11 results about "Alternative complement pathway" patented technology

Provided is a CFB-targeting double-stranded RNA molecule, specifically an siRNA. The provided siRNA can effectively inhibit the CFB expression, and can be used for preventing and treating diseases caused by up-regulated CFB expression or diseases associated with abnormal activation of an alternative complement pathway.

Described herein are isolated monovalent antibodies or antibody fragments thereof that bind human properdin. Such antibodies are useful in methods of treatment for diseases mediated by alternative complement pathway dysregulation.

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 the technical field of medicines, and particularly discloses application of ipropam in preparation of a medicine for treating immune thrombocytopenic purpura. Aiming at the clinical problem that the condition of a patient with refractory recurrent immune thrombotic thrombocytopenic purpura (iTTP) still progresses after the treatment of plasma exchange combined with hormone, repatumab and immune globulin is invalid, the complement abnormal activation marker of the patient is detected to be increased, which indicates that the complement bypass way participates in disease recurrence refractory; ipropam is combined with plasma exchange, hormone and rituximab for treatment. Results show that the hemoglobin and platelet count is significantly increased after treatment for 5 days, mental symptoms are improved in 9 days, creatinine returns to normal in 11 days, ADAMTS13 activity is less than 0.2 IU / dL in 14 days, an inhibitor turns negative, and ADAMTS13 activity, hemoglobin and platelet return to normal in the fifth week. According to the application, it is proved for the first time that ipropam can effectively reverse the pathological process of refractory iTTP by inhibiting complement bypass pathway activation, and a new treatment strategy is provided for the disease.

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 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.

This application discloses a method for determining the functional activity of the alternative complement pathway (AP50) in animal serum and its application. The method includes: preparing a reaction buffer containing magnesium and calcium chelating agents; washing red blood cells with the reaction buffer and adjusting the red blood cell concentration to a preset range by measuring the absorbance value after complete lysis; serially diluting the animal serum to be tested with the reaction buffer, mixing it with a standardized red blood cell suspension, and incubating to induce a hemolytic reaction; after terminating the reaction, measuring the absorbance value of hemoglobin in the supernatant, calculating the hemolysis rate at each dilution, determining the serum dilution factor corresponding to 50% hemolysis, and using its reciprocal as the AP50 value. This application selectively activates the alternative pathway using a disclosed buffer formulation and eliminates batch-to-batch variability in red blood cells through absorbance standardization. It has advantages such as self-controllability, good reproducibility, low cost, and wide species adaptability, and can be widely used in immunological research and drug safety evaluation.

In one aspect, the invention provides methods of inhibiting the effects of MASP-2-dependent complement activation in a living subject. The methods comprise the step of administering, to a subject in need thereof, an amount of a MASP-2 inhibitory agent effective to inhibit MASP-2-dependent complement activation. In some embodiments, the MASP-2 inhibitory agent inhibits cellular injury associated with MASP-2-mediated alternative complement pathway activation, while leaving the classical (C1q-dependent) pathway component of the immune system intact. In another aspect, the invention provides compositions for inhibiting the effects of lectin-dependent complement activation, comprising a therapeutically effective amount of a MASP-2 inhibitory agent and a pharmaceutically acceptable carrier.

Described herein are isolated monovalent antibodies or antibody fragments thereof that bind human properdin. Such antibodies are useful in methods of treatment for diseases mediated by alternative complement pathway dysregulation.

The present invention discloses a piperidine-substituted benzoic acid compound, a pharmaceutical composition thereof, and its use. The piperidine-substituted benzoic acid compound of the present invention is a compound represented by Formula I, a stereoisomer thereof, a tautomer thereof, an isotope-labeled compound thereof, or a pharmaceutically acceptable salt of any of the foregoing. The compound has good inhibitory activity against complement factor B, good affinity for complement factor B, hemolytic activity in the alternative complement pathway, and inhibitory activity in the alternative complement pathway. TIFF2025541667000344.tif11998s

The present invention relates to a crystal form of a multi-membered spiro compound, and a preparation method therefor and a medical use thereof, and in particular to a crystal form of a compound represented by general formula (I), a preparation method, and a pharmaceutical composition containing a therapeutically effective amount of the crystal form and a pharmaceutical use thereof for treating diseases or conditions related to alternative complement pathway activation.