8806results about "Sugar derivatives" patented technology

Provided are compounds of Formula I:nucleosides, nucleoside phosphates and prodrugs thereof, wherein R6 is CN, ethenyl, 2-haloethen-1-yl, or (C2-C8)-alkyn-1-yl. The compounds, compositions, and methods provided are useful for the treatment of Flaviviridae virus infections.

The invention relates to the technical field of oil exploitation, and discloses a compound containing an amide group as well as a preparation method and application thereof. The compound containing the amide group has a structure as shown in a formula (1). The polymer obtained by polymerizing the compound can improve the water-drive recovery efficiency of conventional oil reservoirs and common heavy oil reservoirs. # imgabs0 #

The present invention is directed to a storage-stable formulation of long-chain RNA. In particular, the invention concerns a dry powder composition comprising a long-chain RNA molecule. The present invention is furthermore directed to methods for preparing a dry powder composition comprising a long-chain RNA molecule by spray-drying. The invention further concerns the use of such a dry powder composition comprising a long-chain RNA molecule in the preparation of pharmaceutical compositions and vaccines, to a method of treating or preventing a disorder or a disease, to first and second medical uses of such a dry powder composition comprising a long-chain RNA molecule and to kits, particularly to kits of parts, comprising such a dry powder composition comprising a long-chain RNA molecule.

The invention relates to the field of preparation of novel compounds, and discloses a glycoside group-containing compound as well as a preparation method and application thereof. The glycoside group-containing compound has a structure as shown in a formula (1). The polymer obtained by polymerizing the compound can improve the water-drive recovery efficiency of conventional oil reservoirs and common heavy oil reservoirs. # imgabs0 #

Provided is an antibody-drug conjugate for use in the treatment of cancer in which one or more KRAS mutants, particularly a KRAS G12V mutant, a KRAS G12D mutant, and a KRAS G12C mutant, are expressed. Also provided are a drug and a drug-linker conjugate for use in the antibody-drug conjugate.　The present inventors have produced an antibody-drug conjugate with which a heterocyclic compound represented by formula (II) and having an activity of inducing the decomposition of KRAS mutant proteins can be delivered to cancer in which EGFRs are expressed, the production being achieved by linking the compound to an anti-EGFR antibody. The present inventors have also discovered a drug-linker conjugate for use in the antibody-drug conjugate or a salt thereof. In cancer in which EGFRs are expressed, the antibody-drug conjugate induces the decomposition of one or more KRAS mutants, particularly a KRAS G12V mutant protein, a KRAS G12D mutant protein, and a KRAS G12C mutant protein, thereby inhibiting the KRAS mutants and exhibiting an anti-tumor effect.

This invention provides a halogenated compound for 5' end capping of nucleic acids and its applications, the compound being shown in formula (I). This invention also provides the uses and effects of the above-described compound on nucleic acid transcription and expression.

Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, oligonucleotides comprise various chemical modifications. In some embodiments, the present disclosure provides technologies useful for manufacturing oligonucleotides.

Among other things, the present disclosure provides oligonucleotides, compositions and methods thereof that can bring about specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders, diseases and syndromes that can benefit from adenosine modification.

The present disclosure features useful oligonucleotide compositions and methods related thereto. The present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g. modifications of sugar, base and / or internucleotide linkages) or patterns thereof, can have a significant impact on oligonucleotide properties and activities. The present disclosure also provides methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I):tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

The invention discloses a method for preparing sophorolipid by using candida mycoderma bacteria. The method comprises the following steps: inoculating a seed solution of candida mycoderma bacteria into a fermentation culture medium for fermentation, supplementing materials and the like. According to the method, a lactone-type-based product can be obtained, a high-purity product can be further obtained through a two-stage extraction process, alkali liquor is added according to downstream requirements subsequently for blending, and liquid products with different acid type proportions are obtained and used in the field of home care; in addition, the bacterium-containing wastewater obtained through separation can be subjected to spray drying to obtain a powdery solid, and the powdery solid can be used as a low-end product in the agrochemical direction.

