Alerta

用于向细胞递送治疗性有效载荷的脂质纳米颗粒

Resumen de: WO2025059599A1

The disclosure provides ionizable lipids for constructing lipid nanoparticles. The disclosure further provides conjugates comprising a targeting moiety and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (e.g., payload) for delivery to immune cells, hematopoietic stem cells (HSCs), or liver cells. The conjugates can be delivered to cells ex vivo or formulated in a pharmaceutical composition to be directly administered to a subject in need thereof (e.g., via in vivo administration).

用于向细胞递送治疗性有效载荷的脂质纳米颗粒

Resumen de: WO2025059607A1

The disclosure provides lipid nanoparticles (LNPS) and conjugates comprising a targeting moiety and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (e.g., payload) for delivery to hematopoietic stem cells (HSCs). The conjugates can be formulated in a pharmaceutical composition and can be directly administered to a subject in need of treatment (e.g., via in vivo administration).

蛋白水解靶向嵌合肿瘤疫苗

Resumen de: WO2025059546A1

Disclosed are compounds comprising an active agent covalently linked to a tumor antigen. Also disclosed herein nanoparticles comprising a tumor antigen covalently linked to an active agent and a plurality of lipid or lipidoids.

ホルモン依存性癌の治療用のｎａｂ－シロリムスとエストロゲン抑制剤との組み合わせ

Resumen de: WO2025034870A1

The present application, in certain aspects, pertains to methods and compositions for the treatment of a hormone-dependent cancer (such as an endometrial cancer (e.g., endometrioid endometrial cancer) or hormone-receptor positive breast cancer), using a composition comprising nanoparticles comprising sirolimus and an albumin in combination with an estrogen suppressor (such as letrozole or fulvestrant).

一种pH响应型PLGA-PEG纳米载体及其制备方法

Resumen de: CN122557754A

本发明公开了一种pH响应型PLGA‑PEG纳米载体及其制备方法，包括载体材料、靶向配体和荧光标记，所述靶向配体、荧光标记均与所述载体材料相连接，所述载体材料为PLGA‑PEG‑COOH嵌段共聚物，PLGA‑PEG‑COOH嵌段共聚物包括PLGA和NH2‑PEG‑COOH，PLGA的分子量为10000‑50000 Da，PEG的分子量为2000‑5000 Da，PLGA末端的羧基与NH2‑PEG‑COOH的氨基经酰胺化反应连接形成所述PLGA‑PEG‑COOH嵌段共聚物，PLGA‑PEG‑COOH嵌段共聚物末端的羧基作为pH敏感基团，能够在酸性条件下发生质子化；优点是构建末端羧基作为pH敏感基团的PLGA‑PEG‑COOH嵌段共聚物，并连接叶酸靶向配体和Cy5.5近红外荧光标记，实现了在肿瘤微酸性环境中特异性加速释药、主动靶向肿瘤细胞及体内荧光成像追踪的多功能集成，从而提高药物递送效率、减少系统毒性和监测载体分布。

一种新型可电离脂质SM-RA及其制备方法和应用

Resumen de: CN122562700A

本发明属于脂质纳米颗粒的药物递送技术领域，具体涉及一种新型可电离脂质SM‑RA及其制备方法和应用。该可电离脂质的结构式为：。将其掺入脂质纳米颗粒（LNP）后，所构建的LNPSM‑RA体系具有以下特征：（1）粒径均一，mRNA包封率高达90%以上；（2）具有高效的体内外递送效率；（3）LNPSM‑RA递送幽门螺杆菌mRNA抗原（mNapA）后，LNPSM‑RA在维持系统性体液免疫应答的同时，显著增强黏膜体液免疫应答；此外，该体系还能有效激活系统性及胃肠道局部细胞免疫应答；（4）LNPSM‑RA递送幽门螺杆菌mRNA抗原后可对幽门螺杆菌感染提供保护作用，具体表现为显著降低感染小鼠胃组织中幽门螺杆菌定植量。

