Alerta

PAMAM-PEG-BASED NANOPARTICLES FOR TARGETED PROTEIN DEGRADATION

Resumen de: WO2026016710A1

Disclosed are a substance represented by general formula (I), a substance represented by general formula (II), and a substance represented by general formula (III) for degrading a protein of interest (POI) in a ubiquitin-proteasome system (UPS): PEG-Ra general formula (I) PEG-Rb general formula (II) (Ra-PEG)m-PAMAM-(PEG-Rb)n general formula (III), wherein Ra, Rb, m, and n are as defined in the present disclosure.

PLA-PEG-BASED NANOPARTICLES FOR TARGETED PROTEIN DEGRADATION

Resumen de: WO2026016711A1

The present invention relates to a substance represented by general formula (I) and a substance represented by general formula (II) for degrading a protein of interest (POI) in a ubiquitin-proteasome system (UPS): PLA-PEG-Ra general formula (I) PLA-PEG-Rb general formula (II), wherein Ra and Rb are as defined in the present invention.

THERANOSTIC TARGETED ULTRASONIC BLASTING NANO-BUBBLES, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: WO2026016457A1

Theranostic targeted ultrasonic blasting nano-bubbles, a preparation method therefor, and use thereof, wherein the nano-bubbles are the targeted drug-gene nano-bubbles miRNA/FTY720/PFP@PEI-T7 NBs. The nano-bubbles can be used for preparing a thyroid ultrasound diagnostic agent or a drug for treating thyroid cancer.

Cellular reprogramming to reverse aging and promote organ and tissue regeneration

Resumen de: AU2025283586A1

Abstract Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2 (or any combination thereof) that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging (or any combination thereof). Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2 (or any combination thereof), an engineered protein selected from the group consisting of OCT4; KLF4; SOX2 (or any combination thereof), an antibody capable of activating expression of OCT4; KLF4; SOX2 (or any combination thereof), methods of treating a disease (e.g., ocular disease) or preventing a disease (e.g., ocular disease), and methods of regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, tissue repair, tissue regeneration (or any combination thereof).

LIPID NANOPARTICLES COMPRISING CODING RNA MOLECULES FOR USE IN GENE EDITING AND AS VACCINES AND THERAPEUTIC AGENTS

Resumen de: AU2024312860A1

The present disclosure describes improved LNP-based RNA vaccines, nucleobase editing systems, and therapeutics for use in treating and/or immunization against disease. In particular, the disclosure describes improved LNPs, including novel and improved ionizable lipids for making LNPs, that enhance the targeted delivery of LNP-based RNA vaccines and therapeutics based on linear and/or circular mRNAs. The improved LNPs protect linear and/or circular mRNA payloads from degradation and clearance while achieving targeted systemic or local delivery for use as enhanced vaccines and/or therapeutic agents.

NANOPARTICLE COMPOSITIONS ASSOCIATED WITH PEPTIDES

Resumen de: AU2024314758A1

The present invention relates to compositions comprising sHDL nanoparticles. In particular, the present invention relates to sHDL nanoparticles comprising a phospholipid; an apoplipoprotein mimetic; a thiol-reactive lipid; and a peptide comprising a linker moiety connected with a payload moiety, wherein the linker comprises cysteine (C) and one to five amino acids independently selected from aspartic acid (D), glutamic acid (E), and serine (S), the payload comprises a polypeptide that is 5 to 35 amino acids in length and has a net positive charge at a pH of 7 to 12, and the peptide comprises a net negative charge at a pH of 7 and an isoelectric point of 0.4 to 12; and wherein the peptide is covalently attached to the thiol-reactive lipid by way of the cysteine (C).

NEW METHOD OF PRODUCING AN AQUEOUS MESOPARTICLE COMPOSITION COMPRISING A LIPOPHILIC COMPOUND

Resumen de: AU2024309710A1

Described is a method for the preparation of an aqueous mesoparticle composition comprising a lipophilic compound, comprising the steps of: a. Providing an emulsifier or a blend of emulsifiers in powder form; b. Mixing one or more oils at a temperature above 40°C where all oils have become liquid, wherein said oils differ in melting temperature and which mixture comprises at least a sufficient amount of medium chain triglycerides to enable the composition formed in step g have a partly liquid oil phase at temperatures around about 4°C; c. Adding the hydrophobic or amphiphilic compound in any hydrophobic solvent to the oil mixture; d. Optionally letting the mixture cool down to room temperature; e. Adding the emulsifier powder and water to the oil mixture and letting the mixture emulsify, under optional agitation and heating to 30-40°C; f. Subjecting the emulsified mixture to a sonication and optionally mixing or fluidisation treatment until the average particle size of the mixture remains stable; g. Cooling down the sonicated mixture allowing sufficient time for crystallisation; and h. Optionally, a second sonication treatment while keeping the mixture cold and compositions produced by the above method.

