Nanofármacos

TARGETING NANOSCALE PARTICLE, TARGETING CELL, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: EP4628071A1

The invention discloses a targeted nanoscale particle, a targeted cell, a preparation method therefor, and use thereof. The targeted nanoscale particle is bound to the outer surface of the targeted cell, and is composed of a plurality of proteins interconnected via a first binding site. The targeted nanoscale particle further comprises a second binding site, and is bound to the outer surface of a target cell via the second binding site. In an exemplary embodiment, the targeted nanoscale particle can promote the interaction between the two types of cells by simultaneously binding to a chimeric antigen receptor T cell and a leukemia cell, thereby promoting the recognition and killing of the leukemia cell by the chimeric antigen receptor T cell. In addition, the internal cavities of the proteins in the targeted nanoscale particle provide space for loading of a chemotherapeutic drug, thus realizing the combination therapy of the chimeric antigen receptor T cell and other therapies while loading the drug.

BISPECIFIC ANTIBODIES AGAINST CANINE CD3 AND CD20

Resumen de: MX2025006274A

This disclosure provides a bispecific canine antigen-binding molecule comprising a first antigen binding domain or antigen-binding portion thereof that specifically binds canine CD3, and a second antigen binding domain or antigen-binding portion thereof that specifically binds canine CD20, compositions comprising the same, and methods of their use. The disclosure also provides a canine antibody or antigen-binding portion thereof that binds canine CD3, compositions comprising the same, and methods of their use. The disclosure also provides a canine antibody or antigen-binding portion thereof that binds canine CD20, compositions comprising the same, and methods of their use.

COMPOSITIONS AND METHODS FOR TREATING HUMAN PAPILLOMAVIRUS INFECTIONS

Resumen de: US2024342163A1

The invention provides compositions, compounds, formulations, and methods for treating HPV infections including pre-malignant infections and cancer. Compounds that covalently bind to the HPV E6 protein are disclosed.

VACCINES CONTAINING NOVEL NANOPARTICLE SCAFFOLDS

Resumen de: AU2023399881A1

The present invention provides novel engineered nanoparticle scaffold sequences that are derived from the 13-01 protein. Relative to the known 13-01 protein or variants thereof, the novel 13-01 derived scaffold sequences of the invention contain an extended N-terminal helix. Also provided in the invention are vaccine constructs that contain various immunogenic proteins displayed on the novel nanoparticle scaffold sequences described herein. The vaccine constructs of the invention include, e.g., nanoparticles displaying tandem repeats of influenza M2e proteins or HCV E2 core proteins.

SYNTHETIC SINGLE-STRANDED DNA MOLECULES AND METHODS OF PRODUCING AND USING THE SAME

Resumen de: AU2023406947A1

The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.

TARGETING GPR158 (MGLYR) WITH NANOBODIES FOR THERAPEUTIC BENEFITS

Resumen de: WO2024118636A1

The invention provides antibodies that specifically bind GPR158 and inhibit GAP activity of GPR158 via RGS7/ Gβ5. The antibodies are useful in the diagnosis and treatment of affective disorders, mood disorders, and brain disorders.

COMPOSITIONS AND METHODS FOR MAKING AND USING POLYMER-COATED NANOCAPSULES FOR TARGETED PHARMACEUTICAL AGENT DELIVERY

Resumen de: WO2024118638A2

Embodiments of the present disclosure provide novel compositions and methods for making and using polymer-coated nanocapsules. In certain embodiments, compositions and methods are disclosed for embedding at least one agent in a liquid fatty acid composition to form an inner core of the polymer-coated nanocapsule and coating the at least one agent-containing liquid fatty acid composition inner core with polymer to form at least one coating layer of polymer that further includes at least one positively charged surfactant (e.g., cationic surfactant), forming polymer-coated nanocapsules. In certain embodiments, the at least one positively charged surfactant binds to at least one targeting agent for directed use of the polymer-coated nanocapsules.

SYNTHETIC SINGLE STRANDED NUCLEIC ACID COMPOSITIONS AND METHODS THEREOF

Resumen de: AU2023406947A1

The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.

NOVEL IONIZABLE LIPIDS AND LIPID NANOPARTICLES COMPRISING THE SAME

Resumen de: WO2024119037A1

Provided herein are an ionizable lipid compound, a lipid nanoparticle comprising the ionizable lipid compound, a composition comprising an mRNA formulated in the lipid nanoparticle, and a method of delivering an mRNA to a subject or a cell by administering the composition including an mRNA formulated in the lipid nanoparticle to the subject or cell.

NOVEL POLYGLYCEROL-CONJUGATED LIPIDS AND LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME

Resumen de: AU2023406321A1

The present disclosure provides novel polymer-conjugated lipids, e.g., comprising DODA 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.

LONG NON-CODING RNA LIPTER PRESERVES LIPID METABOLISM OF THE HUMAN HEART

Resumen de: WO2024118378A1

Compositions and methods are disclosed for treating metabolic syndrome-associated heart disease cardiomyopathy and/or heart failure, wherein the method comprises the step of increasing the concentration of LIPTER RNA in the cardiomyocytes of said patient.

PHOSPHATIDYLAMINE COMPOUND COMPRISING MULTIPLE TERTIARY AMINO GROUP STRUCTURES, AND COMPOSITION AND USE THEREOF

Resumen de: EP4628494A1

A phosphatidylamine compound including a plurality of tertiary amino group structures and the composition and use thereof are provided. The phosphatidylamine compound is a phospholipid compound including two or more tertiary amino group structures, the structure of which is represented by the following formula (I), where, the definition of each substituent is detailed in the instructions. The compound works together with other lipid components such as cholesterol, DSPC/DOPE, DMG-PEG2000, and other helper lipids to form lipid nanoparticles (LNPs), which may be used for efficient delivery of drug molecules such as nucleic acids (siRNA, mRNA, pDNA), thereby realizing diagnosis and treatment of diseases such as cancer, fibrosis (e.g., liver, lung, kidney).

STEROID-CATIONIC LIPID COMPOUND AND USE THEREOF

Resumen de: EP4628496A1

A steroid-cationic lipid compound as represented by formula (I). The LNP prepared from the compound can deliver a bioactive substance to a target cell or organ in an effective and stable manner, and the mRNA LNP prepared from the compound has good levels of stability and transfection efficiency, and can trigger a relatively high specific antibody response and cellular immune response in an animal.

STABLE TOPICAL NANOEMULSION COMPRISING MEGLUMINE ANTIMONIATE AND AMPHOTERICIN B FOR THE TREATMENT OF CUTANEOUS LEISHMANIASIS

Resumen de: EP4628102A2

The drugs available for the treatment of cutaneous leishmaniasis have unsatisfactory efficacy, frequent and serious adverse effects, and require long treatment regimens. Thus, the search for new treatment alternatives for cutaneous leishmaniasis is considered a priority by the World Health Organization. Parenteral administration of pentavalent antimonials for the treatment of all forms of leishmaniasis, including cutaneous leishmaniasis, has several limitations. The therapy is long, requires repeated doses, and adverse reactions are frequent. Topical treatment is an attractive alternative for cutaneous leishmaniasis, offering significant advantages over systemic therapy: fewer adverse effects, ease of administration, and lower costs. The present inventors aimed to provide a fixed-dose topical composition containing at least one antileishmanial compound, providing adequate absorption of the active ingredient. Another objective of the present invention is to provide a topical, fixed-dose formulation containing a combination of antileishmanial compounds that has sufficient efficacy and safety to be used in the treatment of cutaneous leishmaniasis.

LIPID NANOPARTICLES COMPRISING NUCLEIC ACIDS AND LIPID-ANCHORED POLYMERS

Resumen de: WO2024119103A1

Provided herein are lipid nanoparticles (LNPs) comprising a therapeutic nucleic acid (TNA) and uses thereof. The LNPs comprise an ionizable lipid; a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, and do not comprise a helper lipid.

LIPID NANOPARTICLE (LNP) DELIVERY SYSTEMS AND FORMULATIONS

Resumen de: WO2024119098A1

The present disclosure describes compositions, nanoparticles (such as lipid nanoparticles), and/or lipid nanoparticle compositions and methods of their use.

SURFACE MODIFIED CARBON NANOTUBES AND USES THEREOF

Resumen de: AU2023401698A1

Provided herein are pharmaceutical compositions comprising surface functionalized carbon nanotube and an active agent. The active agent can be attached to the carbon nanotube covalently or noncovalently. Also provided are methods of preparing the pharmaceutical compositions and methods of use thereof.

LIPID NANOPARTICLES COMPRISING NUCLEIC ACIDS, IONIZABLE LIPIDS, STEROLS, LIPID ANCHORED POLYMERS AND HELPER LIPIDS, THEIR USES

Resumen de: AU2023406303A1

Provided herein are lipid nanoparticle (LNP) compositions (e.g., pharmaceutical compositions) comprising a therapeutic nucleic acid (TNA), wherein the LNP comprises an ionizable lipid; a "helper" lipid, e.g., a ceramide or distearoylphosphatidylcholine (DSPC); a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, as well as uses thereof.

STEALTH LIPID NANOPARTICLE COMPOSITIONS FOR CELL TARGETING

Resumen de: AU2023406483A1

The present disclosure provides stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types,

GOLD NANOZYME COMPRISING GLYCOL CHITOSAN AND GOLD PARTICLES, AND PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING INFLAMMATORY BOWEL DISEASE COMPRISING SAME

Resumen de: EP4628070A1

One aspect according to the present invention relates to: a gold nanozyme including glycol chitosan and gold particles; and a pharmaceutical composition for preventing or treating inflammatory bowel disease, the composition comprising the gold nanozyme. The gold nanozyme and the pharmaceutical composition comprising same according to one aspect of the present invention was found to inhibit the expression of inflammatory factors, inhibit the generation of reactive oxygen species (ROS) and reactive nitrogen species (RNS) inside cells, and reduce the production of nitric oxide (NO) inside cells. In addition, the gold nanozyme was found to inhibit the secretion of Hight Mobility Group Box 1 (HMGB1) when further including glycyrrhizin. In addition, the gold nanozyme and the composition comprising same were found to enable the recovery of the length, weight, etc., of damaged intestines, and thus can be used in the inflammatory bowel disease prevention and/or treatment industry/market.

一种外泌体包覆的共负载金纳米颗粒和抗生素的树状大分子纳米凝胶及其制备方法和应用

Resumen de: CN120732815A

本发明涉及一种外泌体包覆的共负载金纳米颗粒和抗生素的树状大分子纳米凝胶及其制备方法和应用，以树状大分子纳米凝胶为载体，载体内部共负载金纳米颗粒和药物，载体表面包裹外泌体。本发明操作条件简单，易于纯化，可改善抗生素的生物利用度并实现药物在病灶部位的靶向递送和响应性释放，在肺结核治疗中拥有良好的应用前景。

一种靶向放疗增敏剂、仿生纳米递送系统及其制备方法和应用

Resumen de: CN120733052A

本申请涉及生物医用纳米材料技术领域，公开了一种靶向放疗增敏剂、仿生纳米递送系统及其制备方法和应用。靶向放疗增敏剂的制备方法包括：将三苯基磷和多肽缩合剂溶于无水DMF中，搅拌均匀；再加入SH‑PEG2000‑NH2溶液进行反应，得到SH‑PEG2000‑TPP；将氯金酸溶液加热回流，再加入柠檬酸钠溶液，反应后得到AuNPs分散液；将AuNPs分散液、SH‑PEG2000‑NH2和SH‑PEG2000‑TPP混合反应，得到AuNP‑PEG2000‑TPP纳米粒子，即靶向放疗增敏剂。本申请的靶向放疗增敏剂进入肿瘤线粒体后，经过X射线照射，能够有效吸收射线能量，并转化为热能和电子激发能，产生活性氧(ROS)，进而激活炎性小体和caspase‑1，随后产生N端GSDMD蛋白片段，启动焦亡过程，发挥较强的放疗增敏作用；焦亡过程中释放IL‑1β和IL‑18等促炎细胞因子，进一步扩大局部的免疫反应。

一种罗汉果外泌体样纳米颗粒及其制备方法和在改善肠道健康产品中的应用

Resumen de: CN120732915A

本发明公开一种罗汉果外泌体样纳米颗粒及其制备方法和在改善肠道健康产品中的应用，将罗汉果果肉与预冷PBS匀浆，离心取上清液，经超速离心获得粗提液；通过羧基化磁珠偶联磷脂酰丝氨酸靶向多肽，构建免疫磁珠层析柱，特异性捕获并纯化外泌体样纳米颗粒；所得纳米颗粒具有优异的生物相容性和安全性，可显著调节肠道菌群平衡、增强肠道屏障功能、抑制肠道炎症；本发明为肠道疾病治疗及功能性食品开发提供了天然、高效的新方案，具有广阔的医药和食品应用前景。

一种负载葛花异黄酮的纳米颗粒及其制备方法和应用

Resumen de: CN120732816A

本发明公开了一种负载葛花异黄酮的纳米颗粒及其制备方法和应用，属于复合纳米颗粒技术领域。本发明的负载葛花异黄酮的纳米颗粒，包括纳米颗粒中心和多糖外壳；纳米颗粒中心为包覆葛花异黄酮的酒糟醇溶蛋白，多糖外壳由内至外依次为硫酸葡聚糖外壳和壳聚糖外壳。其制备方法包括以下步骤：以碳酸钠为牺牲模版制备负载葛花异黄酮的单层纳米颗粒；通过调节溶液pH，使纳米粒子带正电，以静电沉积效应涂覆负电性的硫酸葡聚糖外壳；然后与壳聚糖溶液混合，进一步涂覆正电性的壳聚糖外壳，形成层层自组装结构。本发明的负载葛花异黄酮的纳米颗粒在体外模拟消化环境中释放率更高，清除自由基活性更强，为葛花异黄酮的递送提供了更高效的技术。

一种可精准质控的干细胞纳米囊泡及其应用

Resumen de: CN119530147A

The invention relates to a stem cell bioactive nano-vesicle with efficient inflammation regulation and tissue repair effects, the average particle size of the nano-vesicle is 100-350nm, the polydispersity index (PDI) is 0.05-0.40, and the expression rate of phosphatidylserine (PS) on the surface of the nano-vesicle is greater than 40%. According to the preparation method, stem cells are subjected to apoptosis induction, and then a programmed serial extrusion process is adopted to prepare the nano-vesicles. The nano vesicles are moderate in particle size, uniform in distribution and high in production efficiency, the preparation process is easy to operate, the quality is stable and controllable, and the nano vesicles have excellent technical effects in the aspects of repair and regeneration of inflammation injury type tissues and organs.

新型细胞因子组合mRNA疫苗佐剂及其应用

Resumen de: CN120733023A

本发明提供了一种新型细胞因子组合mRNA疫苗佐剂及其应用，所述疫苗佐剂包括可电离脂质、胆固醇、磷脂、PEG衍生物，以及含有编码GM‑CSF和IL‑21基因序列的mRNA；其中，GM‑CSF和IL‑21的氨基酸序列分别如SEQ ID NO:1、2所示。将本发明所述新型细胞因子组合mRNA疫苗佐剂应用于疫苗的制备，组合的细胞因子在注射部位对DC的招募和调控促进了LNP递送和表达引发了强烈的抗肿瘤效应。具体的，通过协同作用通过对注射部位免疫微环境的建立提升了抗原被表达、识别和呈递并且促进了抗原的交叉提呈，DC细胞的激活和淋巴结的浸润促进了T细胞的活化，为肿瘤免疫治疗建立了强大了抗肿瘤效应和免疫记忆。

一种生物合成虾青素的马氏副球菌及其色素提取物和应用

Resumen de: CN120738011A

本发明公开了一种生物合成虾青素的马氏副球菌及其色素提取物和应用。该马氏副球菌（Paracoccus marcusii）IHA069于2022年7月15日保藏于中国普通微生物菌种保藏中心，保藏编号为CGMCC No.25302，16s rDNA序列如SEQ ID No.1所示。通过发酵培养基培养48h后，离心并采用无水乙醇萃取菌体得到色素提取物，虾青素在色素提取物中的比例达到40%。马氏副球色素提取物为主料，玉米淀粉、吐温‑80、氯化钙水为配料，调和喷雾干燥得到具有抗氧化效果的纳米颗粒。相较于IHA034，IHA069强化了提高了虾青素产量便于工业生产，提高了在不同动物中使用的可行性。本发明采用纳米颗粒包埋技术提高了色素提取物中虾青素的保留率，提高了保存温度和有效时间。

一种用于金黄色葡萄球菌感染检测灭活的诊疗探针

Resumen de: CN120733070A

本发明公开了一种用于金黄色葡萄球菌感染检测灭活的诊疗探针，构建方法包括：S1：双波长激发量子点的制备；S2：量子点@ZIF‑8的制备，在量子点表面进行PSS修饰，后在ZIF‑8包覆过程中在1小时内匀速缓慢滴加2‑甲基咪唑溶液用以控制纳米颗粒的成核速率，并且加入PVP稳定纳米粒子的形成；S3：量子点@ZIF‑8@Ber的制备，在合成过程中将小檗碱加入溶剂中，采用一锅法直接合成得到负载小檗碱的量子点@ZIF‑8材料。本发明利用夹心法对金黄色葡萄球菌进行检测，结果通过荧光检测仪反馈，并且可以连接手机进行智能化监测，简便快捷；利用抗原抗体反应，靶向捕获金黄色葡萄球菌，其余细菌的影响很小。

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

一种用于治疗急性呼吸窘迫综合征的仿胞葬纳米制剂

Resumen de: CN120732785A

本发明公开了一种用于治疗急性呼吸窘迫综合征的仿胞葬纳米制剂，属于生物医药技术领域。本发明的仿胞葬纳米制剂包括活性成分、脂质体磷脂双分子层和靶头，所述活性成分由ROS清除药物和抗炎药物组成。本发明创新性地运用仿胞葬作用实现纳米制剂的高效精准靶向，在脂质体表面修饰凋亡细胞标志物，通过释放“eat me”信号，模拟巨噬细胞清除凋亡细胞的天然生物学过程被肺泡巨噬细胞识别并吞噬，实现高效特异性靶向，同时炎症部位巨噬细胞PS受体表达上调进一步增强了靶向效果，为急性呼吸窘迫综合征药物制剂高效递送及病理微环境的重塑提供了一种新的途径。

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

编码治疗性多肽的核酸及包含所述核酸的脂质纳米颗粒组合物

Resumen de: WO2024199114A1

Provided are lipid nanoparticle compositions comprising nucleic acids encoding RSV antigenic polypeptides. Also provided are novel antigenic RSV-F polypeptides as well as nucleic acids encoding the antigenic RSV-F polypeptides.

