Resumen de: AU2024263334A1
Disclosed herein are coronavirus (CoV) Spike (S) polypeptides, including naturally and non-naturally occurring polypeptides, and nanoparticles and immunogenic compositions comprising the same, which are useful for stimulating immune responses against various SARS-CoV-2 strains. The nanoparticles present antigens from pathogens surrounded to and associated with a detergent core resulting in enhanced stability and good immunogenicity. Dosages, formulations, and methods for preparing the vaccines and nanoparticles are also disclosed.
Resumen de: WO2024228630A1
The description of the invention discloses a composition comprising 17- AAG and staurosporine for use in the treatment of melanoma, non-small cell lung cancer, pancreatic cancer, mammary gland tumours or gliomas, and a stable polymer-lipid nanoparticle for drug binding and delivery, consisting of a core containing polylactic-co-glycolic acid (PLGA) and polyvinyl alcohol (PVA) and a core envelope containing a mixture of lipids: 1,2-dipalmitoyl-sn-glycero-3-phosphocholine (DDPC), cholesterol and the ammonium salt 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N - amino(polyethylene glycol)-2000 (DSPE-PEG(2000)NH2J.
Resumen de: WO2024228632A1
The description of the invention discloses a stable polymer-lipid nanoparticle for drug binding and delivery comprising a core containing polylactic-co-glycolic acid (PLGA) and polyvinyl alcohol (PVA) and a core envelope containing a mixture of lipids: 1,2-dipalmitoyl-sn-glycero-3- phosphocholine (DDPC), cholesterol and the ammonium salt of 1,2- distearoyl-sn-glycero-3-phosphoethanolamine-N -amino(polyethylene glycol)-2000 (DSPE-PEG(2000)NH2 and a method for producing such particles.
Resumen de: WO2024229321A1
This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects.
Resumen de: WO2024228964A1
The present application, in certain aspects, pertains to methods and compositions for the treatment of cancers, such as undifferentiated pleomorphic sarcoma or leiomyosarcoma, using : (a) a composition comprising nanoparticles comprising an mTOR inhibitor (such as a limus drug, e.g, sirolimus or a derivative thereof) and an albumin; and, optionally, (b) an anti-PD-1 antibody.
Resumen de: CN121285401A
Novel nanogels comprising one or more bioactive molecules, therapeutic molecules or drugs and amphiphilic molecules and methods of making the same. Biomaterial implants, medical devices or bioprostheses coated with nanogels and methods of producing the same.
Resumen de: JP2025105627A
To provide ionizable lipids capable of transfecting cells with genetic material.SOLUTION: The present invention relates to a compound of formula (I), or a pharmaceutically acceptable salt thereof.SELECTED DRAWING: None
Resumen de: KR20260033704A
본 발명은 1) (항암제와 결합하거나 항암제와 결합하지 않은) 분말이나 나노 시트 또는 나노 튜브 형태로 구성된 ① 이황화 몰리브덴(MoS2) ② 이황화 텅스텐(WS2) ③ 이셀렌화 몰리브덴(MoSe2) ④ 이셀렌화 텅스텐(WSe2)이나 ⑤ 산화 아연(ZnO) 나노 와이어 ⑥ 황화 아연(ZnS) 나노 와이어, ①~⑥ 중에서 1가지 이상을 선택하여 구성한 항암소자 표면에 폴리 도파민을 코팅시킨 후 2) 그 위에 암세포에 표적 부착하는 표적 지향성 리간드를 도포하여 결합시켜 구성하여 - (체내에 주입시켜 암세포 부위에 표적 부착시킨 후) 신체 외부에서 암세포 부위에 빛을 조사시키고, 초음파를 조사시켜서 항암소자가 암세포 부위에서 4차 항암치료를 할 수 있도록 구성하여 기존의 항암소자 보다 항암치료에 도움을 줄 수 있는 보다 효능적인 항암소자로 구성한 4차 항암치료 항암소자에 관한 것이다. 색인어 이황화 몰리브덴, 이황화 텅스텐, 이셀렌화 몰리브덴, 이셀렌화 텅스텐, 산화 아연 나노 와이어, 황화 아연 나노 와이어, 4차 항암치료 항암소자, 폴리 도파민, 표적 지향성 리간드.