IONIZABLE LIPID AND USE THEREOF

Resumen de: AU2024307443A1

The present invention provides an ionizable lipid and a drug delivery system comprising the ionizable lipid. Specifically, the present invention provides an ionizable lipid having a structure of formula (I), or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof. Lipid nanoparticles constructed by using the ionizable lipid can realize safe and efficient delivery of nucleic acid drugs, small molecule drugs, peptide drugs and protein drugs.

RAPIDLY-METABOLIZED LIPID COMPOUND

Resumen de: AU2024303397A1

Provided is a rapidly-metabolized lipid compound. The present invention relates in particular to a compound represented by formula (I), or a pharmaceutically acceptable salt, an isotopic variant, a tautomer or a stereoisomer thereof. Also provided are a nanoparticle pharmaceutical composition comprising the compound, and a use of the compound and a composition thereof in delivering nucleic acids.

COMPOSITIONS AND METHODS FOR DELIVERY OF THERAPEUTIC PEPTIDES

Resumen de: AU2024299322A1

Compositions and methods for delivery of therapeutic peptides are disclosed herein. In some embodiments, for example, a composition for treating a disease or condition includes a dynamic hydrogel, and an acylated peptide encapsulated by the dynamic hydrogel. The dynamic hydrogel can include a polymer and a plurality of nanoparticles. The polymer can be non-covalently crosslinked with the plurality of nanoparticles.

FUNCTIONALIZED COMPOSITIONS COMPRISING AN ENGINEERED PHENYLALANINE AMMONIA LYASE (PAL)

Resumen de: AU2024289116A1

The present invention relates to a composition comprising a solid carrier, an engineered phenylalanine ammonia lyase or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the engineered phenylalanine ammonia lyase or a fragment thereof by embedding the engineered phenylalanine ammonia lyase or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses thereof.

PREPARATION METHOD FOR NANOBODY TARGETING TISSUE FACTOR AND CONJUGATE AND USE THEREOF

Resumen de: AU2024252644A1

Disclosed are a new nanobody (Nb) targeting a tissue factor (TF) and a nanobody-drug conjugate (NDC), a preparation method therefor and the use thereof. The monoclonal nanobody and the corresponding NDC can efficiently and high-specifically bind to a purified TF protein and a TF on the surface of various TF abnormally expressed tumor cells, have a high affinity and a low immunogenicity, and have a significant anti-tumor effect in vivo and in vitro.

BROAD SPECTRUM NANOZYMES

Resumen de: US20260022367A1

Disclosed herein are improved broad-spectrum nanozymes for targeting RNA. The disclosed nanozymes are synthesized using recombinant ribonuclease with site-specific cysteine-substituted mutations that can be covalently functionalized with a length-tunable multithiol tether and then loaded onto gold particles through multiple gold-sulfur bonds, or inorganic particles with specific multiple ligand-to-particle-surface bonds. The disclosed nanozymes are also densely loaded with protective DNA oligonucleotides. In some embodiments, the disclosed nanozyme are core-free hollow forms. The removal of the inorganic nanoparticle cores from nanozymes can effectively eliminate the potential long-term toxicity induced by the core, and also creates a cavity for loading and delivery of small molecule drugs.

Asymmetric Piperazine-Based Cationic Lipids

Resumen de: US20260021055A1

The present invention provides, in part, asymmetric piperazine-based lipid compounds of Formula (I′), and sub-formulas thereof or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

LIPID NANOPARTICLE AND PHARMACEUTICAL COMPOSITION

Resumen de: US20260021046A1

The present invention relates to lipid nanoparticles capable of delivering a target substance to hepatic stellate cells. The lipid nanoparticles are for delivering a target substance to hepatic stellate cells and comprise a pH-sensitive cationic lipid including a hydrophilic portion and two hydrophobic portions, wherein an acid dissociation constant pKa of a lipid membrane constituting the lipid nanoparticles is greater than or equal to 6.7 and less than 8.2.

SIV ENVELOPE TRIMER

Resumen de: US20260022141A1

The present application relates to epitope-targeted SIV and HIV vaccines. The invention provides novel envelope glycoproteins which may be utilized as HIV-1 vaccine immunogens, antigens for crystallization, and for identification of broadly neutralizing antibodies. The invention encompasses preparation and purification of immunogenic compositions which are formulated into vaccines of the present invention.

NANOPARTICLES AND PEPTIDES FOR THE DELIVERY OF CARGOS TO CHONDROCYTES

Resumen de: US20260021197A1

Nanoparticles suitable for delivery of a cargo to a chondrocyte, and targeting peptides comprising a chondrocyte targeting sequence, are provided. Further provided are uses of the nanoparticles and targeting peptides, for example, in treating a joint or cartilage disease or disorder.

