Alerta

MODIFIED MYOFERLIN PROTEIN AND METHOD OF USE THEREOF TO INCREASE NANOPARTICLE PAYLOAD RELEASE

Resumen de: AU2024382503A1

A modified myoferlin protein capable of being packaged into extracellular vesicles (EVs) to facilitate release of EV payloads into recipient cells. The modified myoferlin protein enhances the release of payload at the targeted site. The present invention further provides a modified myoferlin-encapsulating nanoparticle comprising an exosome encapsulating any embodiment of the modified myoferlin of the present invention and one or more cargo RNAs to enhance release of the cargo RNAs at the targeted site.

LOW-SUGAR CORONAVIRUS VACCINE AND METHODS THEREOF

Resumen de: AU2024359608A1

The present disclosure relates to a low glycosylated spike protein and a vaccine designed to express the spike protein in vivo. The present disclosure also teaches a method for generating an immune response by utilizing the low glycosylated spike protein, which provides a broader protection across different variants. A method for identifying a glycan-shielded conserved peptide of a glycoprotein is also disclosed.

NANOBUBBLES FOR ULTRASOUND MEDIATED NUCLEIC ACID DELIVERY

Resumen de: AU2024356988A1

Gas-filled nanobubbles for negatively charged genetic material delivery each includes a lipid membrane defining a gas containing internal void, wherein the lipid membrane includes a plurality of cationic lipids for complexing the negatively charged genetic material, an edge-activator incorporated between lipids of the membrane that enhances the flexibility of the membrane, and a membrane stiffener incorporated on an outer surface of the membrane that enhances the membrane's resistance to tearing.

IONIZABLE CATIONIC LIPIDS INCORPORATING SILICON: SYNTHESIS AND LNP FORMULATION

Resumen de: WO2025052296A1

The present invention belongs to the field of biomedicine and drug delivery as well as pest and vector controls. The invention relates to a novel ionizable cationic lipid family incorporating silicon, which belongs to the trademark LipexSil® second generation lipids, wherein the tail is connected to the headgroup with biodegradable silyl acetal linker. Lipids containing silyl acetal linker(s) are state-of-the-art and are effective as ionizable cationic lipids in the formulation of empty or loaded lipid nanoparticles (LNPs). The novel linkers according to the invention are designed by means of proprietary borane catalysts WO2022129966. 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, dsRNA, pDNA, micro RNA, circular DNA, small biologically active molecules) into the cells.

COMPOSITIONS AND ARTICLES COMPRISING (NANO) DIAMOND PARTICLES

Resumen de: AU2026202273A1

Abstract Compositions and articles comprising diamond particles, such as nanodiamond-based pharmaceutical compositions, are generally provided. In some embodiments, the articles and methods comprising (nano)diamond particles may be useful for monitoring and/or treating a disease (e.g., in a subject).

PH-INDUCIBLE STRUCTURE-SWITCHING LIPID NANOVECTORS, SEMI-SYNTHETIC EXTRACELLULAR VESICLES, METHODS OF MAKING SAME AND USES THEREOF

Resumen de: AU2024336703A1

The present disclosure provides a pH-inducible structure-switching non-lamellar lipid nanovector (LNV) comprising: (a) at least one ionizable cationic lipid; (b) at least one phospholipid displaying a critical packing parameter (CPP) value > 1; and (c) at least one non-ionic surfactant displaying a CPP value < 1 at a molar concentration of between 20 % and 50 %, a method of making the LNV, a semi-synthetic extracellular vesicle (ssEV) resulting from the fusion of the LNV with extracellular vesicles at a pH higher than 6 and up to about 10, a kit and a use of the ssEV as a medicament or diagnostic agent.

