Krystal Biotech has announced the initiation of the Phase 1 CORAL-1/US dose escalation trial of its KB407 inhaled gene therapy for the treatment of cystic fibrosis. The Phase 1 study, which is being conducted at the Cystic Fibrosis Institute of Chicago, is expected to enroll approximately 20 CF patients who will receive either KB407 delivered over 30 minutes on 1 day, 2 days in a row, or 4 consecutive days.
According to Krystal, KB407 delivers 2 copies of the CFTR gene, is redosable, and is intended for the treatment of CF patients regardless of underlying mutation. The 1 and 2-day cohort portions of the study are open-label and will involve patients who may use CFTR modulators; the 4-day cohort will involve patients who cannot benefit from CFTR modulators and who will be randomized to receive either KB407 or placebo.
Krystal Biotech Senior VP of Clinical Development Hubert Chen commented, “Dosing our first patient in the KB407 Phase 1 clinical trial is an exciting step forward for the company and for the patients we aim to benefit. Not only is the Phase 1 study designed to provide key insights into the safety and efficacy of KB407, it is also a critical step in expanding our vector platform to tissues beyond the skin. We look forward to continued enrollment into the Phase 1 study with anticipated data in 2024.”
Read the Krystal Biotech press release.
