Platform for AAV Delivery to Skin
Genetic engineering of skin cells poses an entirely new paradigm to gene and immune therapies, as it bypasses the natural immune response and forces in vivo translation, production, and passive secretion of proteins and antibodies locally or directly to the blood stream across the dermal vasculature. George Church's lab is developing a platform technology that doesn’t require genetic engineering ex vivo. Instead, a patch of skin is treated with cavitational ultrasound, which then allows penetration of a topical formulation of viral particles. This delivery platform is broadly applicable to circulating antibody therapies, locally manifesting genetic diseases of the skin, locally manifesting auto-immune diseases, and cosmetic treatment of skin aging. A startup emerging from the Church Lab intends to focus preclinical efforts on HIV vaccines and collagen modulation in skin. Additional anticipated products include gene therapy for a rare genetic and autoimmune skin disease.