一种基于Mn-DNA纳米疫苗的肿瘤免疫治疗方法

Resumen de: CN120733013A

本发明涉及一种基于Mn‑DNA纳米疫苗的肿瘤免疫治疗方法。本发明提供一种用于肿瘤免疫治疗的纳米颗粒，其包含金属离子Mn2+、外源dsDNA、抗原肽和打靶肽。本发明还提供制备所述纳米颗粒的方法、包含所述纳米颗粒的疫苗或药物组合物以及它们用于肿瘤免疫治疗的用途等。本发明的纳米颗粒能够被精准递送到抗原提呈细胞，促进抗原提呈细胞成熟，增强抗原递呈能力，显著增强肿瘤靶向性和抗肿瘤活性。本发明所用原料在体内均可降解，生物安全性高。

一种具有肿瘤能量代谢调控功能的自组装基因纳米药物及其制备方法和应用

Resumen de: CN120733055A

本发明公开了一种具有肿瘤能量代谢调控功能的自组装基因纳米药物及其制备方法和应用，属于纳米材料技术领域。本发明通过将光敏剂与阳离子聚合物偶联，形成基因递送载体，该复合物在特定光照条件下能够增强肿瘤细胞的凋亡效果；通过将光敏剂与siRNA结合，实现了多重治疗机制的协同作用，不仅能精准靶向肿瘤细胞并干预其能量代谢，还能在光照下增强细胞死亡效应，显著提高治疗效果。本发明通过靶向肿瘤细胞中高表达的与能量代谢相关的基因，并结合光动力疗法，解决了现有技术在肿瘤能量代谢干预方面的不足，有望在未来成为靶向细胞器的新型治疗手段，可用于抗癌、抗菌、抗炎等药物治疗及示踪。

经由穿膜肽进行非共价修饰脂质纳米颗粒缀合物及用途

Resumen de: CN120737148A

本发明提供了一种多肽衍生物，其具有结构式CnH2n+1CONH‑CPP‑COOH，其中，n=11，13，15，17；CPP为穿膜肽。本发明还提供了该多肽衍生物的制备方法和与脂质纳米颗粒缀合的用途。本发明通过加入在LNP配方中加入基于CPP的阳离子两亲性分子，由可电离脂质负责mRNA的内体释放而CPP促进LNP与细胞膜的结合，通过调整各组分组成比例以及电荷，显著提升了LNP在体内外的递送效率，经由TAT序列修饰LNP可使得肝脏体内递送效率提升20倍。

一种氨基酸阳离子脂质

Resumen de: CN117460710A

The invention provides a novel amino acid cationic lipid, the structure of which is shown in a general formula (1), and the definition of each symbol is consistent with that described in the specification. The amino acid cationic lipid is a pharmaceutically acceptable, biodegradable or high-biocompatibility lipid, and has the advantages of low toxicity, low immunogenicity and high biocompatibility. The amino acid or the amino acid derivative used in the preparation process is simple and easy to obtain, can be naturally obtained or simply synthesized, and has the advantages of simplicity, convenience, safety and production cost saving. Degradable groups can further be contained between amino acid residues and lipophilic tail chains of the novel amino acid cationic lipid, due to existence of the degradable groups, lipid nanoparticles LNP prepared from the novel amino acid cationic lipid can be degraded in vivo in good time, and the problems that in the prior art, LNP prepared from lipid which cannot be degraded can be stored in vivo, the endosome environment is acidified, and the lipid nanoparticles LNP cannot be degraded in vivo are solved. Endosome escape of drug molecules (such as nucleic acid) is blocked, and the problem that drugs delivered into cells cannot play a role is solved. # imgabs0 #

一种脂质纳米颗粒及其应用

Resumen de: CN120732814A

本发明涉及一种脂质纳米颗粒及其应用。所述脂质纳米颗粒包括：阳离子脂质、阴离子脂质DOPS（1,2‑二油酰基‑sn‑甘油‑3‑磷脂酰‑L‑丝氨酸）、其他脂质和靶向抗体。本发明经过研究发现，掺杂阴离子脂质DOPS并间接偶联靶向抗体可以显著提高纳米颗粒对脾脏T细胞的转染效率。本发明进一步将编码靶向肿瘤血管内皮细胞和肿瘤细胞的CAR蛋白的核酸装载在上述脂质纳米颗粒中并用于肿瘤治疗，结果上述治疗显著促进了免疫细胞的瘤内浸润，显著抑制多种实体瘤进展，为CAR‑T疗法在实体瘤中的有效应用提供了新思路、新手段。

LIPID COMPOUNDS, COMPOSITIONS AND METHODS OF USE THEREOF

Resumen de: WO2025207803A1

Described are compounds, compositions, and methods for delivery of therapeutic, diagnostic, or prophylactic agents (for example, a nucleic acid).

NOVEL LIPID BASED ON OLIGO-GAMMA-GLUTAMIC ACID DERIVATIVE, LIPID NANOPARTICLES COMPRISING SAME, AND USE THEREOF

Resumen de: WO2025206461A1

The present invention relates to a novel lipid derivative compound comprising an oligo-gamma-glutamic acid, a lipid nanoparticle composition comprising the same, and the like. According to the present invention, the compound can form lipid nanoparticles by replacing PEGylated lipids, and thus can prevent side effects such as anaphylaxis and has excellent in vivo stability.

MICROPROJECTION ARRAYS

Resumen de: WO2025199580A1

The present invention relates to formulations, devices and methods for coating surfaces of microprojections on microprojection arrays with nucleic acids. The present invention also relates to formulations and methods for coating surfaces of microprojections with nucleic acids where the nucleic acids are associated with lipid nanoparticles (LNP), in particular where the nucleic acid is mRNA.

POLYMERS FOR ENHANCING LIPID NANOPARTICLE DELIVERY OF NUCLEIC ACIDS

Resumen de: WO2025207986A1

The present invention provides delivery-enhancing polymers capable of being encapsulated in nanoparticles to enhance release of payload from the nanoparticle wherein the delivery-enhancing polymer comprises a polyamine comprising tertiary amine. The delivery-enhancing polymer may comprise methylated polyethylenimine (mPEI), branched PEI (bPEI), PAMAM dendrimer and/or histidine polymer.

CELL THERAPY

Resumen de: WO2025206323A1

The present disclosure provides a novel modality for a new target disease (e.g. pancreatic cancer). The present disclosure applies a mRNA CAR-T vaccine to CAR-T for a new target disease (e.g., pancreatic cancer). In the present disclosure, it has been revealed, by in vitro and in vivo experiments, that a mRNA CAR-T vaccine that targets FAP as an antigen can achieve a potent anti-tumor effect in in vivo editing. As for a mechanism for inhibiting the effect of a mRNA CAR-T vaccine, the pathways involving regulatory T cells and the like have also been revealed. As for a synergistic effect with an immune checkpoint agonist, a "condition" for achieving a potent anti-tumor effect in vivo has also been revealed through much trial and error.

FORMULATIONS FOR ADMINISTERING LAAM, norLAAM AND dinorLAAM AND METHODS OF THEIR USE TO TREAT OPIOID USE DISORDER

Resumen de: US2025302751A1

Rapid-release formulations for administering Levo-alpha-acetylmethadol (LAAM), norLAAM and dinorLAMM and, optionally, magnesium, are provided. The formulations include solid i) core-shell oral dosage forms delivered in capsules or tablets, and ii) electrospun nano/microfiber buccal film dosage forms. Methods of the use of the formulations to treat opioid use disorder (OUD) and pain are also provided.

TREATMENT OF CANCER AND AUTOIMMUNE DISORDERS USING NANO POLYMERS OF HISTONE DEACETYLASE INHIBITORS

Resumen de: US2025302767A1

Provided are compositions that include a histone deacetylase inhibitor (HDACi) encapsulated in and/or otherwise associated with a nanoparticle. In some embodiments, the HDACi is romidepsin, vorinostat, belinostat, panobinostat, and/or chidamide. In some embodiments, the nanoparticle is a poly(D,L-lactide)-PEG-methyl ether (mPEG-PDLLA) nanopolymer. Also provided are methods for treating diseases, disorders, and/or conditions associated with sensitivity to histone deacetylase inhibitors, such as but not limited to tumors and/or cancers; and methods for inhibiting the growth, proliferation, and/or metastasis of a tumor and/or a cancer associated with sensitivity to histone deacetylase inhibitors by administering an effective amount of a composition as disclosed herein, which methods can optionally include administering at least one additional therapeutically active agent, such as but not limited to a chemotherapeutic agent.

IMMUNE ENGINEERING AMPLIFICATION

Resumen de: US2025302763A1

This disclosure provides methods of increasing in vivo transfection efficiency and pharmacologic activity of T cells, by administering multiple small doses within a compact time period of T cell-targeted lipid nanoparticles encapsulating mRNA encoding an antigen receptor that recognizes an antigen of a cell against which immune activity is to be directed. Also provided are methods of depleting B cells, and methods of treating B cell-mediated diseases and disorders by depleting B cells and achieving immunological reset, entailing administration of immune cell-targeted lipid nanoparticles encapsulating mRNA encoding an antigen receptor recognizing a B cell marker as multiple small doses within a compact time period. The antigen receptor can be a T cell receptor or a chimeric antigen receptor.

FORMULATIONS FOR ORAL DELIVERY OF POLYPEPTIDES, ANTIBODIES AND PROTEINS AND USES THEREOF

Resumen de: US2025302766A1

Provided is a nanoparticle comprising a composition comprising a polypeptide having a molecular weight greater than 50,000 g/mol (e.g. IgY antibodies), wherein the composition is encapsulated in a material that includes a biocompatible bioerodible polymer. Also provided is a method of preparing these nanoparticles, and use of the nanoparticles as a therapeutic to treat a disease condition.

SOLID LIPID NANOPARTICLES FOR ENCAPSULATION AND DELIVERY OF BIOACTIVE COMPOUNDS AND METHODS OF MAKING THE SAME

Resumen de: US2025302764A1

Solid lipid nanoparticles (SLNs) for delivery of bioactive compounds are disclosed. The SLNs comprise a lipid matrix and surfactant layer encapsulating at least one bioactive compound selected from vitamins, minerals, enzymes, algae-derived bioactives, proteins, peptides, amino acids, antioxidants, small synthetic molecules, plant-derived volatile compounds, or botanical extracts. The SLNs exhibit submicron particle size, low polydispersity, and sufficient surface charge to ensure colloidal stability and efficient delivery. In one embodiment, algae-based bioactives, such as phycocyanin or fucoxanthin, are encapsulated using only natural and sustainable lipids and surfactants to improve bioavailability and support environmentally friendly formulations. The SLNs may be formulated for oral, topical, transdermal, injectable, ophthalmic, mucosal, textile, veterinary, or agricultural administration. Applications include human and animal health, functional foods, skincare, nutrient supplementation, and crop treatment. The disclosed SLNs offer a biocompatible and scalable delivery system that protects sensitive compounds, enables sustained release, and enhances absorption across diverse industries.

TRIACETYL ANDROGRAPHOLIDE NANOCRYSTAL AND PREPARATION METHOD AND APPLICATION THEREOF

Resumen de: US2025302765A1

The triacetyl andrographolide nanocrystal is mainly composed of triacetyl andrographolide, a stabilizer and an excipient, and an average particle size of drug particles in a triacetyl andrographolide nanocrystal suspension obtained by redissolving the triacetyl andrographolide nanocrystal in water, is less than 500 nm, and a PDI is less than 0.2. The nanocrystal suspension is prepared from the triacetyl andrographolide and the stabilizer by a high-speed shear anti-solvent method in combination with a high-pressure homogenization method, then the excipient is added, and the nanocrystal is prepared through spray-drying.

SELF-EMBEDDING SILVER NANOPARTICLE BIOMASS WASTE COMPOSITIONS

Resumen de: US2025302769A1

Compositions and methods of making and using silver nanoparticles embedded in biomass waste matrixes of various types is described. Exemplified compositions include a silver nanoparticle embedded in a cotton gin waste nanofiber composite. Compositions and methods of making and using aerogels comprising silver nanoparticles in cotton gin waste nanofiber are described. Exemplified uses of compositions include use as antimicrobial agents.

NANOMATERIAL DELIVERY VEHICLE AND METHOD OF USE THEREOF

Resumen de: US2025302762A1

A self-assembled nanomaterial includes a Janus base nanotube, wherein the Janus base nanotube includes at least one compound represented by Formulas I to XII, or a pharmaceutically acceptable salt thereof. Also described are compositions including the Janus base nanotubes.

IN SITU READY-TO-USE INJECTION FORMULATIONS OF POSACONAZOLE FREE OF CYCLODEXTRIN AND ITS DERIVATIVES

Resumen de: US2025302824A1

The present disclosure relates to an in situ ready-to-use injection formulation of posaconazole free of cyclodextrin and derivatives of cyclodextrin, which can be formulated in situ as a nanosuspension injection of posaconazole by a simple dilution operation during clinical use. The formulation has no adverse effects on the renal function of patients, no extreme pH, and low vascular irritation, and can be administrated without the need for central venous cannulation during clinical use. The present disclosure also relates to a method for preparing the formulation and the use of the formulation in the treatment and prevention of fungal infections.

EXTRACELLULAR VESICLES FUNCTIONALIZED WITH AN ERV SYNCITIN AND USES THEREOF FOR CARGO DELIVERY

Resumen de: US2025302761A1

EVs are being recognized as vectors for drug delivery. In particular. EV loading with targeting and therapeutic agents brings along an interesting opportunity to translate EVs into a bio-mimetic selective delivery system. Indeed. EVs constitute a physiological carrier being potentially less immunogenic than artificial delivery vehicles. The inventors now developed a novel method to control the loading of a cargo into EVs on demand. These EVs are equipped, if necessary, with non-viral fusogen, therefore enhancing EV-cargo delivery into acceptor cells. To acutely measure this process, they follow the fate of a luciferase-tagged cargo. Cargo loading was enabled through a drug-reversible inducible dimerization system. Briefly, donor cells were transfected with plasmids encoding for FKBP-tagged CD63, a classical membrane EV marker, and FRB-Nanoluciferase (NLuc) that is normally cytosolic. Upon addition of the dimerizing drug. FRB-Nluc interacts with FKBP-CD63 and is recruited into secreted EVs. This is accompanied by an enhanced delivery into acceptor cells. This phenomenon can be further enhanced when EVs are equipped with syncitin1, a mammalian fusogenic protein that trigger fusion between EV membrane and the plasma membrane of acceptor cells. Using this novel process, the inventors further demonstrated that the catalytic domain of the Diphteria toxin (DTA), that is responsible for protein synthesis inhibition and ultimately cell death, can be delivered to acceptor cells via fun

NANOPARTICLE DOPED POLYETHYLENE GLYCOL BASED GELS AND MEDICAL DEVICES FOR DRUG DELIVERY

Resumen de: US2025302768A1

Provided herein are compositions, kits, and methods of making biodegradable compositions for localized drug delivery. The drug delivery compositions include one or more therapeutic agents that are dispersed within polymerized macromers of the drug delivery composition, loaded within biopolymeric nanoparticles within the drug delivery composition, or both. The release profiles of the one or more therapeutic agents are tunable based on the one or more therapeutic agents for a desired application.

Targeted Lipid Nanoparticles

Resumen de: US2025302987A1

The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention.

LIPIDS, NANOPARTICLES COMPRISING THE SAME AND USES THEREOF

Resumen de: US2025302747A1

Disclosed herein are novel lipids, lipid nanoparticlcs and their uses for the transport of therapeutic agents to a subject, or for the treatment and/or prophylaxis of diseases in the subject.

