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脂质纳米颗粒及其制备方法与应用

Resumen de: CN122604732A

本发明涉及核酸基因治疗技术领域，尤其涉及脂质纳米颗粒及其制备方法与应用。本发明提供的脂质纳米颗粒，其包装材料阳离子脂类、中性脂类、PEG或PEG化脂类、胆固醇或其衍生物的摩尔百分比为(33～50)％：(10～13.33)％：(1.5～2)％：(38.5～51.33)％。在该摩尔百分比例范围进行靶向性LNP合成，具有更高的包封率，并且可以显著提高靶向性LNP转导效率。不仅仅能够实现颗粒的包封率和包载量提高，同时提高了T细胞的靶向功能，实现T细胞的高效率转染。

可电离化脂质及包含其的脂质纳米颗粒

Resumen de: WO2025157953A1

An ionizable lipid of formula (I) or a pharmaceutically acceptable salt thereof, or a stereoisomer of any one of them; a lipid nanoparticle comprising the ionizable lipid, particularly, as an encapsulation agent, optionally comprising a pharmaceutically active agent; and a pharmaceutical composition comprising the lipid nanoparticle. A lipid nanoparticle or a pharmaceutical composition comprising thereof for use in medicine, and the use of the lipid nanoparticles as an encapsulating agent.

将病毒颗粒基于FUS递送至大脑的方法

Resumen de: WO2025160452A1

Provided herein are methods to treat various neurodegenerative disorders comprising delivering a viral particles to the whole brain, including both superficial and deep structures. In some aspects, the viral particles are administered at low dose levels to the CSF, in conjunction with microbubbles followed by application of focused ultrasound (FUS) to a region of interest of the brain, thereby causing entry of the viral particles to the brain.

新型基因递送剂

Resumen de: US2025144233A1

Nanoparticle compositions for delivery of nucleic acids to subjects including aminoalkyl branched lipid-like molecules as carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the nanoparticle containing are described. Also provided are methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects.

一种多肽凝聚体及其制备方法和应用

Resumen de: CN122609644A

本发明涉及一种多肽凝聚体及其制备方法和应用，所述多肽凝聚体为，由融合多肽TAT‑n×NoLS在含有Tris‑HCl、NaCl以及聚乙二醇8000的溶液中，于室温下孵育，自组装形成的体外相分离凝聚体结构，所述多肽凝聚体能够包载核酸或蛋白质并将其递送至细胞中，其中，所述融合多肽TAT‑n×NoLS由具有相分离特性的NoLS多肽序列经n次串联形成n×NoLS模块，n=2‑5；并在N端融合至少一个TAT穿膜肽序列；所述NoLS多肽的序列如SEQ ID NO.1所示，所述TAT穿膜肽的序列如SEQ ID NO.2所示。本发明提供的TAT‑3xNoLS多肽凝聚体具有对核酸和蛋白类生物大分子的装载、胞内递送及功能输出能力，在生物大分子递送方面表现出较好的应用潜力，也为后续开展多功能化拓展与机制优化研究提供了实验基础。

INTERLEUKIN 2 CONJUGATE FOR TREATING INFLAMMATION-RELATED DISEASES

Resumen de: WO2026171131A1

Provided in the present invention is an interleukin 2 (IL-2) conjugate containing an isolated and purified IL-2 polypeptide, and a conjugated moiety attached to a position where the amino acid residue is cysteine on the IL-2 polypeptide sequence, wherein the conjugated moiety contains a vesicle. Using the steric hindrance specificity of the vesicle, on the one hand, the interleukin-2 conjugate of the present invention enables the IL-2 polypeptide to bind to IL-2Rα on the surface of Treg cells, and on the other hand, the IL-2 conjugate of the present invention can simultaneously reduce the binding thereof to IL-2Rβ and/or IL-2Rγ and/or IL-2Rβγ on the surface of CD8 T cells. As a result, the conjugate selectively binds to and activates the Treg cells, promotes the proliferation thereof and the activation of intracellular signaling pathways, and attenuates the activation of the CD8 T cells, thereby regulating the immune microenvironment and maintaining the immunologic balance.

