Resumen de: US2023201363A1
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
Resumen de: CN118662475A
本发明提出了一种纳米载体,所述纳米载体包括:纳米球内核,所述纳米球内核包括角鲨烯;以及纳米球外壳,所述纳米球外壳包括:表面活性剂;所述纳米球内核与纳米球外壳通过疏水作用结合。本发明制备的纳米载体表面性质可进行连续调控,由其制备的疫苗具有高效、低毒、便于制备和储存等优点。克服了传统抗原载体抗原特异性T细胞诱导效率低等弊端。
Resumen de: CN118662631A
本发明属于抗菌材料技术领域,具体涉及一种抗生物膜感染的二氧化锰纳米复合材料及其制备方法与应用。我们将光敏剂(Photosensitizer,PS)与牛血清白蛋白二氧化锰(BSA@MnO2)复合纳米颗粒结合,随后在其表面用壳聚糖季铵盐(HACC)修饰得到BSA@MnO2@PS@HACC纳米复合抗菌材料。该抗菌纳米材料表面修饰的HACC具有细菌靶向性可以与细菌特异性结合,高度促进该抗菌纳米材料和细菌之间的相互作用。本发明实现了光热光动力联合抗生物膜的效果,有效提高抗菌效率。建立了一种能够缓解缺氧增强光动力治疗的靶向抗菌纳米颗粒用于治疗生物膜感染。且该抗菌纳米材料生物相容性良好,生物安全性高,非常适合代替抗生素治疗细菌感染。
Resumen de: CN118666931A
本发明属于生物医药技术领域,涉及基于胆酸类化合物的可电离脂质及其制备方法、组合物与其应用。其化学结构如式(I)所示;#imgabs0#X1、X2、X3分别独立选自氢原子或羟基;Y选自‑O‑、‑NH‑;n为1~8的整数;根据X1、X2、X3的选择,R1、R2分别独立选自取代或未取代的烷基、取代或未取代的烯基、取代或未取代的炔基。本发明提供的基于胆酸类化合物的可电离脂质具有酯键或酰胺键,具有良好的生物可降解性,同时将其制成脂质纳米颗粒,包载核酸药物的效率高、转染效果好。
Resumen de: CN118662479A
本发明涉及抗菌药物纳米递药系统技术领域,具体涉及一种靶向铜绿假单胞菌凝集素的仿生多功能纳米制剂及其制备方法和应用。靶向仿生多功能纳米制剂包括脂质外壳,脂质外壳内包载有纳米载体和抗生素药物。纳米载体由环糊精连接香豆素硼酸基衍生物形成,具有ROS响应的特性,在ROS催化条件下,释放具有抑菌活性的7‑OH香豆素,并且通过破坏纳米粒结构来实现抗菌药物的释放。本技术方案以铜绿假单胞菌特异性凝集素LecA为靶标,在纳米载体表面修饰特异性配体半乳糖,构建主动靶向细菌的仿生型纳米递送系统。本方案可以解决现有抗菌纳米药物递送系统靶向性低、药物释放部位无选择性及对生物膜内细菌杀伤效果差的技术问题。
Resumen de: US2022401364A1
The invention provides novel compositions of fluorocarbon nanoemulsions comprising one or more of fluorosurfactants and phospholipids, and methods of preparation and use thereof for enhanced oxygen delivery.
Resumen de: WO2023084510A1
Particulate compositions comprising low molecular weight chitosan and ss or dsRNA partially complementary to, binding to or at least 90% identical to mRNA targets of viruses pathogenic in farmed crustaceans, compositions and farmed aquatic crustaceans comprising the same, and methods for their use in treating or preventing viral infection in aquaculture are provided.
Resumen de: CN118077010A
A system is provided in one example, the system comprising one or more processors to receive a sequence data structure; applying at least one first metric of the plurality of metrics to the sequence data structure to generate at least one first metric score; determining that the at least one first metric score meets a first condition; in response to the at least one first metric score satisfying a first condition, applying at least one second metric of the plurality of metrics to the sequence data structure to generate at least one second metric score; and outputting an indication of the at least one first metric score and the at least one second metric score.