Resumen de: AU2024290779A1
The present invention generally relates to the field of ionizable (also termed cationic) lipids, and in particular provides a novel type of such lipids as represented by formula (I). The present invention further provides methods for making such lipids as well as uses thereof, in particular in the preparation of nanoparticle compositions, more in particular nanoparticle compositions comprising active agents. It further provides pharmaceutical formulations comprising nanoparticle compositions based on the ionizable lipids disclosed herein.
Resumen de: AU2024279278A1
Disclosed are compositions of lipid nanoparticles (LNP) comprising an ionizable cationic lipid, a phospholipid, a sterol, and a PEG-lipid (non-functionalized and optionally functionalized). The functionalized PEG-lipid can be conjugated with a binding moiety to create a targeted LNP (tLNP). The disclosed tLNP preferentially deliver a nucleic acid molecule or other negatively charged payload to cells expressing a cell surface antigen recognized by the binding moiety of the tLNP, and are better tolerated, as compared to LNPs and tLNPs comprising ionizable cationic lipids found in marketed pharmaceuticals comprising LNPs.
Resumen de: US2025340880A1
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.
Resumen de: MX2022014270A
Polynucleotides, scaffolds, and cassettes are presently disclosed and described. In particular, these polynucleotides may have a formula comprising Signal/Leader-payload-PRM, wherein the Signal/Leader encodes a signal sequence, a leader sequence, or a sorting sequence, in frame with and upstream of a payload; the payload is an antigenic payload region, a detectable agent, and a therapeutic agent; and the PRM encodes all or a portion of at least one parental receptor molecule region from one or more isoforms or proteins selected from the group consisting of CD1d, CD1e, LDLR, LDLRP, and LRP1 proteins.
Resumen de: US2024294918A1
Nucleic acid molecules and compositions thereof targeted to fidgetin-like 2, and methods of their use in treating a wound or scar in a human subject are described.
Resumen de: WO2024182707A1
The present disclosure provides a first polynucleotide (e.g., a recombinant or isolated first polynucleotide) encoding an Interleukin (IL)-2 polypeptide and a second polynucleotide (e.g., a recombinant or isolated second polynucleotide) encoding an IL-12 polypeptide; recombinant nucleic acids and/or gene delivery vehicles comprising the same; compositions, formulations, and medicaments comprising the one or more of the polynucleotides, recombinant nucleic acids, and/or gene delivery vehicles; methods of their use (e.g., for the treatment of cancer); and articles of manufacture or kits thereof.
Resumen de: CN121099985A
The present disclosure provides the use of a compound of Formula I in lipid micron and nanoparticle compositions. The present disclosure also relates to microparticles or nanoparticles comprising a compound of Formula I and optionally a payload molecule, and medical uses thereof.
Resumen de: WO2024178113A1
Recombinant adeno-associated virus (AAV) vectors encoding a modified VP1 protein lacking an immunodominant T cell epitope, as well as AAV vector particles containing the modified VP1 protein, are described. Use of the recombinant AAV vectors and vector particles as improved gene therapy vectors with reduced immunogenicity is also described. Isolated VP1 peptides containing an immunodominant T cell epitope, and use thereof, is further described.
Resumen de: CN120435500A
Disclosed herein, including novel BBB spanning receptors on the blood brain barrier (BBB) interface, targeting peptides and derivatives thereof capable of binding to these novel receptors, and related methods of using these receptors to increase permeability of BBB and deliver agents to the nervous system (e.g., CNS). In some embodiments, the BBB is LRP6 across the receptor. Also disclosed herein are recombinant adeno-associated viruses (rAAVs) with increased specificity and transduction efficiency across BBB, as well as related compositions and methods of treating various diseases and conditions.
Resumen de: MX2025009845A
The present disclosure provides pharmaceutical compositions comprising a peptide- polynucleotide complex, wherein the peptide comprises an amino acid sequence with at least 80%, at least 85%, at least 90%, at least 95%, at least 98%, at least 99%, or 100% identity to the amino acid sequence of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3; and wherein the polynucleotide is a small interfering RNA (siRNA) targeting human KRAS mRNA, wherein the target sequence of human KRAS mRNA does not encode G12, G13, or Q61 with reference to SEQ ID NO: 4 or a mutant amino acid at position 12, 13, or 61 with reference to SEQ ID NO: 4.
