Testing an inhaled gene therapy treatment for cystic fibrosis

This is a Phase 1 safety trial testing KB407, an inhaled gene therapy designed to treat cystic fibrosis. KB407 works by delivering a healthy copy of a gene that cystic fibrosis patients lack, which may help restore normal lung function. This trial will assess whether the medication is safe and well-tolerated when given to adults with CF in increasing doses.

Cystic fibrosis damages the lungs by causing thick, sticky mucus buildup that makes breathing difficult and leads to recurrent infections. Current treatments manage symptoms but don't address the underlying genetic cause. This medication represents a new approach—gene therapy—that aims to fix the root problem by replacing the faulty gene, potentially offering a more lasting benefit.