Resumen de: WO2024072929A1
The present disclosure provides a method of assessing translation efficacy of an mRNA using liquid chromatography-tandem mass spectrometry (LC-MS/MS). The mRNA is first translated into protein either in a cell lysate (cell-free translation; CFT) or inside a cell (cell-based translation; CBT) and analyzed using LC-MS/MS. The method provides advantages such as speed and convenience over traditional immunoassay-based methods of detecting translated proteins. Translation using CBT may be necessary in certain formulations of the mRNA, such as when the mRNA or a mixture of mRNAs is encapsulated inside a lipid nanoparticle.
Resumen de: US2024150306A1
Provided herein are compounds having one or more cleavable disulfide moieties and one or more hydrophobic tail moieties (HTM) for delivery of one or more therapeutic or diagnostic agents. In some embodiments, the one or more therapeutic or diagnostic agents are biologics. In some embodiments, the one or more therapeutic or diagnostic agents are delivered to cancer cells.
Resumen de: EP4595948A1
The present invention refers to a composition comprising BMNPs (Biomimetic magnetic nanoparticles) or BMNP nanoassemblies, characterized in that said BMNPs are disaggregated or dispersed. Alternatively, the first aspect of the present invention refers to a composition comprising: (i) a substantially pure mineral phase of superparamagnetic magnetite, (ii) the MamC protein, and (iii) optionally, the Mms6 and/or Mms7 protein; wherein, at least components (i) and (ii) form superparamagnetic magnetic nanoparticles containing up to 20 wt% of MamC, with a mean particle size between 30 and 120 nm, preferably between 30 and 50 nm, more preferably about 39±7 nm, characterized in that said superparamagnetic magnetic nanoparticles containing MamC are disaggregated or dispersed.
Resumen de: WO2024065043A1
Provided are lipids and nanoparticles containing such lipids and a cargo molecule, such as nucleic acid, methods to formulate said lipids with nucleic acids to produce lipid nanoparticles and chemical routes for making the lipids. The lipids may have the structure of Formula A as defined herein.
Resumen de: CN120051455A
Novel sulfur-containing lipids and nanoparticles containing the lipids and cargo molecules, such as nucleic acids, methods of formulating the lipids with nucleic acids to produce lipid nanoparticles, and chemical pathways for preparing the lipids, are provided. The lipid may have a structure of Formula A as defined herein. # imgabs0 #
Resumen de: WO2024065041A1
The present disclosure relates to a sulfur-containing ionizable lipid or a pharmaceutically acceptable salt thereof that incorporates a dithioacetal or dithioketal moiety in one or more of its lipophilic chains. Further provided is a delivery vehicle, such as a lipid nanoparticle, comprising the ionizable lipid for the delivery of cargo, such as nucleic acid.
Resumen de: EP4595983A1
The present invention relates to functionalised nanoparticles. The present invention also relates to compositions comprising the functionalised nanoparticles, kits, processes for preparing nanoparticles, methods of treating cancer (including prostate cancer), imaging methods, diagnostic methods, methods for killing or attenuating the growth of a cancer cell in vitro, nanoparticles and compositions for use in the treatment of cancer or diagnosis, and a novel pepducin.
Resumen de: EP4596605A1
The present application belongs to the field of drug delivery, and specifically relates to a PEGylated lipid containing a lysine core that can be used as a component of drug carrier, and a lipid composition containing the PEGylated lipid, a LNP pharmaceutical composition and formulations and applications thereof. In the present application, the PEGylated lipid containing a lysine core is more stable. The application of the PEGylated lipid in the preparation of lipid composition and lipid pharmaceutical composition has the advantages of prolonged circulation time, high biocompatibility, low toxicity, high encapsulation efficiency and high delivery efficiency.
Resumen de: US2024415986A1
Nanoparticulate material suitable for administration to a subject, the nanoparticulate material having bound to its surface: (a) copolymeric steric stabiliser that promotes dispersion of the nanoparticulate material in a liquid, wherein the copolymeric steric stabiliser comprises (i) an anchoring polymer segment having one or more binding groups that bind the copolymeric steric stabiliser to the nanoparticulate material, and (ii) a steric stabilising polymer segment that is different from the anchoring polymer segment, and (b) copolymeric mapping moiety comprising (i) an anchoring polymer segment having one or more binding groups that bind the copolymeric mapping moiety to the nanoparticulate material, (ii) one or more mapping groups comprising an agent that specifically binds to fibroblast activation protein (FAP), and (iii) a coupling polymer segment that is different to the anchoring polymer segment, wherein the coupling polymer segment couples the anchoring polymer segment to the one or more mapping groups.
