Alerta

POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS

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.

TREATMENTS COMPRISING AN MTOR INHIBITOR NANOPARTICLE COMPOSITION

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.

DRUG FOR GENETIC MODIFICATION, DRUG DELIVERY METHOD, AND DRUG DELIVERY CARRIER

Resumen de: EP4706637A1

According to one arrangement, a drug(1) for genetic modification according to an arrangement is a drug for performing genome editing on a gene in a hematopoietic stem cell. The drug for genetic modification contains a genome editing molecule(2) and a lipid nanoparticle(3) encapsulating the genome editing molecule. The lipid nanoparticle includes a lipid membrane(30) having a lumen. The lipid composition contains at least a first lipid (FFT-10) and a second lipid (FFT-20) in the lipid composition. The amount of the second lipid is larger than that of the first lipid, the total amount of the first lipid and the second lipid is 40 mol% or less, and the total amount of the cationic lipid is 60 mol% or less.

PHYTIC ACID-BASED TERNARY COMPLEX BIONIC NANO MATERIAL, AND PREPARATION METHOD AND USE THEREOF

Resumen de: EP4706690A1

The present application relates to the field of biological medicines, in particular to a phytic acid-based ternary complex bionic nano material. The ternary complex bionic nano material is composed of phytic acid, metal ions and a protein/polypeptide. The ternary complex is formed by connecting the protein/polypeptide with phytic acid using metal cations as a cation bridge. According to the application, the phytic acid ternary complex having different protein/polypeptide groups is synthesized by using various metal ions as bridges. A novel nano diagnosis and treatment probe having high biocompatibility is constructed, which can be reassembled and gathered for a long time at a tumor part, and has coordination-loaded imaging/therapeutic metal ions. The functions of long-acting tumor imaging, treatment or integrated diagnosis and treatment can be achieved, and the solution can be expected to be used as a novel bionic nano material in the field of biomedicine. In addition, the preparation process of the nano material is simple, and industrial production is easy to implement.

A PROIMMUNOTOXIN THERAPEUTIC NANOPLATFORM AGAINST CANCER

Resumen de: WO2024229147A2

Aspects of the disclosure relate to compositions comprising encapsulated immunotoxins and methods for using the encapsulated immunotoxins. The encapsulated immunotoxins may be capable of reaching otherwise difficult to target tissues, such as the brain, and may resist denaturation and degradation prior to reaching the targeted tissue.

CORTICOSTEROID NANOSUSPENSIONS AND USES THEREOF

Resumen de: MX2025013065A

Suspension formulations of nanoparticles of clobetasol propionate are described. The suspensions can be used therapeutically to treat skin and ocular burns; to enhance wound healing; to prevent or reduce hypertrophic scarring/keloids; to treat allergic rhinitis/sinusitis, asthma, inner ear disorders including hearing loss, tinnitus, or vertigo, tenosynovitis, tendinitis, enthesitis or arthritis.

PEGYLATED RECONSTITUTED HIGH-DENSITY LIPOPROTEIN NANOPARTICLES

Resumen de: EP4706645A1

The present invention relates to PEGylated reconstituted high-density lipoprotein nanoparticles having the effect of preventing or treating neurodegenerative diseases. Specifically, the present invention relates to nanoparticles and a method for producing the same, a phospholipid layer of the produced nanoparticles being protected by PEG due to PEG-lipid or a derivative thereof being included in the process of preparing a fluid comprising a hydrophobic material and a fluid comprising a hydrophilic material. The PEGylated nanoparticles have the ability to avoid the rejection mechanism of the immune response in the body while maintaining the existing transport ability across the blood-brain barrier, thereby having excellent stability and exhibiting long-term pharmacological effect due to high circulation ability in the body, and thus can be effectively utilized as a drug or a drug carrier.

RECONSTITUTED HIGH-DENSITY LIPOPROTEIN NANOPARTICLES COMPRISING CHOLESTEROL

Resumen de: EP4706644A1

The present invention relates to: reconstituted high-density lipoprotein nanoparticles comprising cholesterol; and a composition for preventing or treating Alzheimer's disease and cancer, the composition comprising the nanoparticles. Specifically, the reconstituted high-density lipoprotein nanoparticles comprising cholesterol according to the present invention have an excellent cell influx rate and promote cholesterol efflux from cells, thus having a cancer cell killing effect, and can therefore be used for preventing or treating cancer.

