Krystal Biotech reports positive initial clinical data for inhaled gene therapies KB408 (AATD) and KB407 (CF)

Krystal Biotech, Inc.

KB408: single dose increased lung AAT from 85nM to 729nM in AATD patient; active neutrophil elastase dropped >50%.
Gene delivery confirmed: 39% of airway cells AAT-positive post-dose vs 0% at baseline.
KB407 for CF: Phase 1 CORAL-1 granted conditional sanctioning by CFF TDN; safe and well tolerated.
KB407 enrolling Cohort 3 (4 daily doses); data expected 1H 2025.
No serious adverse events in either program; all adverse events mild to moderate and transient.

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