POLYMER-GEKOPPELTE ANTIKÖRPER UND ANTIKÖRPERFRAGMENTE

Resumen de: DE102024129442A1

Die Erfindung betrifft ein Konjugat umfassend mindestens ein Polymer und mindestens einen Antikörper oder mindestens ein Antikörperfragment, wobei das Polymer ausgewählt ist aus Poly(dimethyl acrylamid) (PDMA), Poly(oligoethylen glykol methacrylat) (POEGMA), Poly(2-hydroxypropyl methacrylamid) (PHPMA), Poly(vinylpyrrolidon) (PVP), Poly(2-methylsulfinyl)ethyl acrylat (PMSEA), Poly(6-O-methacyloyl-D-galactopyranose) (PMAGP), Acrylsäure (PAA), Poly(carboxybetain acrylat) (PCBA), Poly(vinylphosphonsäure) (PVPA), Poly(vinylbenzoesäure) (PVBzA), Poly(propylacrylsäure) (PPAA), Poly(styrolsulfonsäure) (PSS), Polyglycerol (PG) und Polyäpfelsäure (PMA) und das mindestens eine Antikörperfragment vorzugsweise mindestens einen Einzeldomänenantikörper (Nanobody) umfasst. Die Erfindung betrifft ferner die medizinische Verwendung des Konjugats, sein Herstellungsverfahren und Kits für dessen Anwendung.

NEOEPITOPE VACCINE DELIVERY VEHICLE AND METHODS OF MAKING THE SAME

Resumen de: AU2026202277A1

Abstract Disclosed therein are mannan nanogels as a novel vaccine delivery platform as well as a novel method of making a self-assembling mannan nanogel for in vivo delivery of therapeutic agents.

Lipid Nanoparticles For Delivery Of Nucleic Acids, And Related Methods Of Use

Resumen de: AU2025271161A1

The present disclosure provides for improved compositions of ionizable lipid nanoparticles for the delivery of therapeutic nucleic acids to cells. Cationic ionizable lipids are engineered with improved stability to oxidative degradation while in storage, while retaining high transfection activity or potency in cells. These lipids are designed to be biodegradable, thus improving the tolerability of nanoparticles formed with them in vivo. In addition, targeting of these nanoparticles in a highly specific manner to dendritic cells is provided for through inclusion of antibody conjugates directed against cell surface receptors. ov o v

MATERIAL FOR LOCAL DRUG DELIVERY, AND USE THEREOF

Resumen de: AU2024342596A1

The present invention relates to a material for local drug delivery, and use thereof. Specifically provided is a nanoparticle composition comprising a lipid component, the lipid component comprising a compound represented by formula (I). The nanoparticle composition of the present invention is delivered only at the site of administration.

TRICINE AND CITRIC ACID-BASED CATIONIC LIPIDS WITH AROMATIC HEAD GROUPS

Resumen de: WO2024256457A1

The present invention provides, in part, tricene and citric acid-based cationic lipids with aromatic head groups of Formula (I), and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

A NANOPARTICLE COMPOSITION AND RELATED METHODS THEREOF

Resumen de: WO2024253582A1

There is provided a nanoparticle composition comprising a compound represented by general formula (1) or ionized form thereof, wherein R1, R2, and R4 to R8 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl, R3 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene, and R9 and R10 are each independently a hydrophobic tail or contains at least one of the groups defined above for R4 to R8; a therapeutic, prophylactic, and/or biological agent that is encapsulated by the compound of general formula (1) to form nanoparticles; and a cryoprotectant. There is also provided a method of preparing said nanoparticle composition.

IONIZABLE LIPID COMPOUND, LIPID CARRIER COMPRISING SAME AND USE THEREOF

Resumen de: EP4725938A1

The present invention relates to an ionizable lipid compound, a lipid carrier comprising same and the use thereof. Provided in the present invention are a series of compounds as represented by formula (1), a lipid carrier comprising same as an ionizable lipid, a nucleic acid lipid nanoparticle composition and a preparation thereof. A lipid nanoparticle formed from the ionizable lipid can deliver a nucleic acid molecule into the body, so that the transfer rate of a nucleic acid molecule is increased, and the treatment effect of a nucleic acid drug is improved.

LIPID-POLYMER HYBRID NANOPARTICLES

Resumen de: WO2024252406A1

Present disclosure describes a lipid polymer hybrid nanoparticle and a method to synthesize such nanoparticles or such nanoparticle-containing compositions. The nanoparticles are made of biodegradable polymer based micellar core surrounded by lipid-based shell, wherein majority of a pharmaceutical agent is present on the inner periphery of such nanoparticles due to physical adherence with the lipid molecules. Only a minor amount of the pharmaceutical agent is encapsulated in the micellar core. Hence, the lipid-based shell becomes a primary excipient part of the nanoparticle and the biodegradable polymer containing core becomes a secondary excipient part of the nanoparticle.

