Name: VX-809 (Lumacaftor)
Brand: Selleck Chemicals
SKU: S1565
Rating: 5 (234 reviews)

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Controllo Qualità (Quality Control)

Lotto: Purezza: 99.87%

99.87

Target correlati	CRM1 CD markers AChR Calcium Channel Sodium Channel Potassium Channel GABA Receptor TRP Channel ATPase GluR
Altro CFTR Inibitori	Tezacaftor (VX-661) VX-445 (Elexacaftor) Vanzacaftor (VX-121) CFTRinh-172 GLPG1837 Galicaftor (ABBV-2222) GlyH-101 FDL169 IOWH032 PPQ-102

Coltura cellulare, trattamento e concentrazione di lavoro
(Cell Culture, Treatment & Working Concentration)

Linee cellulari	Tipo di saggio	Concentrazione	Tempo di incubazione	Descrizione dell'attività	PMID
CFBE41o	Corrector assay		24 hrs	Corrector activity at CFTR F508-del mutant (unknown origin) expressed in human CFBE41o cells harboring HS-YFP preincubated for 24 hrs followed by forskolin/genistein stimulation for 30 mins by fluorescence assay, EC50 = 2.5704 μM.	29272749
FRT	Corrector assay		25 mins	Corrector activity at human CFTR F508 deletion mutant expressed in FRT cells incubated for 25 mins with forskolin by YFP-based fluorescence analysis relative to control, EC50 = 2.6 μM.	26561003
TC32	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for TC32 cells	29435139
U-2 OS	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells	29435139
A673	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for A673 cells	29435139
DAOY	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for DAOY cells	29435139
Saos-2	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Saos-2 cells	29435139
BT-37	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-37 cells	29435139
RD	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for RD cells	29435139
SK-N-SH	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-SH cells	29435139
BT-12	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-12 cells	29435139
MG 63 (6-TG R)	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for MG 63 (6-TG R) cells	29435139
NB1643	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB1643 cells	29435139
BT-12	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for BT-12 cells	29435139
OHS-50	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for OHS-50 cells	29435139
fibroblast cells	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells	29435139
Rh41	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh41 cells	29435139
Rh30	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh30 cells	29435139
U-2 OS	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for U-2 OS cells	29435139
Rh41	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh41 cells	29435139
RD	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for RD cells	29435139
SJ-GBM2	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SJ-GBM2 cells	29435139
SK-N-MC	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-MC cells	29435139
NB-EBc1	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB-EBc1 cells	29435139
LAN-5	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for LAN-5 cells	29435139
Rh18	qHTS assay			qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh18 cells	29435139
CFBE41o	Corrector assay	1 uM	24 hrs	Corrector activity at CFTR F508-del mutant (unknown origin) expressed in human CFBE41o cells harboring HS-YFP assessed as increase in matured protein levels at cell surface at 1 uM after 24 hrs by electrophoretic mobility assay	29272749
HBE	Corrector assay			Corrector activity at CFTR F508del/F508del mutant in primary HBE cells assessed as increase in chloride ion current across apical membrane measured 18 to 24 hrs post compound treatment on basolateral side of cells in presence of channel potentiator GLPG18	29251932
CFBE41o	Corrector assay			Corrector activity at CFTR F508del mutant (unknown origin) expressed in human CFBE41o cells assessed as increase in fully glycosylated protein by western blot analysis	26041577
CFBE41o	Corrector assay	1 uM	24 hrs	Corrector activity at CFTR F508del mutant (unknown origin) expressed in human CFBE41o cells assessed as increase in size of cAMP-dependent current at 1 uM after 24 hrs measured at +100 mV by whole cell patch clamp assay	26041577
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Informazioni chimiche, conservazione e stabilità (Chemical Information, Storage & Stability)

Peso molecolare	452.41	Formula	C₂₄H₁₈F₂N₂O₅	Conservazione (Dalla data di ricezione)	3 anni-20°Cpolvere
N. CAS	936727-05-8	Scarica SDF		Conservazione delle soluzioni stock	1 anno-80°Cin solvente 1 mese-20°Cin solvente
Sinonimi	VRT 826809			Smiles	CC1=C(N=C(C=C1)NC(=O)C2(CC2)C3=CC4=C(C=C3)OC(O4)(F)F)C5=CC(=CC=C5)C(=O)O

Solubilità (Solubility)

In vitro
Lotto:

DMSO : 90 mg/mL (198.93 mM)
(Il DMSO contaminato da umidità può ridurre la solubilità. Utilizzare DMSO fresco e anidro.)

