3793 results about "Compound s" patented technology

The disclosure features macrocyclic compounds, and pharmaceutical compositions and protein complexes thereof, capable of inhibiting Ras proteins, and their uses in the treatment of cancers.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Aspects of the disclosure relate to methods of reducing fatigue in a subject having myotonic dystrophy type 1 (DM1). Aspects of the disclosure relate to methods of treating one or more symptoms assessable by the MDHI (e.g., a GI symptom, myotonia, upper extremity function impairment, fatigue, mobility impairment, impairment in the ability to perform activities, pain, vision impairment, communication impairment, sleep impairment, emotional issues, cognitive impairment, social satisfaction impairment, social performance impairment, breathing impairment, swallowing impairment, and / or hearing impairment) in a subject having myotonic dystrophy type 1 (DM1). In some embodiments, the methods comprise administering to the subject a composition comprising complexes (e.g., muscle targeting complexes) comprising an oligonucleotide (e.g., a DMPK- targeting oligonucleotide) covalently linked to an antibody (e.g., anti-TfRl antibody).

The disclosure features macrocyclic compounds, and pharmaceutical compositions and protein complexes thereof, capable of inhibiting Ras proteins, and their uses in the treatment of cancers.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The present invention relates to an immunomicrobubble complex and its uses. The immunomicrobubble complex (IMC) according to the present invention comprises microbubbles conjugated with an antibody, wherein the microbubbles have excellent stability and excellent antibody binding strength, and it has been confirmed that when the immunomicrobubble complex is treated with high-intensity focused ultrasound (HIFU), the anti-tumor effect is significantly enhanced and an immune-enhancing effect is exhibited. Therefore, the immunomicrobubble complex according to the present invention is expected to improve the delivery efficiency of the conjugated antibody and can be used for the diagnosis and treatment of cancer, and exhibits various functions in the field of immunotherapy, including a contrast effect, improvement of half-life, improvement of drug delivery, lymphocyte concentrating effect, cancer immunotherapy, and ultrasound-induced immunotherapy.

Provided herein are compositions that are useful in healing wounds and / or treating inflammatory skin conditions comprising (a) a copper-containing peptide; (b) an antioxidant; and (c) a cyclic oligosaccharide-based polymer, where the copper-containing peptide and the antioxidant form a complex. Further provided herein are methods for healing wounds and / or treating inflammatory skin conditions using the compositions of the disclosure.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The invention relates to the technical field of pharmaceutical preparations, and discloses a composition with liver protection and antioxidation effects and a preparation method thereof.The composition is prepared from galactosamine modified astragalus membranaceus exosome, schisandra chinensis nanocrystals, liquorice-chitosan nanoparticles, lactobacillus acidophilus freeze-dried powder, beta-(1, 3) / (1, 3, 4-tetramethyl-1, 3-pentanediol monoisobutyrate) sourced from saccharomyces cerevisiae, and beta-(1, 3, 4-tetramethyl-1, 3-pentanediol monoisobutyrate). And (6) a dextran, grape exosome-curcumin hybrid vesicle freeze-dried powder, a curcumin-mitochondrial targeting peptide compound, a resveratrol-hydroxypropyl-beta-cyclodextrin inclusion compound and reduced panthenol. According to the liver-protecting and anti-oxidation composition disclosed by the invention, multi-dimensional intervention on liver protection is realized through an elaborately designed ternary system. The system is composed of a traditional Chinese medicine extract, a microorganism-plant exosome combination and a high-bioavailability antioxidant, all the parts have a synergistic effect, and the liver protection effect is remarkably improved.

The invention discloses a vitamin K2 composite micro-capsule system with stomach protection and intestine slow release functions and a preparation method and application thereof, the vitamin K2 composite micro-capsule system with stomach protection and intestine slow release functions comprises three layers, namely a core layer, a middle layer and an outer layer from inside to outside in sequence; the core layer is formed by coating vitamin K2 with a zein-lac hydrophobic core, the middle layer is a pea protein-pectin electrostatic compound, and the outer layer is sodium alginate and chitosan double-network gel. The micro-capsule system provided by the invention can be tightly combined in a gastric acid environment after being orally taken, the release of active ingredients in the stomach is reduced, and then the micro-capsule system stays at intestinal mucosa for a long time and slowly releases contents, so that the functions of the micro-capsule system are slowly presented outside, the half-life period is prolonged, and the action effect is improved. Vitamin K2 directly or indirectly participates in and regulates various physiological processes related to health, and plays an important role in the aspects of maintaining bone health, preventing cardiovascular diseases, protecting nerves, improving metabolism and the like.

