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Approval Year

Substance Class Protein
Created
by admin
on Sat Jun 26 17:06:28 UTC 2021
Edited
by admin
on Sat Jun 26 17:06:28 UTC 2021
Protein Sub Type
Sequence Type COMPLETE
Record UNII
PW7453NX3R
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR
Common Name English
POMULMERAN EXPRESSED ABCC7 PROTEIN
Common Name English
CHANNEL CONDUCTANCE-CONTROLLING ATPASE
Common Name English
CAMP-DEPENDENT CHLORIDE CHANNEL
Common Name English
ATP-BINDING CASSETTE SUB-FAMILY C MEMBER 7
Common Name English
CFTR
Common Name English
ABCC7
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 63891
Created by admin on Sat Jun 26 17:06:29 UTC 2021 , Edited by admin on Sat Jun 26 17:06:29 UTC 2021
Code System Code Type Description
UNIPROT
P13569
Created by admin on Sat Jun 26 17:06:29 UTC 2021 , Edited by admin on Sat Jun 26 17:06:29 UTC 2021
PRIMARY
FDA UNII
PW7453NX3R
Created by admin on Sat Jun 26 17:06:29 UTC 2021 , Edited by admin on Sat Jun 26 17:06:29 UTC 2021
PRIMARY
Glycosylation Type HUMAN
Glycosylation Link Type Site
N 1_894
N 1_900
Related Record Type Details
GENE REPLACEMENT->TARGET
TARGET->INHIBITOR OF AGGREGATION
helps with the trafficking of the CFTR protein to the epithelial surface
TARGET->INHIBITOR OF AGGREGATION
helps with the trafficking of the CFTR protein to the epithelial surface
VECTOR->EXPRESSED PROTEIN
ACTIVATOR -> TARGET
TARGET->INHIBITOR OF AGGREGATION
ACTIVATOR -> TARGET
ACTIVATOR -> TARGET
PTI-808 is a small molecule “potentiator” that is intended to rescue mutant cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel activity. The Sponsor intends to develop PTI-808 as part of a triple combination therapy with PTI-801 (CFTR “corrector”) and PTI-428 (CFTR “amplifier”) in cystic fibrosis patients
TISSUE EXPRESSION->TRANSPORTER
APICAL
TARGET->INHIBITOR OF AGGREGATION
helps the CFTR protein open and transport chloride
ACTIVATOR -> TARGET
Name Property Type Amount Referenced Substance Defining Parameters References
MOL_WEIGHT CHEMICAL
Molecular Formula CHEMICAL