KB801

KB801 is a topically administered, replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived gene therapy vector developed by Krystal Biotech. It is engineered to deliver two copies of the nerve growth factor (NGF) transgene to corneal epithelial cells. By transducing these cells, KB801 enables sustained production and secretion of NGF in the front of the eye, aiming to restore corneal health and reduce treatment burden for patients with neurotrophic keratopathy (NK). The drug’s mechanism involves gene transfer leading to local NGF expression. Preclinical studies have demonstrated efficient transduction and sustained NGF production in vitro and in vivo[2][3][5][9].

Other names

NGF gene therapy