Krystal Biotech develops genetic medicines using a replication‑defective, non‑integrating HSV‑1 vector that can be administered multiple times, enabling clinicians to adjust dosing for severe or rare genetic diseases. Its first commercial product, VYJUVEK®, is the only redosable gene therapy for dystrophic epidermolysis bullosa, and the platform is being expanded to a pipeline of other serious genetic disorders. Manufacturing is performed in two GMP‑certified facilities to ensure consistent, high‑quality production.
Funding
$160M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Patients with severe, life‑threatening or rare genetic diseases often have limited or no approved therapies, and existing gene therapies are typically administered as a single, irreversible dose, restricting the ability to adjust treatment over time.
Solution
Krystal Biotech develops genetic medicines that use a clinically proven, replication‑defective, non‑integrating HSV‑1 vector platform capable of being administered multiple times (redosable). This approach enables correction of the underlying genetic defect while allowing clinicians to adjust dosing based on disease progression or patient response. The company’s first commercial product, VYJUVEK®, delivers the first redosable gene therapy for dystrophic epidermolysis bullosa, and its broader pipeline targets other serious and rare genetic disorders. Manufacturing is performed in two GMP‑certified facilities to ensure consistent, high‑quality production of the viral vectors.
Target Audience
Primary customers are healthcare providers and specialists treating patients with severe, rare genetic disorders that lack effective therapies, as well as health systems seeking innovative, repeat‑dose gene‑therapy solutions.
Features
- Modified HSV‑1 viral vector that is replication‑defective and does not integrate into the host genome, enhancing safety
- Redosable dosing regimen that permits multiple administrations to maintain therapeutic effect
- GMP‑compliant manufacturing infrastructure with two dedicated facilities for scalable production
- Platform technology adaptable to a range of severe and rare genetic diseases
- Commercially launched product VYJUVEK® for dystrophic epidermolysis bullosa, demonstrating clinical feasibility