US2022206008A1PendingUtilityA1

Screening methods for parp modulators

73
Assignee: RIBON THERAPEUTICS INCPriority: Apr 30, 2018Filed: Mar 17, 2022Published: Jun 30, 2022
Est. expiryApr 30, 2038(~11.8 yrs left)· nominal 20-yr term from priority
G01N 33/533G01N 33/582C07D 495/04G01N 33/573G01N 2333/91142C07D 491/22G01N 33/542C07F 5/027C07D 493/10
73
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Claims

Abstract

The present disclosure is related to methods of identifying Poly(ADP-ribose) polymerases (PARP) inhibitors, and the methods of using PARP probes.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method of identifying an inhibitor for Poly (ADP-ribose) polymerase (PARP), the method comprising:
 combining (i) a polypeptide comprising a PARP catalytic domain wherein the polypeptide is labeled with a donor fluorophore, (ii) a PARP probe, wherein the PARP probe is labeled with an acceptor fluorophore, and (iii) a test compound;   exposing the donor fluorophore to excitation light;   measuring a signal produced by the acceptor fluorophore; and   identifying the test compound as an inhibitor for PARP based on the signal produced by the acceptor fluorophore.   
     
     
         2 . The method of  claim 1 , wherein the PARP probe is a compound having the structure: 
       
         
           
           
               
               
           
         
         or a salt thereof. 
       
     
     
         3 . The method of  claim 1 , further comprising identifying the test compound as an inhibitor for PARP if the signal produced by the acceptor fluorophore is decreased as compared to a reference level. 
     
     
         4 . The method of  claim 3 , wherein the reference level is the signal produced by the acceptor fluorophore in the absence of the test compound. 
     
     
         5 . The method of  claim 1 , wherein the PARP is PARP1, PARP2, PARP3, PARP4, PARP5a, PARP5b, PARP6, TIPARP, PARP8, PARP9, PARP10, PARP11, PARP12, PARP13, PARP14, PARP15, or PARP16. 
     
     
         6 . The method of  claim 1 , wherein the PARP probe is biotinylated, and the acceptor fluorophore is attached to streptavidin. 
     
     
         7 . The method of  claim 1 , wherein the polypeptide comprises a polyhistidine tag. 
     
     
         8 . The method of  claim 1 , wherein the test compound is a small molecule, a nucleic acid, a polypeptide, or an antibody or antigen-binding fragment thereof. 
     
     
         9 . A method of identifying an inhibitor for Poly (ADP-ribose) polymerase (PARP), the method comprising:
 combining (i) a polypeptide comprising a PARP catalytic domain, wherein the polypeptide is labeled with an acceptor fluorophore; (ii) a PARP probe, wherein the PARP probe is labeled with a donor fluorophore, and (iii) a test compound;   exposing the donor fluorophore to excitation light;   measuring a signal produced by the acceptor fluorophore in the presence of a test compound; and   identifying the test compound as an inhibitor for PARP based on the signal produced by the acceptor fluorophore.   
     
     
         10 . The method of  claim 9 , wherein the PARP probe is a compound having the structure: 
       
         
           
           
               
               
           
         
         or a salt thereof. 
       
     
     
         11 . The method of  claim 9 , further comprising identifying the test compound as an inhibitor for PARP if the signal produced by the acceptor fluorophore is decreased as compared to a reference level. 
     
     
         12 . The method of  claim 11 , wherein the reference level is the signal produced by the acceptor fluorophore in the absence of the test compound. 
     
     
         13 . The method of  claim 9 , wherein the PARP is PARP1, PARP2, PARP3, PARP4 PARP5a, PARP5, PARP6, TIPARP, PARP8, PARP9, PARP10, PARP11, PARP12, PARP13, PARP14, PARP15, or PARP16. 
     
     
         14 . The method of  claim 9 , wherein the test compound is a small molecule, a nucleic acid, a polypeptide, or an antibody or antigen-binding fragment thereof. 
     
     
         15 . A method of identifying an inhibitor for Poly (ADP-ribose) polymerase (PARP), the method comprising:
 contacting a polypeptide comprising a PARP catalytic domain with a PARP probe in the presence of a test compound, wherein the PARP probe comprises a fluorophore;   exposing the probe to polarized excitation light, thereby generating fluorescence;   determining a fluorescence polarization value of the fluorescence; and   identifying the test compound as an inhibitor for PARP based on the fluorescence polarization value of the fluorescence.   
     
