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197
results.
Updated on
15/11/2026 [07:01:00]
Results 25 to 50 of 197
Absstract of: AU2025284498A1
The present invention relates to a lipid composition for lipid nanoparticles (LNP), which comprises: (a) one or more cationic and/or cationically ionizable lipids; (b) a sterol; (c) one or more helper lipids; and (d) a lipid obtainable by a method comprising (i) subjecting a mixture comprising one or more monomers of the following formula (I), (I) wherein R1 represents an ethyl group, R3 and R4, which may be the same or different, independently represent a hydrogen atom, or a C1-2 alkyl group, to cationic ring opening polymerization in the presence of a polymerization initiator for providing a linear polymer; and (ii) introducing a terminal group R2 of the following formula (II): (II) wherein k is an integer of 1 so that a spacer exists between the polyoxazoline chain and Z, whereby the structure of the spacer is determined by the repeating units y and z; y is 0 or an integer of 1 to 3, and z is 0 or an integer of 1 to 3; a which may be the same or different when more than one is present, represents an integer of 1 to 6; b represents 0 or an integer of 1 to 6; X1 which may be the same or different when more than one is present, independently represents an oxygen atom, a group -O-CO-, -N-CO-, or -NR' wherein R' is a hydrogen atom or an alkyl group; X2 which may be the same or different when more than one is present, represents a single bond or a group -CO-; X3 which may be the same or different when more than one is present, independently represents a single bond, an oxygen at
Absstract of: US20260355297A1
The present disclosure relates to methods of treating Type 1 diabetes (T1D) using tolerizing immune modifying nanoparticles encapsulating T1D associated antigens.
Absstract of: US20260355345A1
An antibacterial nanoparticle comprising an inner silica core; a gold (Au) nanosphere surrounding the inner silica core; a surface layer comprising silver (Ag) surrounding the gold nanosphere or the silver is alloyed into the gold nanosphere; and an antimicrobial peptide conjugated to the surface layer, wherein the antimicrobial peptide is selected from the group consisting of a cysteine terminated Syn71 peptide, a cysteine terminated MC1-2 peptide, and a cysteine terminated Syn20 peptide.
Absstract of: US20260355349A1
The invention relates to a lipid-based nanoparticle comprising an antigen-binding domain capable of specifically binding to a target expressed on activated immune cells surface and one or several mRNA molecule(s) encoding an activity-inhibiting protein of said activated immune cells, and to uses thereof.
Absstract of: US20260355123A1
A drug carrier nanoparticle has been synthesized that can specifically target the proximal tubules of the kidneys. The nanoparticles accumulate in the kidneys to a greater extent than other organs (e.g., up to 3 or more times greater in the kidney than any other organ). They can encapsulate many classes of drug molecules. The nanoparticles are biodegradable and release the drug as they degrade. The particles can sustainably release a drug within the kidneys for up to two months. The nanoparticles are useful for the treatment of diseases that affect the proximal tubules, such as heart failure, liver cirrhosis, hypertension, and renal failure; the study of relative blood flow to the renal cortex and medulla; and delivery of agents to treat gout.
Absstract of: AU2025376352A1
The present disclosure provides dual gRNAs for allele-selective or specific gene editing, nucleic acids encoding such gRNAs, vectors comprising such nucleic acids, RNPs comprising such gRNAs and Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective or specific gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition, containing. The present disclosure further provides lipid-based TCVs.
Absstract of: AU2025378053A1
The present disclosure provides gRNAs for allele-selective gene editing, nucleic acids encoding such a gRNA, vectors comprising such a nucleic acid, RNPs comprising such a gRNA and a Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition using such gRNAs. The present disclosure further provides lipid-based TCVs.
Absstract of: AU2025265441A1
The present disclosure relates generally to nucleic acid-lipid particles, in particular functionalised nucleic acid-lipid particles, methods for producing them, and to pharmaceutical compositions containing them and their uses in medicine.
Absstract of: US20260355118A1
The present invention relates to compositions and methods for effective delivery of a therapeutic agent to a cardiomyocyte. The invention also relates to methods of use of the delivery methods of the invention for the treatment of diseases and disorders, including the treatment of cardiac diseases and disorders.