Resumen de: US2024307317A1
Hypoimmunogenic induced pluripotent stem cell (iPSC)-derived exosomes including tailored chimeric antigen receptor (CARs) which can recognize target biomarkers through an antibody fragment scFV region, bifunctional or ByTE antibodies, by a viral epitope recognition receptor (VERR), VHH nanobody, Variable New Antigen Receptor (VNAR), engineered TCR, or by any single heavy chain IgG fragment from which a variable region can be engineered. A method of making exosomes. A method of treating an individual with cancer, by administering the exosomes to an individual, targeting cancer cells, and treating the cancer. Exosomes including tailored CARs which can recognize target biomarkers through a VERR including viral receptors of an oncolytic virus. A method of treating an individual with cancer, by administering exosomes including CAR receptors having a VERR, VHH nanobody, VNAR, engineered TCR, or by any single heavy chain IgG fragment from which a variable region can be engineered with viral receptors of an oncolytic virus to an individual, targeting cancer cells, and treating the cancer. A method of targeting cells in an individual, by administering the exosomes to an individual, and targeting cells to be destroyed or treated for cancer tumors (both liquid and solid), infectious disease, hereditary conditions, autoimmune disease, or metabolic disorders.
Resumen de: WO2024189572A1
The present invention discloses carriers with an onco-suppressive agent and selectively directed to a tumoral target for the treatment of metastatic melanoma resistant to a target therapy.
Resumen de: US2024307309A1
A lipid nanoparticle composition comprising: (i) at least one zwitterionic polymer-containing lipid in which a lipid moiety is covalently attached to a zwitterionic polymer; (ii) at least one non-cationic lipid selected from charged and uncharged lipids, but not attached to a polymer; (iii) at least one cationic or ionizable lipid; and (iv) at least one therapeutic substance, and optionally (v) cholesterol or derivative thereof. Also described herein are methods of delivering a therapeutic substance to a subject, the method comprising administering to the subject a lipid nanoparticle composition described above.
Resumen de: US2024307306A1
This invention relates to a composition for ophthalmic delivery of a therapeutic agent, the composition comprising an oil-in-water (o/w) microemulsion comprising a fatty acid, or fatty acid ester, as the oil phase; an aqueous phase; a surfactant; and a co-surfactant, and wherein the composition further comprise a suspension of therapeutic agent-loaded nanoparticles. Use of the composition for the treatment or prevention of an eye disorder, a method of treatment or prevention of an eye disorder and an eye drop dispenser are also provided.
Resumen de: US2024307299A1
Provided herein are topical ophthalmic preparations which comprise a non-aqueous, self-emulsifying system which can spontaneously give rise to either nanosized emulsions upon contact with an aqueous phase. Also provided herein are methods for the preparation of the same and their use in formulating and delivering poorly water soluble drugs.
Resumen de: US2024307319A1
Disclosed in the present invention are an ionizable lipid molecule, a preparation method therefor, a composition containing same, an application thereof in the preparation of a vector for delivering a nucleic acid to a cell, and an application thereof in the preparation of a lipid nanoparticle (LNP). By improving the structure of the ionizable lipid molecule and adjusting an LNP component solution, the effect of an mRNA-LNP preparation is improved. The ionizable lipid molecule of the present invention has a structure of formula (I). The ionizable lipid molecule is synthesized with phospholipid, cholesterol, and polyethylene glycol by means of microfluidics to obtain the LNP, and the obtained LNP can improve the translation expression level of a load-mRNA in the cell, improve the effect of the mRNA-LNP preparation, and provide a theoretical basis for theoretical treatment of the personalized mRNA-LNP preparation.
Resumen de: US2024307307A1
According to one embodiment, a lipid composition is for delivering an objective substance to a target cell. The composition is contacted with the target cell under an environment of 37° C. or higher. The composition includes a substance delivery carrier having lipid particle and an objective substance encapsulated in the lipid particle. The lipid particle constitutes a liposome and includes FFT-10 and/or FFT-20 as constituents thereof.
