Krystal Biotech operates as a fully integrated, commercial-stage genetic medicines enterprise dedicated to the discovery, development, design, and commercialization of targeted therapeutics for rare and severe diseases. The company leverages its proprietary, non-replicating engineered viral vector platform to deliver corrective genes directly to localized affected tissues, such as the skin, eyes, and lungs. By controlling every phase of the business model—from early-stage laboratory research and advanced clinical trials to specialized in-house current Good Manufacturing Practice production and global commercial distribution—the firm aims to transform the standard of care for patients suffering from genetic disorders that lack effective conventional treatment options.