13568results 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.

Provided are a macrocyclic compound and an antibody-drug conjugate thereof. Specifically, the macrocyclic compound is an inhibitor targeting RAS proteins. Further provided are a method for preparing the above-mentioned macrocyclic compound and antibody-drug conjugate thereof, and the pharmaceutical use thereof.

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.

The present invention provides compounds, compositions thereof, and methods of using the same to treat IRAK4-mediated disorder, disease, or condition in a patient.

The specification relates to conjugates comprising a linker of Formula (IMA): and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the conjugates.

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, the present disclosure provides structural elements including various chemical modifications of sugars, nucleobases and / or internucleotidic linkages that are useful for adenosine editing. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders or diseases that can benefit from editing of an adenosine in a nucleic acid.

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.

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, oligonucleotides comprise various chemical modifications of sugars, nucleobases, and / or intemucleotidic linkages and patterns thereof and are useful for exon skipping. In some embodiments, the present disclosure provides technologies useful for skipping exon 51 of dystrophin (DMD) transcripts. In some embodiments, the present disclosure provides technologies usefill for modulating DMD transcript splicing. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders or diseases including Duchenne muscular dystrophy.

The invention provides a preparation method and application of salidroside. The preparation method comprises the following steps: culturing an engineering strain for synthesizing the salidroside by using a culture medium containing a mixed carbon source to obtain the salidroside, the mixed carbon source comprises glucose and glycerol. The problem that a salidroside preparation method in the prior art is not suitable for industrial large-scale production can be solved, and the method is suitable for the technical field of biosynthesis.

The present invention generally relates to systems and methods for imaging or determining nucleic acids, for instance, within cells. In some embodiments, the transcriptome of a cell may be determined. Certain embodiments are directed to determining nucleic acids, such as mRNA, within cells at relatively high resolutions. In some embodiments, a plurality of nucleic acid probes may be applied to a sample, and their binding within the sample determined, e.g., using fluorescence, to determine locations of the nucleic acid probes within the sample. In some embodiments, codewords may be based on the binding of the plurality of nucleic acid probes, and in some cases, the codewords may define an error-correcting code to reduce or prevent misidentification of the nucleic acids. In certain cases, a relatively large number of different targets may be identified using a relatively small number of labels, e.g., by using various combinatorial approaches.

Described herein are novel trinucleotide cap analogs and methods of making and using the same. Also described herein is an RNA molecule comprising a 5'-cap, wherein the 5'-cap includes a trinucleotide cap analog as described herein. Methods of inducing a therapeutic effect in a subject are also described herein, the methods including a step of administering to the subject an RNA molecule including the trinucleotide cap analog.

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, L1a, L1b, L2, L3, L4, L5, L6, L7, M1, M2, q, w, m and n are as defined herein. Compounds of structure (I) find utility in a number of applications, including use as therapeutic agents for various treatment methods.

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, the present disclosure provides technologies useful for exon skipping in cell populations, e.g., myocyte stem cells. In some embodiments, the present disclosure provides technologies useful for restoring functioning of myocyte stem cells. In some embodiments, the present disclosure provides technologies useful for regeneration of myocyte stem cells. In some embodiments, the present disclosure provides technologies useful for differentiation of myocyte stem cells. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders, or diseases including Duchenne muscular dystrophy.

The invention discloses sesquiterpenoids as well as a preparation method and application thereof. The structure of the sesquiterpenoids is shown in the specification. The sesquiterpenoids of # imgabs0 can be used for preparing drugs for preventing and / or treating cancers, and the cancers comprise at least one of breast cancer, liver cancer, lung cancer, colorectal cancer, leukemia, pancreatic cancer, glioma, osteosarcoma, cervical cancer, kidney cancer and skin cancer. According to the invention, various natural product extracts are subjected to activity screening, the sesquiterpenoids derived from the rattan chrysanthemum have excellent inhibitory activity on various different types of human cancer cells, and an optional scheme is provided for broad-spectrum anti-cancer drugs. The sesquiterpenoids applied in the invention are derived from rattan chrysanthemum, the source is wide, and the production cost is low.

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.

The invention belongs to the technical field of biology, and particularly relates to an extraction method of polydeoxyribonucleotide, which comprises the following steps: carrying out homogenate treatment on pretreated in-vitro testis tissue, filtering the obtained homogenate, adding a pancreas extract into the filtrate, and stirring to obtain enzymatic hydrolysate; and precipitating protein in the enzymatic hydrolysate, removing the protein, adding sodium acetate for treatment, adding absolute ethyl alcohol for precipitation, centrifuging, washing the obtained precipitate, and drying to obtain the product. Common trypsin and lipase in fish pancreas are beneficial to release of DNA, the fish pancreas extract is applied to extraction of the polydeoxyribonucleotide, the extraction time can be remarkably shortened, the yield can be increased, meanwhile, nuclease in the fish pancreas extract can effectively degrade DNA, and small-fragment polydeoxyribonucleotide is obtained.

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.

A nucleoside phosphoramidite compound, which is a 2'-phosphoramidite group and 4'-modified TNA structure compound. The nucleoside phosphoramidite compound can be incorporated into the end and / or middle of an oligonucleotide, so that the modified oligonucleotide is hybridized with a portion of a target mRNA, resulting in the loss or down-regulation of the normal function of the target mRNA.

Provided herein are lipid compounds, e.g., a compound of Formula (I) comprising an amine (heterocyclic) "head group" and a "hydrocarbon tail" moiety, and pharmaceutical compositions thereof. Also provided are lipid nanoparticles, each comprising a lipid compound of Formula (I), as a promising delivery vehicle for a variety of therapeutic agents, including small molecule drugs, proteins, and nucleic acids.

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.

Disclosed in the present invention is a GalNAc delivery molecule as shown in the following formula (I), or a stereoisomer, enantiomer, or pharmaceutically acceptable salt thereof, wherein the definitions of the groups are as described in the description.

The specification relates to conjugates comprising a linker of Formula (IA): and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the conjugates.

The invention provides an adenosine dihydroxysuccinate eutectic crystal and a preparation method and application thereof.In the adenosine dihydroxysuccinate eutectic crystal, the molar ratio of a dihydroxysuccinic acid molecule to an adenosine molecule is 1: (1-4), on the premise that the structure of adenosine is not changed, dihydroxysuccinic acid and adenosine are combined, the water solubility of adenosine is improved, and therefore the adenosine dihydroxysuccinate eutectic crystal is obtained. According to the preparation method, the adenosine which is difficult to dissolve in water is prepared into the water-soluble adenosine dihydroxysuccinate eutectic crystal, so that the water solubility of the adenosine dihydroxysuccinate eutectic crystal can reach 4%, the effects of the adenosine dihydroxysuccinate and the adenosine monomer are not damaged, and the adenosine dihydroxysuccinate and the adenosine monomer have a better synergistic effect; compared with a monomer and a simple mixture, the composition has better effects in the aspects of antibiosis, antioxidation, anti-aging, hair care, hair loss prevention and the like, and has a good application prospect in skin and hair care products.