一种关于LNP设计、制备及应用全过程的集成数据库及构建方法

Resumen de: CN121641162A

本发明公开了一种关于LNP设计、制备及应用全过程的集成数据库及构建方法，集成数据库，包括：物性参数数据集，代表适用于LNP制备的原料物质，每一个物性参数数据对应一个原料物质；工艺参数数据集，代表LNP制备过程中的工艺参数，每一类工艺参数数据包含多个不同数值的工艺参数；配方组成数据集，包括多个配方组成数据，每个配方组成数据包括多个不同的物性参数数据与多个工艺参数数据；以及，递送效率数据集，包括多个递送效率数据，每个递送效率数据与一个配方组成数据对应。本发明整合LNP系统设计‑制备‑应用全过程中涉及的参数，将影响LNP性能的变量全部转化为数值类型的特征参数，便于后续的高通量条件筛选与数据挖掘，可以极大的促进LNP制备过程的自动化改造与LNP性能优化。

环状单链DNA递送载体及其应用

Resumen de: CN121622609A

本发明公开了一种环状单链DNA递送载体及其应用，属于药物制剂技术领域，该环状单链DNA递送载体包括可电离阳离子脂质、脂质聚乙二醇缀合物、甾醇类化合物和1‑硬脂酰基‑2‑油酰基卵磷脂；以环状单链DNA递送载体中存在的总脂质的总摩尔数为1计，可电离阳离子脂质的摩尔百分比为25％‑60％，脂质聚乙二醇缀合物的摩尔百分比为1.0％‑3.5％，甾醇类化合物的摩尔百分比为20％‑50％，1‑硬脂酰基‑2‑油酰基卵磷脂的摩尔百分比为5％‑30％。该递送载体在N/P比为4‑10：1的条件下递送环状单链DNA展现出了卓越的转染效率，在基因治疗领域具有广泛的应用前景。

向细胞内递送遗传物质的三组分脂质纳米颗粒及组合物

Resumen de: CN121622610A

本发明涉及一种基于三组分脂质纳米颗粒(LNP)的新型基因药物递送系统及其制备方法和应用。该三组分LNP由阳离子脂质、结构脂质和聚合物缀合脂质三种成分组成，通过精确配比优化，实现了在简化成分的同时，保持了高效递送性能。与传统四组分LNP相比，该三组分LNP在制备过程中减少了化学成分的种类，从而显著降低了生产工艺的复杂性和成本，使其更适合于大规模工业生产。此外，由于减少了成分种类，三组分LNP的生物相容性得以提升，潜在的免疫原性和毒性风险降低，具有更高的安全性。

一种溶瘤病毒载囊泡及其制备方法和应用

Resumen de: CN121622616A

本发明属于药物递送与肿瘤免疫治疗技术领域，具体涉及一种溶瘤病毒载囊泡及其制备方法和应用。所述制备方法为：溶瘤腺病毒与血小板在含前列腺素E2的缓冲体系中共孵育，诱导形成内膜封装体；在36‑38℃、4‑6%CO2条件下激活释放囊泡，经300g、2000g、10000g、100000g梯度离心收集。所述溶瘤病毒载囊泡粒径50‑200nm，表面含CD41、CD61及P‑selectin，可实现溶瘤病毒的长循环与肺靶向递送，适用于制备治疗乳腺癌及乳腺癌肺转移的药物，抑瘤效果显著。

阳离子脂质化合物及其构建的脂质纳米颗粒、药物组合物和用途

Resumen de: CN121627601A

本发明公开了一种阳离子脂质化合物及其构建的脂质纳米颗粒、药物组合物和用途，由该阳离子脂质化合物（式I）所构建的脂质纳米颗粒具有较小的粒径和较低的多形貌指数，并且对药物分子具有极高的封装效率，同时能够在保持高表达强度的前提下，将其中负载的mRNA药物精准递送至肝脏，且在其它器官，例如肺、心脏和肾脏中几乎不表达，在脾脏少量表达，在癌症治疗中具有重要的临床应用意义。

