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POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE

Resumen de: US2025170070A1

This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE SIGNAL REGULATORY PROTEIN ALPHA (SIRPa) GENE

Resumen de: US2025171786A1

Provided herein, in various embodiments, are methods and compositions comprising a Signal Regulatory Protein Alpha (SIRPα) therapeutic. In certain embodiments, the disclosure provides for methods and compositions for modulating SIRPα expression. In certain embodiments, the methods and compositions are useful in the treatment of a SIRPα-mediated disease or condition.

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.

CABAZITAXEL PRODRUG ANTI-TUMOR PREPARATION

Resumen de: AU2022412640A1

The present invention relates to a cabazitaxel prodrug anti-tumor preparation, designs and synthesizes a cabazitaxel-fatty alcohol small molecule prodrug containing different fatty alcohol side chains and different connecting chains of general formulas (I), (II) and (III), and prepares a self-assembly nanoparticle. A result shows that the cabazitaxel-fatty alcohol small molecule prodrug self-assembly nanoparticle can effectively improve a curative effect of cabazitaxel and reduce toxic and side effects thereof. The length of a side chain of a branched chain fatty alcohol, the structure of the side chain of the fatty alcohol, the composition of a connecting chain element and the length of a connecting chain can significantly affect the pharmaceutical property and the anti-tumor activity of the cabazitaxel prodrug self-assembly nanoparticle. The prodrug self-assembly nanoparticle has higher anti-tumor activity and lower toxicity than a cabazitaxel-linear fatty alcohol small molecule prodrug self-assembly nanoparticle.

OPTIMIZED ENGINEERED MEGANUCLEASES HAVING SPECIFICITY FOR A RECOGNITION SEQUENCE IN THE HEPATITIS B VIRUS GENOME

Resumen de: US2025171757A1

The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and/or efficiency of indel formation, when compared to the first-generation meganuclease HBV 11-12x.26. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or hepatocellular carcinoma.

Cancer vaccines

Resumen de: AU2025203258A1

Abstract The disclosure relates to cancer ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.

PEPTIDE CONJUGATED PARTICLES

Resumen de: AU2025203403A1

0324 The present invention provides compositions comprising peptide-coupled biodegradable poly(lactide-co-glycolide) (PLG) particles In particular, PLG particles are surface-functionalized to allow for coupling of peptide molecules to the surface of the particles (e.g., for use in eliciting induction of immunological tolerance).

LIPID NANOPARTICLE DRUG CONJUGATES

Resumen de: AU2023375377A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

HYALURONIC ACID DERIVATIVE DRUG COMPOSITION AND DRUG COMPOSITION

Resumen de: EP4559483A1

A hyaluronic acid derivative pharmaceutical composition includes an active ingredient suspended in an aqueous carrier containing a hyaluronic acid derivative, in which the active ingredient is a fine particle having a molecular weight of 100 g/mol or greater and 8000 g/mol or less. It is preferable that the fine particle is a microparticle or a nanoparticle.

PH-SENSITIVE CATIONIC LIPID AND LIPID NANOPARTICLE

Resumen de: EP4559896A1

The present invention addresses the problem of providing, without using relatively expensive starting materials, a more conveniently synthesizable pH-sensitive cationic lipid and a lipid nanoparticle that contains this pH-sensitive cationic lipid. The present invention is a pH-sensitive cationic lipid represented by general formula (I). R¹ is a C1-22 hydrocarbon group; the three R¹ groups in one molecule may be the same as or different from each other; Z¹ is a C1-6 alkylene group or a single bond; X is -N(R²)(R³) or is a 5- to 7-membered nonaromatic heterocyclic group (with the proviso that this group is bonded to Z¹ via a carbon atom); and R² and R³ are each independently a hydrogen atom or a C1-4 hydrocarbon group, or R² and R³ may be bonded to each other to form a 5- to 7-membered nonaromatic heterocycle.

NEUTRAL LIPID AND LIPID NANOPARTICLE

Resumen de: EP4559895A1

The present invention provides: a neutral lipid capable of suppressing the phase transition of endosomal membranes caused by phosphatidylcholine from being inhibited; and a lipid nanoparticle containing the neutral lipid. The present invention pertains to an ionic neutral lipid comprising a compound represented by general formula (I). In formula (I), R¹ is a C1-22 hydrocarbon group; three R¹ groups in one molecule may be the same as or different from each other; a1 and a2 are each independently an integer of 0 to 4; b1 and b2 are 0 or 1, satisfying b1+b2=1; R² and R³ are each independently a hydrogen atom or a C1-3 alkyl group; and R4 is an anionic group.

