2025134823A1
The present invention relates to a method for modifying and cross-linking an anticoagulating hydrogel nanofilm for cell encapsulation. According to the present invention, the protection from the external environment and the control of material transfer can be improved due to the anticoagulant functionalization of the material surface. In addition, when applied into the body, it can prevent fibrosis, blood coagulation, protein adsorption, and platelet adhesion caused by immune responses, thereby preventing a decrease in cell viability caused by immune responses and improving cell survival rate.
Resumen de: US2025134824A1
Compositions and methods are provided for stabilization of RNA encapsulated by lipid nanoparticles during lyophilization. The compositions and methods involve the use of empty lipid nanoparticles to stabilize the lyophilized composition. These techniques may be used to prevent the need for cold chain storage and may also simplify the procedure at the clinic for reconstituting the vaccine to prepare an injectable composition.
Resumen de: US2025135072A1
Disclosed herein are compositions comprising a polymer and a metabolic inhibitor, as well as a method of using the composition to modulate an immune response. The composition may be produced in the form of a synthetic tissue for provision in a subject. The composition or synthetic tissue may further comprise additional therapeutic agents.
Resumen de: US2025137169A1
Provided herein are nanofibres including self-assembled cellular components derived from a homogenized plant tissue. Methods for preparing such nanofibres, as well as uses thereof in the treatment or prevention of diseases or disorders in a subject and/or as delivery vehicles are also described.
Resumen de: AU2025202623A1
The present invention provides a pharmaceutical composition comprising a peroxisome proliferator activated receptor (PPAR) modulator and an polymeric nanocarrier component, wherein the polymeric nanocarrier component is capable of solubilising the PPAR modulator in an aqueous medium and, wherein in the polymeric nanocarrier component is a micelle forming non-ionic surfactant. Uses of the same in therapy are also provided.
Resumen de: AU2023365462A1
It is an object of the present invention to provide a lipid composition capable of delivering a nucleic acid such as RNA to a hematopoietic stem/progenitor cell or mesenchymal stem cells, and a method of delivering a therapeutic agent to a cell using the lipid composition. The present invention provides a lipid composition comprising (A) a therapeutic agent and (B) a lipid nanoparticle conjugated to a targeting molecule, wherein the lipid nanoparticle comprises an ionizable lipid, and the targeting molecule specifically binds to a marker of hematopoietic stem / progenitor cells or mesenchymal stem cells.
Resumen de: US2025134822A1
A method to precisely tailor the surface topography of polymeric nanoparticles having bound thereto polyalkylene oxide, based on tuning the architecture of shape-persistent amphiphilic bottlebrush block copolymer (BBCP) building blocks, has been developed. It was demonstrated that nanoparticle formation and surface topography can be controlled by systematically changing structural parameters of BBCP architecture. The surface topography of PEGylated nanoparticles (nanoparticles having PEO or PEG covalently bound thereto) significantly affects their biological performance.
Resumen de: AU2023361218A1
The present invention relates to compounds of formula (I). The invention also extends to micro- or nanoparticles comprising a compound of formula (I). For instance, compounds of formula (I) can be used to produce stable lipid nanoparticles (LNPs). The LNPs have high encapsulation efficiency and can be used to deliver a therapeuticor prophylactic agent to a patient.
Resumen de: AU2023360051A1
The invention relates to oligonucleotides that inhibit Toll-Like Receptor 7 (TLR7) and/or Toll-Like Receptor 8 (TLR8), or potentiate TLR8, and uses thereof.
Resumen de: WO2025088570A1
A compound having the following structure of Formula (I) or a stereoisomer of the compound, tautomer of the compound, pharmaceutically acceptable salt thereof, wherein A1, A2, A3, R1a, R1b, R2a, R2b, m1, m2, m3, and X are as defined herein. Pharmaceutical composition comprising the compounds, and their use in methods of treating diseases are also described.
Resumen de: US2022257767A1
The technology described herein is directed to ionic liquids and methods of drug delivery.
Resumen de: AU2023338228A1
Compounds are provided having the following Structure (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R
Resumen de: EP4545561A1
The present invention relates to a novel nanobody (Nb) and a nanobody-drug conjugate (NDC) targeting CD73, a method for preparing same, and use thereof. The monoclonal nanobody and the corresponding NDC can efficiently bind to isolated CD73, various tumor cells and CD73 on the surface of an immune cell with high specificity and block the catalytic activity of CD73 enzymes, exhibiting high affinity, low immunogenicity, and a significant anti-tumor effect.
Resumen de: WO2017139498A1
The present invention provides compositions comprising particles with a negative zeta potential that encapsulate one or more epitopes associated with Japanese cedar pollen. Methods of inducing immunological tolerance to Japanese cedar pollen by administering said particles are also provided.