INTERFERING RNA FOR INHIBITING B7-H3 GENE EXPRESSION AND USE THEREOF

Resumen de: US20260242784A1

The present invention provides an interfering RNA for inhibiting B7-H3 expression, use thereof, and a method for preparing cells with reduced B7-H3 expression. The interfering RNA is siRNA, a target sequence of which comprises nucleotide sequences set forth in SEQ ID NOs: 1-25. The interfering RNA can effectively inhibit B7-H3 gene expression, thus being useful for preventing or treating B7-H3 related diseases.

METHOD FOR PREPARING NANOPARTICLES BASED ON POLYPEPTIDES ENCAPSULATING AT LEAST ONE ACTIVE PRINCIPLE

Resumen de: WO2026171955A1

The present invention relates to a "one-pot" method for preparing an aqueous composition of nanoparticles of copolymers with amphiphilic blocks and comprising polypeptide units, said nanoparticles comprising at least one active principle encapsulated therein, said method comprising: - at least one step (E1) consisting in preparing a solution (S1) comprising at least one active principle and at least one hydrophilic polymer (P1) comprising at least one amine function; and - at least one step (E2), in an aqueous solvent, consisting in placing the solution (S1) in the presence of at least one hydrophobic α-amino acid N-carboxyanhydride monomer.

NALTREXONE-FATTY ACID CONJUGATE NANOPARTICLES AND LONG-ACTING INJECTION THEREOF

Resumen de: WO2026173309A1

The present invention relates to naltrexone-fatty acid conjugate nanoparticles and a long-acting injection thereof. More specifically, the present invention provides naltrexone-fatty acid conjugate nanoparticles and a long-acting injection composition thereof, the nanoparticles comprising a naltrexone-fatty acid conjugate in which a fatty acid is bonded to naltrexone or a pharmaceutically acceptable salt thereof, and being formed by self-assembly of the conjugate according to a pH change. The composition according to the present invention has improved bioavailability, half-life, injectability, and the like, and exhibits excellent long-term sustained release characteristics, and thus can improve medication compliance of patients with alcohol dependence or opioid dependence.

METHOD FOR PREPARING FUNCTIONALIZED NANOPARTICLES BASED ON POLYPEPTIDES

Resumen de: WO2026171948A1

The present invention relates to a "one-pot" method for preparing an aqueous solution of nanoparticles of copolymers with amphiphilic blocks and comprising polypeptide units, said nanoparticles comprising a chemical agent grafted thereto, said method comprising: • at least one step (E1), in an aqueous solvent, consisting in placing at least one hydrophilic polymer of formula (I): A-NH2, wherein A represents a natural or synthetic polymeric radical comprising the repetition of at least one unit selected from the group consisting of acrylates, ethylenes, oxyalkylenes, ethers, esters, sugars and amino acids, in the presence of at least one hydrophobic α-amino acid N-carboxyanhydride monomer, in order to obtain a copolymer of formula (II), and • at least one functionalization step (E2), in an aqueous solvent, consisting in placing said copolymer of formula (II) in the presence of a chemical agent comprising at least one carboxylic acid function or carboxylic acid derivative function allowing coupling with said copolymer of formula (II).

BIOCOMPATIBLE MONODISPERSED NANOHYDROGELS AND USES THEREOF FOR CONTROLLED DRUG RELEASE AND DRUG DELIVERY

Resumen de: WO2026172347A1

A copolymer nanohydrogel comprising chains made of structural units corresponding to (i) at least one macromonomer consisting of a polyether block copolymer with acrylamide end groups, and (ii) an N-alkylated (meth)acrylamide monomer; characterized in that the chains of the copolymer nanohydrogel are connected by at least one crosslinker that contains biodegradable ester bond. A nanohydrogel comprising poorly water-soluble pharmaceutically active compound, a process for preparing the nanohydrogel, pharmaceutical composition thereof, and method of using the nanohydrogels are also provided by the invention.

