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ＭＲＮＡのためのアルミニウムベース自己組織化送達システム、それを調製するための方法、及びその使用

Resumen de: AU2023221910A1

The present invention relates to the technical field of biopharmaceutics and vaccines, and in particular, to an aluminum-based self-assembled delivery system for mRNA, a method for preparing same, and use thereof. The aluminum-based self-assembled delivery system for messenger RNA is a nanocrystallite formed by in-situ self-assembly of an aluminum ion, an mRNA, and a co-assembled molecule driven by coordination. The delivery system serves as a vector-free self-delivery system serving for interleukin 21 mRNA, and can achieve efficient delivery of interleukin 21 mRNA and improved mRNA stability and targeted mRNA delivery efficiency. The nanoparticle of the present invention features good stability in serum and low toxicity, and can achieve efficient mRNA transfection in melanoma cells and significant inhibition of tumor growth in a melanoma mouse model. Therefore, the present invention provides an efficient and convenient preparation method for nucleic acid delivery systems, and has extremely high practical value.

一种靶向脾脏cDC1的mRNA-LNP疫苗及其制备方法和应用

Resumen de: CN119564639A

本发明属于生物医药技术领域，具体涉及一种靶向脾脏cDC1的mRNA‑LNP疫苗及其制备方法和应用。在本发明中提供了一种靶向性的mRNA‑LNP疫苗，能够通过脂质纳米颗粒(LNP)上连接的目标蛋白精准靶送到具体部位，提高核酸药物mRNA发挥活性的效应，并且整个LNP体系非常稳定，解决了现有技术目标蛋白连接到LNP可能会导致整个LNP体系不稳定的缺陷。实验结果显示，修饰了Spycatcher‑XCL‑1融合蛋白的LNP能够靶向到脾脏的经典一型树突状细胞(cDC1)，并且能有效抑制黑色素瘤的生长，显著延长生存期。本发明对治疗性肿瘤疫苗的开发具有重要的应用价值。

小鼠4T1乳腺癌治疗性肿瘤新生抗原mRNA疫苗及其制备方法与应用

Resumen de: CN119564841A

本发明公开了一种小鼠4T1乳腺癌治疗性肿瘤新生抗原mRNA疫苗及其制备方法与应用，所述乳腺癌治疗性肿瘤新生抗原mRNA疫苗包括：抗原表位组合物，所述抗原表位组合物包括7条与MHC I类分子结合的多肽片段、3条与MHC II类分子结合的多肽片段中的至少一种，所述7条与MHC I类分子结合的多肽片段的氨基酸序列分别如SEQ ID NO.1‑SEQ ID NO.7所示，所述3条与MHC II类分子结合的多肽片段的氨基酸序列分别如SEQ ID NO.8‑SEQ ID NO.10所示。该疫苗对肿瘤生长的抑制效果明显。

一种水痘-带状疱疹病毒mRNA疫苗及其制备方法与应用

Resumen de: CN119564842A

本发明公开了一种水痘‑带状疱疹病毒的mRNA疫苗及其制备方法与应用，本发明提供的mRNA疫苗通过对gE抗原序列进行密码子优化，再选择合适的启动子、5’UTR、3’UTR和3’端PolyA组合成完整的编码水痘带状疱疹病毒的mRNA核酸，筛选获得2组兼顾表达水平和稳定性的编码水痘带状疱疹病毒的mRNA核酸，并将疫苗脂质体采用一种新型的阳离子脂质咪唑并吡啶基脂质化合物，提高了在引流淋巴结中的靶向性，显著提高疫苗在动物体内激发的体液免疫和细胞免疫水平。

一种载氧纳米颗粒及其制备方法与应用

Resumen de: CN119564638A

本发明公开了一种载氧纳米颗粒及其制备方法与应用，所述载氧纳米颗粒包括全氟己烷内核以及包覆在所述全氟己烷内核外的聚合物外壳，所述聚合物的结构通式如下：#imgabs0#其中，x为0‑50之间的任意整数，y为20‑150之间的任意整数，#imgabs1#本发明利用聚合物与全氟己烷之间的氟‑氟相互作用构筑具有全氟己烷内核的胶束纳米颗粒，由于全氟己烷对氧气的高溶解性和稳定性，实现氧气的高效负载和递送，提升乏氧肿瘤的氧气含量。