Resumen de: MX2024012616A
Provided herein are methods for enhancing gene therapy in an individual by administering an IRAK modulator (e.g., an IRAK-4 degrader) with the gene therapy to suppress innate immunity to the gene therapy. In some embodiments, the gene therapy uses an adeno- associated virus (AAV) vector, an adenovirus vector, a lentivirus vector, a Herpes simplex virus (HSV) vector or a lipid nanoparticle. Also provided herein are methods for selecting an individual for treatment with an IRAK modulator in combination with a gene therapy agent.
Resumen de: CN118843482A
The present disclosure relates to a plurality of nanostructures having an average hydrodynamic diameter between 20 nm and 50 nm, wherein each nanostructure is a polymeric nanostructure comprising a central portion, an anchor layer surrounding the central portion, and a coating surrounding the anchor layer. The disclosure also relates to the use of such a plurality of nanostructures as a medicament, in particular for cancer treatment and/or imaging, as well as the use of such nanostructures as carriers for radionuclides. The present disclosure also relates to methods and kits for radiolabeling such nanostructures with multivalent cationic radionuclides.
Resumen de: CN118843456A
The present disclosure relates to a plurality of spherical nanostructures. Each nanostructure comprises a central portion comprising a polymer backbone according to monomer residues of {(OR1) (OR2) PO} 2-(C) {(CH2) mSi (OR3) 3} {(CH2) mSi (OR3) 3} wherein each R1 and R2 are independently selected from the group consisting of a negative charge and H; each R3 is independently selected from the group consisting of a negative charge, H, and a covalent bond bound to a polymer backbone; wherein at least 3 R3 are bonds bound to the polymer backbone; and m is an integer between 1 and 5; and wherein the central portion of the nanostructure has a volume average hydrodynamic diameter of 10 nm to 90 nm; and an anchor layer surrounding the central portion, where the anchor layer includes a polymer according to monomer residues of (RO) 3Si-(CH2) n-Si (OR) 3, where each R is independently selected from the group consisting of negative charges, H, and covalent bonds, wherein at least two R are independently selected from the group consisting of a covalent bond bonded to a monomer residue of the central portion and a covalent bond bonded to a monomer residue of the anchor layer; wherein-represents a covalent bond; n is 1 or 2; and wherein the anchoring layer has a thickness of from 1 nm to 5 nm. The present disclosure also relates to methods of making such nanostructures as well as uses of the nanostructures, and to pharmaceutical compositions comprising such nanostructures.
Resumen de: MX2024012693A
The present disclosure relates generally to the field of molecular virology, and in particular relates to nucleic acid molecules encoding a modified alphavirus viral genome or self-replicating RNA construct (srRNA), recombinant cells and pharmaceutical compositions containing the same, as well as the use of such nucleic acid molecules, recombinant cells and compositions for the production of desired products in cell cultures or in a living body. Also provided are methods of eliciting an immune response in a subject in need thereof, as well as methods of preventing and/or treating rabies virus infection.
Resumen de: MX2024012617A
Provided herein are methods for enhancing gene therapy in an individual by administering an IRAK degrader with the gene therapy to suppress innate immunity to the gene therapy. In some embodiments, the gene therapy uses an adeno-associated virus (AAV) vector, an adenovirus vector, a lentivirus vector, a Herpes simplex virus (HSV) vector or a lipid nanoparticle. Also provided herein are methods for selecting an individual for treatment with an IRAK degrader in combination with a gene therapy agent.
Resumen de: WO2023247047A1
The present invention refers to a peptide, comprising or consisting of SEQ ID NO: 1 (CAYMTMKIRN), for use as a medicament, preferably in the prevention and/or treatment of cardiac damage arising after ischemia followed by reperfusion, or in the prevention and/or treatment of the inflammatory response following acute myocardial infarction. In a preferred embodiment, the peptide is conjugated with a nanoparticle.
Resumen de: AU2022463987A1
The present invention relates to combination therapies for treating cancer, optionally chemotherapy-resistant cancers, in a subject. The combination therapies comprise (a) an antibody or antigen-binding portion thereof that specifically binds to CD40, and (b) chemotherapy. The invention also relates to pharmaceutical compositions, kits and methods of using such therapies.
Resumen de: AU2023288487A1
The disclosure relates to ionizable lipids and compositions comprising the ionizable lipids. Lipid-nanoparticle compositions comprised of an ionizable lipid, optionally in combination with other lipid components such as helper lipids, stabilization lipids and structural lipids, and a therapeutic agent, such as a nucleic acid, for delivery to mammalian cells or organs are described.
Resumen de: AU2023288520A1
Circular RNA, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises group I intron fragments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, the expression sequence encodes an antigen. In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, inventive methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.
