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HYBRID PEPTIDE DENDRIMER SYSTEMS AND EXTRAHEPATIC DELIVERY

Resumen de: AU2023317842A1

The present invention relates to compositions that can deliver therapeutic molecules such as nucleic acids to mammalian cells, and to the human and animal body and methods of preparing and using the same. The compositions comprise nanoparticles comprising a peptide dendrimer, a nucleic acid and a lipid. The compositions of the invention find utility in the field of medicine, such as for treating cancer and autoimmune diseases.

LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS

Resumen de: AU2023338228A1

Compounds are provided having the following Structure (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R

CATIONIC LIPID COMPOUND, COMPOSITION CONTAINING SAME AND USE THEREOF

Resumen de: US2025082576A1

Provided in the present disclosure are a compound of formula (I), or an N-oxide, solvate, pharmaceutically acceptable salt or stereoisomer thereof. Further provided are a composition containing the aforementioned compound, and the use thereof in the delivery of a therapeutic agent or prophylactic agent.

NUCLEOSIDE FORMULATION

Resumen de: US2025082664A1

The present specification relates to a nucleoside formulation which comprises a nucleoside or a phosphate thereof; and an amphipathic cell penetrating RALA peptide. In one embodiment, the nucleoside formulation comprises gemcitabine or a phosphate thereof and the peptide WEARLARALARALARHLARALARALRACEA.

POLYANION COATED LIPID NANOPARTICLES AND METHODS OF USE THEREOF

Resumen de: US2025082776A1

Described herein are nanoparticles comprising an LNP core with electrostatically adsorbed anionic polymers layered on the LNP surface. These nanoparticles comprise varying nucleic acid cargos and may further comprise targeting moieties covalently attached to the anionic polymers. Also provided are methods of administering cargo to a subject, methods of treatment, methods of editing a gene, and methods of reducing non-targeted cell uptake of nanoparticles.

Targeted degradation and removal of amyloid beta plaques for the prevention and treatment of alzheimer’s diseases via engineered nano-scavenger exosomes

Resumen de: US2025082775A1

Exosome-Based Nano-Scavenger (EBNS) are provided to precisely target and remove Aβ plaques. Leveraging exosomes, natural cell-derived vesicles with tissue-penetrating capabilities, these exosomes were engineered to carry disease targeting antibodies and Aβ-degrading enzymes, clearing Aβ specifically, safely and effectively. EBNS can overcome existing treatment limitations and offer a promising avenue for Alzheimer's Disease therapy.

THERAPEUTIC AGENTS FOR TREATMENT OF CORONAVIRUS INFECTION

Resumen de: US2025082667A1

A therapeutic method for treatment of coronavirus infections. The therapeutic method includes using a composition containing a gold compound effective for Rheumatoid Arthritis treatment that both generates gold-S bonds at the active pockets of the main protease of the virus and suppresses the virus induced inflammations in the body.

BIOLISTIC PARTICLE DELIVERY COMPOSITIONS, METHODS, AND SYSTEMS

Resumen de: US2025084440A1

Macrocarriers with a hydrophobic and/or proteinaceous coating are disclosed. Such coated macrocarriers can increase transfer efficiency of microcarriers into targeted cells when used in a biolistic particle delivery device.

TARGETING OF XKR8 IN THERAPIES

Resumen de: US2025084416A1

A therapeutic system or combination includes a first therapeutic agent to treat a disease condition and a second therapeutic agent adnrinistrable within a predetermined time of administration of the first therapeutic agent the second therapeutic agent inhibiting the function of Xkr8.

METHODS OF EX VIVO DOSING AND ADMINISTRATION OF LIPID PARTICLES OR VIRAL VECTORS AND RELATED SYSTEMS AND USES

Resumen de: US2025082777A1

Provided herein are methods of ex vivo administration of a lipid particle or a payload gene, to a subject. In some embodiments, the methods are in-line methods of administration of a lipid particle or payload gene that are performed in a closed fluid circuit. Also provided are related compositions, containers, and systems in connection with the provided methods.

LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS

Resumen de: US2025084030A1

Compounds are provided having the following structure:or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein R1a, R1b, R2a, R2b, R3a, R3b, R4a, R4b, R5, R6, R7, R8, L1, L2, G1, G2, G3, a, b, c and d 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.

glycerylphosphorylethanolamine (GPE) compound and lipid nanoparticle (LNP)-based drug carrier with same

Resumen de: US2025084051A1

A glycerylphosphorylethanolamine (GPE) skeleton compound and a lipid nanoparticle (LNP)-based drug carrier with the same are provided. The GPE skeleton compound has a structure shown in formula (I):A dithiolane structure is modified on a hydrophilic phosphate terminus of the GPE, such that the dithiolane structure is more fully exposed on a surface of the LNP, and the surface of the LNP can expose more dithiolane, thereby further promoting endocytosis of the LNP by mucosal cells. The GPE skeleton compound has a wide adjustment range in an LNP formulation and can increase a content of the dithiolane in the LNP. A nucleic acid-LNP composition and a pharmaceutical preparation are further provided, and can be used for ocular delivery, pulmonary inhalation delivery, and nasal spray of nucleic acid drugs, providing a new option for extrahepatic delivery of the nucleic acid drugs.

METHODS OF INDUCING CELL DEATH OF A POPULATION OF SOLID TUMOR CELLS

Resumen de: US2025084389A1

It was previously demonstrated that the RAC2 G12R mutation rapidly induced HSPCs cell death and hematopoiesis regulation. Now, the invention evaluated the impact of said mutation on three tumor cell lines: MDA-MB-231 (mammary gland adeno-carcinoma), HT29 (colorectal adenocarcinoma) and HepG2 (hepatocellular carcinoma). Briefly, cells were transduced with a lentiviral vector containing the green fluorescent protein (GFP) reporter cDNA (WPI) or a wild type form of RAC2 cDNA (WT) or a RAC2 mutated cDNA form (G12R). The inventors showed that the number of GFP+ cells is drastically lower in the G12R condition as compared to the WT and WPI conditions. The cell morphology and content are particularly disrupted. These observations were confirmed in a time-course proliferation assay performed on MDA-MB-231 and HT29 cell lines. Altogether, these data underlie the deleterious impact of the RAC2 G12R mutation on tumor cell lines proliferation.

LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS

Resumen de: US2025084029A1

Compounds are provided having the following structure:or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein R3, L1, L2, G1, G2 and G3 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.

FORMULATION OF METAXALONE

Resumen de: US2025084043A1

Dosage forms of metaxalone containing submicron particles of metaxalone and uses thereof are described. The submicron dosage forms have improved bioavailability compared to certain conventional metaxalone dosage forms.

NOVEL IONIZABLE LIPIDS AND LIPID NANOPARTICLES AND METHODS OF USING THE SAME

Resumen de: AU2023334610A1

Novel ionizable lipids and lipid nanoparticles that can be used in the delivery of therapeutic cargos are disclosed.

POLYMER COMPOSITE NANOMATERIAL ENCAPSULATION SYSTEM

Resumen de: AU2023342576A1

Generally, a polymer nanomaterial encapsulation system useful in the production of polymer encapsulated nanoparticles comprised of a hydrophobic nanoparticle encapsulated in the hydrophobic region of the polymer with the external hydrophilic region of the polymer ensuring water-solubility and affording a functional group which can be utilized for the production of nanoparticle conjugates. Specifically, particular embodiments include a polymer nanoparticle structure including one or more of: a quantum dot and/or a superparamagnetic iron oxide nanoparticle and/or an upconverting nanoparticle, encapsulated in polystyrene-b-polyethylene glycol amine for the production of antibody conjugates useful in the capture of cellular targets.

KIT FOR MANUFACTURE OF DRUG-CONTAINING NANOPARTICLES AND NANOPARTICLE COMPOSITION FOR DRUG DELIVERY

Resumen de: AU2023338088A1

The present invention relates to a kit for construction of drug-containing nanoparticles and a nanoparticle composition for drug delivery. More specifically, the present invention concerns a kit for construction of drug-containing nanoparticles and a nanoparticle composition for drug delivery, each designed to increase the efficiency of cellular drug delivery by utilizing nanoparticles that include a cationic compound and an anionic polymer compound with at least one acidic functional group.

COMPOSITIONS AND METHODS FOR DELIVERY OF OCULAR THERAPEUTICS

Resumen de: US2025082573A1

Compositions for treating ocular disease are disclosed herein. In some embodiments, the composition comprises a dynamic hydrogel comprising a polymer and a plurality of nanoparticles, wherein the polymer is non-covalently crosslinked with the plurality of nanoparticles. The dynamic hydrogel can also comprise an ocular therapeutic encapsulated by the dynamic hydrogel.

