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Esculentin and its derivatives for the treatment of cystic fibrosis

Cystic fibrosisFibrosi cisticaLung pathologyNtimicrobial peptidesPotentiators CFTR channelTherapeutic agents


The present invention provides the use of synthetic antimicrobial peptides (AMPs) deriving from the frog skin peptide esculentina-1a, to restore the dysregulation of the composition of water and ions of the liquid that bathes the respiratory epithelium, due to mutations in the gene encoding for the ion channel selective for halides, which is defined cystic fibrosis transmembrane conductance regulator (CFTR).

Technical features

This patent describes the use of AMPs as modulators of the mutated CFTR channel in patients with cystic fibrosis (CF) and development of a formulation comprising such AMPs as an active ingredient and pharmaceutically acceptable adjuvants and / or vehicles for the treatment of pulmonary CF. One of the most frequent mutations in CF disease is the loss of phenylalanine 508 in the CFTR channel protein that controls the passage of chloride ions across the membrane of secretory epithelia, including that of the airways. This results in the formation of a protein that is misfolded (rapidly degraded) and with a gating defect. The secretion of chloride ions is inhibited causing the formation of a sticky mucus layer on the respiratory epithelium which favours the development of a chronic lung infection with compromised respiratory functions. AMPs derived from esculentina-1a from frog skin show the ability to recover the activity of CFTR with gating mutations, presumably increasing the probability of opening the channel following their direct interaction with CFTR. It does not directly affect the treatment of lung infections in such patients. Regarding the dosage for the treatment of keratitis, there are data in animal models that have demonstrated efficacy at a concentration of 40 uM equivalent to approximately 0.09 mg / mL.

Possible Applications

  • Treatment of CF pulmonary disease, with greater potential for success than already available drugs;
  • Elimination of lung bacterial infections, with reduced risk of inducing resistance, simultaneously with the recovery of activity of the mutated CFTR protein.


  • Peptides with the ability to restore the mutated CFTR activity, like the current enhancer Ivacaftor;
  • Peptides with antimicrobial and immunomodulatory activity;
  • Reduction of production costs.