Considerations In Dermatology Gene Therapy Development

By Anna Mazuy, MD, Senior Medical Director, Dermatology, IQVIA Biotech

How gene therapy is changing the trajectory of dermatology therapeutics by direct targeting of mutations.

Treatment methods in dermatology have evolved from purely topical medications to include oral treatments, injectables and light and laser therapies. Now, gene therapies offer a new trajectory to address the cause of the disease, rather than simply alleviating symptoms.

Gene therapy is becoming established in oncology and in several inherited disorders, including lipoprotein lipase deficiency, inherited retinal dystrophy, and spinal muscular atrophy. However, expert knowledge in dermatology gene therapy clinical trials can be hard to come by, as the use of gene therapy in dermatology is still a very new concept. The accessibility of the skin, partnered with its regenerative capacity, makes it an attractive target for genetic therapies. In particular, genome editing technologies have the potential to rapidly accelerate the treatment of genodermatoses, a heterogenous group of skin disorders with a variety of phenotypic severity¹.