SYSTEMS AND METHODS FOR SUPER-RESOLUTION OPTICAL IMAGING TECHNOLOGIES AND/OR NANOSENSOR-DRIVEN PATIENT MONITORING AND/OR TREATMENT

Resumen de: US2025303001A1

Described herein are systems and methods for intracellular imaging, assessment, and/or treatment of tissue before, during, and/or after surgical procedures using nanoparticles (e.g., less than 50 nanometers in diameter, e.g., photoswitchable nanoparticles) and/or a super-resolution microscope system. The present disclosure describes nanoparticles (e.g., nanosensors and photoswitchable nanoparticles) that are used to monitor and/or track changes in environmental conditions and/or analytes in the cellular microenvironment before, during, and/or after surgical procedures. The present disclosure also describes systems and methods that provide information related to the distribution and/or delivery of photoswitchable nanoparticles at super resolution (e.g., using super-resolution microscopy).

ENHANCING NON-VIRAL DNA DELIVERY AND EXPRESSION

Resumen de: US2025302990A1

The present invention features method and composition that can be used to facilitate intracellular delivery of DNA to a subject. The provided methods and compositions employ a nanoparticle for intracellular DNA delivery and a type 1 interferon receptor pathway inhibitor. The type 1 interferon receptor pathway inhibitor is provided to decrease the subject's immune response that can be stimulated by the DNA.

FULVESTRANT FORMULATIONS

Resumen de: US2025302966A1

Long term storage stable fulvestrant-containing compositions are disclosed. The compositions can include fulvestrant; a solvent selected from dimethyl sulfoxide (DMSO), glycofurol, N-methyl pyrrolidone, and mixtures thereof; an oil mixture selected from a mixture of caprylic and capric triglycerides, a mixture of caprylic, capric and linoleic triglycerides, a mixture of caprylic, capric and succinic triglycerides, and a mixture of propylene glycol dicaprylate and propylene glycol dicaprate; and a sustained release member selected from benzyl benzoate, dihydrolipoic acid, benzyl alcohol and lipoic acid. The fulvestrant-containing compositions have less than about 5% total impurities, on a normalized peak area response (“PAR”) basis as determined by high performance liquid chromatography (“HPLC”) at a wavelength of 223 nm, after at least about 24 months of storage at a temperature of from about 5° C. to about 25° C.

PEPTIDES AND NANOPARTICLES FOR INTRACELLULAR DELIVERY OF MOLECULES

Resumen de: US2025302963A1

The present invention pertains to peptides and peptide-containing complexes/nanoparticles that are useful for delivering cargo molecules (such as a nucleic acid) into a cell.

DEVICES, COMPOSITIONS AND RELATED METHODS FOR ACCELERATING AND ENHANCING BONE REPAIR

Resumen de: US2025302988A1

The present invention relates to novel therapeutic nanoparticles. In particular, the present invention is directed to nanoparticles associated (e.g., complexed, conjugated, encapsulated, absorbed, adsorbed, admixed) with angiogenesis-activating-agents, methods of synthesizing the same, devices or compositions comprising such nanoparticles, as well as systems and methods utilizing the nanoparticles (e.g., in therapeutic settings for enhancing and/or activating angiogenesis at targeted tissue region).

METHODS AND COMPOSITIONS FOR AGONISING TLR3

Resumen de: US2025302975A1

Provided herein are compositions and methods for inhibiting the growth of a mammalian cancer cell growth or stimulating the immune response of a mammal, by contacting the cell or administering to the mammal an effective amount of a viral nanoparticle comprising at least one TLR agonist and a chemotherapeutic agent.

Sulfur-Containing Lipids

Resumen de: US2025304547A1

Provided herein are novel sulfur-containing lipids having a structure of Formula A or a salt thereof. The compounds may be formulated in a lipid nanoparticle for use in the delivery of charged cargo such as nucleic acids for use in the targeting of a non-liver organ, tissue or cell. Further provided are methods for making the compounds. (Formula A)

NOVEL LIPIDS BASED ON OLIGO-y-GLUTAMIC ACID DERIVATIVES AND LIPID NANOPARTICLES CONTAINING THE SAME, AND USES THEREOF

Resumen de: US2025302991A1

Provided are a novel lipid derivative compound including oligo-γ-glutamic acid, a lipid nanoparticle composition including the same, and the like. According to the present disclosure, the compound may form lipid nanoparticles by replacing PEGylated lipid, thereby preventing side effects such as anaphylaxis and exhibiting excellent in vivo stability, making it useful as a novel drug delivery system.

STING AGONIST-CONTAINING UREASE-POWERED NANOMOTOR-BASED BLADDER CANCER IMMUNOTHERAPY AGENT

Resumen de: US2025302989A1

A chitosan-heparin nanomotor and a method for producing same are disclosed. A STING agonist-encapsulated urease-based chitosan-heparin nanomotor delivers the STING agonist directly to bladder mucosal cells in the bladder, and thus can induce an immune response.

METHOD OF SYNTHESIS OF TARGETED LIPID NANOPARTICLE AND USES THEREOF

Resumen de: AU2024254671A1

The invention relates to a method for producing a lipid-based nanoparticle comprising an antigen binding domain and one or several nucleic acid molecule(s) using a mixing device, to a lipid-based nanoparticle comprising an antigen-binding domain and one or several nucleic acid molecule(s) obtainable trough such method and to uses thereof.

Therapeutic Nanomaterials

Resumen de: US2025302971A1

Disclosed herein is a delivery vehicle based on DNA-inspired Janus based nanotubes (JBNTs) for anti-viral treatment. The nanoparticles (NPs) are based the JBNTs conjugated with targeting moieties such as small molecules, aptamers, and peptides.

Targeted lipid nanoparticles

Resumen de: AU2025201939A1

The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention. The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention. ar a r h e p r e s e n t i n v e n t i o n r e l a t e s t o e n g i n e e r e d t a r g e t e d l i p i d n a n o p a r t i c l e s ( s ) c o m p r i s i n g a n u c l e i c a c i d , a n d c o m p o s i t i o n s t h e r e o f , w h e r e i n t h e s o r c o m p o s i t i o n s a r e c a p a b l e o f t r a v e r s i n g t h e b l o o d b r a i n b a r r i e r ( ) a n d d e l i v e r i n g n u c l e i c a c i d c a r g o e s t o a t a r g e t t i s s u e o r c e l l i n t h e c e n t r a l n e r v o u s s y s t e m n o n e a s p e c t , t h e i n v e n t i o n r e l a t e s t o t h e t r

FERRITIN-BOROCAPTATE SODIUM NANOCAGES FOR THE TREATMENT OF TUMORS

Resumen de: WO2025202984A1

The present invention concerns h-ferritin complexes loaded with anti-tumoral drugs for the treatment of cancer through Boron Neutron Capture Therapy.

MRNA BASED ENZYME PRECURSOR AND PREPARATION METHOD THEREOF

Resumen de: WO2025203087A1

The present disclosure discloses a recombinant construct including a vector and a recombinant nucleic acid molecule (1). The vector including at least one promoter region (13). The recombinant nucleic acid molecule (1) is encoded at least by SEQ ID No. 1. The recombinant nucleic acid molecule (1) is disposed downstream of the at least one promoter region (13) to enable transcription of the recombinant nucleic acid molecule (1) by the promoter region (13) to a plurality of messenger ribonucleic acid (mRNA) molecules encoded by SEQ ID No. 9.

RNA FORMULATION

Resumen de: WO2025202360A1

The present invention relates to aqueous RNA compositions that are suitable for storage, comprising Tris, a saccharide, and phosphate anions. The present invention also relates to methods of producing such aqueous RNA compositions, as well as their use in therapy and prevention of infectious diseases.

LIPID NANOPARTICLE LOADED WITH ANTITUMORAL AGENT AND FUNCTIONNALIZED TO TARGET IMMOSUPPRESSIVE CELLS

Resumen de: WO2025202213A1

The present invention relates to lipid nanoparticle loaded with antitumoral agent and functionalized to target immunosuppressive cells. Inventors developpe valrubicin-loaded immunoliposomes (Val-ILs). A small amount of valrubicin incorporated into Val-ILs induces leukemia cell death in vivo, suggesting that Val-ILs could be used to treat acute leukemia cells. Inventors also demonstrated that Val-ILs could reduce the risk of contamination of CD34+ hematopoietic stem cells by acute leukemia cells during autologous peripheral blood stem cell transplantation. They also highlighted the potential of Val-ILs to target immunosuppressive cell populations in the spleen. The most efficient Val-ILs were found to be those loaded with CD11b,CD223, CD64, TIM1, CD200R3, CD204, CD49b, VEGFR2 and SIGLECF antibodies. This study provides the effectiveness and ease of preparation of Val-ILs as a novel nanoparticle technology. In the context of cancers, Val-ILs have the potential to be used as a precise and effective therapy based on targeted vesicle-mediated cell death.

METHODS AND COMPOSITIONS FOR DENDRITIC CELL TARGETING VACCINES

Resumen de: AU2024250699A1

The present disclosure provides novel compounds, methods, and cell targeting mRNA vaccine formulations for targeted delivery, such as delivery to dendritic cells. The compound and formulation provided herein are designed to have a targeting moiety configured to provide selective delivery features specific for dendritic cells and a lipid tail for incorporated into the bilayer membrane of the formed lipid nanoparticle.

MUCOSAL- AND CELL-MEMBRANE PENETRATING PEPTIDES AND USES THEREOF

Resumen de: AU2024249750A1

Peptides which are capable of penetrating mucosal membranes or cell membranes are provided. In some aspects, functionalized peptide conjugates are provided. Compositions of peptide conjugates are disclosed, and methods of using such compositions are provided.

ENGINEERED NANOCOMPLEXES

Resumen de: AU2024229078A1

The present invention relates to nanocomplexes (NCs) comprising a polysaccharide nanoparticle (NP) and a hormone selected from insulin, glucagon, or glucagon-like protein-1, and uses thereof for reducing the blood glucose level, in particular, for the treatment of diabetes.

LIPID MATERIAL FOR NUCLEIC ACID DELIVERY AND USE THEREOF

Resumen de: WO2025200517A1

The present invention provides a lipid material for nucleic acid delivery, wherein the lipid material comprises a compound having structure I. The present invention also provides use of the lipid material for nucleic acid delivery in the preparation of a therapeutic drug for one or more selected from an infectious disease, a tumor disease, a congenital hereditary disease, and an immune disease. By means of the lipid material provided in the present invention and adopting a nucleic acid drug carrier strategy with high efficiency and low toxicity, a novel ionizable lipid and an auxiliary lipid material are mixed to encapsulate nucleic acid drugs, so that efficient and safe delivery of the nucleic acid drugs in vivo is achieved, and the druggability of the nucleic acid drugs is improved.

SUPEROXIDE DISMUTASE-BASED NANOSCALE TRANSDERMAL DELIVERY SYSTEM AND PREPARATION METHOD THEREFOR

Resumen de: WO2025200113A1

Provided is a superoxide dismutase-based nanoscale transdermal delivery system, which is a capsule composite structure consisting of a small molecule active ingredient, superoxide dismutase, and a polymer coating. The small molecule active ingredient is loaded in the superoxide dismutase, and the surface of the superoxide dismutase is coated with the polymer coating. The superoxide dismutase-based active ingredient transdermal delivery system has a diameter of 20-100 nm, and the polymer coating has a thickness of 7.5-52.5 nm. In the delivery system, the polymer coating protects the superoxide dismutase and the small molecule functional substance having an antioxidant effect, and the capsule composite structure is in the form of nanogel. The nanogel not only promotes the penetration depth of the delivery system, but also mitigates the stimulation of the delivery system to the skin. The delivery system features biofriendly starting materials, simple preparation method, and high yield, and thus can be produced in large scale industrially.

BIOCOMPATIBLE AND BIODEGRADABLE POLYMER NANODISCS AS LIPOPROTEIN-MIMICKING NANOCARRIERS WITH HIGH STABILITY AND LONG CIRCULATION TIME FOR DRUG DELIVERY

Resumen de: WO2025207519A1

Embodiments of the present disclosure pertain to an active agent carrier that includes a disc-shaped membrane with a plurality of self-assembled amphiphilic block copolymers encased by membrane stabilizing agents, where the amphiphilic block copolymers include hydrophilic blocks and hydrophobic blocks, and where at least one active agent is associated with the disc-shaped membrane. Tunable numbers of targeting agents may also be associated with the disc-shaped membrane. Additional embodiments pertain to methods of delivering one or more active agents to a subject by administering to the subject an active agent carrier of the present disclosure. Further embodiments pertain to methods of making an active agent carrier of the present disclosure.

CATIONIC LIPID COMPOUND, PREPARATION METHOD THEREFOR, COMPOSITION COMPRISING SAME, AND USE THEREOF

Resumen de: WO2025200270A1

A cationic lipid compound, a preparation method therefor, a composition comprising same, and a use thereof, relating to the technical field of biomedicine. The cationic lipid compound has relatively high transfection efficiency and relatively low cytotoxicity by introducing at least one protonatable polar headgroup containing a secondary or tertiary amine and at least two hydrophobic tails containing a specified number of carbons, and connecting the polar headgroup and the hydrophobic tails by means of a specific linker chain.

SCALABLE AND HIGH THROUGHPUT ASSEMBLY OF LAYER-BY-LAYER NANOPARTICLES

Resumen de: WO2025207378A1

Surface modification of nanoparticles (NPs) via the layer-by-layer (LbL) technique is a approach to generate targeted drug delivery vehicles. A simple and scalable synthesis method for LbL-NPs that can be adapted for clinical translation is of great interest. Presented herein is a robust and scalable method of polymer deposition onto nanoparticles.

NANOCOMPOSITION COMPRISING MODIFIED EXATECAN AND USE THEREOF

Resumen de: WO2025207828A1

This disclosure is directed to payload bioactive agents (PBAs) that include modified exatecans. This disclosure is also directed to bioactive compositions and pharmaceutical compositions for treating cancer. The pharmaceutical compositions comprise a polymer, a targeting bioactive agent (TBA), a PBA comprising a modified exatecan that is covalently linked to the TBA directly or indirectly, a linker that can comprise a cleavable linker, and a pharmaceutical suitable carrier. The pharmaceutical compositions can be antibody-drug conjugates (ADCs) for treating cancers, with the potential for treating tumors having negative or low (AgLow) tumor antigens.

FREEZE-DRYING COMPOSITION, METHOD FOR PRODUCING FREEZE-DRIED CELLS, AND PHARMACEUTICAL COMPOSITION

Resumen de: WO2025206286A1

The present invention addresses the problem of providing a composition for freeze-drying cells with which it is possible to suppress a decrease in survival rate or a decrease in particle size when the cells are reconstituted after freeze-drying. The composition for freeze-drying cells contains (a) a hydrophobic substance such as (a-1), (a-2), etc., and (b) nanoparticles formed from monolayers or bilayers of amphiphilic molecules, where: (a-1) is one or more hydrophobic amino acids selected from the group consisting of phenylalanine, leucine, glycine, valine, isoleucine, tryptophan, and alanine; and (a-2) is a dipeptide configured from one or two hydrophobic amino acids selected from the group consisting of phenylalanine, leucine, glycine, valine, isoleucine, tryptophan, and alanine. The composition is characterized by being used added to the cells.

ASTRAGALOSIDE NANOFORMULATION FOR TREATMENT OF HEPATITIS B AND PREPARATION METHOD THEREFOR

Resumen de: WO2025200185A1

An astragaloside nanoformulation for the treatment of hepatitis B and a preparation method therefor. The astragaloside nanoformulation for the treatment of hepatitis B comprises astragaloside and an auxiliary material. The auxiliary material comprises at least one of a polyoxyethylene-polyoxypropylene ether triblock copolymer, phospholipid, albumin, and casein. The compounding of these auxiliary materials and astragaloside can result in an astragaloside nanoformulation with good water solubility and high bioavailability, which exhibits higher safety and efficacy for the treatment of hepatitis B.

CTC RNA-LOADED NANOPARTICLES AND USE THEREOF FOR THE TREATMENT OF CANCER

Resumen de: WO2025207807A1

The disclosure provides a nanoparticle comprising a positively-charged surface and an interior comprising (i) a core and (ii) at least two nucleic acid layers, wherein each nucleic acid layer is positioned between a cationic lipid bilayer, and nucleic acid molecules in the nucleic acid layers comprise a sequence of a nucleic acid molecule expressed by a circulating tumor cell (CTC). The disclosure further provides a method of treating cancer in a subject need thereof, the method comprising administering to the subject the nanoparticle described herein, optionally wherein two or more administrations of the nanoparticle are provided, wherein each administration comprises a nanoparticle comprising RNA comprising a sequence of RNA expressed in a CTC isolated from the subject at different points in time.

INHIBITORY NUCLEIC ACIDS AND METHODS OF USE THEREOF

Resumen de: CN120359038A

The present disclosure provides inhibitory nucleic acids, compositions comprising the inhibitory nucleic acids, and methods of using the inhibitory nucleic acids to treat various diseases.

NANOPARTICLE DISPERSIONS COMPRISING THERAPEUTIC AGENTS

Resumen de: AU2023385865A1

Disclosed herein are dense nanolipid fluid (DNLF) dispersions comprising desirable characteristics for incorporating bioactive agents such as peptides into lipid phase of the dispersion for biodelivery of the agents for their typical purpose. Continuous methods for preparing the DNLF dispersions are also disclosed herein to include formation of a crude mill base and passing the base through a twin screw extruder. Dispersions disclosed herein can express a particle size of less than 150 nm under stable storage conditions, while forming lamellar structures after exposure to heat and/or evaporation of the aqueous components of the dispersion.

