Krystal Biotech Presents Preclinical Data on KB801 Gene Therapy for Neurotrophic Keratitis at ARVO 2025

Krystal Biotech, Inc.

KB801 is a topical HSV-1-based vector encoding human NGF for neurotrophic keratitis, disclosed in an 8-K filed May 5, 2025.
In vitro, KB801 transduction of primary human corneal epithelial cells resulted in dose-dependent secretion of mature, functional NGF protein.
In a murine corneal wound model, a single topical KB801 dose produced higher peak NGF (491 pg/eye) and prolonged exposure (t1/2 55.5 hrs) vs. rhNGF (t1/2 83 min).
After eight weekly topical KB801 doses, eyes showed only mild inflammatory infiltrate with no necrosis; all resolved by 27 days post-treatment.
Poster presented at ARVO 2025 Annual Meeting in Salt Lake City, Utah on May 5, 2025.

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