一种用于治疗白桦树花粉症、多发性硬化症、干燥综合征的mRNA脂质纳米颗粒药物组合物

Resumen de: CN122557719A

本发明公开了一种用于治疗白桦树花粉症、多发性硬化症、干燥综合征的mRNA脂质纳米颗粒药物组合物，核心为编码过敏性疾病或自身免疫性疾病相关抗原表位的功能化 mRNA。所述 mRNA 的编码区包含经筛选与验证的白桦树花粉症、多发性硬化症、干燥综合征相关特异性抗原表位编码序列，可通过柔性连接子串联多表位组合；同时 mRNA 搭载优化的 5’UTR、3’UTR、5’帽结构与 polyA 尾，经密码子优化及核苷酸修饰后，兼具高稳定性与高效翻译表达能力。所述 mRNA 由靶向肝窦内皮细胞的脂质纳米颗粒包载递送，可高效诱导抗原特异性免疫耐受，显著改善模型动物疾病指标，为白桦树花粉症、多发性硬化症、干燥综合征提供了新型靶向治疗方案。

含有氨基甲酸酯结构的多酯键脾靶向阳离子脂质及包含其的组合物和用途

Resumen de: CN122562712A

本公开属于医药领域，提供了含有氨基甲酸酯结构的多酯键脾靶向阳离子脂质及包含其的组合物和用途，具体公开了式(I)所示的阳离子脂质。本公开提供的阳离子脂质可用于核酸靶向递送，能够降低肝脏富集的同时显著增强所递送药物的脾靶向性。

賦形剤造粒物

Resumen de: US2025041425A1

0000 Excipient granulations containing a viscosifying agent, a disintegrant, and one or more additional excipients are disclosed. An excipient granulation can be combined with a pharmaceutical granulation to provide a pharmaceutical composition. The excipient granulations can be used to increase the viscosity of an aqueous composition such as an oral pharmaceutical composition. When added to an aqueous solution, the excipient granulation can dissolve to provide a suspension of the pharmaceutical granules in a viscous solution. The excipient granulation can be used to improve the palatability of oral pharmaceutical compositions containing a pharmaceutical granulation.

一种靶向ACE2的酶响应型多肽偶联药物及其制备方法和应用

Resumen de: CN122557761A

本发明属于生物医药技术领域，涉及一种靶向ACE2的酶响应型多肽偶联药物及其制备方法和应用。多肽偶联药物由地塞米松和ACE2靶向肽段通过酶响应性连接子通过共价键连接；其中，酶响应性连接子含有羧酸酯键、硫代酯键或磷酸酯键中的任意一种或几种；ACE2靶向肽段的氨基酸序列为YKYRYL。本发明提供的多肽偶联药物不仅能够形成纳米颗粒，避免迅速降解，而且能够保证与ACE2特异性结合，从而可以主动富集至ACE2高表达的炎症组织等病灶处，且富集后可以在酶作用下特异性释放活性地塞米松，在保持抗炎疗效的同时显著降低地塞米松的全身性副作用，为炎症性疾病等的精准治疗提供了一种安全、有效的通用平台。

一种治疗急性肺损伤的纳米结构脂质载体及其制备方法和应用

Resumen de: CN122557494A

本发明属于医药技术领域，具体涉及一种治疗急性肺损伤的纳米结构脂质载体及其制备方法和应用，本发明以山嵛酸甘油酯为固体脂质、辛癸酸甘油酯为液体脂质，采用吐温80‑大豆卵磷脂复合乳化剂体系，经熔融乳化‑超声分散工艺制备，通过单因素考察结合Box‑Behnken响应面法优化处方。所得制剂平均粒径约100nm，包封率可达87%以上，4℃条件下储存稳定性良好，可显著提高连翘苷的溶解度与口服生物利用度，延长药物体内循环时间。该制剂能够下调IL‑6、IL‑1β、TNF‑α等促炎因子水平，减轻肺组织炎性损伤与肺水肿，对急性肺损伤具有明确治疗作用，且制备工艺简便可控、重复性好，适于产业化开发。

超声激活的铁掺杂钛酸钡基压电芬顿微球及其制备与应用

Resumen de: CN122557737A

本发明公开一种超声激活的铁掺杂钛酸钡基压电芬顿微球及其制备与应用方法与应用，属于生物医药技术领域。本发明以水热法制备铁掺杂钛酸钡纳米颗粒，经苯硼酸脂质包覆后，通过点击化学负载于微流控成型明胶微球得到 PF@MS。超声触发材料产生压电电位，驱动类芬顿反应消耗局部质子，适度上调线粒体膜电位以启动线粒体自噬；反应随局部 pH 升高呈类自限性减弱，防止线粒体过度损伤。纳米颗粒表面硼酸酯修饰可提升能量不足退变髓核细胞的胞内摄取，并实现溶酶体释放。本发明的铁掺杂钛酸钡基压电芬顿微球可恢复线粒体自噬水平，平衡椎间盘细胞外基质代谢，提升退变椎间盘高度保留率，适用于椎间盘退变等线粒体功能障碍类退行性疾病的治疗。

