Krystal Biotech presents preclinical data for KB111 (HHD) and KB112 (Darier) at SID 2025

Krystal Biotech, Inc.

KB111 targets ATP2C1 for Hailey-Hailey disease; KB112 targets ATP2A2 for Darier disease.
Both HSV-1-based vectors transduced keratinocytes with dose-dependent target protein expression and no cytotoxicity.
KB111 functionally corrected ATP2C1 siRNA knockdown in keratinocytes, reversing F-actin loss (p<0.05).
Topical administration in mice showed proper localization of ATP2C1 and ATP2A2 with minimal toxicity.

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