POROUS NANOFIBER/MICROFIBER SPHERES AND METHODS OF USE THEREOF

Resumen de: WO2026174041A1

Porous nanofibrous/microfibrous spheres are provided as well as methods of use thereof and methods of making. In certain embodiments, the method of synthesis comprises a) applying inner air, outer air, and a nanofiber/microfiber suspension to a sphere generator, wherein the nanofiber/microfiber suspension comprises nanofiber/microfiber segments, and b) collecting the spheres from an outlet nozzle of the sphere generator in a media having a temperature of less than about -20°C (e.g., liquid nitrogen), thereby synthesizing the porous nanofibrous/microfibrous spheres.

DENDROBIUM OFFICINALE POLYSACCHARIDE NANOPARTICLE AND PREPARATION METHOD THEREFOR

Resumen de: WO2026170578A1

The present invention belongs to the technical field of the preparation of Dendrobium officinale polysaccharides, and specifically relates to a Dendrobium officinale polysaccharide nanoparticle and a preparation method therefor. The method for preparing the Dendrobium officinale polysaccharide nanoparticle comprises: firstly, extracting Dendrobium officinale polysaccharides by using a non-ionic surfactant-assisted ultrasonic-complex enzyme method, and purifying same to obtain a Dendrobium officinale polysaccharide extract; then dissolving the Dendrobium officinale polysaccharide extract in water to obtain solution 1; dissolving a modified polyacrylic resin in an organic solvent to obtain solution 2; dropwise adding solution 1 to solution 2 under stirring, and performing ultrasonic treatment for 20-30 min after the dropwise addition; and performing spray drying to obtain the final product. The method for extracting Dendrobium officinale polysaccharides provided in the present invention can effectively increase the yield of polysaccharides, and can also improve the inhibition rate of the Dendrobium officinale polysaccharide extract against α-glucosidase. The prepared Dendrobium officinale polysaccharide extract nanoparticle exhibits a good hypoglycemic effect.

SINGLE POLYMER CHAIN NANOPARTICLES TO MIMIC BIOMOLECULAR HYDRATION FRUSTRATION

Resumen de: WO2026174220A1

Single polymer chain nanoparticles (SPCNs) formed by a random heteropolymer composed of three or more components have been developed and shown to display similar levels of hydration frustration as proteins. These SPCNs can be categorized into three types, based on whether either hydrophobic or hydrophilic residues, or both types, display frustrated states. A series of physicochemical rules that determine the state of these SPCNs are proposed. The generality of these rules are demonstrated via atomistic molecular dynamics simulations and simplified Monte Carlo models of SCPNs with different backbones and residues. This disclosure provides insights into the design of SPCNs, an emerging polymer modality that achieves the ease and cost of fabrication of polymeric material with the functionality of biological proteins.

T CELL-TARGETED LIPID NANOPARTICLES (CTLNPS) FOR DELIVERY OF SIRNA AND USE IN TREATING AUTOIMMUNE DISORDERS

Resumen de: WO2026174149A1

The present disclosure provides "stealth" LNP compositions (e.g., T cell-targeted LNPs (ctLNPs)) that surprisingly exhibit physiological characteristics of prolonged blood circulation time (e.g., increased blood t1/2) simultaneously with increased targeting capacity to T cells. The disclosed stealth LNP compositions are used for the treatment of autoimmune diseases and disorders.

