Resumen de: US20260232601A1
Disclosed herein are functionalized polyethylenimine compounds for use as nanoparticles. Methods of making the compounds and nanoparticles comprising a cargo and methods of delivering a nanoparticle comprising a cargo are provided. The nanoparticles of the present invention can be utilized in methods of delivering cargo in a tissue specific manner, providing low toxicity and efficient release of cargo in the cell.
Resumen de: US20260234204A1
The invention relates to truncated isolated BRK peptides and functional peptides thereof that inhibit the phosphorylation of p27Kip1 and the resulting kinase activity of CDK2 and CDK4; and to pharmaceutical compositions thereof. The invention further relates to the use of the isolated peptides in methods of treating cancer in subject in need thereof. The methods of treating cancer include methods of treating pancreatic cancer.
Resumen de: US20260232777A1
The present invention relates to a pharmaceutical combination comprising (i) at least one nucleic acid comprising an antisense and flipped sequence of a fragment of a sequence coding for a kanamycin resistance protein and (ii) at least one non-immunosuppressive inducer of tumor cell apoptosis, wherein said nucleic acid is formulated with a nano-vehicle. Said nucleic acid is preferably pVAX14 plasmid, said non-immunosuppressive inducer of tumor cell apoptosis is preferably ATRA, an arsenic-related compound or azacytidine. The present invention also relates to the pharmaceutical combination as defined above for use in the treatment of cancer or infectious diseases, by activating the innate immune pathway and immunological cell death pathway. Pathway analyses of gene expression profiles revealed a DNA activated gene list regulated in 16 immune pathways. This gene list provides biomarkers of response to DNA treatment.
Resumen de: US20260232602A1
0000 The present invention generally relates to particles, including nanocapsules or other nanoentities, comprising a polymer such as polysialic acid. The particles are able to access the interior of the cells, and/or to procure the intracellular release of the associated drugs. In one aspect, the present invention is directed to nanocapsules or other entities having an exterior or surface comprising a polymer such as polysialic acid. In some cases, targeting moieties such as Lyp-1 or (Lyp-1 peptide are bonded to the polymer, e.g., using aminoalkyl (C<1>-C<4>) succinimide or other linkers. These may be created, for example, by reacting a carboxylate moiety on a polymer with an aminoalkyl maleimide (C<1>-C<4>) or an aminoalkyl (C<1>-C<4>) methacrylamide, and reacting the resulting aminoalkyl (C<1>-C<4>) maleimide or the aminoalkyl (C<1>-C<4>) methacyrlamide to a cysteine or other sulfur group. Targeting moieties are bonded to the polymer, for example, by reacting a carboxylate moiety on a polymer with a N-hydroxysuccinimide or a carbodimide, and reacting the intermediate formed with a lysine or arginine group on a targeting peptide to produce polymer-amide-peptide. Other aspects of the invention are generally directed to methods of making or using such compositions, kits including such compositions, or the like.
Resumen de: US20260234671A1
Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases.
Resumen de: US20260232839A1
The present disclosure is a lipid nanoparticle including a composition including a protein selected from an antibody and an antibody fragment, a polymer, and a lipid, in which the polymer and the protein have opposite charges at a predetermined pH. The lipid nanoparticle preferably has a protein encapsulated therein. In addition, when the particle diameter of the lipid nanoparticles is measured, the peak top of the particle diameter distribution is preferably at 80 nm to 120 nm.
Resumen de: US20260232758A1
An exemplary embodiment of the present disclosure provides an intracellular delivery complex, comprising at least one biomacromolecule, each of the at least one biomacromolecules having a first ionic net charge, and at least one hydrophobic counterion, each of the at least one hydrophobic counterions having a second ionic net charge opposite the first ionic net charge, and wherein the complex is not encapsulated in a nanocarrier. Another embodiment provides a method of delivering a biomacromolecule into a cell, which comprises providing a biomacromolecule as described herein, a hydrophobic counterion as described herein, and mixing the two under conditions effective to form a complex capable of entering a cell. Another embodiment provides a method of administering a biomacromolecule into a cell, which comprises providing the intracellular delivery complex of the present disclosure, and administering the intracellular delivery complex to a subject, such that the complex enters a cell of the subject.