IONIZABLE LIPID AND USE THEREOF

Resumen de: EP4624460A1

Provided in the present invention are an ionizable lipid, and a drug delivery system containing the ionizable lipid. Specifically, provided in the present invention is an ionizable lipid having the structure of formula (I), or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof. A lipid nanoparticle constructed by means of using the ionizable lipid can realize safe and efficient delivery of nucleic acid drugs, small-molecule drugs, peptide drugs and protein drugs.

CARBAMATE-BOND-CONTAINING LIPID COMPOUNDS AND USE THEREOF

Resumen de: EP4624451A1

The present invention belongs to the field of biomedicine, which discloses a class of lipid compounds containing carbamate bond and applications thereof. The lipid compounds have the following structures:wherein the definitions of the various groups are described in the specification. The lipid nanoparticulate carriers prepared from the compounds described in the present invention exhibit excellent biocompatibility and safety as well as unexpected improvements in transfection efficiency, making them suitable for biomedical industrialization.

NOVEL TUMOR-SPECIFIC ANTIGENS FOR LYMPHOID LEUKEMIA AND USES THEREOF

Resumen de: WO2024108303A1

Lymphoid leukemia such as acute lymphoblastic leukemia (ALL) represents a devastating disease especially when it occurs in adults. While dose-intensification strategies have led to a significant improvement in outcomes for pediatric patients, prognosis for the elderly remains very poor, with only 30-40% of adult patients with ALL achieving long-term remission. Novel tumor-specific antigens (TSAs) shared by a large proportion of lymphoid leukemia cells are described herein. Most of the TSAs described herein derives from aberrantly expressed unmutated genomic sequences, such as intronic and intergenic sequences, which are not expressed in normal tissues. Nucleic acids, compositions, cells and vaccines derived from these TSAs are described. The use of the TSAs, nucleic acids, compositions, cells and vaccines for the treatment of leukemia such as lymphoid leukemia is also described.

METHODS AND AGENTS FOR TREATING NEUROTROPHIC KERATITIS

Resumen de: WO2024102746A1

Methods are described for treating neurotrophic keratitis in a subject by administering an agent that inhibits fidgetin-like 2 activity, such as by using an RNA interference agent, e.g., siRNA.

Antimicrobial composition

Resumen de: GB2639579A

An antimicrobial composition comprising an antimicrobial compound and a population of metal nanoparticles is provided. The antimicrobial compound is preferably an antibiotic. The metal nanoparticle may comprise silver and/or copper and may also comprise a polymer coating such as poly(N-vinylpyrrolidone). The antimicrobial composition wherein the population of silver nanoparticles is formed by (a) mixing a first aqueous alkaline solution with an aqueous polymer solution to form an aqueous polymer solution; and (b) mixing the aqueous alkaline polymer solution with an aqueous solution of a metal salt to form a solution of polymer-coated metal nanoparticles is provided. A coating or treatment for a surface or substrate comprising the antimicrobial composition is provided. A medical device comprising the coating or treatment is provided. The medical device may be a wound care dressing or a bandage. A silver nanoparticle coated with poly(N-vinylpyrrolidone) is exemplified. Combinations of the PVP-coated silver nanoparticles (AgNP) and various antibiotics were tested. Compositions comprising silver nanoparticles and aminoglycosides, such as kanamycin and gentamicin, were particularly effective. A combination of silver nanoparticles and ebselen was also particularly effective against E. coli and S. aureus.

COMPOSITIONS OF DRY POWDER FORMULATIONS OF MESSENGER RNA AND METHODS OF USE THEREOF

Resumen de: CN120265279A

The present disclosure provides stable dry powder messenger RNA formulations for therapeutic use and methods of making and using the same.

COMPOSITIONS AND METHODS OF USE THEREOF FOR INCREASING IMMUNE RESPONSES

Resumen de: WO2024112867A1

Nanoparticles having a calcium core, such as calcium hydroxide (Ca(OH)2) and calcium carbonate (CaCO3) particles are provided. The nanoparticles can further include a shell such as one formed of silica or oleic acid. The nanoparticles can further include a coating, such as one formed of polyethylene glycol and optionally further including a lipid. The nanoparticles can further include a targeting agent, such as one that targets dendritic cells, T cells, or other immune cells. The nanoparticles can further include or otherwise be used in combination with an active agent, optionally selected from an antigen, chemotherapeutic drug, immune system modulator, or immune checkpoint modulator. Pharmaceutical compositions including the nanoparticles and methods of use thereof for increasing immune response, e.g., against cancer and infections are also provided.

CONTROL OF NANOCAGE SELF-ASSEMBLY

Resumen de: WO2024110757A1

The invention provides constructs, pharmaceutical compositions, methods of preparing a ferritin nanocage, ferritin nanocages, methods of treating or preventing a disease in a subject, and methods of raising an immune response against an antigen. Exemplary constructs include two ferritin subunits connected by a linker, wherein the linker includes a cleavage site, wherein the linker is arranged to prevent the ferritin subunits from self-assembling into a ferritin nanocage, and wherein cleavage of the linker at the cleavage site does not prevent the ferritin subunits from self-assembling into a ferritin nanocage.

USE OF NANO-CARRIERS FOR DELIVERY OF ACTIVE AGENTS

Resumen de: US2025268840A1

There are disclosed methods of treating a subject or an object in need thereof, the method comprising administering compositions comprising nano-elements containing: a) at least one water-insoluble thermoplastic compound (WITC), capable of forming a core; and b) at least one active agent which can be disposed in said core or in shells surrounding the core. The nano-elements, having an average diameter in the sub-micron range, are constituted of materials having a low vapor pressure and are dispersible in a polar carrier. Methods for preparing these nano-elements, and administering them, so as to treat conditions corresponding to the active agents contained therein, are also provided.

NOVEL CARRIERS FOR NUCLEIC ACID AND/OR PROTEIN DELIVERY

Resumen de: WO2024110492A1

The invention relates to a carrier comprising at least one polar cationizable domain (PCD), two or more apolar cationizable domains (ACD) and at least one branching connector (BC), wherein the two or more ACDs are linked by at least one branching connector to at least one PCD, wherein the PCD is an oligo(alkylamino) acid, an ε-poly-L-lysine or an ε-poly-L-lysine-6-Ahx, and the ACD is a lipo amino fatty acid (LAF) comprising a tertiary amine. The invention further relates to nanoparticles comprising said carrier and a cargo, wherein the cargo comprises a nucleic acid and/or a protein and to a pharmaceutical composition comprising said nanoparticles and to its use in therapy or in in vitro culture.

IONIZABLE LIPIDS AND LIPID NANOPARTICLES CONTAINING THEREOF

Resumen de: CN120225501A

The present invention provides an ionizable lipid of formula (I) or a pharmaceutically acceptable salt thereof or a stereoisomer of any of them; a lipid nanoparticle comprising the ionizable lipid (in particular as an encapsulant), optionally comprising a pharmaceutically active agent; and a pharmaceutical composition comprising the lipid nanoparticles. The present invention also provides a lipid nanoparticle for a drug or a pharmaceutical composition comprising the same, and a use of the lipid nanoparticle as an encapsulant. # imgabs0 #

METHOD OF TREATING OPERATIVE COMPLICATIONS OF CATARACT SURGERY

Resumen de: MX2025011000A

The present disclosure provides a method of treating or ameliorating an operative complication of a cataract surgery. The method comprises administering nanoparticles of clobetasol propionate to a subject in need thereof.

NOVEL LIPID NANOPARTICLE FORMULATIONS FOR DELIVERY OF NUCLEIC ACIDS

Resumen de: MX2025010536A

The present invention provides novel ionizable lipids and novel lipid nanoparticles comprising messenger RNA (mRNA) useful for the delivery of nucleic acids, related pharmaceutical compositions or vaccines as defined herein for use in human or veterinary medicine, in particular for use in the treatment and/or prophylaxis of cancer diseases.

C6'-SUBSTITUTED LOCKED NUCLEIC ACID-MODIFIED CAP ANALOG AND USE THEREOF

Resumen de: EP4624462A2

The present disclosure provides a C6'-substituted locked nucleic acid-modified cap analog and a use thereof, wherein the cap analog improves the stability of mRNA and/or the translation efficiency of mRNA.

NOVEL LIPIDS BASED ON OLIGO-Y-GLUTAMIC ACID DERIVATIVES AND LIPID NANOPARTICLES CONTAINING THE SAME, AND USES THEREOF

Resumen de: EP4624516A1

Provided are a novel lipid derivative compound including oligo-γ-glutamic acid, a lipid nanoparticle composition including the same, and the like. According to the present disclosure, the compound may form lipid nanoparticles by replacing PEGylated lipid, thereby preventing side effects such as anaphylaxis and exhibiting excellent in vivo stability.

EFFICIENT TRANSDERMAL DELIVERY SYSTEM FOR ACIDIC GROUP-CONTAINING BIOMATERIAL

Resumen de: EP4623901A1

Provided is an efficient transdermal delivery system based on an acidic group-containing biomaterial produced by bonding or physically compounding a tertiary amine oxide group-containing polymer to an acidic group-containing biomaterial or an acidic group-containing biomaterial nanogel. The efficient transdermal delivery system does not require a subcutaneous injection. After being smeared or coated on a skin, the transdermal delivery system can effectively penetrate through the stratum corneum of the skin and enter the subcutaneous layers to exert prominent medical aesthetic effects such as wrinkle and fold correction, or to achieve the transdermal delivery of heparin for thrombolysis, or to achieve the delivery of a drug.

COMPOSITIONS AND METHODS FOR TREATING HYPERPROCALCITONEMIA

Resumen de: MX2025008776A

A composition for treating hyperprocalcitonemia is described. The composition comprises a lipophilic or hydrophobic component, an amphiphilic emulsifier, a polar liquid carrier, and with or without one or more electrolytes, where the amphiphilic emulsifier forms micelles having a lipophilic or hydrophobic core comprising the lipophilic or hydrophobic component in the polar liquid carrier, and /or liposomes organized as a lipid bilayer and/or other particle configurations.

TARGETED DELIVERY OF GENE EDITING CONSTRUCTS AND METHODS OF USE THEREOF

Resumen de: CN120390657A

The present invention relates to compositions for efficient delivery of gene editing agents to target cells, and methods of using the same to treat diseases or conditions.

CANCER CYTOTOXIC EXOSOME FORMULATIONS AND METHODS FOR USE IN TREATING CANCER

B5 Novel lipid based on provitamin B5 and lipid nanoparticles containing the same and their uses

Resumen de: KR20250142801A

본 발명은 프로비타민 B5기반의 신규한 지질 유도체 화합물과, 이를 포함하는 지질 나노입자 조성물 등에 관한 것이다. 보다 구체적으로는, 신규한 프로비타민 B5 기반의 지질 화합물이 지질 나노입자의 형성에 있어 이온화지질의 역할을 수행하고 안정적인 지질 나노입자를 형성하여 mRNA등의 약학적 유효성분을 체내로 전달하는 데 이용될 수 있다.

Manufacturing method for self-assembly manganese dioxide nanodrug using drugs interactive with metal ions

Resumen de: KR20250142187A

본 발명은 금속이온과 상호작용이 있는 약물을 사용한 자가조립형 이산화망간 나노약물의 제조 방법에 관한 것으로, 본 발명에 따른 이산화망간 나노약물(Drug/MnO2)은 암세포 및 염증성 세포의 미세환경 조건인 높은 활성산소(Reactive Oxygen Species, ROS, 0.1-1 mM), 낮은 pH(pH 5.5-6.8)에서 분해되어 약물을 방출할 수 있고, 분해 산물인 Mn2+ 이온은 MRI(자기공명영상) T1 조영제로 사용 가능하며, 발생된 산소는 저산소 환경을 개선하여 각종 치료 효율을 증가시킬 수 있다.

지질 나노입자에 접합된 HSC-특이적 항체 및 이의 용도

Resumen de: AU2024214423A1

Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., HSC), and methods of making and using such lipids and targeted lipid. nanoparticles.

Composition for preventing treating or alleviating intestinal permeability syndrome comprising nanoparticles of certain size as an active ingredient

Resumen de: KR20250142140A

본 발명은 특정 크기의 나노입자를 유효성분으로 포함하는 장누수증후군 예방, 치료, 또는 개선용 조성물에 관한 것으로서, 본 발명에 따른 특정 크기를 가지는 나노입자는 비스테로이드성 항염제(Nonsteroidal anti-inflammatory drugs, NSAIDs)를 처리하여 장누수증후군을 모사한 세포 및 동물모델에서 장누수로 인해 증가된 장 투과성을 억제 및 감소시킬 수 있다. 또한, 장누수증후군을 동반하는 비알코올성 지방간 질환 동물모델에서 장 투과성을 억제 및 감소시킴에 따라 간기능을 회복하는 효과가 있다. 이에, 본 발명에 따른 특정 크기의 나노입자는 장누수증후군, 비스테로이드성 항염제 치료의 부작용으로 발생하는 장누수증후군, 및 장누수증후군을 동반한 질환의 예방, 치료, 또는 개선용 조성물 등의 제조에 유용하게 이용될 것으로 기대된다.

SENP SENPDS SENP and SENPDS manufactured from active ingredients and method for manufacturing the same

Resumen de: KR20250142185A

본 발명은 활성성분을 포집한 나노입자(Nanoparticles, NP)와 자가유화 용액(Self-emulsifying solution, SES)으로부터 제조된 자가유화 나노입자(Self-emulsifying nanoparticle, SENP) 및 자가유화 나노입자 운반시스템(Self-emulsifying nanoparticle delivery system, SENPDS)을 제공한다. 경구 투여된 SENPDS는 위장관에서 수성 매체에 희석되면서 형성되는 NP 함유 지질방울은 전신 순환계에 유입되고 체류하면서 활성성분을 지속 방출할 수 있다. 이 과정에서 상기 온전한 NP 함유 지질방울은 자가소수성 반전 및 자가전하 반전을 포함하는 생체장벽 회피 전략으로 활성성분의 생체이용률이 향상됨을 확인하였다. 따라서, 본 발명의 SENPDS는 생체이용률이 향상된 의약품, 건강기능식품 및 화장품을 제공할 수 있다.

CELL TARGETED POLYMER SELF-ASSEMBLY NANOMICELLES AND METHOD OF PREPARING THE SAME

Resumen de: WO2025198324A1

The present invention relates to a polymer self-assembly nanocarrier and a method of preparing same, the nanocarrier binding a peptide that targets a specific cell and, simultaneously, carrying an active ingredient that has been verified. The present invention can exhibit various effects such as a moisturizing effect, an increase in filaggrin production, whitening, skin barrier reinforcement and the like by selectively and efficiently delivering a drug through a targeting peptide, can be applied to skin diseases such as atopy or psoriasis, and is expected to enable the maximum effect of an active substance in the pharmaceutical and cosmetic fields to be obtained.

精製ヒトＲＮＡ編集酵素を使用するための組成物および方法

Resumen de: US2025101427A1

In alternative embodiments, provided are methods for eradicating or reducing the in vivo numbers of cancer stem cells comprising administering to an individual in need thereof an ADAR1 (adenosine deaminase associated with RNA1) inhibiting agent, wherein the ADAR1 inhibiting agent reduces, or significantly reduces, ADAR1 Nano-luc reporter activity in cell lines and in human cancer stem cell assays. In alternative embodiments, provided are methods for inhibiting an RNA virus or a retrovirus, optionally a SARs-COV-2 virus, comprising lentiviral ADAR1 overexpression and in vivo administration, optionally intravenous (IV) administration, of a lentiviral ADAR1 transduced stem cell, optionally the stem cell is a cord blood CD34+ cell or a mesenchymal stromal cell.

ファブリー病の処置のための方法及び組成物

Resumen de: MX2021008131A

The present disclosure provides expression constructs comprising a GLA transgene encoding the at least one α-Gal A protein for use in expressing α-Gal A proteins and preventing, inhibiting or treating Fabry disease or one or more symptoms associated with Fabry disease.

SENP SENPDS SENP and SENPDS manufactured from active ingredients and method for manufacturing the same

Resumen de: KR20250142178A

본 발명은 활성성분을 포함하는 균질하고 견고한 폴리머 복합체 나노입자(Polymer Complexes Nanoparticles, PMNP)와 자가유화 용액(Self-emulsifying solution, SES)로부터 자가유화 나노입자(Self-emulsifying nanoparticle, SENP) 및 비침습적 자가유화 나노입자 운반시스템(Self-emulsifying nanoparticle delivery system, SENPDS)을 제안하였다. 본 발명의 상기 SENPDS는 3중 다층 구조체로 생체 내에서 견고한 PMNP 함유 오일방울로 전환하여 자가소수성 반전 및 자가전하 반전을 하여 기존 SNEDDS(Self-nanoemulsifying drug delivery system)와 다른 생체장벽 회피 전략으로 향상된 생체이용률을 확인하였다. 본 발명은 SENP, SENPDS 및 이들의 제조 방법에 관한 것이다.

글리코겐 합성효소 키나제-3 활성의 조절을 위한 리튬으로 기능화되고 글루타티온-코팅된 금 나노입자(LiG-AuNPs)의 용도

Resumen de: WO2024165949A1

The present invention relates to a method for the production of gold nanoparticles (AuNPs) coated with glutathione and Li+ ions, hereinafter designated as LiG-AuNPs, to a method for the preparation of aggregates of said nanoparticles and to the use of said nanoparticles, aggregates or compositions thereof which comprise them for therapeutic use. LiG-AuNPs then are an effective instrument in inhibiting GSK-3 and its downstream molecular targets, while keeping the lithium extracellular concentration levels below the systemic toxicity threshold (1.5 mEq/L), and exerting an antioxidant action by means of the glutathione present on their surface.

