(R)-Posenacaftor (R)-PTI-801) sodium is the R enantiomer of Posenacaftor. Posenacaftor (PTI-801) is a cystic fibrosis transmembrane regulatory protein (CFTR) modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor may be used in cystic fibrosis (CF) research.

Physicochemical Properties

Molecular Formula	C27H28NNAO5
Molecular Weight	469.504698753357
Exact Mass	468.178
CAS #	2095064-09-6
PubChem CID	168012957
Appearance	Yellow to brown solid powder
Hydrogen Bond Donor Count	1
Hydrogen Bond Acceptor Count	6
Rotatable Bond Count	5
Heavy Atom Count	34
Complexity	681
Defined Atom Stereocenter Count	1
SMILES	O(C1=CC=C(C)C2N=C(C3OC4=CC=CC=C4C=3C)C=C(C(=O)O)C1=2)[C@H](C1CCOCC1)C.[NaH]
InChi Key	GSYMTYPDLBWOIU-LMOVPXPDSA-N
InChi Code	InChI=1S/C27H27NO5.Na/c1-15-8-9-23(32-17(3)18-10-12-31-13-11-18)24-20(27(29)30)14-21(28-25(15)24)26-16(2)19-6-4-5-7-22(19)33-26;/h4-9,14,17-18H,10-13H2,1-3H3,(H,29,30);/t17-;/m0./s1
HS Tariff Code	2934.99.9001
Storage	Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture.
Shipping Condition	Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

Biological Activity

Targets	IC50: CFTR[1]
ln Vitro	The autosomal recessive illness known as cystic fibrosis (CF) is brought on by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR)[1]. The CFTR chloride channel is typically found at the apical membrane of epithelial cells and is controlled by cAMP. A malfunctioning and misfolded CFTR protein is produced when the CFTR gene is mutated, which hinders the movement of ions into and out of cells[1]. Posenacaftor is a CFTR corrector; correctors are made to correct and restore the CFTR protein's malfunction. After then, the modified CFTR travels to the cell surface, where it serves as a chloride channel and aids in preserving the proper fluid balance in the airways[2].
References	[1]. Compositions et procédés pour améliorer la fonction cftr dans des cellules affectées par la fibrose kystique. Patent WO2019156946.

Solubility Data

Solubility (In Vitro)

DMSO: 38.33 mg/mL (81.99 mM)

Solubility (In Vivo)

Solubility in Formulation 1: ≥ 3.83 mg/mL (8.19 mM) (saturation unknown) in 10% DMSO + 40% PEG300 + 5% Tween80 + 45% Saline (add these co-solvents sequentially from left to right, and one by one), clear solution. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 38.3 mg/mL clear DMSO stock solution to 400 μL PEG300 and mix evenly; then add 50 μL Tween-80 to the above solution and mix evenly; then add 450 μL normal saline to adjust the volume to 1 mL. Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH₂ O to obtain a clear solution. Solubility in Formulation 2: 3.83 mg/mL (8.19 mM) in 10% DMSO + 90% (20% SBE-β-CD in Saline) (add these co-solvents sequentially from left to right, and one by one), suspension solution; with ultrasonication. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 38.3 mg/mL clear DMSO stock solution to 900 μL of 20% SBE-β-CD physiological saline solution and mix evenly. Preparation of 20% SBE-β-CD in Saline (4°C，1 week): Dissolve 2 g SBE-β-CD in 10 mL saline to obtain a clear solution.  (Please use freshly prepared in vivo formulations for optimal results.)

Preparing Stock Solutions		1 mg	5 mg	10 mg
	1 mM	2.1299 mL	10.6496 mL	21.2993 mL
	5 mM	0.4260 mL	2.1299 mL	4.2599 mL
	10 mM	0.2130 mL	1.0650 mL	2.1299 mL

*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.