Resumen de: CN120712079A
The present invention relates to the field of vaccine compositions. The invention more particularly relates to a prophylactic vaccine composition comprising killed intact bacteria intended for use in mammals and birds, said bacteria being wrapped with a cationic agent, in particular cationic nanoparticles.
Resumen de: US2024423915A1
Compounds are provided having the following Formula (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3, G1, G2, L1, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Resumen de: CN121622605A
本发明公开了用于靶向治疗具核梭杆菌浸润的结直肠癌的纳米药物体系及其制备方法。本发明属于生物技术领域,具体涉及用于靶向治疗具核梭杆菌浸润的结直肠癌的纳米药物体系及其制备方法。本发明的具有抑制肿瘤细胞活性或抗肿瘤活性的药物活性成分含有NM@PLGA‑MTI‑OXA,NM@PLGA‑MTI‑OXA由中性粒细胞膜囊泡和PLAG‑MTI‑OXA组成,其中PLAG‑MTI‑OXA是由聚乳酸‑羟基乙酸共聚物负载化疗药物MTI及OXA得到的纳米颗粒,中性粒细胞膜囊泡来源于骨髓中性粒细胞。本发明开发的NM@PLGA‑MTI‑OXA能够有效靶向具核梭杆菌浸润的结直肠组织,成功实现抗菌抗肿瘤效果,具有应用价值。
Resumen de: AU2024315222A1
The present disclosure provides a compound of formula (I): (I), or a pharmaceutically acceptable salt thereof, that is useful for forming particles (e.g., lipid nanoparticles) for delivery of nucleic acids. The present disclosure further provides particle compositions comprising the compound of formula I, as well as uses thereof.
Resumen de: CN121628927A
本发明公开了一种狂犬病毒G蛋白mRNA疫苗冻干制剂的制备及应用,涉及兽用生物制品领域。本发明通过体外转录技术规模化制备狂犬病毒G蛋白mRNA,并采用微流控包封工艺成功构建mRNA‑LNP(脂质纳米颗粒)递送系统。通过冻干保护剂配方筛选(涵盖蔗糖、海藻糖及甘露醇)及冻干程序参数优化(包括预冻温度、主干燥速率及二次干燥残留水分控制),最终获得可在4℃条件下稳定存在的冻干mRNA疫苗制剂。
Resumen de: CN121627848A
本发明涉及疫苗领域,特别涉及一种新型佐剂多肽序列及其应用。本发明提供的佐剂多肽序列包含破伤风类毒素表位P2、P16氨基酸序列,还包含P30和/或PX的氨基酸序列。该佐剂多肽序列通过增加表位多样性,旨在扩大免疫反应的覆盖群体。同时,本发明利用LNP(脂质纳米颗粒)递送系统,将编码佐剂多肽与初始抗原蛋白的mRNA高效导入体内,有效提升了初始抗原的免疫刺激能力,从而在减少初始抗原使用剂量的同时,仍能达到或超过传统剂量的免疫效果。进一步,本发明提供的佐剂多肽序列与初始抗原序列优选采用融合的方式,简化了核酸疫苗中mRNA的组成,降低了疫苗制备的复杂性,为疫苗工艺开发提供了新思路,具有显著的技术进步和临床应用潜力。
Nº publicación: CN121628906A 10/03/2026
Solicitante:
无锡市南京大学锡山应用生物技术研究所
Resumen de: CN121628906A
本发明公开了用于降低雷公藤甲素肝毒性的RNAi及其应用,属于生物医药技术领域。本发明将靶向 Cyp2e1 基因的 siRNA 经 2′‑O 甲基修饰及 dTsdT 3′硫代磷酸酯连接修饰后,通过脂质纳米颗粒(LNPs)递送。经静脉注射给药后,24h 内可使小鼠肝脏 Cyp2e1 蛋白表达下降 70% 以上,通过降低血清谷丙转氨酶 / 谷草转氨酶水平、抑制肝脏氧化应激、减少 TNF‑α/IL‑1β/IL‑6 等炎症因子释放,显著减轻雷公藤甲素诱导的亚急性肝损伤。本发明克服了现有减毒方案药效降低或靶向性欠佳的缺陷,提供了靶向调控效果显著、安全性高的临床辅助减毒技术方案,为雷公藤甲素的安全临床应用提供了新路径。