결핍제한 면역반응 치료를 위한 miRNA 기반 입자

Resumen de: AU2024207086A1

A miRNA-mimic based therapeutic particle is disclosed herein. The particles comprise a synthetic miRNA or mimic of miR-187-3p encapsulated in a lipid nanoparticle (LNP) carrier or synthetic miR-193b-5p inhibitor encapsulated in a lipid carrier or their combination encapsulated in a lipid carrier. The lipid nanoparticle carrier is made up of at least four (4) types of lipids, in which the four (4) types of lipids include a) an ionizable cationic lipid selected to be positively charged in a formulation buffer (pH 4), which binds and protects the negatively charged miRNA, and facilitates endosomal escape, and is neutral in a storage buffer, b) a sterol in the structure of the lipid nanoparticle (LNP)., c) a structural helper lipid selected to contribute to lipid nanoparticle stability and/or enhances endosomal release, and d) a PEGylated-lipid selected such that it stabilizes the therapeutic particle and protects it from opsonization.

二硫键桥联的杂环叔胺-紫杉醇前药及其纳米粒的制备方法和应用

Resumen de: CN120717975A

本发明属于药物制剂技术领域，涉及一种二硫键桥联的杂环叔胺‑紫杉醇前药及其纳米粒的制备方法和应用。一种二硫键桥联的杂环叔胺‑紫杉醇前药，前药为式一所示杂环叔胺‑前药或其药学上可接受的盐，本发明二硫键桥联的杂环叔胺‑紫杉醇前药解决疏水药物溶解性差、改善疏水药物利用的表面活性剂容易引起的安全性问题，所述应用二硫键特异性响应释放增加药物选择性和安全性、利用杂环叔胺增加药物自组装能力、组装成纳米粒后通过EPR效应增加药物在肿瘤部位的蓄积、利用杂环叔胺结构具有pH响应的特点并在溶酶体中引起质子海绵效应以提高药物的细胞内递送效率；进而本发明前药及其自组装纳米粒。

一种多糖修饰的锰-没食子酸配合物及其制备和应用

Resumen de: CN120713849A

本发明公开了一种多糖修饰的锰‑没食子酸配合物及其制备和应用，属于金属多酚配合物和医药技术领域。本发明选用锰离子与没食子酸通过直接自组装来制备锰‑没食子酸的配合物，并通过逐层修饰技术引入聚醚表面活性剂和多糖(透明质酸)，聚醚表面活性剂和透明质酸包覆于配合物表面，最终形成了具有类芬顿活性、能够用于治疗肿瘤的多糖修饰的锰‑没食子酸配合物；其中，喜树碱与锰‑没食子酸的配合物协同发挥更强的治疗效果；聚醚表面活性剂的引入有助于促进组分溶解，还能提高稳定性，更容易透过细胞膜被细胞吸收；透明质酸的引入有助于提高生物相容性和对肿瘤细胞的靶向作用。

一种类淀粉样白蛋白涂层修饰的Fe3O4纳米颗粒的制备方法及其应用

Resumen de: CN118873637A

The invention discloses a preparation method of an amyloid-like albumin coating modified Fe3O4 nanoparticle and application of the amyloid-like albumin coating modified Fe3O4 nanoparticle in anti-immune clearance, and relates to the technical field of nano-medicines. The preparation method comprises the following steps: S1, performing amyloid-like transformation on human albumin HSA to prepare an amyloid-like albumin ALH solution; and S2, modifying the amyloid-like albumin solution prepared in the step S1 on the surface of a Fe3O4 nano particle to prepare the nano particle ALH-Fe3O4 nano particle modified by the amyloid-like albumin coating. According to the preparation method of the Fe3O4 nanoparticles modified by the amyloid-like albumin coating and the application of the Fe3O4 nanoparticles modified by the amyloid-like albumin coating in anti-immune clearance, the albumin is subjected to amyloid-like transformation, so that the albumin is quickly self-assembled on the surfaces of the Fe3O4 nanoparticles to realize stable film formation, and meanwhile, the antiagonist performance of the albumin is maintained; the modification method is rapid in operation, wide in application range, free of organic solvents in the preparation process, environmentally friendly and suitable for large-scale commercial production.

一种PLGA封装GE11肽偶联亲水型酞菁纳米光敏剂及其制备方法和应用

Resumen de: CN120713860A

本发明涉及纳米光敏剂以及生物技术领域，尤其涉及一种PLGA封装GE11肽偶联亲水型酞菁纳米光敏剂及其制备方法和应用，包括：制备PLGA预聚物；制备ZnPcS光敏剂，将其磺酰氯化，得到ZnPcS-Cl活性端基；ZnPcS-Cl活性端基与GE11肽的胺基亲核取代，制备GE11-ZnPcS靶向偶联光敏剂；PLGA预聚物与GE11-ZnPcS光敏剂溶于油相体系，缓慢加入水相体系，在超声合成仪下，水包油乳化，得到PLGA@GE11-ZnPcS纳米光敏剂。本发明的PLGA@GE11-ZnPcS纳米光敏剂，利用680nm红光光照，应用于光动力杀灭人表皮鳞状皮肤癌细胞，具有光动力治疗效果。

一种经过工程化改造的通用型铁蛋白载体疫苗及其应用

Resumen de: CN120718133A

本发明公开了一种经过工程化改造的通用型铁蛋白载体疫苗及其应用。本发明公开了一种铁蛋白适配器、人铁蛋白重链突变体、通用型铁蛋白载体系统、抗原融合蛋白和铁蛋白载体疫苗，以及编码它们的核酸、含其的重组表达载体和转化体，以及它们的制备方法及应用。本发明首次明确了NCOA4(residue 484‑499)为FTH1的真实结合表位。设计出亲和力显著提高的Peptide及FTH1的突变体。基于本发明通用型铁蛋白载体系统的狂犬纳米颗粒疫苗，稳定性更高，能诱导更广泛、持久、强烈的免疫应答。2剂次免疫接种能够产生100％的保护效率，比市售的标准灭活疫苗BRP具备更佳的预防效果。

基于脂质体、外泌体与金属有机框架协同靶向治疗脂肪肝的纳米药物递送系统及其制备方法

Resumen de: CN120713866A

本发明提供基于脂质体、外泌体与金属有机框架协同靶向治疗脂肪肝的纳米药物递送系统及其制备方法，涉及新型纳米材料制备技术领域，纳米药物递送系统包括：脂质体、间充质干细胞外泌体和金属有机框架纳米颗粒；脂质体为载体，包载具有缓释特性的金属有机框架纳米颗粒、间充质干细胞外泌体和miR‑204‑3p。通过整合脂质体、外泌体和金属有机框架的优势，实现对MASLD的协同治疗。该系统采用脂质体作为智能载体，包载具有缓释特性的MOF纳米颗粒和间充质干细胞外泌体，同时负载治疗性miR‑204‑3p。MOF材料兼具药物控释和活性氧清除功能，与外泌体及miRNA协同发挥抗氧化、抗炎和抗纤维化作用。该系统具有pH响应特性，能在MASLD病灶的酸性微环境中智能释放药物，实现肝脏靶向治疗。

一种复合脂质化合物及其应用

Resumen de: CN120717973A

本发明提供一种通式A、通式B、通式C、通式D、通式E、通式F、通式B‑1、通式H、通式I、通式J、通式K或通式L所示的复合脂质化合物及其应用。该复合脂质化合物易于与核酸及蛋白等分子结合且易降解。本发明丰富了脂质化合物的种类，以其开发的递送载体对核酸及蛋白等多种分子的包封效率高，能够将药物在体内体外高效递送并高效表达，从而为核酸等药物递送提供了更多选择，对核酸类等药物的发展和应用具有重要的意义。

一种微管蛋白破坏剂二聚体与铜死亡药物联用的纳米颗粒、其制备方法及用途

Resumen de: CN120713867A

本发明提供了一种微管蛋白破坏剂二聚体与铜死亡药物联用的纳米颗粒、其制备方法及用途，属于生物医药领域。本发明提供了一种微管蛋白破坏剂二聚体与铜死亡药物联用的纳米颗粒。所述微管蛋白破坏剂通过破坏细胞微管结构，诱导氧化应激及血流阻断，增强肿瘤细胞对氧化损伤的敏感性；铜死亡药物则促进铜离子积累，引发线粒体蛋白质聚集与功能障碍，最终诱导细胞死亡。两类药物联用，可产生显著协同抗肿瘤效应，显著提高治疗效率；同时，将这两类药物制备成纳米颗粒后用于抗肿瘤的选择性指数好，对细胞的毒副作用小。本发明具有广阔的临床转化前景和重要的社会经济价值。

甲硫氨酸腺苷转移酶2A及其相关物质在制备抗衰产品中的应用

Resumen de: CN120714037A

本发明涉及生物医药技术领域，公开了甲硫氨酸腺苷转移酶2A及其相关物质在制备抗衰产品中的应用。本发明发现甲硫氨酸腺苷转移酶2A(MAT2A)可以通过HMGCS1介导的泛醌合成，诱导线粒体代谢脆性，从而在调节细胞衰老中发挥之前未报道的代谢兼职作用。而在周细胞中特异性提高MAT2A基因表达量有助于提高细胞增殖活性、细胞线粒体呼吸水平、细胞的耗氧率、细胞总ATP水平和核形态标志物LaminB1的表达水平，同时降低衰老细胞百分比和衰老标志物P21的表达水平，提高甲硫氨酸腺苷转移酶2A表达的相关物质可用于制备抗衰药物。

甲硫氨酸腺苷转移酶2A及其相关物质在制备创面修复产品中的应用

Resumen de: CN120714036A

本发明涉及生物医药技术领域，公开了甲硫氨酸腺苷转移酶2A及其相关物质在制备创面修复产品中的应用。本发明通过研究糖尿病创面中的代谢调控网络，发现在周细胞中下调的MAT2A表达与M1型巨噬细胞浸润呈显著负相关，并观察到伤口中周细胞的MAT2A特异性缺失会导致伤口愈合延迟、创面血流灌注减少，并伴有炎症巨噬细胞浸润的增加，而进一步采用负载saMAT2A的周细胞膜包被脂质纳米粒（PMCNPs）进行治疗后，能够显著增强创面的再生过程、减轻糖尿病创面中持续的炎症巨噬细胞浸润，并促进微循环的恢复，且安全性高，在促进创面修复方面具有很好的应用前景。

一种基于受体-配体作用力组装的二维仿生纳米结构及其制备方法

Resumen de: CN120713864A

本发明公开了一种基于受体‑配体作用力组装的二维仿生纳米结构及其制备方法。所述二维仿生纳米结构的核心特征是通过受体‑配体作用将配体分子修饰的超微颗粒锚定于表面表达有相应受体的细胞膜碎片表面，实现超微颗粒与细胞膜的均匀复合，突破了传统细胞膜完全包裹技术中因小尺寸颗粒的高表面曲率导致的复合效率低、功能位点掩蔽等瓶颈。本发明的制备方法使细胞膜与超微颗粒之间具有更强的结合力，可避免超微颗粒在体内循环过程中的脱靶现象，但可实现在高丰度受体表达的病变区受竞争作用而响应释放，在精准药物递送方面具有良好的应用前景。同时，膜负载延长了超微颗粒的体内循环时间，提高了其生物相亲性，确保了药物递送系统的有效性及低毒性。

功能改性的美登木素生物碱及其组合物和使用方法

Resumen de: AU2024203271A1

FUNCTIONALLY MODIFIED MAYTANSINOIDS AND COMPOSITIONS AND METHODS OF USE THEREOF Radiosensitizer prodrugs and formulations and methods of use thereof are provided. Typically, the radiosensitizer prodrug is an analog of a radiosensitizer parent compound having one or more S-nitrosothiol moieties. Typically, the S-N bond of the S-nitrosothiol moiety can be cleaved by radiation during radiotherapy, releasing the parent compound and nitric oxide. One or preferably both the parent compound and the nitric oxide can contribute to death of tumor cells exposed to radiotherapy. Nanoparticle formulations for delivery of the prodrug, and methods of using them in combination with radiotherapy to treat tumors and cancer are also provided.

用于递送的脂质化合物和脂质纳米颗粒

Resumen de: CN120718086A

本发明涉及一种用于递送的脂质化合物和脂质纳米颗粒，公开了一种化合物，结构式如式I所示，以及其药学上可接受的盐或其立体异构体，本申请还公开了一种包含上述化合物或其药学上可接受的盐或其立体异构体的纳米颗粒组合物，本申请的纳米颗粒可以高效递送药物、疫苗至细胞内，发挥药物、疫苗的治疗或预防目的。

一种“抗菌协同-矿化调控”双功能纳米材料及其制备方法与应用

Resumen de: CN120713863A

本发明提供了一种“抗菌协同‑矿化调控”双功能纳米材料及其制备方法与应用，属于生物医用材料技术领域。本发明首先将磷酸氢二钠溶液和氯化钙溶液混合，得到无定形磷酸钙悬浮液；再将无定形磷酸钙悬浮液和单宁酸溶液混合，得到ACP@TA悬浮液；最后将ACP@TA悬浮液和羟丙基三甲基氯化铵壳聚糖溶液混合，得到双功能纳米材料。通过单宁酸与抗菌层羟丙基三甲基氯化铵壳聚糖的静电吸附作用形成交联外壳，将无定形磷酸钙有效包裹，不仅能够显著延缓无定形磷酸钙的结晶进程，确保长效的钙磷离子释放，还能有效发挥抗菌功能，为牙体硬组织的再矿化提供理想的局部微环境，最终实现牙釉质的仿生矿化及牙本质小管的深度矿化封堵。

一种具有诱导铁死亡和递送核酸药物功能的新型载体的制备及在肿瘤治疗中的应用

Resumen de: CN120713861A

本发明公开了一种具有诱导铁死亡和递送核酸药物功能的新型载体及在肿瘤治疗中的应用。本发明所述青蒿琥酯‑鱼精蛋白偶联物载体通过酰胺键将青蒿琥酯与鱼精蛋白偶联。本发明制备的青蒿琥酯‑鱼精蛋白偶联物载体具有成分简单、毒副作用小的特点，可诱导肿瘤细胞铁死亡，同时具有递送核酸的功能，可用于抗肿瘤领域研究中。

一种3-溴丙酮酸离子液体脂质纳米粒及其制备方法和应用

Resumen de: CN120713865A

本发明公开了一种3‑溴丙酮酸离子液体脂质纳米粒及其制备方法和应用，属于肿瘤治疗技术领域，该离子液体脂质纳米粒由3‑溴丙酮酸、阳离子脂质和第一溶剂组成，将其应用于制备治疗肿瘤的药物中。本发明采用上述的一种3‑溴丙酮酸离子液体脂质纳米粒及其制备方法和应用，离子液体脂质纳米粒BPTP提高了3‑BP的生物利用度，增强射频消融，实现了肿瘤糖酵解代谢抑制疗法和射频热消融的精准协同，远优于射频消融或3‑BP单一疗法的抗肿瘤功效，有效抑制肿瘤复发与转移。

腫瘍病変の光音響画像化及び光熱療法のための生体材料被覆ナノ構造

Resumen de: WO2024068100A1

Metal based nanoparticles coated with a polymer functionalized with thiol groups and 5 -NH groups, said polymer preferably being selected among thiolated chitosan, thiolated and aminated alginic acid, thiolated and aminated hyaluronic acid, or a protein preferably selected from the group consisting of albumin and gelatin, or a synthetic thiolated and aminated polymer, preferably α-thio-ω-amino polyethylene glycols; wherein said groups are linked to a ligand of the integrin family receptors, 10 preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a heterobifunctional crosslinker, the crosslinker bearing functional groups able to bind to amino groups, preferably selected from the group consisting of N-hydroxysuccinimidyl ester group (NHS ester), an isocyanate group (-NCO), an isothiocyanate group (-NCS), a Sulfo-N-15 hydroxysuccinimidyl ester group (sulfo-NHS ester), or a carboxylic acid group which is connected by activation with carbodiimide coupling agents; and/or a functional group able to bind thiol groups, preferably selected from the group consisting of: a maleimide group, a terminal vinyl group or a terminal alkyne group; and/or functional groups able to bind alkynes such as azides; and/or functional groups able 20 to bind azides, such as alkynes.

神経筋障害の治療のための組成物及び方法

Resumen de: TW202424194A

The present disclosure relates to adeno-associated virus (AAV)-mediated delivery of nucleic acids for treating neuromuscular disorders, e.g., X-linked myotubular myopathy (XLMTM), in patients in need thereof. The AAV vectors of the disclosure may include, e.g., a transgene encoding a myotubularin protein operably linked to one or more transcription regulatory elements. In some embodiments, the one or more transcription regulatory elements are specifically active in muscle and/or liver tissue.

核酸複合体及びその使用

Resumen de: WO2024068674A1

The present disclosure provides complexes and methods of use. In some embodiments, a complex described herein is a complex comprising a cationic polymer, an anionic polymer, and a monomeric RNA molecule, wherein the cationic polymer and the monomeric RNA molecule form a core complex encapsulated by the anionic polymer. In some embodiments, a complex comprises a linear cationic polymer, an anionic polymer, and a monomeric RNA molecule, wherein the cationic polymer and the monomeric RNA molecule form a core complex encapsulated by the anionic polymer.