一种对乙酰氨基酚喷鼻纳米剂及其制备方法与应用

Resumen de: CN119564637A

本发明涉及医药技术领域，具体涉及一种对乙酰氨基酚喷鼻纳米剂及其制备方法与应用，该纳米剂为对乙酰氨基酚聚乳酸羟基乙酸共聚物纳米粒(AAP‑PLGA‑NPs)；所述对乙酰氨基酚聚乳酸羟基乙酸共聚物纳米粒的外观呈球形，粒径为118nm，多分散系数值为0.221，ζ电位为‑23.7mV。该一种对乙酰氨基酚喷鼻纳米剂的制备方法为：首先以PLGA为载体材料，采用超声乳化法结合溶剂挥发法制备得载对乙酰氨基酚的PLGA纳米粒。本发明的AAP‑PLGA‑NPs经鼻腔给药后能显著提高对乙酰氨基酚在脑组织的蓄积量并且延缓消除，明显提高急进高原小鼠疼痛阈值。

カンプトテシン（ＣＰＴ）を含有するナノ粒子の使用および製剤

Resumen de: CN119013047A

Disclosed herein are compositions, formulations, and methods comprising nanoparticles comprising camptothecin conjugated mucus acid polymers.

一种负载IR780和藤黄酸前药的脂质体的制备方法及应用

Resumen de: CN119564640A

本发明公开了一种负载IR780和藤黄酸前药的脂质体的制备方法，将mPEG‑TK‑NH2溶解在二甲基甲酰胺中，加入藤黄酸、过氧化苯甲酰和三乙胺搅拌反应，接着浓缩、重结晶，得到mPEG‑TK‑GA，将其与大豆磷脂酰胆碱、胆固醇、IR780加入到有机溶剂中，旋转蒸发除去有机溶剂，得到脂质膜，加入磷酸盐缓冲溶液，加热并超声处理，整粒后，得到脂质体溶液，将SIRP‑α基因工程外泌体和IG Lipo混合，然后采用冻融法进行膜融合，即得。与现有方法相比，该方法制备的脂质体具有响应性释放药物作用，能够在提升治疗效果的同时减轻毒副作用，并且该脂质体还负载有IR780,联合光热治疗可以降低剂量并提升治疗效果。

生物可吸收颗粒及其使用方法

Resumen de: US2025057778A1

The present disclosure provides bioabsorbable particles with a nucleic acid. The bioabsorbable particles also include a bioabsorbable polymer matrix and a lipid salt. both of which can be altered to affect the particle properties. The bioabsorbable particles may be administered to a patient for the treatment of diseases. Methods for making and using the bioabsorbable particles are also provided.

一种靶向Trop2的嵌合抗原受体巨噬细胞及其制备方法与应用

Resumen de: CN119570739A

本发明公开了一种靶向Trop2的嵌合抗原受体巨噬细胞及其制备方法和应用。所述巨噬细胞含有靶向Trop2的特异性CAR和阻断免疫抑制信号的阻断剂；所述靶向乳腺癌的Trop2特异性CAR的氨基酸序列如SEQ ID NO.1所示；所述靶向Trop2的特异性CAR的mRNA的核苷酸序列如SEQ ID NO.2所示。本发明采用LNP共递送CAR mRNA与TLR激动剂，在体编辑巨噬细胞原位生成表达Trop2的CAR‑巨噬细胞，省略了繁琐的工程细胞体外制备过程，并规避了免疫细胞系统回输引发的潜在全身毒副作用。

一种载有mir-873模拟物和mir-20a抑制剂的复合纳米药物及其制备方法和应用

Resumen de: CN119564876A

本发明提供了一种载有mir‑873模拟物和mir‑20a抑制剂的复合纳米药物及其制备方法和应用，属于药物制备技术领域；本发明基于前期研究发现mir‑873及mir‑20a可以作为前列腺癌的复合靶点，并基于mir‑873和mir‑20a两个靶点制备了载有mir‑873模拟物和mir‑20a抑制剂的复合纳米药物；所述复合纳米药物以mir‑873模拟物和mir‑20a抑制剂为活性物质，活性物质外部包裹有复合纳米载体，所述复合纳米载体由阳离子脂质体、内质网膜以及蛙皮素多肽复合得到；所述复合纳米药物以高效靶向前列腺癌，抑制前列腺癌的增殖及转移，且所述复合药物能够和多西他赛复合，进一步地高效抑制前列腺癌，具有很好的实用性。

COMBINATION CANCER TREATMENTS WITH A COMPOSITION COMPRISING AN MTOR PATHWAY INHIBITOR

Resumen de: WO2025049962A1

The present application, in certain aspects, pertains to methods and compositions for the treatment of a breast cancer or a gynecological cancer, using a composition comprising nanoparticles comprising an mTOR inhibitor (such as an allosteric mTOR inhibitor, e.g., sirolimus) and an albumin in combination with a second therapeutic agent, e.g., a PI3K inhibitor (such as gedatolisib or alpelisib), an AKT inhibitor (such as miransertib or capivasertib), or mTOR ATP competitive inhibitor (e.g., sapanisertib).

