Drug intelligence / Profile preview

KB104

Development stage
Preclinical
Lead developer
Krystal Biotech
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → 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

SPINK5 (Serine peptidase inhibitor Kazal-type 5)KLK5 (Kallikrein-related peptidase 5)

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