This segment focuses on the research, development, and commercialization of gene therapies for dermatological diseases. Krystal Biotech utilizes its proprietary gene therapy platform to develop treatments for rare skin conditions, including dystrophic epidermolysis bullosa (DEB) with its lead product, B-VEC. Research and development activities include preclinical studies, clinical trials (Phase III for B-VEC), and manufacturing process optimization. The company employs a redosable gene therapy approach, offering potential for repeated administration. This segment aims to improve the quality of life for patients suffering from severe skin disorders by addressing the underlying genetic causes. Market positioning is strong due to the unmet medical needs in rare skin diseases. Future opportunities include expanding the pipeline to treat other dermatological conditions and exploring new delivery methods. Regulatory and clinical aspects involve navigating FDA approval processes and conducting rigorous clinical trials. Partnerships with genetic testing providers enhance patient access and support.

This segment represents Krystal Biotech's expansion into new therapeutic areas, including oncology and aesthetic medicine. Research and development efforts are focused on developing gene therapies for cancer and aesthetic skin conditions. This involves preclinical studies, clinical trials, and exploring new applications of the company's gene therapy platform. The company is developing KB301 for wrinkles and other presentations of aged or damaged skin. The segment leverages the company's expertise in gene delivery and manufacturing to create innovative treatments. Patient impact will be significant, offering new options for cancer patients and those seeking aesthetic improvements. Market positioning is based on the potential to address unmet needs in oncology and the growing demand for aesthetic procedures. Future opportunities include expanding the pipeline to treat other types of cancer and aesthetic conditions. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials. This segment is expected to drive future growth and diversification for Krystal Biotech.