Ethanol : 13 mg/mL

Water : Insoluble

Calcolatore di Molarità

Massa	Concentrazione	Volume	Peso molecolare
=	×	×

Calcolatore di Diluizione Calcolatore del Peso Molecolare

In vivo Lotto:
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Clear solution	30%PEG400 1 0.5%Tween80 2 5%propylene glycol Validato dai laboratori Selleck. Se necessiti di modifiche a questa formulazione, contatta il nostro team di vendita per test personalizzati.	30.000mg/ml (66.31mM)	Taking the 1 mL working solution as an example, add 300 μL of 100 mg/ml clarified PEG400 stock solution to 5 μL of Tween80, mix evenly to clarify it; add 50 μL Propylene glycol to the above system, mix evenly to clarify it; then continue to add 645 μL ddH2O to adjust the volume. to 1 mL. The mixed solution should be used immediately for optimal results.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.
Homogeneous suspension	CMC-NA	≥5mg/ml	Taking the 1 mL working solution as an example, add 5 mg of this product to 1 ml of CMC-Na solution, mix evenly to obtain a homogeneous suspension with a final concentration of 5 mg/ml.

Calcolatore di formulazione in vivo (Soluzione chiara)

Passo 1: Inserire le informazioni di seguito (Consigliato: Un animale aggiuntivo per tenere conto della perdita durante l'esperimento)

Dosaggio: mg/kg Peso medio degli animali: g Volume di dosaggio per animale:μL Numero di animali:

Passo 2: Inserire la formulazione in vivo (Questo è solo il calcolatore, non la formulazione. Contattateci prima se non c'è una formulazione in vivo nella sezione Solubilità.)

% DMSO + % + % Tween 80 + % ddH₂O

%DMSO+ %

Risultati del calcolo:

Concentrazione di lavoro: mg/ml;

Metodo per preparare il liquido master di DMSO: mg farmaco predissolto in μL DMSO ( Concentrazione del liquido master mg/mL, Vi preghiamo di contattarci prima se la concentrazione supera la solubilità del DMSO del lotto del farmaco. )

Metodo per preparare la formulazione in vivo: Prendere μL DMSO liquido master, quindi aggiungereμL PEG300, mescolare e chiarire, quindi aggiungereμL Tween 80, mescolare e chiarire, quindi aggiungere μL ddH₂O, mescolare e chiarire.

Metodo per preparare la formulazione in vivo: Prendere μL DMSO liquido master, quindi aggiungere μL Olio di mais, mescolare e chiarire.

Nota: 1. Si prega di assicurarsi che il liquido sia limpido prima di aggiungere il solvente successivo.
2. Assicurarsi di aggiungere il/i solvente/i in ordine. È necessario assicurarsi che la soluzione ottenuta, nell'aggiunta precedente, sia una soluzione limpida prima di procedere all'aggiunta del solvente successivo. Metodi fisici come il vortex, gli ultrasuoni o il bagno d'acqua calda possono essere utilizzati per facilitare la dissoluzione.

Meccanismo d'azione (Mechanism of Action)