Resumen de: WO2023143601A1
Provided in the present invention are a novel cationic lipid, a lipid nanoparticle and a nucleic acid vaccine. In the present invention, a specific cationic lipid is selected for preparing a lipid nanoparticle mRNA vaccine, which is found to have better in-vitro stability and can stimulate a stronger immunoreaction compared with LNPs prepared from cationic lipids in the prior art.
Resumen de: CN119630390A
Lipid formulations capable of forming lipid-based nanoparticles are provided, the lipid formulations comprising a molar ratio of ionizable lipid to phospholipid bound to a nucleic acid payload of 0.1-1.30, and in some embodiments a stabilizer. In embodiments, the nucleic acid payload is a vaccine genetic element.
Resumen de: US2025099394A1
The present invention provides a lipid nanoparticle (LNP) formulation comprising at least one sialic acid (SA)-containing lipid. The LNP formulation is capable of effectively binding cell surface Siglecs, transfecting certain cells in vitro and targeting certain cells in vivo. The present invention also provides methods of using the LNP compositions described herein for pharmaceutical applications. For example, the LNPs provided herein are useful for the intracellular delivery of a nucleic acid therapeutic to a subject.
Resumen de: WO2024054855A1
The present disclosure relates to a combination therapy comprising an anti-VEGF antibody, a nanoparticle formulated plasmid comprising an IL-12 coding nucleic acid, and, optionally, at least one adjunctive chemotherapeutic drug, and methods of treatment using such combination therapies and/or compositions.
Resumen de: CN120420424A
本专利提供一种高危亚型HPV肿瘤疫苗及其应用,其包括纳米脂质颗粒和表达HPV病毒抗原的RNA,表达HPV病毒抗原的RNA包括编码HPV病毒肽表位的RNA,HPV病毒肽表位包括蛋白E6、E7重组蛋白,所述纳米脂质颗粒包裹RNA。该疫苗的优势在于自主研发的纳米脂质颗粒可以同时包裹多个RNA,有效表达多种高危型HPV的E6、E7抗原。RNA分子设计上通过E6、E7串联的形式进行融合表达,并通过密码子优化实现核酸序列的高效翻译。该疫苗能够有效激活体内肿瘤特异性免疫反应的同时保证安全性,在临床应用中兼顾多种高危型HPV相关癌症的治疗。
Resumen de: AU2023292959A1
Provided herein are therapeutic compositions containing a noncoding RNA BCYRN1 or a derivative thereof, such as a nucleic acid containing a BCYRN1-dervied binding sequence (BDSS). Also provided are methods of treating an immune-related disorder by administering a therapeutically effective amount of a composition containing at least one nucleic acid having a BDSS to a subject in need thereof.
Resumen de: CN119403553A
The present invention relates to a pharmaceutical composition for preventing or treating inflammatory diseases, the pharmaceutical composition comprising a compound of Chemical Formula 1, and to a cosmetic composition comprising the compound. By comprising the compound of Chemical Formula 1, the present invention can scavenge reactive oxygen species and inhibit inflammatory cytokines in a non-toxic manner, thereby protecting protective cells and reducing inflammation.
Resumen de: AU2023310050A1
A minimally invasive method for the reduction of hypertension is provided using focused ultrasound. After injection of ultrasound-responsive nanodroplets loaded with a therapeutic agent (e.g. barbiturates), focused ultrasound is delivered to a norepinephrine-producing region, such as the periaqueductal gray region, thereby locally releasing the therapeutic agent payload from the nanodroplets and achieving a therapeutic reduction in blood pressure. Acoustic emissions from vaporizing droplets may be employed, for example, to infer one or more therapeutic parameters based on a pre-established correlation. For example, plasma hormone content and/or the change in blood pressure may be inferred, thereby providing a means of real-time treatment monitoring. The present example methods may be employed to achieve a reduction in blood pressure for subjects exhibiting drug-resistant hypertension.