COMPOSITIONS COMPRISING ANTI-VEGF AND NANOPARTICLES AND METHODS OF USING THE SAME FOR THE TREATMENT OF ABNORMAL OR EXCESSIVE ANGIOGENESIS

Resumen de: EP4520321A2

A composition for treating abnormal or excessive angiogenesis, such as pyogenic granuloma comprising an anti-vascular endothelial growth factor (anti-VEGF) agent (e.g., an antibody or small molecule inhibitor of VEGF signaling) and a carrier comprising nanoparticles. Methods of treating abnormal or excessive angiogenesis by administering a composition comprising an anti-VEGF agent and nanoparticles, alone or in combination with administering an anti-inflammatory steroid, and administering a non-steroidal anti-inflammatory drug (NSAID) to a subject. Devices for administering the composition for treating pyogenic granuloma are also disclosed.

SILOXANE-BASED LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY

Resumen de: WO2023215796A2

The present disclosure relates, in part, to siloxane-based lipids or lipidoids, lipid nanoparticles (LNPs) comprising the same, and pharmaceutical compositions thereof. In certain embodiments, the LNPs of the present disclosure selectively target cells (e.g., hepatocytes, epithelial cells, endothelial cells, and immune cells, inter alia) and/or organs of interest (e.g., liver, spleen, heart, and lungs, inter alia). In another aspect, the present disclosure relates to methods of treating, preventing, and/or ameliorating one or more diseases and/or disorders in a subject, the method comprising administering to the subject at least one LNP of the present disclosure and/or at least one pharmaceutical composition of the present disclosure.

NANOMATERIAL-STEM CELL COMPOSITIONS AND METHODS OF USE

Resumen de: AU2023265915A1

Disclosed herein are biocompatible and biodegradable nanomaterials combined with molecules of interest and stem cells in a variety of stable and safe compositions. The nanomaterials comprise poly(ethylene glycol)-oligo(ethylene sulfide) (PEG-OES) amphiphilic block-copolymers that self-assemble in supramolecular aggregates of fibrillar shape. The fibrillar architecture of the assemblies allows the easy, fast and not harmful internalization into stem cells, including the preferred umbilical cord derived mesenchymal stem cells (UC-MSC). The OES core enables loading of hydrophobic molecules, such as imaging agents and drugs, which are carried by the nFIB into the stem cells for a final product that comprises a composition of MSC, nFIB and therapeutic molecule (e.g., MSC-nFIB-Rapamycin). The technology can be utilized to enhance the immunoregulatory potency of MSC via intracellular nanomaterial delivery of immunosuppressive drugs, and to obtain active site-targeting and localized delivery of drug-loaded nanofibrils by exploiting the MSC homing ability.

LIPID NANOPARTICLE FORMULATIONS FOR CENTRAL NERVOUS SYSTEM DELIVERY

Resumen de: AU2023264015A1

Provided herein are compositions and methods of delivering nucleic acids, such as therapeutic mRNAs and DNA, to the central nervous system by delivery,

SIGNAL SEQUENCES FOR NUCLEIC ACID VACCINES

Resumen de: AU2023265481A1

Provided herein is a nucleic acid (e.g., messenger RNA) vaccine encoding at least one antigenic prokaryotic polypeptide linked to one or both of a viral secretion signal peptide and a transmembrane domain. Also provided are methods of vaccination against a prokaryotic infection with the nucleic acid described herein.

IONIZABLE CATIONIC LIPIDS INCORPORATING SILICON

Nº publicación: EP4520765A1 12/03/2025

Solicitante:

ALDEXCHEM KFT [HU]
AldexChem Kft

Resumen de: EP4520765A1

The present invention belongs to the field of biomedicine and drug delivery as well as pest and vector controls.The invention relates to a novel ionizable cationic lipid family incorporating silicon, which belongs to the trademark LipexSil<™> 2 generation lipids, wherein the tail is connected to the headgroup with biodegradable silyl acetal linker. Lipids containing silyl acetal linker(s) are state-of-the-art and are effective as ionizable cationic lipids in the formulation of empty or loaded lipid nanoparticles (LNPs). The novel linkers according to the invention are designed by means of proprietary borane catalysts WO 2022/129966. The invention describes the synthesis of the lipids of formula (I), formation and characterization of nanoparticles and biological experiments demonstrating that the lipid nanoparticles prepared with these novel lipids can efficiently deliver their cargo (e.g. RNA, DNA, mRNA, siRNA, dsRNA, pDNA, circular DNA, small biologically active molecules) into the cells.