Krystal Biotech focuses primarily on developing advanced genetic medicines to treat severe, life-altering rare diseases with high unmet medical needs, spanning multiple distinct therapeutic fields. Its lead commercialized program targets Dystrophic Epidermolysis Bullosa, a debilitating genetic skin blistering condition caused by a lack of functional collagen. Beyond dermatology, the company's proprietary viral vector technology platform is actively utilized to tackle severe ophthalmic conditions, such as corneal abrasions and neurotrophic keratitis, and chronic pulmonary disorders, notably cystic fibrosis. Furthermore, the company has expanded its clinical scope into oncology, investigating localized treatments for advanced non-small cell lung cancer and other solid tumors, demonstrating the versatile therapeutic application of its proprietary gene delivery systems.