The disclosure features macrocyclic compounds, and pharmaceutical compositions and protein complexes thereof, capable of inhibiting Ras proteins, and their uses in the treatment of cancers.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

The invention provides a specific T cell receptor targeting KRAS G12V mutant epitope peptide (such as an amino acid sequence as shown in SEQ ID NO: 2) and anti-tumor application of the specific T cell receptor. The specific T cell receptor is composed of two peptide chains alpha and beta and can be specifically combined with a VVGAVGVGK-HLA-A * 11: 01 compound. The invention also provides an antigen binding fragment of the specific T cell receptor, a nucleic acid sequence for coding the T cell receptor, a vector containing the nucleic acid sequence, an engineered cell for expressing the T cell receptor, a composition containing the T cell receptor and application thereof. In addition, the invention also provides a method for screening the KRAS G12V specific T cell receptor. The specific T cell receptor and the antigen binding fragment thereof provided by the invention can be used as an immune effect activator to stimulate the immune response of a body, so that the effect of resisting diseases such as tumors and the like is achieved.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

The disclosure features macrocyclic compounds, and pharmaceutical compositions and protein complexes thereof, capable of inhibiting Ras proteins, and their uses in the treatment of cancers.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., useful for targeting DMPK) covalently linked to an antibody (e.g., anti-TfR1 antibody).

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits activity of a disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The invention relates to an oral mucosa repairing medicine and a preparation method thereof, and belongs to the technical field of medicines, the medicine comprises barnacle peptide, salivary lactobacillus, resveratrol, a biphasic adhesion matrix, hydroxyapatite, vitamin B family and other components. The preparation method comprises the following steps: sequentially carrying out enzymolysis on barnacle gooseneck by pepsin and lumbrukinase to obtain barnacle peptide. The double-phase adhesion matrix comprises a water-phase matrix, an oil-phase matrix and lecithin; the water-phase matrix contains a chondroitin sulfate-chitosan quaternary ammonium salt compound, sodium alginate and other components; the oil-phase matrix comprises sesame oil, beeswax, a polylactic acid-glycolic acid copolymer and the like. According to the invention, barnacle peptide and salivary lactobacillus are prepared by adopting a special method and are matched with resveratrol for use, so that multi-target and multi-path promotion of oral mucosa repair can be realized; the chondroitin sulfate-chitosan quaternary ammonium salt compound is prepared through electrostatic and physical embedding, the chondroitin sulfate-chitosan quaternary ammonium salt compound is matched with other components to prepare the biphasic adhesion matrix, the biocompatibility is good, the adhesion is high, and the action time of a sustained-release drug is well prolonged.

Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., useful for targeting DMPK) covalently linked to an antibody (e.g., anti-TfR1 antibody).

The invention relates to a water-soluble carbon chain substance for adjusting transmembrane transport and fluidity of a cell membrane, and preparation and application thereof. The water-soluble carbon chain substance comprises a carbon chain originally having certain water solubility, and the structure of the water-soluble carbon chain substance comprises at least one or more hydrophobic carbon chain parts and one or more hydrophilic groups or residues; the hydrophobic carbon chain part is coupled with water-soluble molecules to form a high-hydrophilicity compound; the water-soluble carbon chain can be used for preventing and treating infections including viruses, bacteria and fungi; anti-aging; the occurrence of neurodegenerative diseases such as Alzheimer's disease is prevented and reduced; by reducing the non-specific phagocytosis of the nano-drug by cells, the effect of the nano-drug on a target focus is enhanced.

The invention discloses preparation and application of a cationic compound and a compound thereof, belongs to the technical field of medicines, and particularly relates to a cationic compound which can be added into an original lipid compound. The invention also discloses the lipid compound, which can be specifically delivered to the lung, has the characteristics of small toxicity and good targeting property, solves the problem of difficult targeting delivery of nucleic acid drugs, and promotes the development of nucleic acid drugs. The prepared lipid compound can effectively target the lung, lung targeting can be achieved without adding any cationic lipid with positive charges into the lipid compound, and the tail of the cationic lipid needs to be provided with one or two olefins. Compared with a compound formed by a traditional DOTAP lipid material, the cationic lipid developed by the invention has lower hemolytic toxicity and better lung targeting property.