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274
resultados
Última actualización
28/08/2026 [06:57:00]
Resultados 100 a 125 de 274
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.
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.
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.
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.
Resumen de: WO2026171732A1
The invention provides a method for rendering DNA compatible with a hydrophobic environment
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.
Resumen de: WO2026174106A1
The present invention relates to compositions comprising lipid nanoparticles that encapsulate a first nucleic acid comprising (i) a 5' untranslated region (UTR) sequence and a 3' untranslated region (UTR) sequence, and (ii) a cargo sequence; and a second nucleic acid encoding an R2 retroelement polypeptide. The lipid nanoparticles comprise a plurality of lipids wherein 0.1 to 1, or 2.0 to 2.5 mol percent of the plurality of lipids is PEGylated, or comprise amino lipids such as SM-102, have N:P ratios of 1.5 to 6, and/or have diameters of about 40 to 200 nm. The compositions are used RNA-mediated insertion of transgenes in a cell.
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.
Resumen de: WO2026170677A1
Disclosed in the present invention are a J-aggregation-induced emission photosensitizer with NIR-II emission, and a pharmaceutical composition constructed thereby, a preparation method therefor and the use thereof. The photosensitizer can produce cytotoxic type I active oxygen upon light irradiation, and exhibits excellent fluorescence imaging capability in an NIR-II region and superior phototherapeutic performance. The photosensitizer is further co-assembled with sorafenib into a pharmaceutical composition, which has J-aggregation-induced secondary self-assembly property, enables long-term imaging at a tumor site, fulfills dual functions of NIR-II fluorescence imaging and tumor therapy, and exhibits good stability and low toxic and side effects, thereby holding a wide prospect for clinical application.
Resumen de: WO2026171285A1
Provided are a preparation method for a cellulose nanocrystal-based hydrogel, and an application thereof. The hydrogel preparation method comprises: (1) adding cerium ions to a cellulose nanocrystal (CNC) suspension, and controlling the pH to be neutral, to obtain a cerium oxide-modified CNC suspension; (2) mixing the modified CNC suspension in step (1) with a CNC suspension which has not undergone the modification in step (1), to obtain a CNC mixed suspension; and (3) mixing the CNC mixed suspension in step (2) with a prepolymer, and irradiating with ultraviolet light, to obtain a hydrogel.
Resumen de: WO2026171211A1
Disclosed are a hMPV fusion protein F variant, an mRNA vaccine, and a use thereof. The hMPV fusion protein F variant has an amino acid residue difference at one or more sites relative to an hMPV fusion protein F having the accession number AGJ74096.1 in the GenBank database, wherein the amino acid sequence of the hMPV fusion protein F variant is as shown in SEQ ID NO: 1 or 2. The mRNA vaccine designed on the basis of the hMPV fusion protein F variant can induce a strong specific antibody response, and the persistence of an antibody in vivo is strong. The induced antibody can better neutralize an hMPV strain and control the amplification of the hMPV in the lungs. Therefore, the specific antibody induced by the mRNA vaccine provides an effective basis for prevention of hMPV infection-related diseases and development of combination vaccines.
Resumen de: US20260240889A1
Methods and compositions for the transamniotic delivery of mRNA encoding alpha-1 antitrypsin protein to a fetus for the prenatal treatment of alpha-1 antitrypsin deficiency.
Resumen de: WO2025038853A1
The disclosure relates to compositions, systems, and methods related to repair RNA sequences, as well as related systems for trans-splicing a target nucleic acid using a repair RNA sequence. Various compositions and systems are described herein, including compositions comprising a repair RNA sequence comprising one or more exons and/or introns, a splice donor and/or splice acceptor, wherein the repRNA is suitable for trans-splicing. The compositions, systems, and methods described herein are suitable for targeting one or more Usher syndrome-associated genes, and treating Usher syndrome.
Resumen de: WO2025038475A1
The present disclosure provides compositions and methods comprising recombinant particles suitable for specifically delivering one or more engineered receptors to immune effector cells in vivo.
