CFTR

CFTR Corrector 16 targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein, functioning as a corrector to enhance its trafficking and function. It exhibits significant biological activity in restoring chloride ion channel function, making it a valuable tool for studying cystic fibrosis pathology. This compound is useful for research applications focused on the development of therapies for cystic fibrosis and related ion channel disorders.

ARN23765 is a CFTR corrector specifically designed to target the F508del mutation. With an EC50 of 38 pM in human bronchial epithelial cells, ARN23765 enhances the maturation and membrane expression of F508del-CFTR, thereby improving ion transport and secretion. This compound plays a significant role in addressing the underlying pathological mechanisms associated with cystic fibrosis, making it a valuable tool for research in therapeutic interventions for this condition.

Riselcaftor is a CFTR modulator that exhibits an EC50 value of 20.1 nM in human bronchial epithelial cells. This compound is primarily utilized in research focused on cystic fibrosis, facilitating investigations into the modulation of cystic fibrosis transmembrane conductance regulator activity. Its potent effects on CFTR function make it a valuable tool for studying therapeutic pathways in the treatment of cystic fibrosis.

GLPG2737 is a potent CFTR type 2 corrector that enhances the functional activity of the CFTR protein in cystic fibrosis models. This compound can be utilized alongside a type 1 co-corrector to investigate synergistic effects on protein correction and improve chloride transport. Its application in research aids in elucidating therapeutic avenues for cystic fibrosis treatment and understanding CFTR-related pathophysiology.

VRT-532 is a potent modulator of the cystic fibrosis transmembrane conductance regulator (CFTR) channel. It significantly enhances channel activity and intrinsic ATPase activity in G551D-CFTR variants, providing valuable insights into cystic fibrosis mechanisms. This compound is suitable for research applications focused on developing therapeutic strategies for cystic fibrosis and understanding CFTR-related dysfunction.

CFTR Corrector 18 is a potent cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It enhances the processing and trafficking of CFTR, thereby increasing the presence of functional CFTR protein on the cell surface. This compound is a valuable tool for research applications focused on cystic fibrosis, aiming to elucidate pathways for therapeutic intervention and improve understanding of CFTR-related pathophysiology.

(R)-Olacaftor, a CFTR modulator, functions by enhancing the activity of the cystic fibrosis transmembrane conductance regulator. This compound demonstrates significant potential in the therapeutic investigation of cystic fibrosis, making it a valuable tool for research into CF-related pathophysiology and treatment strategies. Its role in restoring CFTR function supports studies targeting the underlying mechanisms of cystic fibrosis.

CFTR Corrector 9 is a small molecule modulator targeting the cystic fibrosis transmembrane conductance regulator (CFTR). This compound is designed to enhance the trafficking and functionality of mutant CFTR proteins, making it valuable for research into cystic fibrosis and other CFTR-related disorders. Its application can facilitate studies aimed at understanding disease mechanisms and developing therapeutic strategies for CF.

AF-2785 is a potent blocker of the cystic fibrosis transmembrane conductance regulator (CFTR). It effectively inhibits the cAMP-activated chloride current in rat epididymal cells, exhibiting an IC50 of 170.6 μM. This reagent is primarily utilized in research focused on contraceptive mechanisms and related studies.

Zatonacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator designed to enhance the activity of the CFTR protein. This compound is primarily used in research studies focusing on cystic fibrosis, providing insights into disease mechanisms and potential therapeutic strategies. Its role in restoring CFTR function may aid in the development of new treatment approaches for cystic fibrosis patients.

Bamocaftor potassium is a CFTR corrector that targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein, specifically aimed at restoring the function of the F508del mutant variant. This compound is particularly useful in cystic fibrosis research when used in combination with Tezacaftor and Ivacaftor, providing insights into pharmacological interventions for improving chloride ion transport in affected patients. Its role in potentiating drug efficacy makes it significant for developing combination therapies in the treatment of cystic fibrosis.

CP-628006 is a small molecule CFTR potentiator that enhances ATP-dependent gating of the cystic fibrosis mutant G551D-CFTR. This compound has demonstrated key biological activity in restoring channel function, making it a valuable tool for research focused on cystic fibrosis pathophysiology and potential therapeutic interventions. Its utility in studying CFTR modulation may aid in the development of novel treatments for cystic fibrosis and related disorders.

CFTR Activator 2 (WAY-326769) is a selective activator of the cystic fibrosis transmembrane conductance regulator (CFTR), specifically targeting mutant forms of the protein. This compound enhances chloride ion transport across epithelial cell membranes, thereby improving ion permeability and function in cells expressing mutant CFTR. CFTR Activator 2 is valuable for research applications aimed at understanding cystic fibrosis pathophysiology and developing potential therapeutic strategies.

PGD97 is a selective cyclic peptide inhibitor that targets the CAL/CFTR interactions, exhibiting a KD value of 6 nM for its desulfide cyclized form at the CAL PDZ domain. Demonstrating a selectivity of ≥ 130-fold compared to NHERF1/2 PDZ domains, PGD97 effectively stabilizes F508del-CFTR at the cell membrane, enhancing CFTR function essential for maintaining fluid homeostasis in the lung. This compound is valuable for research applications focused on cystic fibrosis.

CFTR Corrector 11 is a chemical compound that acts as a corrector for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. It is primarily utilized in research focused on cystic fibrosis, where it aids in the rescue of misfolded CFTR proteins, thereby improving chloride ion transport. This compound is instrumental in studying therapeutic strategies for cystic fibrosis and evaluating the functional restoration of CFTR activity in affected cells.

Steviol is a selective inhibitor of the aquaporin-2 (AQP2) and cystic fibrosis transmembrane conductance regulator (CFTR) proteins. This compound impedes renal cyst growth by inhibiting CFTR activity, which leads to decreased AQP2 expression and promotes the degradation of both AQP2 and CFTR. Steviol is relevant for research focused on polycystic kidney disease and mechanisms underlying renal cyst development.