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用于体内转染的表达增加的RNA

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.

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。

一种用于无表面活性剂癌症药物递送的Pickering纳米片包裹层

Resumen de: CN122557488A

本发明公开了一种用于无表面活性剂癌症药物递送的Pickering纳米片包裹层，属于癌症药物递送技术领域。该包裹层以经十八烷基功能化的单层磷酸锆ZrP‑C18纳米片为Pickering乳化剂，通过乳化‑扩散‑挥发法在疏水性化疗药物多西他赛DTX核心表面自组装形成致密的纳米屏障。本发明摒弃了传统的有毒分子表面活性剂和有机溶剂，ZrP‑C18纳米片通过界面拥塞效应赋予载药纳米颗粒卓越的胶体稳定性，同时实现药物的可控缓释；该包裹层可使载药颗粒被紫杉烷耐药癌细胞高效内化，绕过耐药机制，且无明显溶血效应，血液相容性优异。本发明为疏水性癌症化疗药物的无表面活性剂递送提供了新方案，适用于卵巢癌、乳腺癌等多种癌症的治疗，具有良好的临床转化前景。

脂质体纳米颗粒组合物及其用途

Resumen de: WO2025157076A1

The present disclosure relates to liposome nanoparticle (LNP) compositions comprising a therapeutical cargo (e.g., a viral vector such as a rAAV vector). The LNP can shield the surface antigen of the cargo and reduce the clearance of the cargo by the immune system. It also relates to the use of immunosuppressants to suppress the immune response in the body and further reduce the clearance of the cargo by the immune system.

表面修饰的白蛋白纳米平台、其制备方法及利用其的用于诊断或治疗疾病的组合物

Resumen de: AU2024419247A1

The present invention relates to a surface-modified albumin nano-platform, a method for preparing same, and a composition for diagnosing or treating a disease using same. The surface-modified albumin nano-platform according to the present invention can reduce ingestion by the reticuloendothelial system even when an excess amount of click-functional groups and various delivery substances are conjugated to albumin, by inactivating a click reaction functional group to which a delivery substance on the surface of albumin is not bound, so that the in vivo half-life is greatly increased, and a target molecule or an active substance can be effectively delivered to a target tissue or cell, leading to excellent therapeutic and diagnostic effects.

包含内封有核酸的脂质纳米粒子的冷冻组合物

Resumen de: WO2025150516A1

The present invention provides a frozen composition containing nucleic acid-encapsulated lipid nanoparticles, wherein the nucleic acid-encapsulated lipid nanoparticles contain an ionic lipid represented by formula (1), and the molar ratio of amino groups of the ionic lipid represented by formula (1) to nucleic acid phosphate groups is 7-250 inclusive (the definitions of the symbols in formula (1) are as described in the description).

用于药物递送的纳米颗粒组合物

Resumen de: WO2025143892A1

The present invention relates to a composition for drug delivery, and a preparation method therefor, and, more specifically, to a composition for drug delivery, and a preparation method therefor, the composition having a form such that a drug is encapsulated inside a nanoparticle structure formed by a polymer and a cationic lipid with a specific structure.

脂质纳米颗粒组合物的用途

Resumen de: WO2025130965A1

A method of preventing, treating or managing a disease in an organ or a tissue in a subject, comprising administration of a lipid nanoparticle comprising a messenger RNA (mRNA) encoding a protein to the subject having the disease in an organ or tissue via a systemic administration route, wherein the half-life of the protein is less than certain value as determined in mouse serum, and wherein the comparative accumulation level of the protein in the organ or tissue when the lipid nanoparticle comprising the mRNA encoding the protein is administered via the systemic administrative route is higher than the comparative accumulation level of the protein in the organ or tissue when the protein is administered via the same systemic administration route.

脂质纳米颗粒组合物及其用途

Resumen de: US2025177562A1

0000 Provided are aerosolized pharmaceutical compositions including comprising aerosol particles, the aerosol particles comprising lipid nanoparticles (LNPs). Also provided herein are methods of administering the aerosolized pharmaceutical compositions described herein.

一种负载PROTAC的仿生神经靶向植物外泌体及其制备与应用

Resumen de: CN122557486A

本发明公开了一种负载PROTAC的仿生神经靶向植物外泌体及其制备与应用，属于生物医药技术领域。该纳米药物以植物来源外泌体为核心载体，内载PROTAC分子并天然携带内源性活性分子，在此基础上通过细胞膜仿生包被及pH响应性溶酶体逃逸聚合物修饰构建工程化外泌体递送系统。该系统经鼻腔给药后，能够依次克服鼻黏膜屏障，实现脑部病灶区域的富集，并在细胞内高效释放治疗载荷。本发明通过PROTAC对病理相关蛋白的选择性降解作用，结合植物外泌体内源性活性分子对细胞代谢与线粒体功能相关通路的调控作用，实现对神经元蛋白稳态与代谢稳态的协同调节，从而对神经退行性疾病的病理过程进行多靶点、多层级干预。

一种蛋白靶向降解嵌合体CytoTAC及其应用

Resumen de: CN122562961A

本发明公开了一种溶酶体靶向蛋白降解嵌合体CytoTAC及其应用。所述CytoTAC由三个功能性蛋白结构域组成，包括：靶向溶酶体结构域、靶向靶蛋白结构域以及Linker。CytoTAC在靶蛋白降解率高达约80％，并具备多靶点设计的灵活性。通过串联设计，CytoTAC能够同时降解2‑3种靶蛋白。在应用方面，CytoTAC展示了针对包括肿瘤和阿尔茨海默症等疾病的显著治疗潜力。本发明创新性地结合了高特异性、快速降解和模块化设计，突破了传统蛋白降解技术的局限。CytoTAC在细胞靶向治疗、疾病机制研究及遗传工程等领域具有重要的应用价值，为疾病治疗提供了全新的技术解决方案。

