Krystal Biotech is a commercial-stage company focused on developing innovative genetic medicines to address severe and rare diseases with significant unmet medical needs. Their flagship product, VYJUVEK®, is the first and only redosable gene therapy specifically designed for the treatment of dystrophic epidermolysis bullosa (DEB). The company employs a proprietary platform based on a modified, replication-defective HSV-1 vector, which allows for the delivery of genetic material in a way that minimizes cytotoxicity and evades the immune system. This platform is characterized by its large genetic payload capacity, efficient transduction of various cell types, and the ability to provide durable expression of therapeutic genes without the risk of integration into the host genome.

Krystal's pipeline includes a range of drug candidates targeting multiple therapeutic areas, including dermatology, respiratory diseases, oncology, and aesthetics. The company is particularly focused on conditions such as cystic fibrosis, alpha-1 antitrypsin deficiency, and various genetic skin disorders, for which there are currently no approved treatments. Additionally, through its subsidiary Jeune Aesthetics, Krystal is exploring gene therapies aimed at rejuvenating aged skin by enhancing the natural production of collagen and elastin. This comprehensive approach underscores Krystal's commitment to leveraging advanced genetic technologies to create transformative therapies for patients in need.

United States

Krystal Biotech, Inc. is using gene therapy to develop effective and novel treatments for skin diseases. Our goal is to make a meaningful difference in the lives of underserved patient populations with debilitating skin diseases. We work to accomplish that through scientific innovation, operational excellence and believe that “nature operates in the shortest way possible”. (Aristotle)

Cancer

Eye Diseases

Genetic Diseases

Lung Diseases

Metabolic Diseases

Skin Diseases

Krystal Biotech

Dystrophic epidermolysis bullosa

Epidermolysis bullosa

Skin disease/disorder

Beremagene geperpavec ("B-VEC", previously "KB103"), a replication-defective, non-integrating HSV-1 that is based on the company's viral gene therapy platform, is Krystal's lead product candidate currently being evaluated in humans and is the subject of a planned phase III clinical trial. B-VEC has been engineered to deliver wild-type human type VII collagen genes (and thus, functional COL7 protein) directly to a DEB patient's dividing and non-dividing skin cells, providing molecular correction of the underlying genetic deficiency to stabilize the patient's otherwise extremely fragile skin (<a href="https://www.krystalbio.com/focus/our-focus/">Learn more HERE</a>).&nbsp;

VYJUVEKTM (B-VEC) (Topical Beremagene Geperpavec)

KB803 (Ophthalmic B-VEC)

Wrinkles

KB301 is designed to deliver the gene for full length type III collagen (COL3). It is injected directly into the area of interest, with the goal of targeted collagen production by the body’s own cells.
Gene therapy

KB301 

Autosomal recessive congenital ichthyosis

Ichthyosis

KB105 is Krystal’s second product candidate, currently in clinical development, and seeks to use gene therapy to treat patients with TGM1-deficient ARCI. KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s technology platform to deliver functional human TGM1 gene directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.
Gene therapy

KB105 

Cystic fibrosis

KB407 is an investigational, redosable gene therapy designed to molecularly correct the underlying cause of CF by delivering two copies of the&nbsp;CFTR&nbsp;gene directly to the airways via nebulization. By enabling the expression of full-length, healthy CFTR protein in the lung, treatment with KB407 has the potential to restore proper anion transport within transduced cells to correct the lung manifestations of the disease. Because KB407 is engineered to enable expression of wild-type CFTR protein, treatment is agnostic to patients’ underlying CFTR mutations. The US Food and Drug Administration and the European Commission have granted orphan drug designation for KB407 to treat patients with CF.

KB407

Multiple tumors

Solid tumor/s

KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance. Two formulations of KB707 are in development, a solution formulation for transcutaneous injection and an inhaled (nebulized) formulation for lung delivery.

KB707 (IL-2 and IL-12)

Severe alpha-1 antitrypsin deficiency

KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.

Krystal Biotech

Company Details