Caratteristiche	Higher specificity and efficacy relative to other CFTR defect drugs.
Targets/IC₅₀/Ki	F508del-CFTR (Fisher rat thyroid cells) 0.1 μM(EC50)
In vitro	Lumacaftor (VX-809) acts at the level of the ER to allow a fraction of the F508del-CFTR to adopt a properly folded form, to exit the ER and mobilize to the cell surface for normal functioning. In Fischer rat thyroid (FRT) cells expressing F508del-CFTR, this compound treatment significantly improves F508del-CFTR maturation by 7.1 fold with an EC50 of 0.1 μM, and enhances F508del-CFTR-mediated chloride transport by approximately 5 fold with EC50 of 0.5 μM, while VRT-768 has higher EC50 values of 7.9 μM and 16 μM, respectively. In HEK-293 cells expressing F508del-CFTR, it (3 μM) increases F508del-CFTR exit from the ER by 6 fold, reaching levels comparable to 34% of CFTR. In primary human bronchial epithelial (HBE) cells with F508del-CFTR mutation, the agent increases CFTR maturation and enhances chloride secretion with EC50 of 350 nM and 81 nM, respectively, more efficacious than Corr-4a and VRT-325. F508del-CFTR corrected by it exhibits single-channel open probability of 0.39 similar to normal CFTR of 0.40. Unlike VX-770, it is not a CFTR potentiator, as acute addition has no effect on F508del-CFTR function. In contrast to VRT-325 and Corr-4a, it does not improve the processing of the normal or mutant forms of hERG or P-gp, as well as other disease-causing mislocalized proteins, including α1-antitrypsin Z mutant (E342K-α1-AT) or N370S-β-glucosidase, suggesting that it is specific for CFTR. It in combination with VRT-325 or Corr-4a has additive effect on CFTR-mediated chloride transport in cultured F508del-HBE.
Saggio chinasico	maturazione del F508del-CFTR
Saggio chinasico	Le cellule FRT che esprimono stabilmente F508del-CFTR vengono trattate con concentrazioni crescenti di Lumacaftor (VX-809) per 48 ore. Dopo l'incubazione, le cellule vengono raccolte in soluzione D-PBS ghiacciata (senza calcio e magnesio) e centrifugate a 1.000 × g a 4 °C. I pellet cellulari vengono lisati in 1% Nonidet P-40, 0,5% desossicolato di sodio, 200 mM NaCl, 10 mM Tris, pH 7,8 e 1 mM EDTA più miscela di inibitori di proteasi (1:250) per 30 minuti su ghiaccio. I lisati vengono centrifugati per 10 minuti a 10.000 × g a 4 °C per pelletizzare i nuclei e il materiale insolubile. Circa 12 μg di proteine totali vengono riscaldate in tampone di Laemmli con 5% β-mercaptoetanolo a 37 °C per 5 minuti e caricate su un gel Tris-acetato dal 3% all'8%. Il gel viene trasferito su nitrocellulosa e processato per Western blotting utilizzando un anticorpo monoclonale CFTR o policlonale per GAPDH. I blot vengono sviluppati mediante chemiluminescenza potenziata. La quantificazione delle quantità relative delle bande C e GAPDH viene eseguita utilizzando l'analisi NIH ImageJ dei film scansionati.
In vivo	Lumacaftor (VX-809; VRT 826809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein.
Riferimenti	[1] https://pubmed.ncbi.nlm.nih.gov/21976485/

Applicazioni (Applications)

Metodi	Biomarcatori	Immagini	PMID
Immunofluorescence	CFTR / USP13 Cell surface kAE1 / kAE1		30618756

Informazioni sullo studio clinico (Clinical Trial Information)

(dati da https://clinicaltrials.gov, aggiornato il 2026-07-17)

Numero NCT	Reclutamento	Condizioni	Sponsor/Collaboratori	Data di inizio	Fasi
NCT07695090	RECRUITING	Heart Failure With Reduced Ejection Fraction	Qanatpharma AG	2026-07	PHASE2
NCT04581408	COMPLETED	Long QT Syndrome	Istituto Auxologico Italiano	2021-06-15	PHASE2
NCT05968612	COMPLETED	Healthy Volunteer	Qanatpharma Canada LTD	2023-11-17	PHASE1
NCT04235140	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2020-02-24	PHASE3
NCT03601637	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-09-07	PHASE3
NCT03625466	COMPLETED	Cystic Fibrosis	Vertex Pharmaceuticals Incorporated	2018-08-10	PHASE2

C1=C0/X	C1:	LOG(C1):
C2=C1/X	C2:	LOG(C2):
C3=C2/X	C3:	LOG(C3):
C4=C3/X	C4:	LOG(C4):
C5=C4/X	C5:	LOG(C5):
C6=C5/X	C6:	LOG(C6):
C7=C6/X	C7:	LOG(C7):
C8=C7/X	C8:	LOG(C8):

VX-809 (Lumacaftor) CFTR Modulatore

Peso molecolare: 452.41

Controllo Qualità (Quality Control)

Coltura cellulare, trattamento e concentrazione di lavoro
(Cell Culture, Treatment & Working Concentration)

Informazioni chimiche, conservazione e stabilità (Chemical Information, Storage & Stability)

Solubilità (Solubility)

Calcolatore di Molarità

Calcolatore di formulazione in vivo (Soluzione chiara)

Meccanismo d'azione (Mechanism of Action)

Applicazioni (Applications)

Informazioni sullo studio clinico (Clinical Trial Information)

VX-809 (Lumacaftor) CFTR Modulatore

Peso molecolare: 452.41

Controllo Qualità (Quality Control)

Coltura cellulare, trattamento e concentrazione di lavoro (Cell Culture, Treatment & Working Concentration)

Informazioni chimiche, conservazione e stabilità (Chemical Information, Storage & Stability)

Solubilità (Solubility)

Calcolatore di Molarità

Calcolatore di formulazione in vivo (Soluzione chiara)

Meccanismo d'azione (Mechanism of Action)

Applicazioni (Applications)

Informazioni sullo studio clinico (Clinical Trial Information)

Coltura cellulare, trattamento e concentrazione di lavoro
(Cell Culture, Treatment & Working Concentration)