Recessive Dystrophic Epidermolysis Bullosa

The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum. Fewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.

The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in serum. Fewer wounds, more rapidly healing wounds, and decreased C7 and HSV-1 antibodies could improve quality of life.

This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa). The GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts. It is made in a laboratory using a small sample of the patient's own skin. To help the patient's skin cells produce a missing protein called type VII collagen, scientists grow the patient's cells in the lab and use a virus-like tool (called a retroviral vector) to give the cells the correct instructions. This allows the cells to make the normal protein that is missing in people with RDEB. The graft is designed to be permanent, and the goal is to improve wound healing by replacing damaged skin cells with healthy ones.

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).