Krystal Biotech
Krystal Biotech develops genetic medicines for severe, life-threatening, or rare diseases and has introduced VYJUVEK®, the first redosable gene therapy for dystrophic epidermolysis bullosa.
Company Overview
Krystal Biotech develops genetic medicines for severe, life-threatening, or rare diseases with limited or no approved therapies. The company focuses on creating treatments for debilitating genetic conditions, employing innovative technologies such as a modified HSV‑1 vector. This approach involves a replication‑defective and non‑integrating vector to deliver therapeutic genes.
Key Products
The company's first commercial product, VYJUVEK®, is the first and only redosable gene therapy for dystrophic epidermolysis bullosa. In addition, Krystal Biotech is developing other gene therapies across multiple therapeutic areas including dermatology, respiratory, oncology, ophthalmology, and aesthetics.
Manufacturing Facilities
Krystal Biotech operates two GMP manufacturing facilities to support its gene therapy production. The ASTRA facility, operational since H1 2023, spans approximately 155,000 sq. ft. The Ancoris facility, covering around 21,100 sq. ft., is tailored for the global launch of VYJUVEK. These facilities ensure the capacity and quality necessary for commercial-scale manufacturing.
Subsidiaries and Specialized Programs
Jeune Aesthetics, a wholly-owned subsidiary of Krystal Biotech, focuses on treating aesthetic skin conditions through gene therapy. Krystal Biotech also supports patients through its personalized program, Krystal Connect, which offers resources and assistance to individuals receiving its therapies.
Research and Development Pipeline
Krystal Biotech is actively developing a diverse pipeline of gene therapies targeting various conditions. This includes treatments for Autosomal Recessive Congenital Ichthyosis (ARCI), Netherton Syndrome, respiratory conditions like Cystic Fibrosis and Alpha-1 Antitrypsin Deficiency, and solid tumors. The company is also working on therapies for ocular complications and developing aesthetic products aimed at boosting collagen and elastin production in aged skin.