Krystal Biotech KB407 Phase 1 positive: CFTR delivery confirmed in all 6 biopsied patients

Krystal Biotech, Inc.

Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations (modulator ineligible).
Transduction rates ranged 29.4% to 42.1% across six patients; all biopsies positive for CFTR or viral marker.
KB407 well tolerated; one SAE (asthma exacerbation) deemed procedure-related, resolved in 5 days.
Registrational CORAL-3 study design submitted to FDA; enrollment expected 1H 2026 after alignment.
Four of seven highest-dose patients were modulator ineligible; data supports mutation-agnostic potential.

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