がんの処置において有用なＴＧＦＢアンタゴニストプロドラッグを含有する製剤化および／または共製剤化リポソーム組成物ならびにその方法

Resumen de: WO2021167703A1

Formulated and/or co-formulated liposomes (LNP) and solid-lipid nanoparticles (SLNP) comprising TB Prodrugs and methods of making the LNPs and SLNPs are disclosed herein. The TB prodrug compositions comprise a drug moiety, a lipid moiety, and linkage unit that inhibit ALK5. The TB Prodrugs can be formulated and/or co-formulated into a liposome or solid-lipid nanoparticle to provide a method of treating cancer, immunological disorders, and other disease by utilizing a targeted drug delivery vehicle.

ALBUMIN NANOPLATFORM FOR BORON NEUTRON CAPTURE THERAPY AND COMPOSITION FOR BORON NEUTRON CAPTURE THERAPY COMPRISING SAME

Resumen de: AU2025215292A1

The present invention relates to an albumin nanoplatform for boron neutron capture therapy and a composition for boron neutron capture therapy comprising same. The albumin-based nanoplatform according to the present disclosure can effectively deliver boron to specific tumor tissues by simultaneously conjugating a boron compound containing an excessive amount of boron and a targeting molecule as a carrier for specifically targeting tumor tissues via click chemistry functional groups introduced into the albumin surface. In particular, the nanoplatform enables sufficient delivery of boron molecules to target tumor tissues even at doses less than one-tenth of those required by conventional boron neutron capture therapy (BNCT) drugs, and thus can be applied as a composition for boron neutron capture therapy and as an anticancer therapeutic agent.

METABOLIC INHIBITORS AND METHODS OF MAKING AND USING THEREOF

Resumen de: US20260232707A1

Disclosed are compounds, nanoparticles, and compositions for effective delivery of metabolic inhibitors to disease state cells. The compounds, nanoparticles, and compositions disclosed herein show trackability in biological system, extended stability, and other advantageous physicochemical properties to treat various disease states. Also disclosed are methods of treating a subject in need thereof, such as a subject with cancer.

LIPID NANOPARTICLES FOR THE TREATMENT OF OCULAR DISEASES

Resumen de: US20260232590A1

The present invention relates to a composition comprising lipid nanoparticles comprising rosehip oil, at least one solid lipid, at least one surfactant, and optionally at least one active ingredient, as well as their use in a method of treating ocular diseases, such as for example dry eye disease.

Lipid nanoparticles encapsulation of large RNA

Resumen de: AU2026208137A1

A method of producing lipid-encapsulated RNA nanoparticles includes flowing an aqueous solution comprising an RNA through a 1st tube having a first inner diameter (ID); the RNA comprises from about 6,000 to about 13,000 nucleotides; flowing an ethanol solution comprising lipids through a 2nd tube having a second inner diameter (ID), at a flow rate of about 0.2 to about 1 times relative to the aqueous solution through the 1st tube, the lipids comprise a cationic lipid; and mixing the ethanol solution with the aqueous solution; the first ID and second ID and flow rates through the 1st tube and 2nd tube are selected to produce a shear force sufficiently low to preserve the integrity of the RNA; the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about ethanol, the lipid-encapsulated RNA nanoparticles having a bilayer structure. ul u l

NANOPARTICLE-HYDROGEL COMPOSITE FOR NUCLEIC ACID MOLECULE DELIVERY

Resumen de: AU2024417973A1

This disclosure provides novel peptide hydrogels containing encapsulated nanoparticles comprising nucleic acid molecules (such as miRNA) that can undergo multiple gel-to-solution (gel-sol) and solution-to-gel (sol-gel) phase transitions, and their use, such as for controlled delivery of nucleic acid molecules to a subject. Also provided are novel peptides for use in disclosed peptide hydrogels.

PHARMACEUTICAL FORMULATIONS OF GENE DELIVERY VEHICLES

Resumen de: US20260234617A1

0000 The invention relates to formulations comprising miRNA that have improved stability for the treatment of diseases including neurodegenerative diseases such as spinocerebellar ataxias type 3.

