99 results about "Complement factor B" patented technology

The invention provides a complement factor B inhibitor compound, pharmaceutically acceptable salts thereof, a medicinal composition thereof and application of the complement factor B inhibitor compound in preparation of medicines related to complement factor B. The compound has a structural formula shown in a formula (I), and experimental results show that the compound has good complement factor B inhibitory activity, good in-vivo C3a level inhibition rate, bioavailability and safety. Particularly, the polypeptide has more remarkable binding capacity with target protein. # imgabs0 #

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.

The present invention 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 sense strand contains at least fifteen consecutive nucleotides in any one sequence shown as SEQ ID NO: 1-SEQ ID NO: 255 in Table 1 or a nucleotide sequence which differs from the at least fifteen consecutive nucleotides in respect of not more than three nucleotides; and / or, the antisense strand contains at least fifteen consecutive nucleotides in any one sequence shown as SEQ ID NO: 256-SEQ ID NO: 510 in Table 1 or a nucleotide sequence which differs from the at least fifteen consecutive nucleotides in respect of not more than three nucleotides. The double-stranded oligonucleotide can inhibit CFB gene expression in cells, thereby achieving the effects of relieving, treating and / or preventing diseases or disorders mediated by abnormal CFB gene expression.

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.

Compositions and methods useful to reduce expression of CFB gene and for treatment of CFB-associated diseases and conditions 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.

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.

Provided herein are compounds of formula (I) and pharmaceutical compositions thereof that are useful for treating diseases or disorders mediated by complement factor B. [Formula 1] TIFF2025511100000139.tif32170

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.

The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the complement factor B (CFB) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a CFB gene and to methods of treating or preventing a CFB-associated disease in a subject.

The invention relates to nucleic acid products that interfere with complement factor B (CFB) gene expression or inhibit its expression. The nucleic acids are preferably for use in the prophylaxis or treatment of complement associated diseases, disorders or syndromes, particularly C3 glomerulopathy (C3G), paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), lupus nephritis, IgA nephropathy (IgA N), myasthenia gravis (MG), and primary membranous nephropathy.

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 invention belongs to the technical field of chemical drugs, and relates to a compound as shown in a general formula (I), or a racemate thereof, or an isomer thereof, or a pharmaceutically acceptable salt thereof, as a complement factor B inhibitor, and a method for treating various specific diseases or symptoms by using the complement factor B inhibitor. # imgabs0 #

Described herein is an oligonucleotide (e.g., RNAi oligonucleotide) that comprises sense and antisense strands for targeting complement factor B (CFB) mRNA.RNAi oligonucleotide can be used to inhibit CFB expression, level and / or activity in cells.Also described is a method of using oligonucleotide (e.g., RNAi oligonucleotide) for prevention or treatment of disease, disorder or condition mediated by complement pathway activation or dysregulation.

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.

The invention relates to a nitrogen-bridge-containing heterocyclic compound, a preparation method thereof and application of the nitrogen-bridge-containing heterocyclic compound in medicine. Specifically, the invention relates to a nitrogen-containing bridge heterocyclic compound as shown in a general formula (I), a preparation method thereof, a pharmaceutical composition containing the compound, and application of the nitrogen-containing bridge heterocyclic compound as a therapeutic agent, especially application of the nitrogen-containing bridge heterocyclic compound as a complement factor B (Factor B) inhibitor and application of the nitrogen-containing bridge heterocyclic compound in preparation of drugs for treating and / or preventing Factor B-mediated diseases or disorders. # imgabs0 #

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 nitrogen-bridge-containing heterocyclic compound, a preparation method thereof and application of the nitrogen-bridge-containing heterocyclic compound in medicine. Specifically, the invention relates to a nitrogen-containing bridge heterocyclic compound as shown in a general formula (I), a preparation method thereof, a pharmaceutical composition containing the compound, and application of the nitrogen-containing bridge heterocyclic compound as a therapeutic agent, especially application of the nitrogen-containing bridge heterocyclic compound as a complement factor B (Factor B) inhibitor and application of the nitrogen-containing bridge heterocyclic compound in preparation of drugs for treating and / or preventing Factor B-mediated diseases or disorders. # imgabs0 #