RNA LIPID NANOPARTICLES (LNPs) COMPRISING A POLYOXAZOLINE AND/OR POLYOXAZINE POLYMER

Resumen de: US20260021199A1

The present disclosure relates to RNA nanoparticles (LNPs) for delivery of RNA to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous, intratumoral, intraarterial, intradermal, dermal, intranasal, rectal or oral administration, and compositions comprising such RNA LNPs. The RNA LNPs in some embodiments comprise single-stranded RNA such as mRNA which encodes a peptide or protein of interest, such as a pharmaceutically active peptide or protein. The RNA is taken up by cells of a target tissue and the RNA is translated into the encoded peptide or protein, which may exhibit its physiological activity. Furthermore, the present disclosure relates to certain conjugates of (a) a polyoxazoline (POX) and/or polyoxazine (POZ) polymer and (b) one or more hydrophobic chains, compositions comprising such conjugates, and the uses of such conjugates and compositions.

COMPOSITIONS CONTAINING ABIOTICALLY-STRESSED PLANT-DERIVED EXOSOME-LIKE NANOPARTICLES

Resumen de: US20260021157A1

The present disclosure provides a composition containing a purified population of plant-derived exosome-like nanoparticles isolated from tissue of a vascular plant, wherein the exosome-like nanoparticles comprise a tuned cargo comprising a protein signature and an miRNA signature, wherein the tuned cargo of the plant-derived exosome-like nanoparticles is a result of exposure of the plant to combinations of abiotic stress conditions that cause the plant to modulate its signaling pathways and metabolism to ensure its survival in a challenging environment. The tuned cargo of the plant-derived exosome-like nanoparticles can modulate bioactivities of mammalian cells directly or indirectly. The present disclosure also provides a method for improving appearance of human skin and human hair health, including eyelashes and eyebrows, comprising applying a composition comprising the abiotically stressed plant-derived exosome-like nanoparticles containing the tuned cargo and a carrier; and applying the composition topically.

Chelation Therapy to Limit Cell Senescence

Resumen de: US20260021066A1

The present application is generally directed to methods for chelating calcium deposits within calcified blood vessels, as well as methods and compositions for use in regulating various senescence-related inflammatory pathways.

METHODS AND COMPOSITIONS FOR TREATING PARASITIC DISEASES USING NANOBUBBLES AND ANTI-PARASITIC AGENTS

Resumen de: US20260021054A1

Provided herein are methods and compositions for treating parasitic diseases using nanobubbles in combination with at least one anti-parasitic agent.

IMMUNOGENIC COMPOSITIONS AND USES THEREOF

Resumen de: US20260021175A1

The present disclosure relates to RSV F, hMPV F, PIV3 F, PIV3 HN, PIV1 F and PIV1 HN protein mutants, nucleic acids or vectors encoding them, compositions comprising a RSV F, hMPV F, PIV3 F, PIV3 HN, PIV1 F and/or PIV1 HN nucleic acid or combinations thereof, and uses thereof.

EXTRACELLULAR VESICLES AND PARTICLES FROM IMMUNE CELLS FOR PROVIDING ANTICANCER ACTIVITY

Resumen de: US20260021138A1

The present invention is related to the methods for obtaining compositions of extracellular vesicles and particles for providing an anticancer activity which consists of recognising and eliminating cancerous cells. The compositions are obtained by a set of methods that include immune cell stimulation by Toll-like receptor agonist, microenvironment stiffness and adhesion peptides, extracellular vesicle and particle collection, decreasing size of large extracellular vesicles for applicability adaptation and loading of an anticancer agent for delivery to cancer cells.

DIBLOCK POLYMER

Resumen de: WO2026017913A1

A nanoparticle comprising a diblock polymer comprising a first component covalently bound via a linker to a second component; wherein said first component is an oligomer comprising at least 50 mol% L-5 guluronic acid residues or at least 50 mol% galacturonic acid residues and having a degree of polymerisation n where n is at least 3; said second component is a second polymer having no more than 30 mol% L-guluronic acid residues or galacturonic acid residues and having a degree of polymerisation of at least 4;10 wherein said nanoparticle comprises a chelator such as DOTA; wherein said nanoparticle comprises at least one metal ion such as a radionuclide or Gd ions.

LIPID NANOPARTICLES FOR DIRECT DELIVERY

Nº publicación: WO2026017865A1 22/01/2026

Solicitante:

PROVIREX GENOME EDITING THERAPIES GMBH [DE]
PANTHERNA THERAPEUTICS GMBH [DE]
PROVIREX GENOME EDITING THERAPIES GMBH,
PANTHERNA THERAPEUTICS GMBH

Resumen de: WO2026017865A1

The present invention relates to the field of lipid nanoparticles (LNP) and direct delivery of an active agent to a cell. It provides lipid nanoparticle constructs that are particularly useful for delivery of active agents, e.g., of RNA, preferably, mRNA, to cells. The lipid nanoparticle constructs comprise a specific neutral hydrophilic solid lipid nanpoparticle (SLP) and a targeting agent associated with the nanoparticle, e.g. a single domain antibody (sdAb), which may, for example target CD4. The lipid nanoparticle construct may comprise an active agent, and it may be used for targeting a cell such as a T cell. Pharmaceutical compositions comprising the nanoparticle constructs are also provided, in particular for use in targeting a cell. The invention also provides a composition or kit suitable for preparing the lipid nanoparticle construct of the invention.