MICRORNA-15B ANTAGOMIR AND CERIUM OXIDE NANOPARTICLES, AND USES OF SAME FOR TREATMENT OF INTRAUTERINE GROWTH RESTRICTION

Resumen de: WO2025050037A1

The compositions, methods, and systems described herein are useful in treating subjects suffering from or at risk of developing Intrauterine Growth Restriction.

TARGETING LIPID NANOPARTICLES FOR NUCLEIC ACID DELIVERY

Resumen de: WO2025049765A1

Targeting lipid nanoparticles for nucleic acid delivery, In one embodiment, the targeting lipid nanoparticle includes an ionizable lipid, a cholesterol or cholesterol derivative, a helper lipid, a PEG lipid, a targeting lipid, and a nucleic acid. In other embodiments, the targeting lipid nanoparticle including an ionizable lipid, a cationic lipid, a cholesterol or cholesterol derivative, a helper lipid, a PEG lipid, a targeting lipid; and a nucleic acid. In other embodiments, the targeting lipid nanoparticle including an ionizable lipid, a cholesterol or cholesterol derivative, a helper lipid, a PEG lipid, a functionalized lipid; and a nucleic acid. In other embodiments, the targeting lipid nanoparticle including an ionizable lipid, a cationic lipid, a cholesterol or cholesterol derivative, a helper lipid, a PEG lipid, a functionalized lipid; and a nucleic acid. Also provided are kits for preparing targeting lipid nanoparticles and functionalized lipid nanoparticles for nucleic acid delivery.

CIRCULAR POLYETHYLENE GLYCOL LIPIDS

Resumen de: WO2025049690A1

The present application provides, circular polymeric lipid compounds, and methods for preparing and using the same. The circular polymeric lipid compounds can comprise a macrocycle comprising one or more polymer blocks, such as one or more polyethylene glycol (PEG) blocks, and at least one lipid. In some embodiments, one or more lipids are covalently attached to the macrocycle and is outside the macrocycle's backbone. In some embodiments, the macrocycle includes two lipids within the macrocycle's backbone, wherein all the polymeric blocks are between the two lipids. The present application also provides methods of preparing the subject circular polymeric lipid compounds, and their use in lipid nanoparticle formulations.

INTRANASAL FORMULATION FOR PULMONARY DELIVERY OF NANOPARTICLES, COMPOSITIONS, USES, AND MANUFACTURING METHODS THEREOF

Resumen de: WO2025049579A1

The invention provides intranasal formulation for pulmonary delivery of nanoparticles, compositions, uses, and manufacturing methods thereof. In one aspect, an intranasal formulation for intranasal or intrapulmonary administration includes a viscosity agent, an epithelial adherence agent, and a foaming agent. The viscosity agent may include carboxymethylcellulose, the epithelial adherence agent may include poly-lysine, and the foaming agent may include gelatin hydrolysate.

LIPID NANOPARTICLE WITH NUCLEIC ACID CARGO AND IONIZABLE LIPID

Resumen de: WO2025046121A1

Disclosed is a lipid nanoparticle (LNP) encapsulating a nucleic acid cargo preferably comprising messenger ribonucleic acid (mRNA). The LNP comprises at least an ionizable lipid fraction, and a stabilizer fraction. The stabilizer fraction preferably comprises at least one polyethylenglycol (PEG) lipid. Furthermore, the ionizable lipid fraction comprises at least one ionizable glycerol dialkyl glycerol tetraether (GDGT) lipid. Also disclosed is a pharmaceutical composition comprising the LNP, such as an mRNA vaccine. In a further aspect, the invention relates to the ionizable GDGT lipids and methods for producing them.

CIRCULAR RNA ENCODING VEGF POLYPEPTIDES, FORMULATIONS, AND METHODS OF USES

Resumen de: WO2025045142A1

Disclosed herein are immunogenic compositions having circular RNAs encoding VEGF polypeptides. Related methods for manufacture and therapeutic uses thereof are also provided herein.

