82 results about "Complement factor B" patented technology

Provided are a compound as shown in general formula (I), or a stereoisomer, deuterate, solvate, prodrug, metabolite, pharmaceutically acceptable salt or co-crystal thereof, an intermediate thereof, a preparation method therefor, and the use thereof in the preparation of a drug for treating a disease associated with the activity or expression quantity of complement factor B.

Provided is an siRNA for inhibiting the gene expression of complement factor B (CFB), or a pharmaceutical composition or conjugate containing the siRNA. The siRNA contains a sense strand and an antisense strand. Each nucleotide in the siRNA is an independent modified or unmodified nucleotide. The sense strand contains a segment of nucleotide sequence I, and the nucleotide sequence I and a nucleotide sequence show in SEQ ID NO: 1 have an equal length and differ by no more than 3 bases. The antisense strand contains a nucleotide sequence II, and the nucleotide sequence II and a nucleotide sequence shown in SEQ ID NO: 2 have an equal length and differ by no more than 3 bases. The siRNA or the pharmaceutical composition or conjugate containing the siRNA can effectively treat and / or prevent diseases related to the mRNA level of the gene expression of the CFB.

The present invention belongs to the field of biotechnology. Disclosed is a dosage composition of a complement factor B inhibitor. Specifically, the present invention provides a pharmaceutical composition comprising a compound represented by formula I, an isomer thereof, or a pharmaceutically acceptable salt thereof in a unit dosage form, wherein the mass of the compound or the pharmaceutically acceptable salt thereof is 5-1500 mg based on the free base.

The present invention belongs to the technical field of biology. Disclosed are a crystal form of a complement factor B inhibitor, a preparation method therefor and the use thereof. Specifically, the present invention provides a crystal form of a compound represented by formula I, a preparation method therefor and the use thereof. The crystal form prepared by the present invention has good stability, for example, crystal form stability and thermal stability.

The present invention relates to RNAi agents for inhibiting the expression of complement factor B, methods of preparation and uses thereof, which can inhibit the expression of complement factor B for the treatment of complement factor B related diseases and disorders.

Aspects of the disclosure provide compounds, compositions, and methods for modulating the expression or activity of Complement Factor B (CFB). In certain aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB mRNA in a cell or animal. In certain aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB protein in a cell or animal.

Provided herein are methods of ameliorating a disease or condition associated with a disorder of a complement bypass pathway by administering to a subject a complement factor B (CFB) specific inhibitor.

The invention discloses application of CFB polypeptide as a target spot in preparation of diabetic nephropathy diagnosis and treatment medicines, and belongs to the technical field of biological medicines. The invention provides application of CFB (circulating fluid bed)-sourced polypeptide in preparation of a medicine for treating diabetic nephropathy and application of the CFB-sourced polypeptide as a possible target in development of a novel diabetic nephropathy biomarker. According to the invention, a complement factor B-derived polypeptide which is significantly up-regulated in diabetic nephropathy is found through deep peptidomics analysis; furthermore, a functional experiment proves that the polypeptide shows remarkable anti-fibrosis activity in kidney cells for the first time, the active oxygen level can be reduced, and an HIF-1 alpha / glycolytic pathway can be regulated and controlled.

The present invention relates to the technical field of biology. Disclosed are a salt of a complement factor B inhibitor, a preparation method therefor and a use thereof. Specifically, the present invention provides a salt of a compound as shown in formula (A), a preparation method therefor and a use thereof.

Relates to the technical field of nucleic acid drugs, and particularly provides a double-stranded oligonucleotide for inhibiting complement factor B (CFB) gene expression, the double-stranded oligonucleotide comprises a sense strand and an antisense strand, the positive-sense strand comprises at least 15 continuous nucleotides in any sequence as shown in SEQ ID NO: 1-SEQ ID NO: 255 in a table 1 or a nucleotide sequence which is different from the at least 15 continuous nucleotides by not more than 3 nucleotides; and / or the antisense strand comprises at least 15 contiguous nucleotides in any sequence as shown in SEQ ID NO: 256-SEQ ID NO: 510 in the table 1 or a nucleotide sequence which is different from the at least 15 contiguous nucleotides by not more than 3 nucleotides. The double-stranded oligonucleotide can inhibit CFB gene expression in cells and achieve the effects of relieving, treating and / or preventing diseases or symptoms mediated by CFB gene expression abnormity.

This disclosure relates to RNAi agents capable of inhibiting complement factor B (CFB) gene expression. Pharmaceutical compositions containing CFB RNAi agents and methods of use thereof are also disclosed. The CFB RNAi agents disclosed herein may be conjugated to a targeted ligand containing an N-acetyl-galactosamine ligand to facilitate in vivo delivery to hepatocytes. RNAi agents can be used in methods of treating diseases, disorders, or conditions partially mediated by CFB gene expression, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), immune complex-mediated membrane proliferative glomerulonephritis (IC-MPGN), lupus nephritis (LN), anti-glomerular basement membrane antibody disease (anti-GBM), ischemia-reperfusion injury and T-cell-mediated rejection in kidney transplantation (TCMR), anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, age-related macular degeneration (AMD) including early and / or intermediate-stage AMD, geographic atrophy (GA), glaucoma, Doyne honeycomb retinal dystrophy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), pre-eclampsia, rheumatoid arthritis (RA), and / or other complement-mediated disorders.

Compositions and methods for reducing complement activation by introducing one or more alterations into a complement factor B (CFB) polynucleotide in a cell. In particular embodiments, the invention of the disclosure features a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying a CFB polynucleotide, where the modification is associated with reduced expression, and / or reduced activity of the CFB polypeptide encoded by the polynucleotide.