RNA-LOADED LIPID NANOPARTICLES

Resumen de: WO2024253741A1

Compositions are provided that include a lipid nanoparticle (LNP);a self-replicating RNA encoding a gene of interest loaded within the LNP; and an inhibitory nucleic acid, such as a small interfering RNA (siRNA) targeting IFN-α/β receptor l(Ifharl) gene loaded within the LNP, and use of the compositions for generating an immune response.

POLYMERIC PRODRUG NANOPARTICLE FOR TUMOR-TARGETED ULTRASOUND-ACTIVATED CANCER THERAPY

Resumen de: EP4725504A1

A polymeric prodrug nanoparticle for the targeted accumulation and activation in tumor tissue, consisting of the following monomers:a) at least one platinum(IV) complex andb) at least one sonosensitizer, andc) a compound having a hydrophilic group providing this hydrophilic group at each terminal end of the prodrug nanoparticle, wherein a), b) and c) are each covalently bonded to one another by means of a linker and a) and b) are arranged stochastically distributed within the polymeric prodrug nanoparticle can be used to treat tumors and in particular metastases in a way that is well tolerated by the body. Upon irradiation of the polymeric prodrug nanoparticle with ultrasound, the platinum(IV) prodrug is activated in the tumor region, resulting in the release of cytotoxic platinum and killing of tumor cells.

SUBMICRON PARTICLE COMPRISING A PEPTIDE ACTIVE, PREPARATION METHOD AND USES THEREOF, IN PARTICULAR COSMETIC USES

Resumen de: FR3149503A1

La particule submicronique est sensiblement sphérique comprenant un cœur huileux renfermant l’actif peptidique et une enveloppe de cire solide à température ambiante. La particule est stabilisée par une couche extérieure d’un tensio-actif non-ionique à haut HLB. L’invention propose un procédé pour obtenir une suspension aqueuse desdites particules présentant un taux d’encapsulation satisfaisant et une stabilité à long terme améliorée, pour une utilisation notamment dans l’industrie des cosmétiques. Des combinaisons de peptides sont possibles intégrées à l’intérieur des particules et adsorbés à l’extérieur. Fig. 1

EFFICIENT CATIONIC POLYESTER AND USE THEREOF

Resumen de: EP4725977A1

Disclosed in the present invention are an efficient cationic polyester and the use thereof. The cationic polyester of the present invention is a hyperbranched polymer formed by means of polymerizing three monomers of monomer P, monomer S and monomer M, or two monomers of monomer P and monomer S, with monomer T. The hyperbranched polymer has an end group modified with group E, and the group E has a relative concentration of 0.48-0.75 to group E of a hyperbranched polymer with complete end-group modification. Monomer P is cyclic lactone. Monomer S is an organic acid with an end group containing two or more carboxyl. Monomer M is a compound containing two hydroxyl and one secondary amine or tertiary amine. Monomer T is a compound containing at least three hydroxyl. The end-group modifying compound E which provides group E modification is a compound containing at least one primary amine, secondary amine or tertiary amine group. The cationic polyester of the present invention only has a part of the end groups modified with group E, has higher nucleic acid transfection efficiency when being used for nucleic acid drug delivery, has low cytotoxicity, and can be used in clinical practice.

BACKPACKED-ANTIBODY CONSTRUCTS AND THEIR USES

Resumen de: WO2024251979A1

The present invention is comprised within the fields of molecular biology and biotechnology. It specifically relates to polypeptides for the generation of Backpacked-antibody (B-ab) molecules for a variety of uses, in particular, for the specific delivery of drugs to tumor cells.

Veterinary compositions

Resumen de: GB2644423A

The invention relates to sterile veterinary formulations for treating or preventing mastitis in cattle. The formulations comprise a suspension of Derivatised Microparticles, optionally in combination with Microparticles, in an amount of 5 percent to 60% w/v. The Derivatised Microparticles and Microparticles are biodegradable and are formed from an organic polycationic polymer and a saturated or unsaturated fatty dicarboxylic acid. Suitable polymers may include organic polymeric amines, quaternary ammonium compounds, polypeptides and carbohydrates, with preferred antimicrobial polymers such as nisin, ε‑polylysine or chitosan. In preferred embodiments, the microparticles are formed from sebacic acid and ε‑polylysine and the fatty dicarboxylic acid to polymer molar ratio is around 1:1. The suspension may comprise 20 % to 50 % microparticles, preferably around 30 %, in a veterinary acceptable carrier such as water, mineral oil, liquid paraffin or white soft paraffin. The microparticles may be present as a mixture, with the ratio of Derivatised Microparticles to Microparticles ranging from 90:10 to 10:90. The formulation may be administered during the dry period of the cow and provides an effective method for treating or preventing mastitis. The invention further includes mixtures of Derivatised Microparticles and Microparticles and veterinary formulations containing such mixtures.