Compositions and methods for pulmonary surfactant-biomimetic nanoparticles

Resumen de: AU2026208121A1

Abstract Compositions and methods comprising pulmonary surfactant (PS)-biomimetic nanoparticles are disclosed. In particular, the disclosure relates to a composition comprising a negatively charged nanoparticle having an average size of 200 to 400 nm and comprising a plurality of pulmonary surfactant-biomimetic molecules, and one or more cargo molecules enveloped by the nanoparticle, wherein the cargo molecule has a molecular weight of up to 1200 Da. Abstract 40x ** wo 2021/071823 Perth Body weight change (%) Perth+PS-GAMP IgG titer Log10 = -5 -2.0 -10 SUBSTITUTE SHEET (RULE 26) Days post immunization Days post immunization PBS Perth GAMP Perth Michigan ** HAI titer PBS Perth+PS. Perth .GAMP PBS Perth+PS. Perth .GAMP PCT/US2020/054377 FIGS. 37A-37E ul u l e m p e r a t u r e h a n g e ( ° ) e r t h + - ( ) Days post immunization Days post immunization e r t h t i t e r t i t e r e r t h -

NANOMATERIALS CONTAINING CONSTRAINED LIPIDS AND USES THEREOF

Resumen de: US20260232587A1

Compositions for delivering nucleic acids to cells or tissue microenvironments are provided. In one embodiment, the compositions are lipid nanoparticle compositions formulated to have reduced splenic and hepatic clearance. It has been discovered that the chemical composition of lipid nanoparticles significantly influences the natural trafficking of the lipid nanoparticles. More specifically, it has been discovered that conformationally constrained ionizable lipids can modify the tropism and clearance profile of lipid nanoparticles without the need of a targeting ligand. It has also been discovered that tropism of the disclosed lipid nanoparticles is size-independent.

COMPOSITIONS AND METHODS OF MANUFACTURING TRIVALENT FILOVIRUS VACCINES

Resumen de: AU2026206234A1

Abstract Disclosed is a stable immunogenic composition capable of eliciting a robust and durable immune response, comprising at least one antigen consisting of a filovrus glycoprotein and at least one nano-emulsion adjuvant which are co-lyophilized and can be reconstituted immediately prior to use. Also disclosed is a vaccine composition comprising at least two antigens, wherein each antigen is specific to a different genus of filovirus and which also comprises at least one nano-emulsion adjuvant. Abstract

DEGRADABLE CATIONIC LIPID CONTAINING A DISULFIDE BOND AND APPLICATIONS THEREOF

Resumen de: US20260234104A1

0000 A cationic lipid containing a disulfide bond of Formula (1), wherein, —S—S— is a disulfide bond; L<1 >and L<2 >are each independently a degradable divalent linking group, X<1 >and X<2 >are each independently an acyclic divalent linking group containing a tertiary amine group; G<1 >and G<2 >are each independently a linking bond or a trivalent branching group; when G<1 >is a linking bond, k1 is 1; when G<1 >is a trivalent branching group, k1 is 2; when G<2 >is a linking bond, k1 is 1; when G<2 >is a trivalent branching group, k2 is 2; R<1 >and R<2 >are each independently a substituted or unsubstituted C<5-30 >hydrocarbon group or C<5-30 >hydrocarbon derivative residue. The lipid composition prepared with the cationic lipid can more effectively exert the therapeutic effect of the LNP-nucleic acid pharmaceutical composition formulation, thereby improving immunological or therapeutic outcomes. 0000

LIPIDS AND LIPID NANOPARTICLE FORMULATIONS

Resumen de: US20260232588A1

The present disclosure relates generally to lipids, lipid nanoparticle formulations, and methods of using the same for delivering nucleic acids, such as mRNA.

Nuclear Targeted DNA Delivery and Compositions for Use in Practicing the Same

Nº publicación: US20260234655A1 13/08/2026

Solicitante:

SEAWOLF THERAPEUTICS INC [US]
Seawolf Therapeutics, Inc.

Resumen de: US20260234655A1

0000 Methods of nuclear targeted DNA delivery are provided. Aspects of the methods include contacting a cell with: (a) a nuclear targeted deoxyribonucleic acid (NTDNA) that includes a DNA nuclear targeting sequence (DTS) and a cargo nucleic acid heterologous to the DTS. Also provided are compositions for use in practicing methods of the invention.