Resumen de: US2024309366A1
Provided herein are compositions and methods for treating metabolic disorders, such as hepatic steatosis, adipose tissue dysfunction, and insulin resistance. Small interfering RNAs (siRNAs) targeting LPS-binding protein (LBP) that include unlocked nucleotides (UNA) and their therapeutic applications for the treatment of metabolic disorders are provided herein.
Resumen de: US2024309367A1
To treat disease, DNA and RNA therapeutics need to be delivered to target tissues and provide lasting benefit without side effects. Lipid nanoparticle spherical nucleic acids address this unmet need by using DNA and RNA sequences for nanoparticle targeting and tissue specificity. The lipid SNA structure has markedly different biodistribution properties than both lipid particles (loaded with nucleic acid) or even conventional SNAs (liposome and gold core).
Resumen de: US2024309382A1
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.
Resumen de: US2024309376A1
Compositions of polynucleotide(s), pharmaceutical compositions thereof, and methods of use thereof are disclosed. A polynucleotide may be or encode a synthetic transfer ribonucleic acid (tRNA). The polynucleotide may be assembled with a lipid composition for delivery to a cell or an organ, such as a lung cell or a lung of a subject. Methods for enhancing an expression or activity of cystic fibrosis transmembrane conductance regulator (CFTR) protein in a cell are provided. Methods for treating a subject having or suspected of having a CFTR-associated condition are also provided.
Resumen de: US2024309291A1
The present disclosure provides compounds useful as ionizable cationic lipids. The ionizable cationic lipids are useful for preparing lipid nanoparticles for the delivery of therapeutic nucleic acids to cells. Cationic ionizable lipids were engineered with improved stability to oxidative degradation while in storage.
Resumen de: WO2024191532A2
This invention relates to drug-device core-shell microneedle devices containing anti-diabetes and anti-obesity medications, and transdermal delivery of said medications. The microneedle drug-device system comprises: (a) a two-dimensional array of conical bilayer containing an inner layer with the base diameter ranging from about 100 mm to about 500 mm and the height ranging from about 100 mm about 1200 mm capable of accommodating a known amount of anti-diabetes and/or anti-obesity medications, and a suitable, photostable chromophore or a fluorophore whose absorption and/or emission occurring in the range of 400-900 nm; (b) a larger outer layer that adheres to and encapsulates the inner layer, and protects the inner layer, and (c) a two-dimensional array of cylindrical 'cap layer' that aligns with and adheres to the basal surface of core shell bilayer and seals the bilayer to prevent leakage of said medication(s). The apical (i.e., sharp) ends of the conical bilayer array inserts into the skin and are programmed to allow the said medications to effuse out of the bilayer and into the skin in a controlled fashion and to be monitored by photodetection devices.
Resumen de: AU2024200012A1
This invention relates to repeated administration of antigen-specific immunotherapeutics using protocols, or elements thereof, that do not induce immunosuppression. In some embodiments, the protocol has been previously shown not to induce immunosuppression in a subject.
Resumen de: WO2024192288A2
Described herein are methods, systems and compositions for the production and use of a class of highly branched, nanostructured particles, generally referred to herein as dendritic particles (DPs), synthesized from biodegradable materials that are specifically adapted for drug release in vivo.
Resumen de: WO2024191103A1
The present invention relates to a self-assembling nanoparticle containing a dexamethasone and polyester polymer conjugate, a preparation method therefor, and a drug delivery composition comprising the same, wherein the self-assembling nanoparticle contains a polymer conjugate where dexamethasone is conjugated to a polyester polymer, and a drug (anticancer agent, anti-inflammatory agent), providing enhanced drug efficacy, easy nuclear entry, and simultaneous anticancer and anti-inflammatory activities.
Nº publicación: WO2024191271A1 19/09/2024
Solicitante:
QMINE CO LTD [KR]
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Resumen de: WO2024191271A1
The present invention relates to double-stranded RNA that regulates the expression of WFDC2. The double-stranded RNA regulating the expression of WFDC2 according to the present invention can exhibit anticancer effects in various types of cancer.