KB407 is Krystal Biotech's redosable inhaled gene therapy candidate for cystic fibrosis, designed to deliver full-length wild-type CFTR to the lung independent of mutation class. Interim Phase 1 CORAL-1 data reported successful lung delivery and expression of wild-type CFTR in treated patients, and the company is advancing repeat-dosing registrational-intent CORAL-3 planning. The program remains investigational.
- Drug Class
- Investigational inhaled redosable gene therapy
- Approval Status
-
Investigational; in Phase 1 with registrational-intent repeat-dosing study design submitted to FDA
- Mechanism of Action
- Inhaled delivery of full-length wild-type CFTR transgene to airway cells to restore CFTR protein function and ion transport
Indications
- \Cystic fibrosis (mutation-agnostic investigational setting)\