CF is caused by a mutation in the cystic fibrosis transmembrane conductance (CFTR) gene, which codes for the protein that regulates salt and water balance. It is characterized by the buildup of mucus in the respiratory and digestive track leading to progressive damage to these organs. There are five classes of CF representing more than 1,700 different mutations in the CFTR gene.
Class 1 nonsense mutations result in no production of CFTR protein
• 12% of mutations are class 1, affecting between 7,000 and 9,000 people globally1
There are no approved treatments for Class 1 CF; management is palliative
address Class 1 CF: ELX-02 and oral TURBO-ZM™ based RMAs.