IP Library Patent Application 14532791
Patent Application
App. No. 14/532,791

Modulators of ATP-Binding Cassette Transporters

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Quick Facts
Patent No.
US None
App. No.
14/532,791
Abstract

Compounds of the present invention and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (“CFTR”). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.

Claims (17)

1 - 39 . (canceled)

40 . A compound which is:

41 . A pharmaceutical composition comprising the compound as described in claim 40 and a pharmaceutically acceptable carrier.

42 . A kit for use in measuring the activity of an ABC transporter or a fragment thereof in a biological sample in vitro or in vivo, comprising:

(i) a composition comprising the compound according to claim 40 ; and

(ii) instructions for:

a) contacting the composition with the biological sample; and

b) measuring activity of said ABC transporter or a fragment thereof.

43 . The kit of claim 42 , further comprising instructions for:

a) contacting an additional composition with the biological sample;

b) measuring the activity of said ABC transporter or a fragment thereof in the presence of said additional compound; and

c) comparing the activity of the ABC transporter in the presence of the additional compound with the density of the ABC transporter in the presence of a composition of formula (I).

44 . The kit of claim 42 , wherein the kit is used to measure the density of CFTR.

45 . A method of modulating ABC transporter activity comprising the step of contacting said ABC transporter with a compound which is:

46 . The method of claim 45 , wherein the ABC transporter is CFTR.

47 . A method of treating or lessening the severity of a disease in a patient, wherein said disease is selected from cystic fibrosis, hereditary emphysema, COPD, dry-eye disease, pancreatic insufficiency or male infertility, said method comprising the step of administering to said patient an effective amount of a compound which is:

48 . The method of claim 47 , wherein the disease is cystic fibrosis.

Assignments (2)
CHANGE OF ADDRESS Recorded Jul 11, 2017
From: VERTEX PHARMACEUTICALS INCORPORATED
To: VERTEX PHARMACEUTICALS INCORPORATED
Reel/Frame 043151/0445 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 26, 2015
From: HADIDA RUAH, SARA S.; GROOTENHUIS, PETER D.J.; VAN GOOR, FREDRICK; ZHOU, JINGLAN; BEAR, BRIAN; MILLER, MARK T.; MCCARTNEY, JASON; NUMA, MEHDI MICHEL JAMEL; YANG, XIAOQING
To: VERTEX PHARMACEUTICALS INCORPORATED
Reel/Frame 035266/0531 →