Krit1 mRNA在治疗脑海绵状血管畸形中的应用

Resumen de: CN121622940A

本发明涉及生物医学和基因治疗技术领域，具体涉及一种Krit1 mRNA在治疗脑海绵状血管畸形中的应用；KRIT1 mRNA通过脂质纳米颗粒形成LNP@Krit1 mRNA进行递送，可以应用于制备预防和/或治疗脑海绵状血管畸形试剂、制备恢复脑血管内皮细胞的屏障功能试剂和制备减少或抑制脑部血管的渗漏和出血试剂；在体外和体内CCM模型中均能有效恢复Krit1功能，显著改善血管病变，具有良好的开发前景和临床转化价值。

지질 나노입자 제형 및 조성물

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.

이온화 가능한 지질

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.

アジュバントを全く含まないカチオン性多糖ナノ粒子を介して不活性化全細菌を送達するための系を含むワクチン組成物

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.

4차 항암치료 항암소자

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.

IONIZABLE LIPIDS FOR NUCLEIC ACID DELIVERY

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

疾患の処置のためのＫＲＡＳ阻害のための組成物および方法

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.

治療薬の生体内分布および有効性の受容体媒介制御のための標的

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.

一种多巴胺类阳离子脂质化合物及其制备方法和应用

Resumen de: CN121627529A

本发明公开了一种多巴胺类阳离子脂质化合物及其制备方法和应用，属于有机合成技术领域。本发明以盐酸多巴胺为初始原料，随后与二碳酸二叔丁酯或氯甲酸叔丁酯进行亲加成核反应，然后在碱性催化条件下与1‑溴卤代烃反应，得到多巴胺衍生物；接着通过三氟乙酸将多巴胺衍生物上的叔丁氧羰基脱去，在碱性条件下与卤代烃反应，得到多巴胺类阳离子脂质化合物。本发明制备的多巴胺类阳离子脂质化合物能够增强生物活性分子和治疗分子的细胞内递送效率，将多巴胺类阳离子脂质化合物作为递送载体制备的药物制剂可将治疗有效剂量的生物活性分子递送至靶细胞处，具有潜在的应用价值。

胍基衍生化合物及包含其的组合物和应用

Resumen de: CN121627754A

本发明属于药学与生物材料技术领域，具体涉及胍基衍生化合物及包含其的组合物和应用。本发明的化合物具有如式I所示的通式。本发明还提供了包含其的脂质载体、脂质纳米颗粒以及药物组合物及其应用。本发明具有如下技术效果：本发明提供了一类胍基衍生化合物及包含其的组合物和应用，该类新型胍基衍生化合物可用于制备脂质纳米粒递送载体。与含有其他可电离脂质的脂质纳米粒相比，由该类胍基衍生化合物制备的脂质纳米粒或药物组合物，具有以下优点：包封率高、转染效率高且弱趋肝性、稳定性好、细胞相容性良好等。

含酰胺的脂质

Resumen de: US2024423914A1

Compounds are provided having the following Formula (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein G1, R1, R2, R3, 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.

防治心肌肥大的膜性细胞器纳米囊泡、其制备方法及应用

Nº publicación: CN121628811A 10/03/2026

Solicitante:

重庆理工大学昆明医科大学

Resumen de: CN121628811A

本发明涉及生物医药技术领域，具体公开了防治心肌肥大的膜性细胞器纳米囊泡、其制备方法及应用，所述膜性细胞器纳米囊泡包括从哺乳动物肝脏组织中提取的内质网，经超声破碎处理后得到的具有钙离子缓冲功能的纳米级囊泡。本发明通过提取肝脏源性的内质网纳米囊泡及其衍生体，直接与心肌细胞内质网发生物理融合，从根本上实现了从化学信号调控到细胞器功能重建的技术跨越，实现了精准、高效地增强SERCA2a钙泵活性，直接修复内质网钙稳态，同时协同改善能量代谢并抑制细胞凋亡，从而在动物模型中显著逆转心肌肥大病理进程，且因其生物源性的特点具备优异的生物安全性与产业化潜力。