IMMUNE ENGINEERING AMPLIFICATION

Resumen de: AU2025225163A1

This disclosure provides methods of increasing in vivo transfection efficiency and pharmacologic activity of T cells, by administering multiple small doses within a compact time period of T cell-targeted lipid nanoparticles encapsulating mRNA encoding an antigen receptor that recognizes an antigen of a cell against which immune activity is to be directed. Also provided are methods of depleting B cells, and methods of treating B cell-mediated diseases and disorders by depleting B cells and achieving immunological reset, entailing administration of immune cell-targeted lipid nanoparticles encapsulating mRNA encoding an antigen receptor recognizing a B cell marker as multiple small doses within a compact time period. The antigen receptor can be a T cell receptor or a chimeric antigen receptor.

OXYTOCIN-ENCODING MRNA FORMULATIONS FOR NON-OPIOID PAIN RELIEF

Resumen de: WO2026170266A1

The present invention provides mRNA-lipid nanoparticle (mRNA-LNP) compositions and methods for achieving controlled, localised, and transient production of therapeutic peptides, particularly oxytocin and oxytocin derivatives, within nasal epithelial tissues following intranasal administration. Upon uptake by respiratory and olfactory epithelial cells, the mRNA directs endogenous synthesis and secretion of bioactive peptide capable of engaging central nervous system pathways via nose-to-brain transport mechanisms. This approach circumvents the short systemic half-life, poor bioavailability, and inconsistent central penetration that limit the clinical utility of exogenously administered oxytocin peptides. The mRNA-LNP formulations disclosed herein provide extended analgesic and neuromodulatory effects after a single dose, including relief of mechanical, thermal, and postoperative pain, with efficacy comparable to standard analgesics and a notably longer duration of action. The compositions further enhance social and behavioural outcomes without impairing motor function or inducing inflammatory responses in nasal tissues. The invention thus offers a safe, non-invasive, and scalable platform for treating pain, neurological disorders, behavioural conditions, and other indications in which central or peripheral peptide signalling is therapeutically beneficial. Additionally, the modular nature of the mRNA platform allows rapid adaptation to encode alternative neuroactive peptides or

LIPID COMPOSITION, PHARMACEUTICAL COMPOSITION, AND METHOD FOR DELIVERING LIPID COMPOSITION TO IMMUNE CELLS

Resumen de: WO2026173073A1

It is an object of the present invention to provide a lipid composition for delivering a drug to immune cells selected from the group consisting of B cell, plasma cell and NK cells; a pharmaceutical compositions which comprises the above-mentioned lipid composition; and a method for delivering a lipid composition to immune cells selected from the group consisting of B cell, plasma cell and NK cell. The present invention provides a lipid composition comprising (A) a drug and (B) a lipid nanoparticle bound to a targeting molecule, wherein the lipid nanoparticle contain an ionizable lipid, the targeting molecule specifically binds to a surface antigen of an immune cell, and the immune cell is selected from the group consisting of B cell, plasma cell and NK cell.

ENGINEERED PARTICULATE SYSTEMS FOR ANTIGEN-SPECIFIC INDUCTION OF TOLERANCE

Resumen de: WO2026174059A1

The present invention provides dual particle compositions comprising lipid nanoparticles (LNP) comprising mRNA encoding a dendritic cell chemoattractant and an immunosuppressive tolerogenic agent, and microparticles comprising an immunomodulatory agent and a disease-relevant antigen and methods of making the dual particle compositions. Some dual particle compositions comprise lipid nanoparticles (LNP) comprising mRNA encoding a dendritic cell chemoattractant (GM-CSF) and an immunosuppressive tolerogenic agent (TGF-β1), and poly(lactic-co-glycolic acid) (PLGA) microparticles comprising an immunomodulatory agent (1α,25-Dihydroxyvitamin D3) and a rheumatoid arthritis disease-relevant antigen, for example collagen II or citrullinated fibrinogen. The invention provides methods of using the dual particle compositions to deliver disease-relevant antigens to a subject. The invention provides methods of using the dual particle compositions to treat an immune disorder, including an autoimmune disorder, for example rheumatoid arthritis, in a subject.

