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Posenacaftor

Chemical Structure : Posenacaftor

CAS No.: 2095064-05-2

Posenacaftor (PTI-801, PTI 801)

Catalog No.: PC-22022Not For Human Use, Lab Use Only.

Posenacaftor (PTI-801) is a newly developed p.Phe508del-CFTR corrector with EC50 of 0.52 uM (HS-YFP quenching rate).

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Purity & Documentation Purity: >98% (HPLC)

Biological Activity

Posenacaftor (PTI-801) is a newly developed p.Phe508del-CFTR corrector with EC50 of 0.52 uM (HS-YFP quenching rate).
Posenacaftor (PTI-801) at 1 and 3 μM concentrations also demonstrated greater efficacy in rescuing p.Phe508del-CFTR function than VX-661 at 5 μM concentration.
Posenacaftor (PTI-801) rescues p.Phe508del-CFTR processing, PM trafficking, and channel function (upon agonist stimulation) with greater correction effects in combination with ABBV-2222, FDL-169, VX-661, or VX-809, but not with VX-445.
Posenacaftor (PTI-801) exhibits no potentiator activity on low temperature- and corrector-rescued p.Phe508del-CFTR, displays similar behavior to that of VX-445 on genetic revertants.

Physicochemical Properties

M.Wt 445.52
Formula C27H27NO5
Appearance Solid
CAS No.
Storage
Solide Powder
-20°C 12 Months; 4°C 6 Months
In Solvent
-80°C 6 Months; -20°C 6 Months
Shipping
Solubility

10 mM in DMSO

Chemical Name/SMILES

8-Methyl-2-(3-methyl-2-benzofuranyl)-5-[(1R)-1-(tetrahydro-2H-pyran-4-yl)ethoxy]-4-quinolinecarboxylic acid

References

1. Filipa C Ferreira, et al. Eur J Pharmacol. 2024 Mar 15:967:176390.

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