US2022098272A1PendingUtilityA1

Pharmaceutical composition or food composition, and method for assessing effect of active ingredient in vivo

Assignee: KARYDO THERAPEUTIX INCPriority: Mar 29, 2016Filed: Sep 17, 2021Published: Mar 31, 2022
Est. expiryMar 29, 2036(~9.7 yrs left)· nominal 20-yr term from priority
Inventors:Narutoku Sato
G01N 2800/347G01N 2333/75C12Q 1/6883G01N 33/6893G01N 2333/47C12N 2310/20A01K 2207/25C07K 14/70503A23L 33/00G01N 33/6812A61K 38/00A61P 13/12C12N 2320/30A01K 2217/075C12N 15/1138A01K 67/0276A61K 48/00C12N 15/11C12N 2800/80A61K 31/713C12N 9/22A01K 2267/035A01K 2227/105C07K 2319/30A61K 31/7105C12Q 2565/60
60
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Claims

Abstract

An object of the present invention is to provide a pharmaceutical composition or food or drink composition comprising an active ingredient that suppresses functional expression of Oscar protein. Another object of the present invention is to provide a pharmaceutical composition or food composition for preventing or treating kidney disease. A further object of the present invention is to provide a pharmaceutical composition or food or drink composition that suppresses functional expression of Oscar in a living organism in order to suppress functional expression of FGF23. A still further object of the present invention is to provide a method for evaluating an effect, in the body, of an active ingredient that suppresses functional expression of Oscar protein. The above objects are achieved by at least one member selected from the group consisting of antagonists of the Oscar protein; genome editing systems that target Oscar gene; at least one RNA molecule selected from the group consisting of siRNA, shRNA, and miRNA that target Oscar mRNA, or vectors capable of expressing the RNA molecule; and antibodies that specifically bind to the Oscar protein and suppress function of the Oscar.

Claims

exact text as granted — not AI-modified
1 .- 34 . (canceled) 
     
     
         35 . A device for evaluating an effect, in the body, of an active ingredient that suppresses functional expression of Oscar protein, the device comprising:
 a memory for storing a program;   an interface; and   a processor, during execution of the program, configured to:   obtain a measurement value relating to at least one protein selected from the group consisting of proline-rich proteins (PRPs) and fibrinogens contained in a specimen collected from a subject to which the active ingredient has been administered (a treated specimen), and/or a measurement value of mRNA of the protein contained in a specimen collected from the subject; and   evaluate the effect of the active ingredient based on the measurement value(s) obtained.   
     
     
         36 . The device according to  claim 35 ,
 the processor, during execution of the program, further configured to:   obtain a measurement value relating to the at least one protein selected from the group consisting of proline-rich proteins (PRPs) and fibrinogens, and/or a measurement value of mRNA of the protein in a specimen collected from a subject, test tissue, or test cell that is not treated with the active ingredient (an untreated specimen); and   compare the measurement value(s) of the treated specimen with the measurement value(s) of the untreated specimen,   wherein the processor evaluates the effect of the active ingredient in the body based on the comparison result obtained.   
     
     
         37 . The device according to  claim 36 , wherein, in the evaluation, the processor determines that the active ingredient is effective in the body when the measurement value(s) of the treated specimen are lower than the measurement value(s) of the untreated specimen. 
     
     
         38 . The device according to  claim 35 , wherein the active ingredient is at least one member selected from the group consisting of antagonists of the Oscar protein;
 genome editing systems that target Oscar gene;   at least one RNA molecule selected from the group consisting of siRNA, shRNA, and miRNA that target Oscar mRNA, or vectors capable of expressing the RNA molecule; and   antibodies that specifically bind to the Oscar protein and suppress function of the Oscar.   
     
     
         39 . A method for supporting the evaluation of an effect, in the body, of an active ingredient that suppresses functional expression of Oscar protein, the method comprising the following steps:
 a first obtaining step of obtaining a measurement value relating to at least one protein selected from the group consisting of proline-rich proteins (PRPs) and fibrinogens contained in a specimen collected from a subject to which the active ingredient has been administered (a treated specimen), and/or a measurement value of mRNA of the protein contained in a specimen collected from the subject; and   a step of evaluating the effect of the active ingredient based on the measurement value(s) obtained by the obtaining step.   
     
     
         40 . The method according to  claim 39 , further comprising the following steps:
 a second obtaining step of obtaining a measurement value relating to the at least one protein selected from the group consisting of proline-rich proteins (PRPs) and fibrinogens, and/or a measurement value of mRNA of the protein in a specimen collected from a subject, test tissue, or test cell that is not treated with the active ingredient (an untreated specimen); and   a step of comparing the measurement value(s) of the treated specimen with the measurement value(s) of the untreated specimen,   wherein in the evaluation step, the effect of the active ingredient in the body is evaluated based on the comparison result obtained by the measurement value comparison step.   
     
     
         41 . The method according to  claim 40 , wherein in the evaluation step, it is determined that the active ingredient is effective in the body when the measurement value(s) of the treated specimen are lower than the measurement value(s) of the untreated specimen. 
     
     
         42 . The method according to  claim 39 , wherein the active ingredient is at least one member selected from the group consisting of antagonists of the Oscar protein;
 genome editing systems that target Oscar gene;   at least one RNA molecule selected from the group consisting of siRNA, shRNA, and miRNA that target Oscar mRNA, or vectors capable of expressing the RNA molecule; and   antibodies that specifically bind to the Oscar protein and suppress function of the Oscar.

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