Resumen de: CN119816517A
Circular RNAs and related compositions and methods are described. In some embodiments, the circular RNA of the invention comprises a Group I intron fragment, a spacer, an IRES, a duplex forming region, and an expression sequence. In some embodiments, the expression sequence encodes an antigen. In some embodiments, the circular RNAs of the invention have improved expression, functional stability, immunogenicity, ease of manufacture, and/or half-life when compared to linear RNAs. In some embodiments, the methods and constructs of the invention result in improved cyclization efficiency, splicing efficiency and/or purity when compared to existing RNA cyclization methods.
Resumen de: NL2040524A
The present invention relates to the field of nanomaterial technology, and more particularly, to a preparation method, product, and application of a targeted responsive nanomaterial. The preparation method comprises the following steps: Step 1: Dissolve a surfactant in a buffer solution, then add perfluoro-lS-crown-S-ether, an anticancer drug, and an oxidant, followed by vortexing and sonication to obtain a suspension; Step 2: Add a dopamine hydrochloride solution to the suspension and sonicate to obtain solution A; Step 3: Stir solution A and then centrifuge it; remove the supernatant and perform ultrafiltration to obtain a concentrate; Step 4: Mix the concentrate With cRGD- PEGSK-NHS, stir, and ultrafilter to concentrate, thereby obtaining the targeted responsive nanomaterial. The preparation method of the present invention is simple. The nanomaterials prepared using the method of the invention are not only expected to become a safer and more effective approach to tumor treatment but also lay a solid foundation for the future development of similar multifunctional composite nanopharmaceutical products.
Resumen de: CN120423968A
本申请提供阳离子脂质化合物、包含其的组合物及应用。为了给基因药物、核酸疫苗、小分子药物等制剂的递送提供更多的选择,本申请提出通式#imgabs0#所示的阳离子脂质化合物,或其药物可用的盐、前药或立体异构体。本申请的阳离子脂质化合物可用于递送核酸药物或小分子药物,丰富了阳离子脂质化合物种类,对核酸预防剂及治疗剂的发展和应用具有重要的意义。
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.
Resumen de: CN120424104A
本发明公开了一种活性氧激活型近红外区光热试剂及其制备方法和应用,通过将酚羟基功能化的氮杂氟硼荧类染料与对溴苯硼酸频哪醇酯通过成醚反应,构建出具有活性氧响应的近红外区光热试剂,并进一步将其制备成纳米粒子,该纳米粒子与活性氧反应后,在808nm激光光照下,其光热效率显著提升,具有优异的光热成像效果和光热治疗功能。
Resumen de: CN120420299A
本发明公开了一种甘草次酸白蛋白纳米粒,所述纳米粒呈均一的球形;所述纳米粒包括甘草次酸、白蛋白;所述纳米粒粒径为≦200nm,包封率90%~95.10%,载药量为30.56%~63.40%,所述纳米粒24小时内释放的甘草次酸为60%~70%;本发明还公开了一种甘草次酸白蛋白纳米粒的制备方法,该方法得到的纳米粒粒径小且分布均一,其稳定性和安全性随白蛋白占比增加而增加;其中,富含白蛋白的纳米粒在预防和治疗急性肝损伤实验中展现双模式药理活性。
Resumen de: CN120420339A
本发明属于生物医药领域,具体涉及一种治疗银屑病及复发性银屑病的新型mRNA核酸药物。本发明提供一种治疗银屑病及银屑病相关疾病的mRNA,所述mRNA核苷酸序列如SEQ ID NO.1或SEQ ID NO.2所示。本发明还提供包括前述的mRNA的核酸药物,所述药物采用脂质体纳米颗粒包裹mRNA。本发明还提供前述的mRNA或前述的核酸药物在制备预防和/或治疗银屑病及银屑病相关疾病的药物中的应用,所述银屑病相关疾病包括银屑病关节炎、儿童性银屑病、复发性银屑病。以及,本发明提供前述的mRNA或前述的核酸药物在制备抗炎症药物中的应用。本发明的mRNA药物能够起到治疗银屑病的效果,实现银屑病症状缓解;控制银屑病复发,克服目前银屑病生物制剂对耐药性以及停药无法的难题。
Nº publicación: CN120423969A 05/08/2025
Solicitante:
摩登纳特斯有限公司
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.