Peptide for the delivery of anionic materials

Resumen de: GB2644439A

The invention provides a peptide or a salt or amide thereof, for use as a cell delivery agent, comprising, or consisting of: (Xaa1)a-(Xaa2)b-LYRLFRKS-(Xaa3)c-(Xaa4)d-(Xaa4)e-NLKPFERHARAC, wherein: a, b, and c can be either 0 or 1; d and e are 1; Xaa1-2 represent Ala, Val or Gly; and Xaa3-5 represent His or Trp. An exemplary sequence, AALYRLFRKSWHNLKPERHARAC, when combined with mRNA, forms 103 nm nanoparticles with a charge of 19 mV and polydispersity index of 0.06, and can transfect NCTC-929 cells with an efficiency of up to 81%. The particles are also shown to encapsulate miRNA and plasmid DNA with an encapsulation efficiency up to 95%. Also claimed is the first medical use of the peptide in gene therapy, and second medical use of the peptide in the treatment of prophylaxis, infection, cancer and wounds.

RSV疫苗

Resumen de: WO2025017142A1

Provided herein is a vaccine against RSV. The vaccine comprises an mRNA encoding a stabilised prefusion RSV F protein immunogen linked to a scaffold based on lumazine synthase.

CD177靶向膜修饰脂质体在制备预防和/或治疗系统性红斑狼疮的药物中的应用

Resumen de: CN121846047A

本发明公开了CD177靶向膜修饰脂质体在制备预防和/或治疗系统性红斑狼疮的药物中的应用。所述CD177靶向膜修饰脂质体，其结构分为三层，包括：负载PADI4抑制剂的脂质体、在所述脂质体的表面偶联CD177靶向肽形成的靶向识别层、以及最外层包覆天然中性粒细胞膜形成的仿生功能层。在咪喹莫特（IMQ）诱导的狼疮模型中，所述药物治疗显示出强大的疾病缓解能力：1）改善全身表型：显著缩小狼疮小鼠肿大的脾脏和淋巴结。2）修复肾脏功能：显著缓解肾小球肾炎，减少肾小球内炎性细胞浸润、IgG及补体C3沉积。3）显著降低血清中促炎细胞因子水平及狼疮标志物抗dsDNA抗体的滴度。

一种肉桂醛纳米颗粒的制备方法及其应用

Resumen de: CN121846049A

本发明公开了一种肉桂醛纳米颗粒制备方法及其应用，制备方法以肉桂醛为功效成分，玉米醇溶蛋白为载体，酪蛋白酸钠为稳定剂，果胶为稳定剂和粘合剂，制备获得所述肉桂醛纳米颗粒。经试验验证，本发明制备的肉桂醛纳米颗粒能显著提高肉桂醛的稳定性，同时增强其对于金黄色葡萄球菌的抗菌活性，为肉桂醛的开发利用提供了更多选择。

一种修饰可电离胺基的双介孔二氧化硅纳米颗粒及其制备方法和应用

Nº publicación: CN121846313A 14/04/2026

Solicitante:

华东理工大学

Resumen de: CN121846313A

本发明公开了一种修饰可电离胺基的双介孔二氧化硅纳米颗粒及其制备方法和应用，其核心技术流程包括：首先通过嵌段共聚物与表面活性剂的双模板法合成介孔二氧化硅；然后将可电离胺基通过硅烷偶联剂接枝到介孔二氧化硅表面；最后利用静电吸附将RNA负载到修饰后的介孔二氧化硅上，最终实现肿瘤高效治疗。本发明还公开一种上述二氧化硅纳米颗粒在RNA递送中的应用，根据本发明方法制备的二氧化硅纳米颗粒有着高效的RNA负载效率以及细胞摄取效率。根据本发明制备的修饰可电离氨基团的双介孔二氧化硅纳米颗粒具有药物负载量大、细胞摄取率高、生物相容性高、合成简单、反应条件温和、便于裁剪等优点，具有良好的应用前景。