Krystal Biotech, Inc.

This early-stage study tests an inhaled gene therapy called KB407 in 20 adults with cystic fibrosis. The treatment uses a harmless virus to deliver a working copy of the CF gene directly to the lungs. The main goal is to check safety, but researchers will also measure if it impro…

This study tests an experimental eye drop called KB801 for people with stage 2 or 3 neurotrophic keratitis, a condition where the cornea loses feeling and develops persistent sores. The drop delivers a gene that produces nerve growth factor to help heal the cornea. About 60 adult…

This study tests an inhaled therapy called KB707 for people with advanced solid tumors that have spread to the lungs. KB707 is a modified virus designed to trigger the immune system to attack tumors. The trial involves 250 adults who have not responded to or cannot tolerate stand…

This study tests an eye drop called KB803 in 16 people aged 6 months and older with dystrophic epidermolysis bullosa (DEB), a rare genetic condition causing fragile skin and recurrent corneal abrasions. The trial compares KB803 to a placebo to see if it safely reduces the number …