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Preclinical trials in vivo
The agent is an inhalation solution, a biosimilar of Pulmozyme® (Roche, Switzerland). Its active substance is recombinant human deoxyribonuclease I (dornase alpha). Purulent pulmonary discharge contains very high concentrations of extracellular DNA, a viscous polyanion released from disintegrating leukocytes accumulating during the course of infection . GNR-039cleaves extracellular DNA contained in large amounts in the viscous bronchial discharge accumulating in respiratory tract of cystic fibrosis patients. It also it decreases sputum viscosity, thus improving pulmonary function and diminishing infection risk in these patients.
Cystic fibrosis is a systemic congenital disease affecting all organs that secrete mucus: bronchopulmonary system, pancreas, liver, perspiratory, salivary intestinal and reproductive glands. Cystic fibrosis is one of the most prevalent congenital diseases. In cystic fibrosis patients pathological impairments are caused by mutation in the transmembrane cystic fibrosis conductance regulator gene (CFTR).
This protein controls transmembrane electrolyte (mostly chlorine) transport in excretory duct lining in exocrine glands. Mutation results in impaired protein structure and function, therefore, themucus secreted from these glands becomes excessively thick and viscous.
In Russia average incidence rate of cystic fibrosis is 1:10 000 of newborns.