Atopic dermatitis (AD), also known as atopic eczema, is a major skin disorder affecting 15%–20% of children*, and is well-recognized as a complex trait where a combination of genetic factors and environmental stimuli interact to produce the disease. Filaggrin (FLG), a 12.5kb gene, has recently been identified as a major player in AD, and it is estimated that 50% or more of the AD patients carry a mutation in AD. Due to its size and complexity, packaging of FLG in other viral vectors is highly inefficient and not feasible. Our proprietary backbone with a payload capacity ≥30Kb can accommodate FLG as well as other effectors for a combinatorial approach to target AD.**
*Nutten S. Atopic Dermatitis: Global epidemiology and risk factors. Ann Nutr Metab 2015;66(suppl 1):8-16
**Weidinger S. Loss-of-function variations within the filaggrin gene predispose for atopic dermatitis with allergic sensitizations. J Allergy Clin Immunol. 2006 Jul;118(1):214-9.