STABILIZED LIPID AND LIPIDOID NANOPARTICLE FORMULATIONS WITH SPECIFIC SURFACTANT PROPERTIES FOR ENHANCED PHARMACEUTICAL APPLICATIONS

Resumen de: WO2025045767A1

This invention presents novel lipid and lipidoid nanoparticle (LNP and LiNP) formulations enhanced with specifically characterized surfactants. These surfactants exhibit unique Langmuir isotherm properties, ensuring optimal stabilization of the nanoparticles. The described formulations improve the stability, reduce aggregation, and mitigate challenges faced during purification, such as filtration clogging or fouling. Integral to the innovation is a method for determining the suitability of surfactants as stabilizers based on predetermined Langmuir isotherm values and filtration speeds. The stabilized nanoparticles, when formulated with therapeutic agents, have demonstrated potential in medical applications, particularly in the realm of mRNA delivery, vaccination and immunization.

FACTOR VIII (FVIII) GENE THERAPY METHODS

Resumen de: US2025073353A1

Methods of using vectors comprising nucleic acid and nucleic acid variants encoding FVIII protein are disclosed. In particular embodiments, a method of treating a human having hemophilia A includes administering a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding Factor VIII (FVIII) or nucleic acid variant encoding Factor VIII (FVIII) having a B domain deletion (hFVIII-BDD). In some aspects, a nucleic acid variant has 95% or greater identity to SEQ ID NO:7 and/or a nucleic acid variant has no more than 2 cytosine-guanine dinucleotides (CpGs). In other aspects, a rAAV vector is administered to the human at a dose of less than about 6×1012 vector genomes per kilogram (vg/kg).

LIPID NANOPARTICLES FOR OLIGONUCLEOTIDE DELIVERY

Resumen de: US2025073177A1

The current invention relates to ionizable lipid-like compound according to Formula (I) or pharmaceutically acceptable salt, tautomer, or stereoisomer thereof.The present invention also provides a lipid nanoparticle comprising an ionizable lipid-like compound according to Formula I and one or more RNA molecules, as well as a pharmaceutical composition or vaccine, comprising such lipid nanoparticles.

TARGETING THE YTHDF1/ARHGEF2 AXIS FOR CANCER TREATMENT

Resumen de: US2025075210A1

There is described herein methods for the treatment of cancer in a subject comprising downregulating YTHDF1 or ARHGEF2 and also compounds and compositions for achieving the same.

NANOPARTICLES COMPRISING FUSION PROTEIN OF SINGLE-CHAIN VARIABLE FRAGMENT AND FERRITIN, AND USE THEREOF

Resumen de: US2025075003A1

The present invention provides: ferritin; a single-chain variable fragment bound to the N-terminus of the ferritin; and a fusion protein self-assembling nanoparticle structure in which an N-terminus RNA-interaction domain (RID) in a human-derived lysyl-tRNA synthetase is bound to the N-terminus of the single-chain variable fragment by means of a novel fusion partner. Therefore, the present invention provides: a fusion protein having enhanced water solubility; nanoparticles; a vector coding same; a host cell transformed by means of the vector, and a pharmaceutical composition, for treating a disease, using the same.

POLYMER NANOPARTICLES VIA CONDENSED DROPLET POLYMERIZATION

Resumen de: US2025073169A1

Provided is a method of synthesizing polymer particles, including: introducing vapor-phase reagents into a reactor having a substrate; forming condensed droplets of the reagents on the substrate; initiating polymerization; and polymerizing the condensed droplets of the reagents, thereby forming polymer particles. Polymer particles are also provided, including those incorporating therapeutic agents.

PEPTIDES, HYDROGEL COMPOSITIONS AND METHODS OF USE THEREOF

Nº publicación: US2025074956A1 06/03/2025

Solicitante:

REGENTS OF THE UNIV OF MINNESOTA [US]
Regents of the University of Minnesota

Resumen de: US2025074956A1

The invention provides antibiotic BPI Fold Containing Family A Member 2 (BPIFA2) peptides, compositions comprising a BPIFA2 peptide, hydrogels comprising a BPIFA2 peptide, nanofibrillar networks comprising a BPIFA2 peptide, and tissue scaffolds comprising BPIFA2 peptides. The peptide, compositions, hydrogels nanofibrillar networks and tissue scaffolds are useful for medical therapy in an animal.