면역치료를 위한 항원-포획 나노입자들 및 제형들

Resumen de: AU2024219072A1

The present invention provides novel antigen-capturing nano-particles (ACNPs) and pharmaceutical formulations containing such ACNPs. Also provided herein are methods for preparing the ACNP formulations, and methods for the treatment of a disease (such as cancer) in a subject with the ACPNs or a pharmaceutical formulation containing the ACPNs.

화합물 또는 그 염, 지질 조성물, 의약 조성물 및 송달 캐리어

Resumen de: WO2024190817A1

The present invention addresses the problem of providing: a compound, or a salt thereof, which constitutes a lipid composition that can realize a high nucleic acid encapsulation rate and excellent nucleic acid delivery when mRNA is used; and a lipid composition, a pharmaceutical composition, and a delivery carrier that can realize a high nucleic acid encapsulation rate and excellent nucleic acid delivery when mRNA is used. The present invention provides a compound represented by formula (1), or a salt thereof. In the formula, R1 represents a hydrocarbon group having 1-24 carbon atoms; R2 represents a hydrocarbon group having 1-6 carbon atoms, R3 represents a hydrocarbon group having 6-24 carbon atoms and having a branched chain; R4 and R5 each represents a hydrocarbon group having 1-6 carbon atoms which are optionally substituted; R6 represents a hydrogen atom or a hydrocarbon group having 1-18 carbon atoms; L represents *-O-C(O)- or *-C(O)O -; a represents an integer of 1-12; and b, c, and d each represents an integer of 1-4.

一种具有解毒功能的黄芩苷酵母微胶囊及其制备方法和应用

Resumen de: CN120694967A

本发明属于生物制药领域，具体涉及一种具有解毒功能的黄芩苷酵母微胶囊及其制备方法和应用。通过薄膜水化和酸碱‑有机制备出搭载黄芩苷的阳离子脂质体及酵母细胞壁，两者静电自沉积形成黄芩苷酵母微胶囊。实验表明本发明所述的黄芩苷酵母微胶囊可通过改善肾脏纤维化、机体炎症反应及促进毒素排出等途径改善了毒素对机体的损伤。本发明为黄芩苷的低生物利用度提供了一种纳米载体的改善策略，为黄芩苷改善赭曲霉毒素A造成的肾损伤提供科学依据。

一种用于肾损伤的纳米硒制剂及其制备方法和应用

Resumen de: CN120694969A

本发明涉及一种用于肾损伤的纳米硒制剂及其制备方法和应用，所述纳米硒制剂包括芯和壳，所述芯包括硒，所述壳包括二硬脂酰基磷脂酰乙醇胺‑聚乙二醇，所述壳包裹于所述芯的表面。制备方法包括如下步骤：包裹：将芯的原料、壳的原料分别溶于溶剂，得到芯材溶液、壳材溶液；将芯材溶液与壳材溶液混合，使壳的原料包裹于芯的原料的表面，得到含有纳米硒制剂前体的溶液；制备用于肾损伤的纳米硒制剂：将含有纳米硒制剂前体的溶液蒸干，加水重悬，得到用于肾损伤的纳米硒制剂。本发明的纳米硒制剂对AKI有较好的保护作用，具有良好的肾靶向性和生物安全性，能有效改善肾功能，减少肾组织病理学损伤，显著减轻线粒体损伤和细胞凋亡，具有剂量依赖性。

一种透明质酸-柚皮素自组装纳米颗粒的制备方法及应用

Resumen de: CN120694957A

本发明公开了一种抗肿瘤的透明质酸‑柚皮素自组装纳米颗粒的制备方法，属于医药领域。该方法通过透明质酸上的羧基与柚皮素上的羟基结合形成酯基，得到透明质酸‑柚皮素聚合物来进行。亲水性的透明质酸结合疏水性的柚皮素，形成亲疏水性聚合物，然后在水溶液中通过超声波的作用，疏水端排斥水分子而相互聚集，亲水端便包裹在疏水端外围，与水接触，从而形成内核是疏水性的柚皮素，外核是亲水性的透明质酸的纳米粒子。该纳米粒子的粒径为140~200nm，粒子稳定、均匀，体外释放实验结果说明能缓慢释放，细胞实验显示制剂能明显提高柚皮素对肿瘤细胞的毒性，说明其有良好的抗肿瘤活性。

一种负载乏氧前药的上转换纳米颗粒的制备方法与应用

Resumen de: CN120694966A

本发明涉及一种负载乏氧前药的上转换纳米颗粒的制备方法与应用。纳米颗粒由基于上转换纳米颗粒的纳米晶体与有机光敏剂复合，负载甘草酸和乏氧前药后组成。制备方法包含上转换纳米颗粒的制备，有机光敏剂的复合，甘草酸和乏氧前药的负载。本发明制备方法简单，能够有效负载光敏剂和乏氧前药，在808nm近红外光照射下具有高效的光动力治疗性能。甘草酸使纳米颗粒功能化靶向线粒体，光动力治疗引起的乏氧环境激活乏氧前药，引起肿瘤细胞的DNA损伤。进一步引起肿瘤免疫原性细胞死亡，增敏免疫检查点抑制剂的肿瘤免疫治疗疗效，为肿瘤免疫治疗提供新策略。

REACTIVE OXYGEN SPECIES-RESPONSIVE DRUG DELIVERY PARTICLES WOUND HEALING METHOD USING REACTIVE OXYGEN SPECIES-RESPONSIVE DRUG DELIVERY PARTICLES AND Photobiomodulation AND DEVICE FOR WOUND HEALING

Resumen de: US2025295780A1

Disclosed are reactive oxygen species-responsive drug delivery particles including: a ferrocene particle containing a polymer containing ferrocene; and a reactive oxygen species-scavenging drug loaded in the ferrocene particle, wherein the ferrocene particle contains ferrocenylmethyl methacrylate, polyethylene glycol methacrylate bound to the ferrocenylmethyl methacrylate, and methacrylic acid bound to the polyethylene glycol methacrylate, the ferrocenylmethyl methacrylate, the polyethylene glycol methacrylate, and the methacrylic acid having a molar ratio of 1:1:1 to 1:5:10.

脂质化合物、脂质纳米颗粒和药物组合物

Resumen de: CN120698908A

本申请提供了脂质化合物，例如式(I)的化合物。还提供了脂质纳米颗粒和药物组合物，其各自包含脂质化合物，例如下式的化合物。

一种植物来源的挤压纳米囊泡的制备方法和应用

Resumen de: CN120699882A

本发明公开了一种植物来源的挤压纳米囊泡的制备方法和应用，涉及生物医药技术领域。植物来源的挤压纳米囊泡的制备方法包括以下步骤：取洗净的植物根、茎、叶、花或果实榨汁得到植物汁液，用纱布过滤得到第一滤液；将第一滤液经过低速300‑6,000 ×g离心得第二滤液；将第二滤液于0‑5℃下依次经过8,000‑20,000 ×g离心0.5‑3 h，得到第一沉淀；将第一沉淀加至缓冲液中，超声，稀释，依次通过孔径0.4‑10 μm和孔径0.1‑0.4 μm的滤膜，得到第三滤液；将第三滤液离心，取沉淀重悬，得到挤压纳米囊泡。经实验检测，发现上述挤压囊泡特征与天然囊泡相似，但产量远远高于天然囊泡，可作为纳米载体应用于基础或临床。

自组装纳米颗粒及制备方法和应用、外泌体包裹的纳米材料及应用

Resumen de: CN120694984A

本发明公开了一种自组装纳米颗粒及制备方法和应用、包含自组装纳米颗粒的外泌体包裹的纳米材料及应用，其中自组装纳米颗粒包括五味子酚和绿原酸，所述五味子酚和绿原酸混合形成纳米颗粒。本申请通过五味子酚和绿原酸自组装形成纳米颗粒，自组装纳米颗粒具备优异的抗氧化性能，自组装纳米颗粒被外泌体包裹形成纳米材料，纳米材料能够治疗肝损伤，促进特异性免疫耐受的形成。

可用于增溶膜蛋白的脂质纳米盘

Resumen de: AU2024209122A1

Provided herein are composition including lipids and copolymers in the form of a nanodisc assembly. The copolymers include monomer units of methacrylic acid and styrene. Also provided herein is an aqueous solution comprising the subject composition, methods of producing the nanodisc assembly. Further provided are methods of solubilising hydrophobic constituents such as membrane proteins in aqueous solution, including forming nanodisc assemblies of a lipid, the hydrophobic constituent, and the subject copolymer.

一种以融合前构象GPC为靶标的拉沙病毒mRNA疫苗及其制备方法

Resumen de: CN120695172A

本发明提供一种以融合前构象GPC为靶标的拉沙病毒mRNA疫苗及其制备方法，涉及疫苗制备技术领域。所述拉沙病毒mRNA疫苗的氨基酸序列如SEQ ID NO.1所示，核酸序列如SEQ ID NO.2所示，且该疫苗为通过确定拉沙病毒的保守GPC氨基酸序列，并编码融合前构象GPC的氨基酸序列得到。本发明克服了现有技术的不足，能够设计并制备一款对LASV感染具有较好预防效果且安全稳定，研发周期短，成本低的疫苗。

一种规避人体预存抗PEG抗体的方法

Resumen de: WO2024148785A1

A method for evading a preexisting anti-PEG antibody in a human body, and the use of a PEGylated nano-carrier containing terminal hydroxyl in the preparation of a drug for evading a preexisting anti-PEG antibody in the human body. A PEGylated nano-carrier and nano-preparation containing terminal hydroxyl are included. The PEGylated nano-carrier and nano-preparation containing terminal hydroxyl have low binding with the preexisting anti-PEG antibody in the human body, and can thus evade being quickly cleared from the blood of the human body, so as to have a better treatment effect. In addition, by means of evading binding of the PEGylated nano-carrier and the nano-preparation containing terminal hydroxyl to the preexisting anti-PEG antibody in human blood, complement activation can be reduced, and side effects such as clinical injection reaction are reduced.

用于药物用途的RNA制剂

Resumen de: AU2024210882A1

The present disclosure relates to RNA comprising one or more miRNA binding sequences, wherein the one or more miRNA binding sequences bind to miRNA that is present in cells in which expression of the RNA is not desired. Delivering the RNA to cells after administration, in particular after intramuscular or intravenous administration, allows expression of a polypeptide encoded by the RNA in certain cells while expression in other cells is repressed. In some embodiments, such cells comprise endothelial cells. RNA compositions described herein allow expression of a pharmaceutically active peptide or polypeptide by the RNA in a subject while reducing or avoiding the risks of undesired effects resulting from expression of the pharmaceutically active peptide or polypeptide in certain cells or tissues.

牡荆素在制备弱精症药物中的应用

Resumen de: CN120694987A

本发明公开了牡荆素在制备弱精症药物中的应用，具体是以人类重链铁蛋白HFn纳米笼负载牡荆素VI形成HFn@VI纳米颗粒，用于制备弱精症药物。本发明统一了牡荆素的药理学和诊断潜力，为男性不育症的精准医学提供一个新的平台。

一种ALKBH5在抑制结直肠癌PHF20表达中的用途

Resumen de: CN120695207A

本发明提出了一种ALKBH5在抑制结直肠癌PHF20表达中的用途，包括以下步骤：步骤一：选择合适ALKBH5过表达载体、并敲低PHF20、使用ALKBH5小分子激活剂或CRISPR、Cas9系统调控ALKBH5活性，从而显著抑制结直肠癌细胞的增殖和转移；步骤二：将调控元件递送至结直肠癌细胞或肿瘤组织，递送方式包括病毒载体、脂质体、局部注射或静脉注射，以提高治疗的精确性和有效性；步骤三：检测ALKBH5与PHF20表达水平的变化，评估肿瘤增殖、转移或耐药性的抑制效果。本发明核心在于证实ALKBH5抑制结直肠癌细胞中PHF20表达的作用，通过多种调控手段和递送系统，实现对结直肠癌细胞的增殖、转移和耐药性的抑制。此外，还提供了相应的药物组合物及诊断评估方法，为结直肠癌的治疗和预后提供了新的策略。

脂质纳米粒、靶向脂质纳米粒及制备方法和应用

Resumen de: CN120694968A

本发明公开了一种脂质纳米粒、靶向脂质纳米粒及制备方法和应用。该脂质纳米粒具有核壳结构，内核为由含药物分子的PLGA纳米粒形成，外壳为由包括磷脂、胆固醇和修饰材料的原料形成；其中，含药物分子的PLGA纳米粒为由包括PLGA和药物分子的原料形成；PLGA与磷脂的质量比为1～5:1；磷脂与胆固醇的质量比为3～10:1，磷脂与修饰材料的质量比为3～10:1；药物分子选自难溶性药物、水溶性药物、基因和蛋白质中的一种或多种；所述修饰材料为PEG‑DSPE。本发明的脂质纳米粒的硬度可以调控，可以通过调控硬度以改善细胞摄取；本发明的靶向脂质纳米粒可以更好地改善细胞摄取，促进肠上皮细胞吸收，提高口服疗效。

一种雨生红球藻外泌体包埋类胡萝卜素的制备方法

Resumen de: CN120695198A

本申请设计了一种雨生红球藻外泌体包埋类胡萝卜素的制备方法，包括步骤S1、将雨生红球藻藻种接种于基础培养基进行育种培养并扩增；步骤S2、对基础培养基进行离心分离，并进行过滤浓缩获取雨生红球藻外泌体；步骤S3、将类胡萝卜素用二甲基亚砜或丙二醇溶解后，将雨生红球藻外泌体与类胡萝卜素混合，于32～40℃自然孵育1～4h，得到含有外泌体的类胡萝卜素。通过上述设计，本申请能够首次利用雨生红球藻外泌体作为载体包埋类胡萝卜素，显著提升脂溶性活性成分的稳定性和生物利用度，虾青素包封率达0.071％、岩藻黄素包封率达2.87％，且工艺全程可规模化操作，在医药、化妆品及功能食品领域具有广阔工业化应用前景。

包含用于在没有任何佐剂的情况下经由阳离子多糖纳米颗粒递送灭活的完整细菌的系统的疫苗组合物

Resumen de: MX2025009500A

The invention relates to the field of vaccine compositions. The invention more particularly relates to a prophylactic vaccine composition that is intended for mammals and birds and comprises a killed whole bacterium, said bacterium being covered with a cationic agent, in particular cationic nanoparticles.

一种铈基靶向纳米药物及其应用和制备方法

Resumen de: CN120695167A

本发明公开了一种铈基靶向纳米药物及其应用和制备方法。本发明通过无载体配位驱动自组装技术，将水溶性葡萄糖氧化酶溶液和铈离子溶液与脂溶性替拉扎明溶液混合在一起，并通过官能团配位键获得无载体配位自驱动的铈基靶向纳米药物。本发明进一步通过反复冻融和差速离心方式高效制备了血小板膜，将其与铈基靶向纳米药物通过共挤出方式制备得到血小板膜仿生铈基靶向纳米药物，能够有效增强铈基靶向纳米药物的靶向性和生物安全性。本发明制备的药物稳定性好，具有优异的生物相容性、低毒性、高靶向性和高肿瘤杀伤性。

PHARMACEUTICAL COMPOSITIONS COMPRISING HIV INTEGRASE INHIBITORS

Resumen de: US2025296932A1

The present disclosure relates generally to pharmaceutical compositions (e.g., solid oral dosage forms) of the compound of Formula I:Also disclosed are methods of treating or preventing human immunodeficiency virus (HIV) infection in a human, including orally administering to the human the solid oral dosage forms or pharmaceutical compositions disclosed herein.

DRUG-POLYMER COMPLEX, PHARMACEUTICAL COMPOSITION, AND DRUG DELIVERY POLYMER

Resumen de: WO2025197935A1

The present invention provides a drug-polymer complex that includes a drug and a drug delivery polymer having a phosphocholine group, and that exhibits a dissociation constant (KD) of 1.0×10-6M or less with respect to STARD7.

METHOD FOR PREPARING NANO-STRUCTURED LIPID CARRIERS, NANO-STRUCTURED LIPID CARRIERS AND USE THEREOF

Resumen de: WO2025194234A1

The present invention relates to a method for preparing nano-structured lipid carriers that are nanometric systems compatible with hydrophobic drug products and that do not exhibit toxicity due to their excipients. These nano-structured lipid carriers contain co-encapsulated cannabidiol and paclitaxel and have applications in the pharmaceutical field, more specifically in antitumour therapy.

USE OF MAGNETOELECTRICALLY DRIVEN MAGNETOSTRICTIVE PIEZOELECTRIC NANOPARTICLE IN ANTIVIRAL THERAPY

Resumen de: WO2025195465A1

Provided is use of a magnetoelectrically driven magnetostrictive piezoelectric nanoparticle in antiviral therapy. The magnetostrictive piezoelectric nanoparticle comprises a shell structure with piezoelectric properties and a core structure with magnetoelectric response properties. The magnetostrictive piezoelectric nanoparticle, by means of up-regulating multiple immunomodulatory pathways, effectively enhances the expression of type I interferon and a host's innate immune response, ultimately inhibiting the viral activity, ameliorating hepatic and pulmonary tissue damage, and improving the survival rates. The magnetostrictive piezoelectric nanoparticle has the advantages such as high controllability, deep penetration, and non-invasiveness, and offers broad application prospects in the antiviral field.

cGMP EXOSOME LOADED THERAPEUTICS FOR TREATING SICKLE CELL DISEASE

Resumen de: US2025297256A1

A composition for treating sickle cell disease includes a cGMP exosome having a size range between 60 nm to 120 nm, which may be extracted from a human mesenchymal stem cell (hMSC) or human PBMC at 320,000 g, wherein the cGMP exosome may be loaded with a Hemoglobin Subunit Beta (HBB) DNA plasmid carrying a gene encoding a normal beta chain of hemoglobin and alone or in combination with a short interference RNA (siRNA) that silences the translation of the SNP rs334 (A) mutation of the beta chain of a Hemoglobin A protein.