Resumen de: AU2026210866A1
Abstract The present invention provides a multivalent compound for targeted molecular imaging and/or targeted drug delivery, wherein two components or targeting molecules each interacts with one or more biomarkers on a cell. The present invention further provides a multifunctional chelator to combine the targeting molecules. The present invention also provides an in vitro high-throughput screening assay to determine the length of the spacer molecules. The present invention also relates to compounds/probes, kits and methods for use in targeted molecular imaging and/or targeted drug delivery. ug u g
Resumen de: US20260240786A1
0000 The invention generally relates to polymer-based nano-structures. More particularly, the invention relates to novel, surface-functionalized, guest-host polymer nano-assemblies and nano-delivery vehicles useful in diverse fields including drug delivery, diagnostics and specialty materials. The nano-assemblies and nano-delivery vehicles of the invention are afforded via simplify and reliable approaches.
Resumen de: US20260242803A1
The application provides circular DNA vectors comprising promoters, scaffold matrix attachment region (S/MAR) sequences, and transgenes derived from proteins expressed in respiratory epithelium and which lack drug resistance genes. The application provides pharmaceutical compositions comprising therapeutic amounts of the DNA vectors.
Resumen de: US20260240959A1
The first systemic administration of a CRISPR/Cas9-based therapeutic for in vivo editing of a liver KLKB1 gene in a clinical trial is described. Described herein are methods for in vivo editing of a liver KLKB1 gene by systemically administering a lipid nanoparticle composition comprising an mRNA encoding a Cas9 nuclease and a guide RNA that targets the gene. Assessment of biosafety metrics and clinical efficacy metrics, as well as methods of treatment, are also described herein.
Resumen de: US20260241032A1
0000 Provided herein are systems comprising Class 2, Type V CRISPR proteins and guide nucleic acids (gRNA) useful in the modification of a PCSK9 gene. The systems are also useful for introduction into cells, for example eukaryotic cells. Also provided are methods of using such systems to modify cells having such mutations.
Resumen de: US20260241030A1
The present invention refers to a multifunctional CNC*-Au-based nanoplatform, related conjugates and/or composition containing thereof.The present invention also refers to the use of such a multifunctional CNC*-Au-based nanoplatform, related conjugates and/or composition containing thereof in medicine
Resumen de: US20260240788A1
The present invention deals with a method for preparing a nanosystem vehicle encapsulating one or more active ingredients, preferably selected from pharmaceutical active ingredients, subsequently comprising cryomilling one or more amphiphilic compounds, one or more of said active ingredients and a frozen aqueous medium in order to obtain a uniform frozen mixture, and forming said nanosystem vehicle upon thawing. The invention further relates to a nanosystem vehicle obtainable by the aforementioned method and its uses for therapeutic and non-therapeutic applications.
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.
Resumen de: AU2026213957A1
The present invention relates to lipid nanoparticles (LNP) or compositions thereof for delivery of mRNA molecules encoding CAR, nucleic acid molecule, and/or therapeutic agents to selected targets, such as cells. Thus, in various aspects, the present invention also provides methods of preventing or treating diseases or disorders in a 5 subject in need thereof using the said LNPs or compositions thereof. ug u g
Resumen de: US20260242769A1
0000 Provided herein are methods and compositions and kits related to identification and/or treatment of subjects, including subjects with hyperuricemia, gout or a condition associated with gout, and for preventing gout flare.
Nº publicación: US20260241040A1 20/08/2026
Solicitante:
LAHJAVIDA INC [US]
Lahjavida, Inc.
Resumen de: US20260241040A1
0000 The design, synthesis, and functionalization of a conjugate including a tumor-targeting near-infrared (NIR) dye and a therapeutic agent and/or a diagnostic agent, whereby the NIR dye can function to target the therapeutic agent and/or diagnostic agent to tumor cells.
BOPI
Sede Electrónica
