Krystal Biotech, Inc. (NASDAQ: KRYS)

Krystal Biotech, Inc. (KRYS) is a commercial-stage biotechnology company operating in the genetic medicine industry. With a focus on the discovery, development, and commercialization of genetic medicines, Krystal Biotech aims to treat diseases with high unmet medical needs. The company's technology platform is based on engineered herpes simplex virus type 1 (HSV-1), which is designed to deliver therapeutic transgenes to cells of interest in multiple organ systems.

Krystal Biotech's main business activities involve the development of gene therapies for rare and severe diseases. The company's primary products include VYJUVEK, ophthalmic B-VEC, KB407, KB104, and KB301. VYJUVEK, approved by the FDA in May 2023, is a redosable, off-the-shelf gene therapy for dystrophic epidermolysis bullosa (DEB). Ophthalmic B-VEC is a gene therapy candidate for ocular complications of DEB, with no approved therapies available at this time. KB407 is a gene therapy candidate for cystic fibrosis (CF), targeting patients ineligible or intolerant to modulator regimens. KB104 is a gene therapy candidate for Netherton Syndrome, while KB301 is a gene therapy candidate for aesthetic skin conditions.

The company generates revenue through the development and commercialization of its gene therapies, as well as through partnerships and collaborations with other biotechnology companies and research institutions. Krystal Biotech's customer base includes patients with rare and severe diseases, as well as pharmaceutical companies and research institutions that are interested in developing and commercializing gene therapies.

Krystal Biotech's competitive advantages include its proprietary HSV-1 vector platform, which offers several advantages, such as repeat administration, non-integrating nature, high payload capacity, direct delivery, stability, and reproducible and scalable manufacturing. These features allow the company to deliver therapeutic transgenes to cells of interest in multiple organ systems, enabling the treatment of a wide range of diseases.

In the DEB space, Krystal Biotech's position within the industry is strong, as VYJUVEK is the first and only approved gene therapy for DEB in the United States. The company's ophthalmic B-VEC is the only gene therapy candidate in development for ocular complications of DEB, further emphasizing its leadership in this area.