53 results about "Complement factor I" 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.

Disclosed in the present invention are a preparation method for a complement factor D inhibitor, an intermediate thereof, and a preparation method for an intermediate. The present invention provides a preparation method for a new complement factor D inhibitor, a new intermediate, and a preparation method for an intermediate. The selectivity of a reaction is improved by means of the new intermediate, the material conversion rate of each step of a route is high, a reaction type is common, the length of the route is moderate, and the obtained products can all be purified by means of a conventional method to obtain a target product, thereby reducing the production costs, and facilitating industrial production.

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.

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.

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.

The present invention relates to the prediction and early diagnosis of acute kidney injury (AKI). The methods provide biomarkers that correlate with a patient's risk of developing AKI, or the presence of early AKI. The invention also provides devices and kits for predicting the risk of development of AKI and for diagnosing AKI.SOLUTION: A method for predicting the risk of occurrence of acute kidney injury (AKI) or for early diagnosis of AKI in a subject, the method comprising: a. determining in a sample obtained from the subject the amount of at least one biomarker selected from the group consisting of complement factor D and insulin-like growth factor binding protein 1 and combinations, isoforms, fragments and variants thereof.SELECTED DRAWING: None

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 invention relates to the field of medicines, in particular to application of a reagent for detecting a complement factor in preparation of a tinnitus diagnostic product, the complement factor is selected from a complement component, a complement regulatory protein or a complement related chemotactic factor, the complement component is selected from C3, C5 and / or C1q, and tinnitus is chronic tinnitus. The peripheral blood can be used as a sample, the chronic tinnitus can be accurately diagnosed, theoretical and experimental bases are provided for clinical diagnosis of the chronic tinnitus, diagnosis and intervention can be carried out in the early stage of tinnitus, and further deterioration is prevented.

An isolated polynucleotide comprising a nucleotide sequence encoding a codon-optimised Complement Factor I (CFI).

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 present invention provides monoclonal antibodies that bind to complement factor 5 (C5) protein, and methods for using the same. In various embodiments of the invention, the antibodies are fully human antibodies that bind to the C5 protein. In some embodiments, the antibodies of the invention are useful for inhibiting or neutralizing C5 activity, thereby providing a means for treating or preventing a C5-related disease or disorder in humans. In some embodiments, the invention provides an anti-C5 antibody that has improved pharmacokinetic and pharmacodynamic properties, for example, a half-life of more than 10 days.

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.

Disclosed are compounds of formula I and II and pharmaceutically acceptable salts thereof. Also disclosed are methods of treating a neurodegenerative disorder, an inflammatory disease, an autoimmune disease, an ophthalmic disease or a metabolic disorder using the compounds disclosed herein.

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 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.

Disclosed are compounds of formula I and II and pharmaceutically acceptable salts thereof. Also disclosed are methods of treating a neurodegenerative disorder, an inflammatory disease, an autoimmune disease, an ophthalmic disease or a metabolic disorder using the compounds disclosed herein.

Methods of treating ocular diseases such as age-related macular degeneration may include the administration of peptides comprising complement factor H and factor H-like protein domains to reduce an amount of geographic atrophy in a subject in need thereof. These methods may be administered by intraocular, intervascular or subcutaneous injection.

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 dual-antigen binding molecules, and nucleic acid sequences encoding such molecules, that bind human complement factor D (FD) and human complement component 2 (C2). In particular embodiments, the FD binding region of the dual-antigen binding molecules comprises light and heavy chain variable regions, and the C2 binding region comprises a first single monomeric variable antibody domain (aka VHH), and optionally second single monomeric variable antibody domain. In certain embodiments, the dual-antigen binding molecules are used to treat complement-related diseases (e.g., dysregulated complement activation diseases).

The present application relates to an RNA inhibitor for inhibiting gene expression of a complement factor B (CFB) in a cell, comprising an antisense strand. The antisense strand and at least 15 consecutive nucleotides in an mRNA (SEQ ID NO: 8) encoding a CFB form a target sequence complementary region, the target sequence complementary region has 0, 1, 2, 3, 4 or 5 mismatches, and preferably, the complementary region has a length of 15-30 nucleotide pairs, and more preferably 17-23 nucleotide pairs.

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.

The present invention relates to complement factor B inhibitors of formula (I), pharmaceutical compositions thereof and uses thereof. [Formula 1] TIFF2026502231000352.tif47167

Provided herein are compounds of Formula (I) and pharmaceutical compositions thereof useful for treating diseases or disorders mediated by the complement factor B. (I)

Provided herein are substituted indole compounds. In certain embodiments, the compounds are inhibitors of the alternative pathway of the complement system, and in particular, inhibitors of complement factor B (CFB). Also provided are compositions comprising the compounds and methods of use thereof. The compounds provided are useful in the treatment, prevention or amelioration of a disease, condition or disorder through inhibition of the complement alternative pathway.