CFTR Modulators for Treating Rare Forms of Cystic Fibrosis


Small molecules to treat cystic fibrosis and other pulmonary diseases.

Key Benefits

  • Rescue rare variants of CFTR gene.
  • Serve as PDE4 inhibitors with strong anti-inflammatory effects.

Market Summary

Cystic fibrosis (CF) is a progressive, genetic disorder that arises from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, resulting in dysfunction at the plasma membrane. Wild type CFTR acts as an ion channel in epithelial cells, and regulates fluid transport in lung, pancreas, and other organs. Since CF has an autosomal recessive inheritance pattern, if a patient receives two mutated copies of the CFTR gene, the CFTR protein fails to act appropriately, causing thick secretions of mucus in the affected organs and profound inflammation in lung. The disease is progressive and leads to difficulty breathing, persistent lung infections, gastrointestinal issues, and male infertility. There is currently no cure for CF.

Technical Summary

Researchers at Emory University and Children’s Healthcare of Atlanta have developed small molecules that treat CFTR dysfunction as a result of rare variants for which no current CFTR modulator therapies exist. The compounds also enhance activity of G551D and F508del CFTR, and strongly augment the wild-type protein. Because these compounds function as PDE4 inhibitors, they are expected to have robust anti-inflammatory effects in cystic fibrosis respiratory disease, as well as conditions such as COPD and COVID-19.

Developmental Stage

  • Lead compounds identified and optimized by medicinal chemistry.
  • IND enabling studies in progress, with IND in preparation.
  • Clinical trial planned.

Patent Information

App Type Country Serial No. Patent No. File Date Issued Date Patent Status
PCT PCT PCT/US2021/043956   7/30/2021   National Phase Entered
Nationalized PCT - Foreign Australia 2021315786   1/24/2023   Pending
Nationalized PCT - Foreign Canada 3,187,651   1/30/2023   Pending
Nationalized PCT - United States United States 18/018,997   1/31/2023   Pending
Nationalized PCT - Foreign EP 21850804.2   2/17/2023   Pending
Tech ID: 19229
Published: 11/2/2020