一种FeN-T纳米酶复合材料及其制备方法和应用

Resumen de: CN122557594A

本发明公开了一种FeN‑T纳米酶复合材料及其制备方法和应用，属于药物制剂技术领域。所述FeN‑T纳米酶复合材料由铁基纳米酶、他克莫司和聚乳酸‑羟基乙酸共聚物组成。本发明将铁基纳米酶（FeN）与抗炎药物他克莫司（TAC）通过PLGA包裹进行复合制备得到纳米酶复合材料（FeN‑T）。经实验验证，FeN‑T可促进细胞迁移，在有效治疗浓度下几乎无细胞毒性；且能够提高H2O2的抑菌率；同时，FeN‑T与H2O2联用治疗在9天内的创面愈合率能够达到95%，显著优于单一治疗。因此，本发明提供了一种兼具高效抗菌、良好生物相容性及抗炎促修复功能的多功能复合材料。

一种基于植物细胞外囊泡的药物组合物及其制备方法与应用

Resumen de: CN122557493A

本发明公开了一种基于植物细胞外囊泡的药物组合物及其制备方法与应用，属于生物医药技术领域，包括植物来源细胞外囊泡和载药脂质体；所述植物来源细胞外囊泡包括蛋白质；所述载药脂质体为负载有小分子药物的脂质体；所述蛋白质和脂质体的质量比为（1:10）‑（10:1）。本发明使用了物理共挤出法作为灵活简便的杂化工艺，无需复杂的化学修饰，制备方法简单可控，可在保持各组分生物活性的前提下获得粒径均一、结构稳定的杂化纳米粒，易于放大生产，显著降低了规模化制备的成本。并且，本发明提供的药物组合物在4T1原位乳腺癌模型表现出显著的抑瘤效果，具有良好的临床应用前景。

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).

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.

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).

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.

AFFIBODY MICELLE DRUG CONJUGATE

Resumen de: US20260232835A1

A copolymer includes a copolymer X and a target-specific molecule bonded to the copolymer X. The copolymer X includes structural units of formulas (A), (B) and (C),where R1, R2, and R3 are the same or different and are hydrogen or C1-3 alkyl, R4 is C1-3 alkyl, R5 is hydrogen, C1-18 alkyl, a 3- to 8-membered cycloalkyl optionally having a substituent, an adamantyl, a C6-18 aryl optionally substituted, or a 5- to 10-membered heteroaryl group optionally substituted, X1, X2, and X3 are the same or different and are oxygen, sulfur, or N—R7, R6 is hydrogen, leaving group, or linker, R7 is hydrogen or C1-3 alkyl group, m is 1 to 100, and n is 0 to 3.

IONIZABLE CATIONIC LIPID AND USE THEREOF

Resumen de: WO2026166561A1

Disclosed in the present application are an ionizable cationic lipid and the use thereof. In the ionizable cationic lipid of the present application, adsorption with a biological drug such as a nucleic acid is formed by means of the structural design of the head group, thereby improving the encapsulation efficiency, a branched structure is formed at the tail group by using a saturated or unsaturated fatty chain, and the head group and the tail group are linked by using an ester bond, which facilitates in-vivo degradation to reduce toxicity. The design of the unsaturated fatty chain increases the membrane fluidity during endosomal release, and effectively improves the release efficiency of a biological drug. Moreover, the ionizable cationic lipid of the present application has a good temperature stability, which not only improves the product quality, but also expands the product accessibility. In summary, the ionizable cationic lipid of the present application has the advantages of a high encapsulation efficiency, a low toxicity, a high endosomal release efficiency, a good temperature stability, etc., and can improve the quality and therapeutic effect of a lipid nanoparticle drug, thus providing a new solution and choice for biological drug delivery.

A THERMORESPONSIVE INJECTABLE HYDROGEL FOR INTRAVASCULAR ADMINISTRATION

Resumen de: AU2025265466A1

Disclosed herein is a flowable polymer solution for intravascular (arterial and venous) administration comprising the polymer and at least one solvent, the polymer comprising a first monomer for binding water; a second monomer for imparting mechanical properties; optionally, a third monomer for binding to a natural or synthetic peptide or protein (NSPP); and a fourth monomer for imparting thermoresponsive phase-transition behaviour. In an embodiment, the solution further comprises at least one radiographic intravenous contrast agent is selected from contrast media for digital subtraction angiography (DSA), computer tomography (CT) and fluoroscopy such as iodinated contrast media, tantalum, and bismuth-based materials (e.g., bismuth chelate), heavy metal chelates contrast media for MRI including gadolinium agents (e.g., gadobutrol) or MRI nanoparticles.

COMPOSITIONS TARGETING KLKB1 AND METHODS OF USE THEREOF

Resumen de: US20260232843A1

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.

LIPID NANOPARTICLE WITH NON-COVALENT BIFUNCTIONAL BINDERS FOR ACTIVE TARGETING

Resumen de: US20260232837A1

A nanoparticle complex comprises a bifunctional binder capable of non-covalently binding to the lipid nanoparticle in the nanoparticle complex.

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

Nº publicación: AU2025215292A1 13/08/2026

Solicitante:

CLICHEMBIO INC
CLICHEMBIO, INC.

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.