CATIONIC LIPID COMPOUNDS AND COMPOSITION AND USE THEREOF

Resumen de: WO2025195308A1

The present application provides new cationic lipid compounds and a composition containing the compounds. A nanoparticle composition contains new cationic lipid compounds and other lipids, such as phospholipids, structural lipids and PEG lipids. Additionally, the nanoparticle composition of the present application can be used to deliver a therapeutic agent and/or a prophylactic agent to mammalian cells or organs to, for example, regulate polypeptide, protein or gene expression.

RGD-PEPTIDE MODIFIED COVALENT ORGANIC FRAMEWORKS FOR TARGETED CANCER THERAPY

Resumen de: WO2025196660A1

The present disclosure provides nanoparticles having RGD-based peptides conjugated thereto. The nanoparticles may be formed one or more covalent organic frameworks (COFs) of the present disclosure. Also disclosed are methods of making the COFs and nanoparticles. The COFs have one or more binding partners and/or COF (following conjugation of the binding partner) may have an RGD-based peptide. The RGD- based peptide may be selected based on the desire to treat a specific cancer. COFs may have the following structure: Formula (I), where L is a linker group and Peptide is an RGD-based peptide.

HYBRID PARTICLES, METHOD FOR PRODUCING SAME AND USE

Resumen de: WO2025196641A1

The invention relates to the field of medicine, and more particularly to bioengineering, genetic engineering, gene editing, molecular medicine, nanotechnology, biotechnology, nanoengineering and protein engineering. The invention can be used for packaging any RNA, DNA, or a mixture thereof into biological nanoparticles of any origin and composition, inter alia nanoparticles enriched with a protein of interest.

ENHANCED COMPOSITION FOR INHIBITING VIABILITY OF TRIPLE-NEGATIVE BREAST CANCER (TNBC) CELLS AND PREPARATION THEREOF

Resumen de: WO2025196687A1

According to an aspect of the present disclosure, a composition for use in inhibiting viability of triple negative breast cancer (TNBC) cells contains 1,8-cineole as an active ingredient and β-cyclodextrin as a carrier, where the β-cyclodextrin encapsulates the 1,8-cineole forming a nanoformulation. According to another aspect of the present disclosure, a method of preparing a composition for use in inhibiting viability of TNBC cells includes forming a first solution by dissolving β-cyclodextrin in a solvent, forming a second solution by adding Elettaria cardamomum essential oil to the first solution, where the Elettaria cardamomum essential oil contains 1,8-cineole as an active ingredient. The method also includes sonicating the second solution to form a homogenized second solution, centrifuging the homogenized second solution to form a pellet, deep freezing the pellet, and lyophilizing the deep frozen pellet to form a nanoformulation, where the nanoformulation contains the 1,8-cineole encapsulated by the β-cyclodextrin.

CELL TARGETED POLYMER SELF-ASSEMBLY NANOMICELLES, AND METHOD OF PREPARING SAME

Resumen de: WO2025198324A1

The present invention relates to a polymer self-assembly nanocarrier and a method of preparing same, the nanocarrier binding a peptide that targets a specific cell and, simultaneously, carrying an active ingredient that has been verified. The present invention can exhibit various effects such as a moisturizing effect, an increase in filaggrin production, whitening, skin barrier reinforcement and the like by selectively and efficiently delivering a drug through a targeting peptide, can be applied to skin diseases such as atopy or psoriasis, and is expected to enable the maximum effect of an active substance in the pharmaceutical and cosmetic fields to be obtained.

LYMPHATIC VESSEL CONTRAST AGENT WITHOUT VENOUS CONTAMINATION

Resumen de: AU2024217151A1

The present invention relates to a non-vascular injection formulation containing nanoparticles of which the hydrodynamic diameter is adjusted to 2 to 20 nm and the surface charge is adjusted to -20 mV to 0 mV through the hydration of hydrophilic functional groups exposed to the surface of the nanoparticles, so that the nanoparticles are selectively discharged only into peripheral lymphatic vessels without infiltrating or being discharged into the capillaries at an injection site, wherein the nanoparticles (i) themselves are a drug and/or (ii) a carrier for other drugs, and the nanoparticles are discharged only into peripheral lymphatic vessels when injected into tissue areas and not intravenously administered, and thus drug modality in which residence time at the injection site is extended is provided. Nanoparticles of which the hydrodynamic diameter and the surface charge are adjusted, according to the present invention, can be excreted from the body within 1 week after administration without any residue at the administration site, and, when the nanoparticles are designed as a contrast agent exhibiting T1-MRI contrast effects and are injected into tissue areas adjacent to blood vessels, peripheral or central lymphatic vessels can be selectively imaged without venous contamination.

SILOXANE-BASED LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY

Resumen de: US2025295788A1

The present disclosure relates, in part, to siloxane-based lipids or lipidoids, lipid nanoparticles (LNPs) comprising the same, and pharmaceutical compositions thereof. In certain embodiments, the LNPs of the present disclosure selectively target cells (e.g., hepatocytes, epithelial cells, endothelial cells, and immune cells, inter alia) and/or organs of interest (e.g., liver, spleen, heart, and lungs, inter alia). In another aspect, the present disclosure relates to methods of treating, preventing, and/or ameliorating one or more diseases and/or disorders in a subject, the method comprising administering to the subject at least one LNP of the present disclosure and/or at least one pharmaceutical composition of the present disclosure.

IMMUNOSTIMULATORY NANOPARTICLE

Resumen de: US2025295806A1

An immunostimulatory nanoparticle comprising a biocompatible lipid shell that defines an outer surface of the nanoparticle and a core, which is loaded with a Toll-like Receptor 9 (TLR9) agonist and a nucleic acid inhibitor of V domain Immunoglobulin Suppressor of T cell activation (VISTA), and optionally a plurality of targeting moieties linked to the outer surface, wherein the optional targeting moieties are configured to direct the nanoparticle to tumor-resident myeloid cells in a tumor microenvironment upon administration of the nanoparticle to a subject with cancer.

COMPOSITIONS AND METHODS FOR DELIVERY OF BIOMACROMOLECULE AGENTS

Resumen de: US2025295805A1

The present invention relates to nanoparticles complexed with biomacromolecule agents configured for treating, preventing or ameliorating various types of disorders, and methods of synthesizing the same. In particular, the present invention is directed to compositions comprising nanoparticles (e.g., synthetic high density lipoprotein (sHDL)) carrying biomacromolecule agents (e.g., nucleic acid, peptides, glycolipids, etc.), methods for synthesizing such nanoparticles, as well as systems and methods utilizing such nanoparticles (e.g., in diagnostic and/or therapeutic settings).

BIO-NANOSHELLS AND METHODS FOR PREPARATION AND APPLICATIONS THEREOF

Resumen de: US2025295779A1

The present invention provides a bio-nanoshell for binding specifically to a target cell. The bio-nanoshell may comprise a cell-derived biological membrane from a donor cell and a nanoshell having an exterior surface coated with the cell-derived biological membrane. The cell-derived biological membrane may comprise a phospholipid bilayer and an adhesion protein specific for the target cell. Also provided is a method for preparing the bio-nanoshells and a method for delivering the bio-nanoshells to target cells.

MRNA VACCINE COMPOSITION

Resumen de: US2025295755A1

Disclosed herein are nucleic acid vaccine compositions including one or more polynucleotides encoding one or more antigenic polypeptide, formulated within a lipid reconstructed plant messenger packs (LPMPs) comprising natural lipids and an ionizable lipid. The disclosure also includes a method for making a nucleic acid vaccine, comprising reconstituting a film comprising purified PMP lipids in the presence of an ionizable lipid to produce a LPMP comprising the ionizable lipid, and loading into the LPMPs with one or more polynucleotides encoding one or more antigenic polypeptides.

CROSS-LINKED TUMOR LYSATE SPHERICAL NUCLEIC ACIDS AS CANCER VACCINES

Resumen de: US2025295745A1

The disclosure is generally related to cross-linked tumor lysate spherical nucleic acids (CLSNAs), nanostructures comprising a core to which a shell of oligonucleotides is attached. Methods of making and using the CLSNAs are also provided herein. In some aspects, the disclosure provides a cross-linked tumor lysate spherical nucleic acid (CLSNA) comprising: (a) a core comprising a plurality of cross-linked tumor cell antigens; and (b) a shell of oligonucleotides attached to the external surface of the core, the shell of oligonucleotides comprising one or more immunostimulatory oligonucleotides.

RNA VACCINES AGAINST INFECTIOUS DISEASES

Resumen de: US2025295751A1

The disclosure provides compositions, methods of treatment, and methods of making and using compositions to deliver a nucleic acid to a subject. Methods of using these compositions as a vaccine for treatment of an infectious disease are also provided.

POLYMER PARTICLES FOR NEUTROPHIL INJURY

Resumen de: US2025295589A1

Provided herein are methods of treatment, compositions, systems and kits using polymer particles as restraints of neutrophil function. Such methods include, but are not limited to, methods of preventing, treating, and/or ameliorating inflammatory diseases, infections, autoimmune diseases, malignant diseases, and other diseases or conditions in which neutrophils may be implicated. In some embodiments, polymer particles are useful for diagnosing neutrophil related diseases or conditions.

COMPLEX LIPID NANOPARTICLES ENCAPSULATING POLYPEPTIDES AND USES THEREOF

Resumen de: US2025295587A1

Disclosed herein are complex lipid particles encapsulating one or more exogenous peptides, polypeptides, or proteins, as well as methods of producing a complex lipid formulation comprising an exogenous peptide, polypeptide, or protein. Also, disclosed herein are modified plant messenger packs (PMPs) formulation encapsulating one or more exogenous peptides, polypeptides, or proteins, and methods of producing a modified PMP formulation comprising an exogenous peptide, polypeptide, or protein.

DEFORMABLE NANO-SCALE VEHICLES (DNVS) FOR TRANS-BLOOD BRAIN BARRIER, TRANS-MUCOSAL, AND TRANSDERMAL DRUG DELIVERY

Resumen de: US2025295586A1

In various embodiments deformable nano-scale vehicles (DNV) are provided that are useful for the delivery of therapeutic agents. In certain embodiments the DNVs are capable of transdermal delivery and can additionally cross the blood-brain barrier.

Multifunctional Nanoparticles For Prevention and Treatment of Atherosclerosis

Resumen de: US2025295605A1

This disclosure relates to nanoparticles for preventing, treating and reversing atherosclerosis.

FORMULATIONS OF BENDAMUSTINE

Resumen de: US2025295637A1

Long term storage stable bendamustine-containing compositions are disclosed. The compositions can include bendamustine or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable fluid which can include in some embodiments PEG, PG or mixtures thereof and an antioxidant or chloride ion source. The bendamustine-containing compositions have less than about 5% total impurities, on a normalized peak area response (“PAR”) basis as determined by high performance liquid chromatography (“HPLC”) at a wavelength of 223 nm, after at least about 15 months of storage at a temperature of from about 5° C. to about 25° C.

FUNCTIONALIZED POLYMERIC PARTICLES FOR TREATMENT OF GLIOMAS

Resumen de: US2025295604A1

Nanoparticle compositions including one or more active agents, and strategies for enhanced delivery of the active agents, are provided. In preferred embodiments, the nanoparticles are composed of block copolymers of one or more hydrophobic polymers that form the core, and a hyperbranched polymer that forms a shell or corona. In some embodiments, the particles include an acid-sensitive, poly(amine-co-ester) (PACE) that can increase release of the active agent in acidic environments, for example within endosomes. The compositions can include one or more targeting moieties. Preferred targeting moieties include adenosine agonists and pHLIP which can enhance delivery to tumor cells. Methods of using the compositions to treat diseases and disorders of the central nervous system, for example, brain cancers such as glioma, are also provided.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

Resumen de: US2025295603A1

Disclosed herein compositions and methods for the treatment of neurodegenerative disorders, such as Alzheimer's disease.

A PEPTIDE AND THE SELECTION METHOD THEREOF

Resumen de: US2025297035A1

Provided is a peptide, wherein the peptide comprises a first functional module, a second functional module and a third functional module. Provided is a method of selecting a candidate peptide. Provided is a selecting method thereof. The peptide helps deliver nucleic acids efficiently.

EXTRACELLULAR VESICLES DERIVED FROM MILK AND PROCESS FOR ISOLATING THE SAME

Resumen de: US2025295600A1

The present invention relates to the field of biotechnology, and particularly to milk derived extracellular vesicles, and provides a process for isolating such extracellular vesicles from milk and milk related fluids. The present invention is also related to compositions containing said extracellular vesicles derived from milk, particularly suitable for use in pharmaceutical, veterinary, cosmetic and/or nutraceutical applications.

PEPTIDE COMPOUNDS AND PEPTIDE CONJUGATES FOR THE TREATMENT OF CANCER THROUGH RECEPTOR-MEDIATED CHEMOTHERAPY

Resumen de: US2025296957A1

The present disclosure relates to peptide compounds and conjugate compounds, processes, methods and uses thereof for treating cancer. For example, the compounds can comprise compounds of formula(I)(SEQ ID NO: 1)X1X2X3X4X5GVX6AKAGVX7NX8FKSESY(II)(SEQ ID NO: 2)(X9)nGVX10AKAGVX11NX12FKSESY(III)(SEQ ID NO: 3)YKX13LRRX14APRWDX15PLRDPALRX16X17L(IV)(SEQ ID NO: 4)YKX18LRR(X19)nPLRDPALRX20X21L(V)(SEQ ID NO: 5)IKLSGGVQAKAGVINMDKSESM(VI)(SEQ ID NO: 6)IKLSGGVQAKAGVINMFKSESY(VII)(SEQ ID NO: 7)IKLSGGVQAKAGVINMFKSESYK(VIII)(SEQ ID NO: 8)GVQAKAGVINMFKSESY(IX)(SEQ ID NO: 9)GVRAKAGVRNMFKSESY(X)(SEQ ID NO: 10)GVRAKAGVRN(Nle)FKSESY(XI)(SEQ ID NO: 11)YKSLRRKAPRWDAPLRDPALRQLL(XII)(SEQ ID NO: 12)YKSLRRKAPRWDAYLRDPALRQLL(XIII)(SEQ ID NO: 13)YKSLRRKAPRWDAYLRDPALRPLLwherein X1 to X21 and n can have various different valuesand wherein at least one protecting group and/or at least one labelling agent is optionally connected to said peptide compound at an N- and/or C-terminal end.

Immunoreactant carrier

Resumen de: AU2025226757A1

The immunoreactive substance carrier of the present invention may stably deliver various immunoreactive substances, including antibodies and cytokines, to a target site, thereby exhibiting excellent immunotherapeutic effects. Therefore, a composition comprising a carrier of the present invention can be used for immunotherapy, thereby having excellent effects in the prevention or treatment of cancer or various immune diseases. The immunoreactive substance carrier of the present invention may stably deliver various immunoreactive substances, including antibodies and cytokines, to a target site, thereby exhibiting excellent immunotherapeutic effects. Therefore, a composition comprising a carrier of the present invention can be used for immunotherapy, thereby having excellent effects in the prevention or treatment of cancer or various immune diseases. ep h e i m m u n o r e a c t i v e s u b s t a n c e c a r r i e r o f t h e p r e s e n t i n v e n t i o n m a y s t a b l y d e l i v e r v a r i o u s i m m u n o r e a c t i v e s u b s t a n c e s , i n c l u d i n g a n t i b o d i e s a n d c y t o k i n e s , t o a t a r g e t s i t e , t h e r e b y e x h i b i t i n g e p e x c e l l e n t i m m u n o t h e r a p e u t i c e f f e c t s h e r e f o r e , a c o m p o s i t i o n c o m p r i s i n g a c a r r i e r o f t h e p r e s e n t i n v e n t i o n c a n b e u s e d f o r i m m u n o t h e r a p y , t h e r e b y h a v i n g e x c e l l e n t e f f e c t s i n t h e

RECOMBINANT AAV VECTORS AND USE THEREOF

Resumen de: US2025297279A1

Provided is a recombinant adeno-associated virus (rAAV) capsid protein, comprising an amino acid sequence of 11 contiguous amino acids X1X2X3X4X5X6X7X8X9X10Q, rAAV vector comprising the same, and the use thereof.

MICROFLUIDIC PREPARATION OF DUAL-PHASE NANODROPLETS WITH FLUORINATED COMPOUNDS

Resumen de: US2025295818A1

The present invention relates to calibrated dual-phase nanodroplets comprising an outer layer and an inner core, said outer layer comprising a biocompatible fluorinated surfactant and said inner core comprising a fluorinated compound and a biocompatible oil. The invention further relates to a method of preparation of said calibrated dual-phase nanodroplets through microfluidic technique, and to their use for in vivo or in vitro diagnostic and/or for therapy.