CATIONIC LIPID COMPOUNDS FOR USE IN LIPID NANOPARTICLES

Resumen de: AU2025223661A1

The present disclosure provides cationic lipid compounds having the following Structure (I): where G1, G2, R1, R2, R3a, R3b, R3c, R3d, R4a, R4b, R4c, R4d, L1, L2, and L3 are as defined herein. The present disclosure also discloses pharmaceutically acceptable salts or stereoisomers thereof. Also provided by the present disclosure are uses 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.

PROTEIN NANOPARTICLES TO INCREASE EXERCISE ENDURANCE, FUNCTIONALITY AND LONGEVITY IN CANCER AND NON-CANCER PATIENTS

Resumen de: US20260240787A1

0000 This invention discloses the beneficial effects of the intravenous administration of protein nanospheres particularly Fibrinogen-coated Albumin Spheres, including the mobilization of stem cells from the bone marrow or other sites, leading to an effective build-up of muscle mass and/or repair of other dysfunctional conditions. The benefits can be produced in the healthy individual, regardless of age, which can lead to greater muscle mass, better exercise endurance and stronger performance, resulting in increased health and even life span. The invention can potentially be applied to mitigate the harm expected in space travel and habitation, including the effects of microgravity and cosmic radiation. In cancer patients with pre-cancer-treatment impairments, FPS can improve deficient domains such as poor physical performance, cognitive, nutritional and psychological deficiencies before and after cancer treatment.

NANOMATERIALS COMPRISING TRIOLS

Resumen de: WO2026174164A1

Compounds of Formula (I) or a pharmaceutically acceptable salt thereof are provided, wherein X1, X2, R4, L1, L2, and L3 are as defined herein. The present disclosure also describes compositions, preparations, lipid nanoparticles comprising compounds of Formula (I) and methods of their use.

MRNA COMBINATION FOR USE IN PREVENTING AND/OR TREATING AN AUTOIMMUNE DISEASE

Resumen de: WO2026171886A1

The invention relates to the prevention and/or treatment of autoimmune diseases using a combination of an mRNA molecule encoding a disease-causing autoantigen and one or more mRNA molecules encoding an immunosuppressing protein. More specifically, the present invention provides for a combination of an mRNA molecule encoding a disease-causing ?(4 particular selected from the list comprising a human IL2 (interleukin 2) mutein, CD39, CD73, IDO (Indoleamine 2,3 dioxygenase), CCL1 (CC motif ligand 1), IL10 (interleukin 10), IL27 (interleukin 27) and TNFAIP3 (tumor necrosis factor alpha-induced protein 3) more in particular CCL1 (CC motif ligand 1). Pharmaceutical compositions comprising said combinations and uses thereof are also provided.

PROCESS FOR ENCAPSULATING DNA

Resumen de: WO2026171732A1

The invention provides a method for rendering DNA compatible with a hydrophobic environment

RADIATION-ACTIVATED PHOTODYNAMIC THERAPY FOR CANCER TREATMENT

Nº publicación: WO2026170260A1 20/08/2026

Solicitante:

NEWSOUTH INNOVATIONS PTY LTD [AU]
NORTHERN SYDNEY LOCAL HEALTH DISTR [AU]
THE UNIV OF SYDNEY [AU]
UNIV OF TECHNOLOGY SYDNEY [AU]
NEWSOUTH INNOVATIONS PTY LIMITED
NORTHERN SYDNEY LOCAL HEALTH DISTRICT
THE UNIVERSITY OF SYDNEY
UNIVERSITY OF TECHNOLOGY SYDNEY

Resumen de: WO2026170260A1

Disclosed herein is a radiation-activated photodynamic therapy for cancer treatment using lipid-polymer nanoparticles. The lipid-polymer nanoparticles comprise: a lipid shell comprising one or more lipids; and an inner core comprising a polymer and a high energy electromagnetic radiation induced photodynamic therapy agent. The mass percentage of the polymer in the lipid-polymer nanoparticle is about 80% or less based on the combined mass of the polymer and lipids.