     
         16 . The method of  claim 15 , further comprising identifying the test compound as an inhibitor for PARP if the fluorescence polarization value of the fluorescence is decreased as compared to a reference level. 
     
     
         17 . The method of  claim 16 , wherein the reference value is the fluorescence polarization value of the fluorescence in the absence of the test compound. 
     
     
         18 . The method of  claim 15 , wherein the PARP probe is a compound having the structure: 
       
         
           
           
               
               
           
         
         or 
         or a salt thereof. 
       
     
     
         19 . The method of  claim 15 , wherein the PARP is PARP1, PARP2, PARP3, PARP4, PARP5a, PARP5b, PARP6, TIPARP, PARP8, PARP10, PARP1l, PARP12, PARP13, PARP14, PARP15, or PARP16. 
     
     
         20 . The method of  claim 15 , wherein the test compound is a small molecule, a nucleic acid, a polypeptide, or an antibody or antigen-binding fragment thereof. 
     
     
         21 . A method of identifying an inhibitor for Poly (ADP-ribose) polymerase (PARP), the method comprising:
 contacting a fusion polypeptide with a PARP probe that comprises a fluorophore, wherein the fusion polypeptide comprises a PARP catalytic domain and a luciferase enzyme;   contacting the luciferase enzyme with a substrate to produce light, wherein the light can excite the fluorophore;   measuring a signal produced by the fluorophore in the presence of a test compound; and   identifying the test compound as an inhibitor for PARP based on the signal produced by the fluorophore.   
     
     
         22 . The method of  claim 21 , further comprising identifying the test compound as an inhibitor for PARP if the signal produced by the fluorophore is decreased as compared to a reference level. 
     
     
         23 . The method of  claim 22 , wherein the reference level is the signal produced by the fluorophore in the absence of the test compound. 
     
     
         24 . The method of  claim 21 , wherein the PARP probe is a compound having the structure: 
       
         
           
           
               
               
           
         
         or a salt thereof. 
       
     
     
         25 . The method of  claim 21 , wherein the fusion polypeptide comprises a sequence that is at least 85%, 90% or 95% identical to SEQ ID NO: 11. 
     
     
         26 . The method of  claim 22 , wherein the PARP is PARP1, PARP2, PARP4, PARP5a, PARP5b, PARP3, PARP6, TIPARP, PARP8, PARP9, PARP10, PARP11, PARP12, PARP13, PARP14, PARP15, or PARP16. 
     
     
         27 . The method of  claim 22 , wherein the test compound is a small molecule, a nucleic acid, a polypeptide, or an antibody or antigen-binding fragment thereof. 
     
     
         28 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 456 to 657 of NP_056323.2 (SEQ ID NO: 1). 
     
     
         29 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 808 to 1025 of NP_116178.2 (SEQ ID NO: 2). 
     
     
         30 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 489 to 684 of NP_073587.1 (SEQ ID NO: 3). 
     
     
         31 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 1611 to 1801 of NP_060024.2 (SEQ ID NO: 4). 
     
     
         32 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 481 to 678 of NP_689828.1 (SEQ ID NO: 5). 
     
     
         33 . The method of  claim 1 , wherein the polypeptide comprising a PARP catalytic domain comprises a sequence that is at least 85%, 90% or 95% identical to amino acid residues of 5 to 279 of NP_060321.3 (SEQ ID NO: 6). 
     
     
         34 . A PARP probe having a structure according to Formula (I): 
       
         
           
           
               
               
           
         
         or a salt thereof, 
         wherein:
 L is a linking group having 5-30 spacer atoms selected from C, N, O, and S connecting the N atom of the piperidinyl group of Formula (I) with group A; 
 A is a fluorophore or an affinity tag. 
 
       
     
     
         35 . The PARP probe of  claim 34  wherein L is 
       
         
           
           
               
               
           
         
         a is 0, 1, or 2; 
         b is 1-26; and 
         c is 0, 1, or 2; 
         wherein the sum of a+b+c is 1 to 26, or 
         L is a chain of 5-30 atoms in length comprising —(CH 2 CH 2 O) d — wherein d is 2-10. 
       
     
     
         36 . The PARP probe of  claim 34  wherein A is: 
       
         
           
           
               
               
           
         
         or a salt thereof. 
       
     
     
         37 . The PARP probe of  claim 34  which is a compound having the structure: 
       
         
           
           
               
               
           
         
         or a salt thereof.

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