Resumen de: US20260232781A1
0000 Described herein is a composition for immunization of a mammal against an infection by a parasite. The composition includes an extracellular vesicle produced by a red blood cell infected with the parasite; and a pharmaceutically acceptable carrier for parenteral administration. Also described herein is a method for immunizing a subject against an infection by a parasite. The method includes administering to the subject an effective amount of an extracellular vesicle exuded by the parasite, which resides with the host red blood cell.
Resumen de: US20260232589A1
A nanopreparation loaded with trabectedin and paclitaxel is provided. The nanopreparation loaded with trabectedin and paclitaxel includes a non-targeted drug-loaded micelle loaded with paclitaxel and a first targeting group linked to a first micelle carrier, where the non-targeted drug-loaded micelle has a first shell-core structure, and the first micelle carrier is prepared from poly(ε-caprolactone)-poly(2-ethyl-2-oxazoline) (PCL-PEOz), where a hydrophobic block of PCL and paclitaxel jointly form a core of the first shell-core structure, and a hydrophilic block of PEOz forms a shell of the first shell-core structure; and the first targeting group is linked via an amide bond formed between —NH2 on a surface of the first targeting group and —COOH at a terminus of the PEOz on a surface of the shell.
Resumen de: US20260232604A1
Methods for preparing particles and related compositions are provided. In some embodiments, the particles include at least one polynucleotide (e.g., mRNA), and in certain embodiments, the particles may include at least one ionizable molecule (e.g., a lipid). A method for preparing a suspension including the particles may comprise one or more filtration steps. In some such embodiments, prior to or during filtration, one or more properties of the particles (e.g., surface charge) and/or one or more properties of the suspension (e.g., pH) may be altered. In some embodiments, altering one or more properties of the particles and/or suspension may improve yield, improve a characteristic of the resulting composition, and/or prevent or reduce certain problems, such as fouling during the filtration process.
Resumen de: US20260233124A1
0000 The present invention relates to methods for the purification of lipid nanoparticles (LNPs) encapsulating a nucleic acid, comprising the steps of subjecting a solution containing said LNPs to a chromatographic medium with convective flow properties in the presence of at least one kosmotropic agent; and eluting LNPs from said chromatographic medium. The present invention further relates to respective uses of a chromatographic medium with convective flow properties for the purification of lipid nanoparticles (LNPs) encapsulating a nucleic acid.
Resumen de: US20260232847A1
0000 Therapeutic methods, imaging methods, and inorganic nanotherapeutic imaging agents and inorganic nanoimaging agents. Inorganic nanotherapeutic imaging agents and inorganic nanoimaging agents are used in therapeutic methods and imaging methods. The inorganic nanotherapeutic imaging agents and inorganic nanoimaging agents are, for example, silica or aluminosilicate nanoparticles functionalized with fluorescent group(s), targeting groups, and therapeutic groups. Therapeutic methods can be used to treat and imaging methods can be used to investigate or diagnose inflammatory joint diseases, such as, for example, rheumatoid arthritis, osteoarthritis, or both, in a subject.
Resumen de: US20260232707A1
Disclosed are compounds, nanoparticles, and compositions for effective delivery of metabolic inhibitors to disease state cells. The compounds, nanoparticles, and compositions disclosed herein show trackability in biological system, extended stability, and other advantageous physicochemical properties to treat various disease states. Also disclosed are methods of treating a subject in need thereof, such as a subject with cancer.
Resumen de: US20260232788A1
0000 The present invention relates to a dengue virus messenger ribonucleic acid (mRNA) vaccine. In particular, the vaccine includes an mRNA encoding a mutant envelop (E) protein of dengue virus at position 8 and/or 101 formulated in lipid nanoparticles. The mRNA vaccine of the present invention is safe and effective, which causes enhanced production of neutralizing antibodies against multiple serotypes of dengue virus and reduced antibody dependent enhancement (ADE) response.
Resumen de: US20260232592A1
0000 The present invention relates to a method for the production of gold nanoparticles (AuNPs) coated with glutathione and Li<+> ions, hereinafter designated as LiG-AuNPs, to a method for the preparation of aggregates of said nanoparticles and to the use of said nanoparticles, aggregates or compositions thereof which comprise them for therapeutic use. LiG-AuNPs then are an effective instrument in inhibiting GSK-3 and its downstream molecular targets, while keeping the lithium extracellular concentration levels below the systemic toxicity threshold (1.5 mEq/L), and exerting an antioxidant action by means of the glutathione present on their surface.
