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Pharmaceutical Composition for the Treatment of Cystic Fibrosis

Active pharmaceutical ingredient, with anionophore capacity for the treatment of patients with cystic fibrosis, regardless of the mutation

Technology Overview

This technology is a pharmaceutical composition for the preparation of a medicament for the treatment and/or prophylaxis of diseases derived from the abnormal transport of anions at the cellular level, comprising a compound of formula ().

There are a group of diseases related to abnormal function of channel proteins facilitating the transport of ions through biological membranes. These conditions, known as “channelopathies”, are very diverse and include cystic fibrosis (CF). Currently, approved drugs such as Kalydeco or Orkambi target pharmacological correction of CFTR protein. However, the therapeutic outcome of these depends dramatically on the specific mutations of the patients. More than 1,700 mutations have been identified and there is a cohort of patients lacking CFTR production for

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