Absstract of: EP4837591A1
0001 The present invention relates to a nanomaterial for preventing tumor bone metastasis, a preparation method therefore and use thereof. The present invention discovers a spatiotemporal coupling interaction between tumor cells and osteoclasts, and provides a tumor-bone initial metastasis behavior-targeting strategy, which may accurately prevent tumor metastasis based on the source, and may avoid drug resistance and biochemical resistance. Based on this, the present invention designs a nanomaterial for physical killing targeting tumor-osteoclast conjugates, and the nanomaterial is a bone-targeting group-modified nanovesicle encapsulating a carbonate compound and a phosphate compound. The nanomaterial can be effectively enriched in bone tissue; when the tumor cells are activated, an acid secretion function of tumor-related osteoclasts in the tumor-osteoclast conjugates in close contact triggers the carbonate compound to generate a carbon dioxide gas, and promotes the phosphate compound to release to form calcium phosphate crystals with a calcium ion, so as to kill nearby tumor cells, thereby achieving a specific very-early tumor metastasis inhibition effect.
Absstract of: EP4837902A1
The present application discloses a compound with a structural formula shown as formula (I), and a salt and an isomer thereof. The present application further discloses a nanoparticle composition including the compound, or the salt or the isomer thereof. Nanoparticles of the present application may efficiently deliver a drug or a vaccine into a cell, to exert a therapeutic or prophylactic effect of the drug or the vaccine.
Absstract of: EP4837917A2
0001 The present disclosure provides a capping analog for self-replicating mRNA and uses thereof, wherein the capping analog for self-replicating mRNA is capable of enhancing the stability and/or translation efficiency of the self-replicating mRNA.
Absstract of: EP4837897A1
0001 The present disclosure relates to a PEG lipid and the use thereof. The PEG lipid can be used for preparing a lipid nanoparticle that delivers an active ingredient. The present disclosure further relates to a pharmaceutical composition and a lipid nanoparticle comprising the PEG lipid, as well as the use thereof in preparing a pharmaceutical.
Absstract of: AU2025205865A1
A composition for genetic editing of a hydroxyacid oxidase 1 (HAO1) gene, comprising: (a) a messenger RNA (mRNA) encoding a Cas12i2 polypeptide, (b) a guide RNA (gRNA) targeting the HAO1 gene; and (c) lipid excipients, which optionally form lipid nanoparticles. Also provided herein are methods for genetic modification of the HAO1 gene in host cells with the composition provided herein.
Absstract of: WO2025147528A1
The present invention is based on the surprising discovery that a lipid nanoparticle (LNP) bearing sialic acid residues on the surface can withstand nebulization and maintain its integrity. The invention provides an LNP composition comprising a lipid bearing, or substituted by, a sialic acid (SA) residue such as a ganglioside, a ganglioside derivative, a mimetic, a SA-bearing entity or a combination thereof. The lipid nanoparticles described herein can incorporate nucleic acid molecules, or cargos, withstanding nebulization, and delivering the cargos to the lung via a nebulization-mediated delivery. 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 treatment of various pulmonary diseases. The LNPs provided herein are also useful for prophylactic applications including vaccines for infectious diseases, allergies, autoimmune diseases and cancer.
Absstract of: WO2025147545A1
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.
Absstract of: WO2025147516A1
The present invention provides a lipid nanoparticle comprising an entity that contains one or multiple sialic acid (SA) moieties, the entity is preferably a ganglioside, a ganglioside derivative, a ganglioside mimetic, or a combination thereof. The lipid nanoparticle of the present invention can effectively present SA moieties on the surface to bind Siglecs and complement factors to, inter alia, mitigate inflammation. The present invention also provides methods of using said lipid nanoparticle compositions described herein for pharmaceutical applications. For example, the lipid nanoparticles provided herein are useful for treating inflammatory and autoimmune diseases.
Absstract of: WO2025146681A1
The present invention relates to pro-nano-emulsion compositions comprising at least one type of Omega 3 fatty acid capable of converting to nano-emulsion composition having average droplet size of below Ip, typically below 0.5p, and use thereof, particularly as dietary supplement, for preferential delivery of the Omega 3 fatty acid to heart and/or liver tissue of a subject consuming the composition, thereby maintaining normal cardiovascular/hepatic function and/or improving cardiovascular/hepatic disease or disorders.
Absstract of: EP4837568A1
The present invention relates to a surface-modified albumin nanoplatform, a method for preparing the same, and a composition for diagnosing or treating a disease using the same. The surface-modified albumin nanoplatform according to the present invention can reduce uptake by the reticuloendothelial system even when an excessive amount of click functional groups and various transmitters are conjugated to albumin, by deactivating the click-reactive functional groups on the surface of the albumin to which a transmitter is not conjugated, and accordingly, the in vivo half-life is greatly increased, so that a target molecule or active material can be effectively delivered to a target tissue or cell, thereby achieving excellent therapeutic and diagnostic effects.