Resumen de: US20260232590A1
The present invention relates to a composition comprising lipid nanoparticles comprising rosehip oil, at least one solid lipid, at least one surfactant, and optionally at least one active ingredient, as well as their use in a method of treating ocular diseases, such as for example dry eye disease.
Resumen de: US20260234617A1
0000 The invention relates to formulations comprising miRNA that have improved stability for the treatment of diseases including neurodegenerative diseases such as spinocerebellar ataxias type 3.
Resumen de: US20260234655A1
0000 Methods of nuclear targeted DNA delivery are provided. Aspects of the methods include contacting a cell with: (a) a nuclear targeted deoxyribonucleic acid (NTDNA) that includes a DNA nuclear targeting sequence (DTS) and a cargo nucleic acid heterologous to the DTS. Also provided are compositions for use in practicing methods of the invention.
Resumen de: US20260234614A1
Inhibitors, pharmaceutical compositions and methods for inhibiting the expression of Fos-related antigen-1 (Fra-1) or Fos-related antigen-2 (Fra-2) in fibrogenic tissue cells for use in the treatment or prevention of organ or tissue fibrosis. The inhibitor of the present invention is characterized in that it interferes with Fra-1 or Fra-2 expression, resulting in a reduction in the amount of Fra-1 or Fra-2 in the fibrogenic cells to levels found in non-fibrotic healthy cells or tissues.
Resumen de: WO2026169759A1
The present disclosure relates generally to compositions and methods for in vivo gene therapy, and more specifically to delivering mRNA encoding anti-CD19 chimeric antigen receptors (CARs) to immune cells (e.g., T cells, such as CD8+ T cells) in vivo.
Resumen de: WO2025073055A1
Provided is a method for inducing an immune response in a subject to treat or prevent a disease or disorder, the method comprising administering a vaccine comprising a lipid nanoparticle encapsulating nucleic acid encoding an antigenic protein, peptide or fragment thereof, a neutral lipid content of from 30 mol% to 70 mol%, a sterol or derivative thereof and an ionizable cationic amino lipid at between 5 and 50 mol%, and a hydrophilic polymer-lipid conjugate that is present at a lipid content of 0 mol% to 5 mol%, wherein the administering of the lipid nanoparticle results in the immune response against the antigenic protein, peptide or fragment thereof expressed by the mRNA in the subject, wherein each mol% is measured relative to a total lipid content of the lipid nanoparticle. The disclosure further provides vaccine compositions and use of such compositions to induce an immune response in a subject.
Resumen de: EP4790140A2
0001 The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells.
Resumen de: WO2025074120A1
The present invention is directed to nanoparticle compositions obtainable from the polymerisation of oligolactoglycolic acid dimethacrylates (OLGADMAs), methods for preparing said OLGADMAs, methods for preparing said nanoparticle compositions, and uses thereof. In one aspect the present invention provides a nanoparticle composition obtainable from precipitation polymerisation of one or more oligomers of formula (I), as defined herein.
Resumen de: WO2025076415A1
Artificial expression constructs including a dnSHP1, caBCL2, dnNRDP1, PCK1, cFLIP.v3, caAKT, survivin, or dnFADD transgene under the control of an inducible promoter are described. The artificial expression constructs can be used to enhance the function of a recombinant receptor-immune cell (e.g., CAR T-cell). The dnSHP1, caBCL2, dnNRDP1, PCK1, cFLIP.v3, caAKT, survivin, or dnFADD transgene disclosed herein enhances the potency of the recombinant receptor-immune cell (e.g., CAR T-cell), resulting in potentiated cell killing ability, proliferation, cytokine output, and/or maintenance of artificial expression construct positivity.
Nº publicación: EP4788666A1 12/08/2026
Solicitante:
CAPSTAN THERAPEUTICS INC [US]
Capstan Therapeutics, Inc.
Resumen de: WO2025076127A1
Ionizable cationic lipids, methods for synthesizing the same, intermediates useful in synthesis of the ionizable cationic lipids and methods of synthesizing the intermediates are disclosed. The ionizable cationic lipids are useful as a component of lipid nanoparticles (LNP), which in turn can be used for the delivery of nucleic acids into cells in vivo or ex vivo. LNP compositions are also disclosed, including LNP comprising a functionalized lipid to enable conjugation of a binding moiety, and targeted LNP (tLNP), that is an LNP in which a binding moiety has been conjugated to the functionalized lipid and can serve as a targeting moiety to direct the tLNP to a desired tissue or cell type.