The present disclosure includes ionizable lipids suitable for lipid nanoparticle compositions and pharmaceutical formulations thereof. The lipids have general formula (I).

Compounds useful as biologically active compounds are disclosed. The compounds have the following structure (I):or a stereoisomer, tautomer or salt thereof, wherein R1, R2, R3, R4, R5, L, L1, L2, L3, L4, M, q, w and n are as defined herein. Methods associated with preparation and use of such compounds is also provided.

The present disclosure provides, in part, double stranded compounds, including oligonucleotides such as siRNA, that comprise one or more independent occurrences of a modified nucleotide components of Formula (I), Formula (II), Formula (III), or Formula (IV) as described herein, as well as their methods of use in inhibiting gene expression and the treatment of various disorders.

The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.

Compounds having the structure of Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof; wherein R1, R2, R3, R4, R5, L1, L2, a and z are as defined herein, are disclosed. Use of these 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.

The present disclosure, among other things, provides technologies for synthesis, including reagents and methods for stereoselective synthesis. In some embodiments, the present disclosure provides compounds useful as chiral auxiliaries. In some embodiments, the present disclosure provides reagents and methods for oligonucleotide synthesis. In some embodiments, the present disclosure provides reagents and methods for chirally controlled preparation of oligonucleotides. In some embodiments, technologies of the present disclosure are particularly useful for constructing challenging internucleotidic linkages, providing high yields and stereoselectivity.

The invention discloses a method for extracting and purifying flavone in litsea coreana through an ultrasonic-assisted eutectic solvent, and belongs to the technical field of green processing of natural products. The invention provides a method for efficiently preparing litsea coreana flavone by integrating computer prediction, green solvent extraction and resin purification technologies. The method comprises the following steps: firstly, calculating and screening a deep eutectic solvent system with the highest affinity with the litsea coreana flavone on the basis of a COSMO-RS theoretical model, then coupling the optimized DES with an ultrasonic-assisted extraction (UAE) technology, and through the synergistic effect of the DES and the UAE technology, remarkably improving the extraction rate of the litsea coreana flavone and furthest keeping the biological activity of a target component; then, a macroporous resin dynamic adsorption-desorption purification process is linked, so that high-selectivity enrichment and impurity removal of the flavonoid compounds in the crude extract are realized, a high-purity product is obtained, and a complete process route from intelligent screening, efficient extraction to precise purification is finally formed.

The present disclosure provides multivalent molecules (e.g., compositions of Formula (I), an ionized form thereof, an isomer thereof, or a salt thereof. The present disclosure further provides methods for the use of the compositions, e.g. in the sequencing of poly-nucleic acid molecules.

The invention provides a benzofuranone compound as well as a pharmaceutical composition and application thereof, and relates to the technical field of medicines. The benzofuranone compound is a compound as shown in a formula I, a stereoisomer thereof, a tautomer thereof, a crystalline hydrate thereof, a solvate thereof, a prodrug thereof or a pharmaceutically acceptable salt thereof. The benzofuranone compound and the pharmaceutical composition and the pharmaceutical preparation prepared from the benzofuranone compound can prevent or treat organ injury diseases, and have good treatment effects in kidney injury, liver injury, lung injury, brain injury and heart injury.

Described herein are programmable DNA proteolytic target chimera complexes that can be used both for the direct treatment of cancer by inhibiting biochemical pathways that are overexpressed in cancer cells, and for the indirect treatment of cancer by recruiting the E3 ligase complex to engage with a protein of interest or a mutant thereof and initiating proteolysis. Also described herein are methods of using the complexes in the treatment of cancer, as well as compositions comprising the complexes.

The invention provides a double-target degradation molecule based on a functionalized nucleic acid connexon and application thereof, and relates to the technical field of biological medicine, the double-target degradation molecule comprises an E3 ubiquitin ligase ligand at one end, a first target protein ligand at the other end, and the functionalized nucleic acid connexon located between the E3 ubiquitin ligase ligand and the first target protein ligand; the functionalized nucleic acid linker is a nucleotide sequence capable of specifically recognizing and combining a second target protein or a coding gene thereof, so that the protein level degradation of the first target protein and the nucleic acid level or expression level inhibition of the second target protein / gene are realized in the same molecule. Functionalized nucleic acid and a PROTAC strategy are organically combined, single-molecule double-target collaborative intervention is achieved, targeting efficiency and treatment potential are improved, higher flexibility and expandability are provided in synthesis and design, and a new molecular platform and technical route are provided for multi-target accurate treatment.