HYDROPHOBIN-BASED CAPSULES WITH TARGET-SENSITIVE RELEASE MECHANISM, COMPOSITIONS COMPRISING THEREOF, AND RELATED METHODS

Resumen de: WO2025196666A2

The present invention relates to hydrophobin (HFB) fusion polypeptides, HFB-based capsules comprising said HFB fusion polypeptides and having a target-sensitive opening mechanism, and compositions comprising the HFB-based capsules. The invention further relates to the use of said HFB capsules as a platform for the delivery of a variety of compounds for agronomical, gastronomical and/or pharmaceutical purposes and its use for the detection of pathogens and/or microbes.

NOVEL AMYLOID-BETA AGGREGATE DEGRADER AND BRAIN-TARGETING DRUG DELIVERY SYSTEM USING SAME

Resumen de: US2025295612A1

The present invention comprises: a novel molecule capable of disaggregating amyloid-beta plaques; and a brain-targeting amyloid-beta plaque disaggregation nano platform loaded with the molecule. An amyloid-beta plaque disaggregating agent developed according to the present invention exhibits high amyloid-beta plaque disaggregation efficacy, and the brain-targeting amyloid-beta plaque disaggregation nano platform shows high potential in the medical field on the basis of the effects of effectively targeting the brain and disaggregating amyloid-beta plaques present in the brain.

BIOSOLUBLE POLYMER OR PARTICLE FOR DELIVERY OF AN ACTIVE AGENT AND A METHOD FOR THE PRODUCTION

Resumen de: US2025295606A1

The present invention refers to a method for producing a polymer in form of a gel or a particle, and to the resulting polymer, gel and particle, respectively. The polymer comprises a carbon donor and a metal oxide precursor, a metal oxide or a combination thereof and optionally an active agent. The invention is further directed to a composition and film comprising such polymer, and their use as a medicament for example in treating diabetes, obesity, neuronal disease, viral infection or cancer.

IN VIVO NICKASE-BASED EDITING OF THE LPA GENE FOR TREATMENT OF CARDIOVASCULAR DISEASE

Resumen de: WO2024197065A2

Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the LPA gene. Treatment or prevention of cardiovascular disease through disruption of the production of apo(a) through genetic editing and the reduction of the blood lipoprotein(a) Lp(a) concentration is disclosed herein. Disclosed are nickase-based gene editing systems designed to effectuate the installation of insertions and/or deletions (indel variants) and/or non-synonymous variants in the coding sequence of LPA. The nickase-based gene editing systems generally comprise one or more mRNAs that encode one or more nickases and a plurality of guide oligonucleotides (e.g., gRNAs) and may be delivered in vivo to a mammalian subject in need thereof via a suitable delivery system, such as lipid nanoparticles (LNPs) (with or without GalNAc targeting moieties) intravenously, or otherwise, administered to a patient as potentially a once-and-done therapeutic. The manufacturing, use, and formulation of the gene editing systems and compositions are also disclosed.

非ウイルス性ＤＮＡ送達及び発現の促進

Resumen de: WO2024054882A1

The present invention features method and composition that can be used to facilitate intracellular delivery of DNA to a subject. The provided methods and compositions employ a nanoparticle for intracellular DNA delivery and a type 1 interferon receptor pathway inhibitor. The type 1 interferon receptor pathway inhibitor is provided to decrease the subject's immune response that can be stimulated by the DNA.

核酸送達のための２－アミノプロパン－１，３－ジオールでキャップされたカチオン性ペプトイド

Resumen de: CN119894918A

The present disclosure provides delivery vehicle compositions comprising a hydroxyalkyl terminated cationic peptoid, such as a 2-aminopropane-1, 3-diol terminated cationic peptoid, and complexes of the delivery vehicle with a polyanionic compound, such as a nucleic acid. The disclosure further provides methods of making and using the delivery vehicle compositions and complexes, such as for delivering polyanionic compounds (e.g., nucleic acids) to cells. The present disclosure also provides methods of eliciting an immune response with the delivery vehicle complexes of the present disclosure.

薬物含有ナノ粒子製造用キット及び薬物送達用ナノ粒子組成物

Resumen de: CN119768157A

The present invention relates to a kit for preparing nanoparticles containing a drug and a nanoparticle composition for delivering a drug, and more particularly, to a kit for preparing nanoparticles containing a drug and a nanoparticle composition for delivering a drug, the kit is designed to increase the cellular delivery efficiency of a drug by using nanoparticles comprising a cationic compound and an anionic polymer compound having at least one acidic functional group.

放射線治療シート

Resumen de: MX2025002804A

A sheet for radiotherapy, which includes a sheet base configured for placement on tissue of a patient and a radium-binding material coupled to the base sheet. Alpha-emitting radium radionuclides are coupled to the radium-binding material. The radium radionuclides do not leave the sheet, but radon and lead resulting from decay of the radium radionuclides easily leave the sheet.

ピペラジン化合物

Resumen de: WO2024054901A1

The present invention relates to novel piperazine compounds, in particular, of formula (I) or formula (II). These piperazine compounds can be used, for example, in lipid nanoparticle compositions for drug delivery and cancer treatments.

ＢＲＣＡ陰性／相同修復熟達癌を治療するためのＩＬ－１２遺伝子療法

Resumen de: MX2025002927A

The present disclosure relates to a method for treating cancer in a subject that is BRCA-negative and homologous repair proficient (HRP), the method comprising administering to the subject a nucleic acid vector (e.g., a plasmid) comprising a polynucleotide that encodes an interleukin-12 (IL-12) formulated with a lipopolymer (e.g., a nanoparticle). In some aspects, the method further comprises administering to the subject an anticancer agent (e.g., a chemotherapeutic agent), an antibody or antigen-binding fragment thereof that specifically binds a vascular endothelial growth factor (VEGF) (anti-VEGF antibody), an immune checkpoint inhibitor, or any combination thereof.

POLYMER-DRUG CONJUGATE, INTERMEDIATE THEREOF, AND USE THEREOF

Resumen de: AU2024261935A1

A nano-size controllable and stable polymer-drug conjugate, an intermediate thereof, and a use thereof. Provided is a polymer-drug conjugate as represented by formula (I) and having different polymer bodies, a controllable coupled group quantity, controllable group coupling sites and a controllable nano-size. The drug conjugate has one or more of the following advantages: a low renal clearance rate, a low liver-spleen clearance rate, long plasma half-life, strong drug accumulation capability at lesion tissues, strong drug permeability at lesion tissues, low toxic and side effects, and an excellent treatment effect.

NOVEL LIPID NANOPARTICLE FORMULATIONS FOR DELIVERY OF NUCLEIC ACIDS

Resumen de: AU2024233180A1

The present invention provides novel ionizable lipids and novel lipid nanoparticles comprising messenger RNA (mRNA) useful for the delivery of nucleic acids, related pharmaceutical compositions or vaccines as defined herein for use in human or veterinary medicine, in particular for use in the treatment and/or prophylaxis of cancer diseases.

METHOD FOR TRANSIENT ACIDIFICATION OF WOUNDS

Resumen de: AU2024228558A1

This disclosure provides the use of nanoparticles of self-assembled acidic molecular clusters (NP-AMCs) of ammonium salts to lower the pH of skin and underlying tissue of a human or other mammal as a means of stimulating the localized immune response to a wound.

POLYSACCHARIDE CROSS-LINKED COLLOIDAL PARTICLES SQUEEZABLE BETWEEN INTERNAL BODY STRUCTURES WITHOUT AGGREGATION, AND USE THEREOF AS MODIFIER FOR IN VIVO INJECTION

Resumen de: AU2024219315A1

The present invention relates to polysaccharide cross-linked colloidal particles and a use thereof as a modifier for in vivo injection. The present invention provides a polysaccharide cross-linked colloidal particle platform technology that can be designed to not only act as a drug delivery carrier for functional nanoparticles or drugs for in vivo introduction, but also control in vivo distribution and excretion.

分泌タンパク質の産生のための修飾ポリヌクレオチド

Resumen de: JP2025024198A

To provide nucleic acid based compounds or polynucleotides having features which are useful for optimizing formulation and delivery of nucleic acid based therapeutics.SOLUTION: An isolated polynucleotide comprises: (a) a first region of linked nucleosides, the first region encoding a polypeptide of interest, the polypeptide of interest having prescribed sequences; (b) a first flanking region located at a 5' terminus of the first region, the first flanking region comprising (i) a sequence of linked nucleosides selected from a native 5' UTR and (ii) at least one 5' terminal cap; and (c) a second flanking region located at a 3' terminus of the first region, the second flanking region comprising (i') a sequence of linked nucleosides selected from a native 3' UTR and (ii') a 3' tailing sequence of linked nucleosides.SELECTED DRAWING: Figure 1

USE OF A CHOLESTEROL-BINDING PEPTIDE TO IMPROVE THE CELLULAR UPTAKE OF A PEPTIDE COACERVATE DELIVERY SYSTEM

Resumen de: WO2025198537A1

The present invention provides methods for enhancing cellular binding or entry of a peptide coacervate composition utilising a peptide that comprises a cholesterol binding domain/motif. The invention also covers novel peptide coacervates that comprise a peptide that comprises a cholesterol binding domain/motif, optionally peptide coacervates that also comprises a payload, such as an active agent to be delivered into the cell, and their use in therapy or diagnosis.

PH-SENSITIVE CATIONIC LIPIDS, LIPID NANOPARTICLES COMPRISING THE SAME AND METHODS OF DELIVERING NUCLEIC ACIDS

Resumen de: WO2025199302A1

A pH-sensitive cationic lipid, a lipid nanoparticle including the pH-sensitive lipid, and a method of delivering a nucleic acid encapsulated in the lipid nanoparticle to a cell or a subject. The lipid nanoparticle may include a compound represented by general formula (I) or pharmaceutically acceptable salt thereof: (R1)(R2)C(OH) -(CH2)a-(O-CO)b-X (I) wherein a represents an integer of 3-5; b represents 0 or 1; R1 and R2 each independently represent a group represented by general formula (A): (R11)(R12) - CH-(CH2)w - (CO-O)c - (CH2)v - (A), wherein R11 and R12 each independently represent C5-15 alkoxy group; each c independently represents 0 or 1; each v independently represents an integer of 4-12; each w independently represents an integer of 0-3; and X represents a 5- to 7-membered non-aromatic heterocyclic group, wherein a carbon atom of the heterocyclic group is bound to (O-CO)b- and one or two hydrogen atoms of the heterocyclic group may optionally be replaced with C1-4 alkyl group or C2-4 alkenyl group.

NOVEL PROVITAMIN B5-BASED LIPID, LIPID NANOPARTICLE COMPRISING SAME, AND USES THEREOF

Resumen de: WO2025198369A1

The present invention relates to a novel provitamin B5-based lipid derivative compound, a lipid nanoparticle composition comprising same, and the like. More specifically, the novel provitamin B5-based lipid compound functions as an ionizable lipid in the formation of lipid nanoparticles and forms stable lipid nanoparticles, which can be used for delivering pharmaceutically active ingredients such as mRNA into the body.

IONIZABLE CATIONIC LIPID COMPOUNDS FOR DELIVERY SYSTEM AND USES THEREOF

Resumen de: WO2025194409A1

The present disclosure relates to novel lipids for delivering one or more biologically active molecules to a subject. The present application also relates to a composition or a nanoparticle comprising said lipid, methods of preparing said lipid, use of said lipid and method of using said lipid.

POLYOXYALKYLENE-1,2-DIMYRISTOYL-GLYCEROL COMPOUNDS, WHEREIN THE POLYOXYALKYLENE IS A POLY(ETHYLENE OXIDE) HAVING C1 TO C3-ALKYLOXYMETHYL SIDE CHAINS

Resumen de: MX2025005575A

The present invention refers to novel polyoxyalkylene based compounds and their manufacturing method as well as compositions comprising at least one novel polyoxyalkylene based compound and at least one active agent. Furthermore, the present invention refers to the manufacture of the compositions of the present invention as well as their use for the treatment of an illness in mammals or humans.

NANOCONJUGATES CONTAINING PDGFR-BETA LIGANDS AND USES THEREOF

Resumen de: WO2024105116A1

The present invention relates to the field of nanostructured protein materials, more specifically to therapeutic agents carrying fusion proteins which can be used for therapy.

COMPOSITIONS AND METHODS FOR FIBROBLAST GROWTH FACTOR RECEPTOR 3-MEDIATED DELIVERY TO ASTROCYTES

Resumen de: AU2023379457A1

The present invention provides, in part, protein-drug conjugates comprising an anti-fibroblast growth factor receptor 3 (FGFR3) (e.g., human FGFR3) antigen-binding protein (e.g., scFv, Fab) conjugated to a molecular cargo (e.g., polynucleotides, polypeptides, liposomes or lipid nanoparticles) for delivery of the molecular cargo to a targeted tissue (e.g., brain). Methods for treating various diseases or disorders, such as neurological diseases, with the conjugates are provided.

AEROSOLIZED LIPID NANOPARTICLES AND USES THEREOF

Resumen de: AU2023382622A1

Lipid nanoparticles (LNPs) containing particular cationic ionizable lipids with a biologically active polynucleotides (e.g., RNAs) are provided. In some aspects, the LNP complexes are provided as aerosols and/or dry powders, such as for delivery to the lungs. Methods of making and using such compositions are provided.

CATIONIC LIPIDS AND METHOD FOR PREPARING SAME

Resumen de: EP4620945A1

The present invention relates to cationic lipids and a method for preparing same, more specifically to cationic lipids that facilitate forming a complex with anionic medicinal material to thus be useful for drug delivery, and to a method for preparing the cationic lipids.

NANOPARTICLE COMPOSITION FOR DRUG DELIVERY

Resumen de: EP4620460A1

The present invention relates to a nanoparticle composition for drug delivery and, more specifically, to a composition for drug delivery, wherein the composition comprises an amphiphilic block copolymer and a lipid with a specific structure that can easily form complexes with anionic drugs, whereby the composition can easily form drug-containing nanoparticles and thus is useful for drug delivery, especially for targeted delivery of drugs to the lungs.

LIPID COMPOUND AND COMPOSITION FOR DELIVERY OF ACTIVE SUBSTANCE

Resumen de: EP4620948A1

Disclosed are a new lipid compound and a new lipid nanoparticle (LNP) composition comprising the lipid compound. The lipid compound according to the present disclosure may enhance the structural stability of an active substance by directly or indirectly binding to the active substance through multivalent interactions to encapsulate the active substance. In addition, the lipid nanoparticle composition comprising the lipid compound may significantly improve the intracellular delivery efficiency and activity of the active substance, therefore may be useful for the treatment and prevention of the disease.

NANOPARTICLE COMPOSITIONS FOR SUSTAINED THERAPY

Resumen de: NZ738043A

This disclosure provides compositions and methods for promoting the formation, expansion and recruitment of TR1 cells and/or B cells in an antigen-specific manner and treating diseases and disorders in a subject in need thereof. In particular, the invention provides a complex comprising a nanoparticle coupled via a less than 5 kDa PEG linker with maleimide end to a plurality of antigen relevant MHC class II complexes, where the 1nm-100nm nanoparticle core comprises iron oxide and where the pMHCII density per nanoparticle is 0.4 - 50 pMHC/100nm^2 and where the pMHCII complexes are from a list.

IONIZABLE LIPIDS AND LIPID NANOPARTICLE COMPOSITIONS FOR THE DELIVERY OF NUCLEIC ACIDS

Resumen de: MX2025005703A

Novel ionizable lipids are provided. Also provided are novel lipid nanoparticle compositions for the delivery of nucleic acid material to cells in vitro and in vivo with different and improved pharmacokinetic profiles as compared to what is typically observed in the art. Also provided are methods for using the compositions in research and as therapeutics.

PRODRUG STRATEGY THAT ENHANCES EFFICACY AND LOWERS SYSTEMIC TOXICITY OF MERTANSINE

Resumen de: WO2024108173A1

A nanoparticle and methods of using the same wherein the nanoparticle includes a prodrug comprising a targeting moiety-lipid conjugate, a polyethylene glycol-lipid conjugate, a sterol, a bulk lipid, and a drug-lipid conjugate, wherein a chemical linker group is positioned between the drug moiety and the lipid moiety of the drug-lipid conjugate. The nanoparticle can be used to treat cancer, for example, multiple myeloma.

POLYOXYALKYLENE-N,N-DITETRADECYLACETAMID COMPOUNDS, WHEREIN THE POLYOXYALKYLENE IS A POLY(ETHYLENE OXIDE) HAVING C1 TO C3-ALKYLOXYMETHYL SIDE CHAINS

Resumen de: MX2025005656A

The present invention refers to novel polyoxyalkylene based compounds and their manufacturing method as well as compositions comprising at least one novel polyoxyalkylene based compound and at least one active agent. Furthermore, the present invention refers to the manufacture of the compositions of the present invention as well as their use for the treatment of an illness in mammals or humans.

Compositions for cell-specific expression and uses thereof

Nº publicación: GB2639326A 24/09/2025

Solicitante:

MYELOID THERAPEUTICS INC [US]
Myeloid Therapeutics, Inc

Resumen de: GB2639326A

Compositions and methods for making and using engineered NK cells, T cells and B cells that express a chimeric antigen receptor.