Krystal Biotech, Inc.

This early study tests a new gene therapy gel called KB111 for Hailey-Hailey disease, a rare genetic condition that causes painful blisters and skin lesions. The gel uses a modified virus to deliver a working copy of the faulty gene directly to the skin. The trial enrolls about 7…

This early-stage study tests a new gene therapy called KB407, given as a mist through inhalation, in 20 adults with cystic fibrosis. The goal is to see if it is safe and can improve lung function by delivering a working copy of the CF gene to lung cells. Participants will receive…

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. Participants will receive both KB803 and a placebo in random order to see if …

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 cannot heal itself. The treatment uses a harmless virus to deliver nerve growth factor directly to the eye. About 60 participants will receive…

This study tests an inhaled treatment called KB707, a modified herpes virus designed to trigger the immune system against lung tumors. It is for adults with advanced solid tumors that have spread to the lungs and who have run out of standard options. The trial will test KB707 alo…