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 any improve…

This study tests an inhaled treatment 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 cancer cells. The trial involves 250 adults who have not responded to standard treatme…

This study tests a new gene therapy called KB801 for people with stage 2 or 3 neurotrophic keratitis, a rare eye disease where the cornea loses feeling and can develop sores that won't heal. The treatment is an eyedrop that delivers a protein to help the cornea repair itself. Abo…

This study tests an eye drop called KB803 for people with dystrophic epidermolysis bullosa (DEB), a rare genetic condition that causes fragile skin and repeated cornea scratches. The trial will include 16 people aged 6 months and older. Each person will receive both KB803 and a p…