がん送達用医薬組成物または免疫賦活用組成物

Resumen de: TW202404641A

The present invention addresses the problem of providing lipid nanoparticles that are useful as a pharmaceutical composition for delivery to cancer or as an immunostimulating composition. The problem is solved by a pharmaceutical composition for delivery to cancer, an immunostimulating composition, or the like that contains: a pH sensitive cationic lipid represented by formula (I); a stereoisomer thereof; or a stereoisomer mixture. In formula (I), 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) (in formula (A), R11 and R12 each independently represent a straight chain or branched chain C1-15 alkyl group; c represents 0 or 1; v represents an integer of 4-12); X represents a group represented by general formula (B) (in formula (B), d represents an integer of 0-3; and R3 and R4 each independently represent a C1-4 alkyl group or a C2-4 alkenyl group (said C1-4 alkyl group or C2-4 alkenyl group may have one or two hydrogen atoms substituted with phenyl groups), but R3 and R4 may be bonded to each other to form a 5 to 7-membered non-aromatic hetero ring (said ring may have one or two hydrogen atoms substituted with a C1-4 alkyl group or a C2-4 alkenyl group) or a 5 to 7-membered non-aromatic heterocycle group (where said group is bonded to (O-CO)b- via a carbon atom, and one or two hydrogen atoms of said ring may be substituted with a C1-4 alkyl group or a C2-4 alkenyl group).

AMINO LIPID COMPOUND, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: EP4559894A1

The present application relates to an amino lipid compound having the following structural formula (I) or a pharmaceutically acceptable salt or stereoisomer thereof, and use thereof as a component of a lipid nanoparticle preparation for delivering a therapeutic agent. The present application further relates to a composition comprising the amino lipid compound, and particularly, to a lipid nanoparticle, and use thereof.

NOVEL IONIZABLE CATIONIC LIPIDS CONTAINING THIOETHER LINKAGE

Resumen de: EP4559898A1

The invention relates to novel ionizable amine lipids incorporating one or more sulphur atoms in the tail section. These lipids can be used in combination with other components to form lipid nanoparticles with oligonucleotides. The invention describes the synthesis of the lipids of formula (I), formation and characterization of nanoparticles and biological experiments demonstrating that the lipid nanoparticles prepared with these novel lipids can efficiently deliver their cargo (e.g. RNA, DNA, mRNA, siRNA, miRNA, pDNA, circular DNA, dsRNA, small biologically active molecules) into the cells.

LIPID FORMULATIONS

Resumen de: CN119604279A

A method of making hybrid lipid particles suitable for delivery of brittle active ingredients, such as nucleic acids, the method comprising the steps of preparing hybrid lipid particles and then "spraying" their surfaces with particles of an inorganic material. The invention also relates to hybrid lipid particles, uses and products, characterized in that the inorganic material particles are present on and in the lipid particles instead of being encapsulated by the lipid particles, and the active ingredient does not need to be encapsulated.

ENVELOPED VIRUS LIKE PARTICLES COMPRISING SARS-COV-2 S PROTEIN

Resumen de: WO2024018364A1

Provided is an enveloped virus-like particle (eVLP) comprising a substantially full-length recombinant SARS-CoV-2 spike (S) protein. The eVLP may further comprise an additional recombinant SARS-CoV-2 S protein having a different sequence, another recombinant viral antigen, or a recombinant non-viral protein. The eVLP is derived from an animal cell, such as a CHO cell, expressing the recombinant SARS-CoV-2 spike protein. Also provided are methods of producing such eVLPs, compositions including such eVLPs, and methods and uses for the induction of an immune response against a SARS-CoV-2 spike protein and/or prevention of COVID-19 or SARS-CoV-2 infection, employing such eVLPs.

胸腺靶向肽基可离子化脂质及其纳米颗粒

Resumen de: CN120040543A

本发明涉及胸腺靶向肽基可离子化脂质及其纳米颗粒。更具体而言，本发明涉及肽基可离子化脂质、包含其的胸腺靶向的脂质纳米颗粒及其用途。

一种载SOX9 siRNA脂质纳米粒、其制备方法及在治疗结直肠癌中的应用

Resumen de: CN120037203A

本发明公开了一种载SOX9siRNA脂质纳米粒，该脂质纳米粒由肿瘤靶向载体材料和SOX9siRNA组成。其中，所述的肿瘤靶向载体材料由cRGDfK肽修饰的磷脂‑聚乙二醇2000(DSPE‑PEG2000‑cRGDfk)、4‑(N,N‑二甲基氨基)丁酸(二亚油基)甲酯(DLin‑MC3‑DMA)、二硬脂酰磷脂酰胆碱(DSPC)、二肉豆蔻酰甘油‑聚乙二醇2000(DMG‑PEG2000)和胆固醇组成。本发明制备的载SOX9siRNA脂质纳米粒能被结直肠癌细胞摄取，实现溶酶体逃逸，抑制结直肠癌细胞的增殖、迁移和侵袭。同时在结直肠癌荷瘤小鼠体内也具有良好的肿瘤靶向性、抗肿瘤活性和安全性。

