5242 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 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 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.

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 (e.g., wherein each complex is of the exemplary formula shown below) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody). In some embodiments, the complexes are formulated with histidine (e.g., L-histidine) and sucrose at a specified pH (e.g., about 5.0 to 7.0). Also provided are uses of these formulations for treating a subject having a mutated DMD allele associated with Duchenne Muscular Dystrophy.

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 oligonucleotides designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease (e.g., FSHD). Wherein a complex comprises an anti-transferrin receptor 1 (TfR1) antibody covalently linked to an oligonucleotide configured for reducing expression or activity of DUX4.

Aspects of the disclosure relate to methods of promoting expression or activity of a dystrophin protein and / or methods of treating DMD in a subject. In some embodiments, the methods comprise administering to the subject a composition comprising complexes (e.g., muscle targeting complexes) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) 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 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 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 provides a pyridopyrimidine derivative as a small GTP enzyme KRAS mutation inhibitor, and particularly provides a pyridopyrimidine derivative with a structural formula as shown in a general formula (A) and pharmaceutically acceptable salts thereof, and the pyridopyrimidine derivative has inhibitory activity on mutants such as KRAS G12D and the like. The invention also provides a preparation method of the derivative, a pharmaceutical composition and a salifying compound of the derivative, and medical application of the derivative as a small GTP enzyme KRAS mutation inhibitor in treatment of different types of tumors.

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 invention discloses a polypeptide and application thereof in preparation of a medicine for preventing and / or treating cancers. The amino acid sequence of the polypeptide is shown as SEQ ID No. 1 or SEQ ID No. 2. The cancer is at least one of breast cancer, colorectal cancer, leukemia, esophageal cancer, pancreatic cancer, brain glioma, kidney cancer, skin cancer, osteosarcoma, ovarian cancer and prostate cancer. The invention designs and synthesizes the polypeptide of a targeted staphylococcus tumor protein compound SND1-MTDH containing a nuclease structural domain 1 (SND1 and a heteromucin (MTDH), finds that the polypeptide has excellent inhibitory activity on various different types of human cancer cells for the first time, and provides an optional scheme for broad-spectrum anti-cancer drugs. The polypeptide applied in the invention is simple in synthesis process and low in production cost.

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.

The invention relates to self-healing double-layer hydrogel for promoting healing of diabetic wounds as well as a preparation method and application of the self-healing double-layer hydrogel, and belongs to the technical field of medical materials of surgical supplies containing macromolecular materials. The hydrogel comprises an inner layer and an outer layer, the inner layer is self-healing hydrogel in which microalgae cells are uniformly distributed, and the outer layer is self-healing hydrogel in which metal ion-humic acid coordination nanoparticle compounds are uniformly distributed, the self-healing hydrogel is formed by self-crosslinking of a macromolecular polymer containing an aldehyde group and a macromolecular polymer containing an amino group. The hydrogel provided by the invention has excellent biocompatibility, adhesion and self-healing performance, can meet the requirements of actual production and application, and has a relatively great application prospect as a dressing for chronic diabetes wounds; and the preparation process has the characteristics of simplicity in operation, low cost and mild reaction conditions, and is suitable for large-scale popularization and application.

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.

The invention provides combination therapies comprising JAK / STAT pathway inhibitor compounds and mitochondrial complex I modulator (MCIM) compounds, which find utility in the medical field of inflammatory and progressive disorders such as RA, IBD, idiopathic pulmonary fibrosis, interstitial lung disease, and MS.

The invention belongs to the technical field of immunodetection, and discloses a T3 sandwich method rabbit monoclonal antibody mAb3 and application thereof. The T3 sandwich method monoclonal antibody mAb3 provided by the invention comprises a light chain variable region and a heavy chain variable region, the light chain variable region comprises LCDR1-3, the LCDR1 comprises an amino acid sequence NIYNS, the LCDR2 comprises an amino acid sequence YAS, and the LCDR3 comprises an amino acid sequence QSHYGFSSSYGAA; the heavy chain variable region comprises HCDR1-3, the HCDR1 comprises an amino acid sequence GFDVSYN, the HCDR2 comprises an amino acid sequence IDTGANA, and the HCDR3 comprises an amino acid sequence ARVFYDNSFDP. The monoclonal antibody can be combined with a T3 compound in a high-specificity mode, is suitable for double-antibody sandwich immunodetection and is applied to an immunodetection kit prepared through a double-antibody sandwich ELISA or a chemiluminescence method, a T3 standard antigen is detected through a double-antibody sandwich chemiluminescence platform, the detection sensitivity is lower than 0.3 ng / ml, clinical samples are detected through a magnetochemiluminescence method, and the detection sensitivity is lower than 0.3 ng / ml. In the sample range of 1-80 pmol / L, the correlation with clinical comparison is good.

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.