KB407

Drug

Drug Profile

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
Brand Names
  • KB407
Indications
  • \Cystic fibrosis (mutation-agnostic investigational setting)\

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