The present invention provides compositions and methods that can be used to reduce CFB gene expression and to treat CFB-related diseases and conditions. It also provides CFB dsRNA agents, CFB antisense polynucleotide agents, compositions containing CFB dsRNA agents, and compositions containing CFB antisense polynucleotide agents that can be used to reduce CFB expression in cells and subjects.

Compositions and methods useful for reducing CFB gene expression and for treating CFB-related diseases and disorders are provided. Provided are CFB dsRNA agents, CFB antisense polynucleotide agents, compositions comprising CFB dsRNA agents, and compositions comprising CFB antisense polynucleotide agents that can be used to reduce CFB expression in cells and subjects.

Aspects of the disclosure provide compounds, compositions, and methods for modulating the expression or activity of Complement Factor B (CFB). In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB mRNA in a cell or animal. In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB protein in a cell or animal.

The present disclosure relates to RNAi agents able to inhibit Complement Factor B (CFB) gene expression. Also disclosed are pharmaceutical compositions that include CFB RNAi agents and methods of use thereof. The CFB RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that comprise N-acetyl-galactosamine, to facilitate the in vivo delivery to hepatocyte cells. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by CFB gene expression, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), lupus nephritis (LN), Anti-Glomerular Basement Membrane disease (anti-GBM), ischemia reperfusion injury and T-cell mediated rejection (TCMR) in kidney transplantation, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, age-related macular degeneration (AMD), including early and / or intermediate AMD, geographic atrophy (GA), glaucoma, Doyne honeycomb retinal dystrophy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), pre-eclampsia, rheumatoid arthritis (RA), and / or other complement-mediated diseases.

A pharmaceutically acceptable salt type and crystal form of a compound of formula (I), and a preparation method of the compound and an application thereof are provided.

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

The invention discloses preparation, application and application of a benzo spiro indole compound, and particularly discloses a compound as shown in a formula (I), an optical isomer or pharmaceutically acceptable salt of the compound, and application of the compound to treatment and / or prevention of complement factor B activity and expression quantity related diseases. # imgabs0 #

Provided herein are methods of ameliorating a disease or disorder associated with dysregulation of the alternative complement pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

The invention relates to a siRNA double-stranded molecule targeting complement factor B (CFB) mRNA, a siRNA-GalNAc delivery carrier conjugate and application of the siRNA double-stranded molecule and the siRNA-GalNAc delivery carrier conjugate. And using such siRNA example compositions to inhibit expression of CFB mRNA and treat diseases associated with abnormal expression of complement components.

The invention relates to a polysubstituted indole compound as well as a preparation method and application thereof. Specifically, the invention discloses a compound as shown in a formula (I), or a pharmaceutically acceptable salt thereof, or a prodrug thereof, the compound is a complement factor B inhibitor, has a significant inhibitory effect on complement factor B, and is high in bioavailability and excellent in pharmacokinetics.

The invention discloses siRNA (small interfering Ribonucleic Acid) for inhibiting CFB (circulating fluid bed) gene expression and application of the siRNA. The siRNA and the nucleic acid conjugate provided by the invention can effectively prevent and / or treat complement factor B mediated related diseases.

The invention provides a small interfering RNA (Ribonucleic Acid) for inhibiting expression of a complement factor B (CFB) and application of the small interfering RNA to preparation of a medicine for treating diseases related to expression of the complement factor B.

The invention provides a complement factor B inhibitor, the structure of the complement factor B inhibitor is basically shown in a general formula (I), and the definition of each substituent group in the formula is shown in the specification. The compound provided by the invention has obvious complement factor B inhibition activity, and can be used for preventing and / or treating complement factor B inhibitor mediated diseases or disease states. # imgabs0 #

Disclosed in the present invention are a piperidine-substituted benzoic acid compound, and a pharmaceutical composition and the use thereof. The piperidine-substituted benzoic acid compound of the present invention is a compound as shown in formula (I) or a stereoisomer, a tautomer or an isotopic label thereof, or a pharmaceutically acceptable salt of any one of the foregoing. The compound has a good inhibitory activity on complement factor B, a good affinity activity on complement factor B, and a hemolytic activity and an inhibitory activity of an alternative complement pathway.

Provided is use of a complement factor B inhibitor in the preparation of a drug for treating or preventing IgA nephropathy or a disease or condition related thereto. Specifically, provided is use of a compound represented by formula I or a pharmaceutically acceptable salt thereof or a hydrate thereof in the preparation of a drug for treating or preventing IgA nephropathy or a disease or condition related thereto. The drug can effectively reduce creatinine and urea nitrogen levels in patients with IgA nephropathy, reduce renal inflammation and / or fibrosis in patients with IgA nephropathy, and provide a novel, effective and safe option for the treatment of IgA nephropathy.

Provided herein are methods for ameliorating a disease or disorder associated with dysregulation of the alternative complement pathway by administering a complement factor B (CFB)-specific inhibitor to a subject.

The present disclosure relates to RNAi agents able to inhibit Complement Factor B (CFB) gene expression. Also disclosed are pharmaceutical compositions that include CFB RNAi agents and methods of use thereof. The CFB RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that comprise N-acetyl-galactosamine, to facilitate the in vivo delivery to hepatocyte cells. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by CFB gene expression, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), lupus nephritis (LN), Anti-Glomerular Basement Membrane disease (anti-GBM), ischemia reperfusion injury and T-cell mediated rejection (TCMR) in kidney transplantation, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, age-related macular degeneration (AMD), including early and / or intermediate AMD, geographic atrophy (GA), glaucoma, Doyne honeycomb retinal dystrophy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), pre-eclampsia, rheumatoid arthritis (RA), and / or other complement-mediated diseases.