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PharmSci Research Seminar Series: Prasuna Paraselli

February 2 @ 12:00 pm - 1:00 pm

Free

Speaker

Prasuna Paraselli, Vertex Pharmaceuticals

Title

Advances in Cystic Fibrosis Therapy: A Case History Study of the Ivacaftor and Lumacaftor Combo

Abstract

Quinoline-3-carboxamide, a novel CFTR potentiator, was discovered using high-throughput screening in NIH-3T3 cells expressing the F508del-CFTR mutation. Extensive medicinal chemistry and iterative structure-activity relationship       (SAR) studies to evaluate potency, selectivity, and pharmacokinetic properties resulted in the identification of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, Ivacaftor), an investigational drug    candidate approved by the FDA for the treatment of CF patients 6 years of age and older carrying the G551D mutation. It took over 22 years from the discovery of the CFTR gene in 1989 until approval of the first drug that targets the defective CFTR         mutant protein, which is the underlying cause of cystic fibrosis. Additional CFTR modulators have been approved to treat other mutations such as F508del in combination with VX-770, and a case history study of the combination therapy with   Ivacaftor and Lumacaftor will be discussed.

Details

Date:
February 2
Time:
12:00 pm - 1:00 pm
Cost:
Free
Event Categories:
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