Resumen de: CN119899835A
本申请公开了一种高效表达GBA1的mRNA及其应用。本申请高效表达GBA1的mRNA由5’端至3’端依序包括5’甲基化帽子、5’UTR、目的基因序列、3’UTR和polyA尾巴；其中，目的基因序列为编码GBA1蛋白或GBA1突变蛋白的序列。本申请高效表达GBA1的mRNA，通过修改mRNA不同元件来获得更稳定、表达效率更高的GBA1mRNA。
Resumen de: CN119896651A
本发明属于生物技术技术领域，具体涉及一种基于磷酸钙介导的外泌体药物递送系统，并进一步公开其制备方法与应用。本发明所述基于磷酸钙介导的外泌体药物递送系统，通过Ca2+和磷酸盐构建形成磷酸钙介导的外泌体递送体系，通过氯化钙转染miRNA后可以将核酸药物载入到外泌体中，外泌体中miRNA的含量显著提高，进而借助于外泌体的递送性能将药物运输，有效提高了药物的载药效率和递送效率，具有装载效率高的优势。
Resumen de: CN119899206A
本发明公开了一种基于脐带间充质干细胞囊泡负载I型光敏前药的纳米囊泡及其制备方法与应用，其包括来源于人脐间充质干细胞的囊泡及其负载的所述I型光敏前药。本发明的优点包括：纳米囊泡EVs@NBS‑PB的I型光敏前药可与糖尿病慢性伤口微环境中高水平的H2O2响应释放I型光敏剂，再通过激光激发后产生PDT效应释放活性氧达到抗菌的目的，实现对糖尿病慢性创面的靶向抗感染作用，结合来源于人脐间充质干细胞的囊泡调节伤口微环境来协同治疗慢性创面；干细胞囊泡不仅能够提高光敏前药的生物相容性，同时还具有促细胞增殖、迁移及血管生成的作用；用水凝胶微针贴片装载给药的方式不仅具有高药物递送效率和微创性，同时也能提高患者的依从性。
Resumen de: CN119896731A
本发明属于光热材料制备技术领域，具体涉及一种液态金属纳米颗粒光热材料的制备方法，包括：步骤1、配制海藻酸钠水溶液；步骤2、在海藻酸钠水溶液中加入镓基液态金属，先通过搅拌混合，再通过超声处理充分混合；步骤3、干燥后得到生成物。本发明的制备过程是在常规环境下制备，对环境的要求低。另外，本发明制备的纳米颗粒具有稳定性好、光热性能佳、制备方法简单、形成速率快、易控制的特点，可制备具有高效光热能力的材料。综合以上有益效果，本发明在光热材料制备技术领域具有良好的应用前景。
Resumen de: CN119899218A
本文描述了作为光热转导剂的环金属化铁(II)络合物。所公开的络合物表现出高结构稳健性和在近红外(NIR)和可见光区域的显著吸收。所描述的络合物可以自组装以形成金属超分子粒子，其具有优异的光热性能并可以通过EPR效应靶向肿瘤。例如，本文公开的Fe NP具有强的近红外(NIR)吸光度，在近红外(例如，808nm)激光照射下具有至少30％(例如约60％)的高光热转换效率和/或优异的光热稳定性。Fe NP可以用包衣剂(例如牛血清白蛋白)包衣以形成包衣的Fe NP，这可以进一步增强金属超分子粒子在体内的肿瘤积累和生物相容性。Fe NP的优异光热性能使其能够解决与大多数现有光热材料相关的光热转换效率低的问题。
Resumen de: WO2025083657A1
The present invention relates to a novel lipid compound for tissue-specific delivery and a lipid nanoparticle (LNP) including same. The lipid nanoparticle includes, as a component, a lipid compound modified such that an active substance therein is selectively delivered into cells of a specific tissue such as lymph nodes, the spleen, the retina, cancer, the brain, the liver, and the like in vivo, thereby preventing side effects and safely exhibiting a desired level of effects. These tissue-specific, non-viral LNP carriers can be effectively utilized for the prevention of infectious diseases and the treatment of rare and intractable (hereditary) diseases (macular degeneration, diabetic retinopathy, hereditary retinal degeneration, cancer, brain diseases, liver diseases, and etc.), where targeted and selective delivery within the body is crucial.
Nº publicación: KR20250057862A 29/04/2025
Solicitante:
더트러스티스오브더유니버시티오브펜실베니아
Resumen de: AU2023334056A1
The present disclosure provides modified immune cells or precursors thereof comprising chimeric antigen receptors and surface-bound, biologically inert molecules. Also included are methods of preparing said modified immune cells and methods of treating CAR treatment-related toxicities in subjects in need thereof comprising said modified immune cells.
BOPI
Sede Electrónica
