Krystal Biotech

Redosable gene therapy, epidermolysis bullosa, topical

USpublicest. 2015krystalbio.com (opens krystalbio.com)Checked 11 Oct

The brief

Analyst view
  • Krystal raised $39.6M in September 2017 IPO; $160M total funding.
  • Vyjuvek: FDA approved May 2023; Q2 2026 revenue $119.2M with 95% gross margin.
  • Pipeline: KB407 (CF) in Phase 1 with 2027 registrational study planned; programs in ophthalmology, dermatology, oncology.

Technical approach

As reported
Topical delivery

Applied directly to wounds

High local concentration without systemic exposure; enables redosability.

Redosable design

Repeat applications

Can be reapplied as wounds recur, solving one-time-only limitation.

COL7A1 restoration

Replace missing collagen VII

Delivers functional gene so cells produce collagen VII anchoring skin layers.

Primer