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KB104
- Development stage
- Preclinical
- Lead developer
- Krystal Biotech
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Topical, Injectable
01
Overview
KB104 is a herpes simplex virus type 1 (HSV-1) vector-based gene therapy developed by Krystal Biotech for the treatment of Netherton syndrome, a rare genetic skin disorder. The therapy is engineered to deliver a functional copy of the SPINK5 gene directly to the skin of patients with mutations in this gene, aiming to restore normal skin barrier function. The underlying technology platform is STAR-D, which enables targeted delivery and expression of therapeutic genes in affected tissues. As of early 2024, KB104 remains in preclinical development and has not yet entered clinical trials[1][2][8][9].
- Other names
- KB 104KB104KB-104
02
Targets
KLK5 (Kallikrein-related peptidase 5)SPINK5 (Serine peptidase inhibitor Kazal-type 5)
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