Absstract of: EP4837915A1
The present invention is directed to silicon containing ionizable lipids, lipid nanoparticles (LNPs) comprising at least one ionizable lipid according to the invention, pharmaceutical compositions comprising a lipid nanoparticle according to the inventio and a pharmaceutically acceptable carrier as well as their pharmaceutical use.
Absstract of: EP4837592A1
0001 The present invention discloses a lipid nanoparticle (LNP) and its application, comprising cationic ionisable lipids, auxiliary lipids, ligand-modified PEGylated lipids, and cholesterol. The auxiliary lipids and ligand-modified PEGylated lipids target receptors on the surface of liver sinusoidal endothelial cells (LSECs). The LNPs enhance LSEC-specific uptake through a targeted design involving the lipid-specific interaction pathway.
Absstract of: CN123063463A
本发明提供了用于治疗ADSL基因缺陷相关疾病的腺相关病毒及其应用。本发明提供了包含5' ITR，启动子，ADSL基因编码序列，转录调控元件，polyA，3' ITR的质粒载体，以及由该质粒和包装质粒、辅助质粒包装而成的重组腺相关病毒。本发明通过实验证明所述重组腺相关病毒可以有效治疗ADSL基因缺陷相关疾病，具有广阔的应用前景。
Absstract of: CN123059006A
本发明公开一种外泌体组合物及其应用，涉及生物药品与肽类药物递送领域，该组合物包括：人脐带间充质干细胞源外泌体、包封于外泌体膜内腔中的AnnexinA1模拟肽Ac2‑26，以及由海藻糖、组氨酸缓冲盐和L‑亮氨酸中的一种或多种构成的药学上可接受吸入稳定体系；通过控制单位颗粒载荷量和游离Ac2‑26比例，使活性肽受到膜性结构保护并适于肺部递送；该组合物制成液体雾化制剂、复溶型冻干制剂或吸入粉末，用于制备肺部炎症性损伤相关吸入制剂。本发明组合物到达肺部后释放Ac2‑26，调节FPR2/ALX相关炎症消退信号，减少中性粒细胞过度募集，维持肺泡‑毛细血管屏障。
Absstract of: CN123059152A
本发明公开了一种肿瘤细胞膜修饰的超声响应型纳米异质结的制备方法及其应用，属于生物医学工程领域。该仿生纳米异质结具有纳米级的“壳”‑“核”结构，其中(1)“核”结构为内部荷载携氧全氟化碳的CaO2/CoS纳米粒子，(2)“壳”结构为脂质体和同源肿瘤细胞膜的包覆层。其主要特征在于：(1)利用同源肿瘤细胞膜的生物活性，能够发挥肿瘤同源靶向性，渗透到肿瘤深处；(2)在外部超声刺激下，经外部超声刺激，能够有效释放出内容物Ca2+，引发线粒体膜电位改变，从而促进细胞凋亡。(3)此外，载氧全氟化碳释放氧气改善肿瘤部位的局部乏氧，同时异质结结构在超声刺激下能够有效分离电子和空穴，声动力性能得到大幅提高，与钙死亡产生协同治疗效应，有效抑制了肿瘤的生长。
Nº publicación: CN123059072A 10/11/2026
Applicant:
南方科技大学
Absstract of: CN123059072A
本申请涉及肿瘤药物技术领域，尤其涉及一种PTRHD1作为肿瘤免疫治疗靶向位点的应用。提供了一种PTRHD1作为肿瘤免疫治疗靶向位点的应用，所述PTRHD1蛋白包含SEQ ID No.1所示的氨基酸序列，或与SEQ ID No.1具有至少85%氨基酸序列同一性的蛋白。PTRHD1可调节线粒体磷酸变位酶PGAM5和线粒体动力蛋白DRP1的蛋白功能来调控线粒体分裂与细胞坏死，PTRHD1缺失的肿瘤细胞因此发生炎症性免疫原性增加；因此，以PTRHD1作为肿瘤免疫治疗靶向位点，通过靶向抑制PTRHD1相关基因或编码蛋白的表达，可以有效激发机体抗肿瘤免疫，具有广泛应用。
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