治療薬の細胞内送達のための分岐状尾部脂質化合物及び組成物

Resumen de: MX2022003269A

The application relates to lipids of Formula (A), Formula (B) and Formula (1-1), and to lipid nanoparticles (empty or loaded LNPs) including such a lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用

Resumen de: CN120037202A

本发明公开了一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用，属于医药技术领域。所述所述脂质纳米颗粒的原料组成包括：连接处含有分支结构的阳离子脂质、辅助脂质、胆固醇，所述阳离子脂质的结构式如式(Ⅰ)所示。本发明提供的基于连接处含有分支结构的可电离阳离子脂质的脂质纳米颗粒，可以在动物体内有效地递送如mRNA等核酸类药物。相较于市售的可电离脂质SM‑102及其配方，本发明提供的脂质纳米颗粒在动物体内具有更好的mRNA递送效率，有利于改善mRNA等核酸药物的临床应用效果。

用于递送生物活性成分的氨基脂质化合物和脂质纳米颗粒

Resumen de: AU2023359279A1

Provided are an amino lipid compound for preparing a lipid nanoparticle for delivering an active ingredient and a preparation method therefor, a lipid nanoparticle and a pharmaceutical composition containing the amino lipid compound, and the use thereof.

一种具有内质网靶向及免疫激活功能的聚合物-金属离子复合物纳米颗粒

Resumen de: CN120037201A

本发明公开了一种具有内质网靶向及免疫激活功能的聚合物‑金属离子复合物纳米颗粒，所述含氧化三级胺的两性离子聚合物的结构式为I所示#imgabs0#，其中，X为聚合单元，包括但不限于（甲基）丙烯酸酯（或）酰胺，氨基酸等；Y为N‑氧化三级胺连接基团，R1和R2选自甲基、乙基、丙基、丁基、戊基、己基，n=3‑300；所述具有免疫激动剂功能的为金属离子，金属离子选自锰离子、钙离子、锌离子、铁离子、铂离子中的一种。本发明将纳米颗粒有效递送至内质网并激活cGAS‑STING通路，增强抗肿瘤免疫反应，抑制肿瘤生长。

一种GSH响应型限域聚集金纳米囊泡及其制备方法和在放射增敏中的应用

Resumen de: CN120037370A

本发明提供一种GSH响应型限域聚集金纳米囊泡及其制备方法和在放射增敏中的应用，所述金纳米囊泡为介孔二氧化硅包被AuNP@MnO2Ve@mSiO2，通过在AuNPs表面原位形成具有GSH响应性的MnO2，得到AuNP@MnO2；接着在AuNP@MnO2表面修饰上磷酸‑聚苯乙烯嵌段共聚物P‑PS；然后将其进行自组装，获得等离子体金纳米囊泡AuNP@MnO2Ve；最后用介孔二氧化硅包覆在AuNP@MnO2Ve表面进行修饰稳定，最终得到GSH响应型限域聚集金纳米囊泡AuNP@MnO2Ve@mSiO2；所述金纳米囊泡在放射治疗增敏剂中的应用。本发明制备的纳米金囊泡通过优化AuNPs的颗粒大小，特别是可通过GSH响应限域聚集策略改变AuNPs颗粒间的间距，增强其放射增敏效果，从而减少放疗辐射剂量，可有效地较少肿瘤细胞的放射抗性，避免X射线损伤相邻的正常组织。

一种pH和光热双响应性聚多巴胺纳米载药微球及其制备方法和应用

Resumen de: CN120037375A

本发明提供了一种pH和光热双响应性聚多巴胺纳米载药微球及其制备方法和应用，涉及医药技术领域。本发明提供的pH和光热双响应性聚多巴胺纳米载药微球，包括疏水性药物、包覆所述疏水性药物的聚多巴胺和接枝在所述聚多巴胺表面的聚甲基丙烯酸N,N‑二甲基氨基乙酯。该载药微球利用肿瘤微环境酸性与正常生理条件的pH差异设计，不依赖肿瘤特异性抗原，因此具有广泛的适用性和较低的脱靶风险。本发明提供的载药微球具有优异的pH和光热响应性及良好的生物相容性，其能够在具有酸性微环境的肿瘤部位聚集并迅速释放药物，达到靶向治疗的目的，具有靶向肿瘤的普遍适用性；同时在局部近红外光照射下，发挥光热治疗作用，协同药物增强抑瘤作用。

ピコルナウイルスをコードするＲＮＡポリヌクレオチドの製造

Nº publicación: JP2025516241A 27/05/2025

Solicitante:

エレベートバイオテクノロジーズ，インコーポレイテッド

Resumen de: CN119233824A

The present disclosure relates to the use of ribozyme to generate a recombinant RNA molecule encoding an oncolytic viral genome, such as a picornavirus, which recombinant RNA molecule has the native 5 '-terminus of the viral genome The disclosure also relates to the design of a corresponding DNA template and the use of the recombinant RNA molecule and/or a corresponding particle for the treatment and prevention of cancer.