The invention relates to a ligand-effector moiety provided with at least one saponin and antibody-effector moiety provided with at least one saponin. An aspect of the invention is a composition comprising the ligand-effector moiety provided with at least one saponin or the antibody-effector moiety provided with at least one saponin of the invention. The invention also relates to an antibody-drug conjugate comprising covalently linked saponin and to an antibody-oligonucleotide conjugate comprising covalently linked saponin. An aspect of the invention relates to a pharmaceutical composition comprising the ligand-effector moiety provided with at least one saponin or the antibody-effector moiety provided with at least one saponin of the invention, and optionally further comprising a pharmaceutically acceptable excipient. The invention also relates to the ligand-effector moiety provided with at least one saponin or the antibody-effector moiety provided with at least one saponin, for use as a medicament. The invention also relates to the ligand-effector moiety provided with at least one saponin or the antibody-effector moiety provided with at least one saponin of the invention for use in the treatment or prophylaxis of a cancer.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, Pemphigus, e.g., Pemphigus vulgaris (PV) and Pemphigus foliaceus (PF), and C3 glomerulopathy.

The invention belongs to the field of preparation of medical intermediates, and particularly relates to a preparation method of an On-DNA sulfinic acid structure compound. The method for preparing the On-DNA sulfinic acid structure compound comprises a step of oxidizing On-DNA thiol (phenol) into the On-DNA sulfinic acid structure compound under alkaline and heating or illumination conditions, a solvent used in the reaction step is a mixed solution of water, dimethyl sulfoxide, N, N-dimethylformamide, acetonitrile, ethanol or tetrahydrofuran, the reaction temperature is not more than 80 DEG C, and the reaction time is not more than 2 hours. And the illumination wavelength ranges from 365 nanometers to 535 nanometers. The On-DNA sulfinic acid structure compound obtained in the invention can be directly used for constructing a structure compound with a sulfonyl group, the reaction condition is mild and convenient, a new method and a new idea are provided for construction of a DNA coding compound library, and the On-DNA sulfinic acid structure compound has a very good application prospect in lead drug development.

The invention discloses modified polystyrene resin as well as a preparation method and application thereof, and belongs to the technical field of medicine purification. The modified polystyrene resin contains phenolic hydroxyl groups and sulfonic acid groups at the same time, electrostatic interaction and hydrogen-bond interaction can be generated on pyridine, the two actions are complementary, and pyridine molecules in different protonation states can be captured; moreover, a benzene ring of the modified polystyrene resin can be combined with an aromatic ring of pyridine through pi-pi stacking, and the introduction of a sulfonic acid group and a phenolic hydroxyl group further polarizes an electron cloud of the benzene ring of polystyrene, so that the conjugation effect can be enhanced, and the bonding strength of the benzene ring and the aromatic ring of pyridine is improved. The modified polystyrene resin prepared by the method has nanoscale pores and ultrahigh specific surface area, can form a'pore capture-group immobilization 'synergistic path when pyridine is adsorbed, and can effectively remove residual pyridine in a reaction system.

Disclosed herein are activatable inactive sonosensitizer compounds, referred to as locked sonosensitizer compounds, designed with a detachable stimuli-responsive tether acting as a trigger or safety, as these compounds restore their inherent sonosensitization activity upon the detachment of the tether. The invention further encompasses pharmaceutical compositions comprising the same, offering a strategic approach to sonodynamic therapy, whereas the compounds and compositions are formulated to be administered and applied under favorable conditions, enhancing their efficacy in the context of therapeutic applications, since the therapeutic methods presented herein are more versatile and efficient compared to presently known SDT methods due to the controlled activation of the sonosensitizer.