What Krystal Biotech does

Krystal Biotech is a commercial-stage biotechnology company that develops and commercializes redosable gene therapies for rare and severe genetic diseases. Its lead product, VYJUVEK (beremagene geperpavec-svdt), is the first and only FDA-approved redosable gene therapy and is indicated for dystrophic epidermolysis bullosa (DEB), a devastating skin-fragility disorder caused by COL7A1 mutations. The company was co-founded in 2016 by Krish S. Krishnan (Chairman & CEO) and Suma Krishnan (President of R&D) and is headquartered in Pittsburgh, PA, with operations listed across the US, Germany, France, Japan, Switzerland, and the UK.

The company's technology is built on an engineered replication-deficient HSV-1 viral vector platform that delivers genetic payloads episomally without integration risk. The platform supports payloads exceeding 30kb (VYJUVEK alone carries over 19kb), exhibits broad cell tropism across keratinocytes, fibroblasts, and lung/eye tissue, and evades host immunity to enable repeat dosing—a property most gene therapy vectors lack. Two in-house GMP manufacturing facilities (ASTRA in Sunnyvale and Ancoris in Pittsburgh) provide full vertical integration from virus banks to commercial packaging, supporting sustained gross margins of 94-95%. The platform underpins a pipeline spanning inhaled gene therapies for cystic fibrosis (KB407) and Alpha-1 Antitrypsin Deficiency (KB408), oncology candidates (KB707 in NSCLC and solid tumors), ophthalmology assets (KB801, KB803), and dermatology indications (KB111 for Hailey-Hailey disease), plus a wholly-owned aesthetics subsidiary (Jeune Aesthetics).

Krystal operates a direct commercialization model with a US field sales force targeting dermatologists and healthcare providers, supported by the Krystal Connect patient support program. As of Q1 2026, 570+ unique prescribers had prescribed VYJUVEK and 695+ reimbursement approvals had been secured in the US. Internationally, Krystal has launched through subsidiary operations in Germany, France, Japan, and the UK, and is scaling a global distributor network from 20 to over 40 countries by year-end 2026. Full-year 2025 revenue reached $389.1M (+34% YoY) with $204.8M in net income, and Q1 2026 revenue was $116.4M (+32% YoY), reflecting 11 consecutive quarters of positive EPS and a cash and investments balance of $955.9M.

Name

Krystal Biotech

Legal name

Krystal Biotech, Inc.

Website

https://krystalbio.com

Company type

Public

Founded year

2016

Operating status

Operating

Headcount range

251–500 employees

Short description

Krystal Biotech is a commercial-stage biotechnology company that develops redosable gene therapies using a proprietary HSV-1 vector platform, with its lead product VYJUVEK approved for dystrophic epidermolysis bullosa and a broader pipeline across respiratory, oncology, and ophthalmology indications.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical and Medicine Manufacturing (3254), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Redosable gene therapy
• HSV-1 vector platform
• Rare disease treatment
• Topical gene therapy
• Biopharmaceutical products

Headquarters

HQ city

Pittsburgh

HQ country

United States

HQ region

North America

Offices7 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Marketing or Sales, Operations, Infrastructure, Supply Chain

Revenue model

1. VYJUVEK Product Sales: Krystal Biotech generates product revenue from sales of VYJUVEK (beremagene geperpavec-svdt), an FDA-approved gene therapy for dystrophic epidermolysis bullosa. The therapy is administered as a topical treatment. Revenue has shown strong growth with 34% year-over-year increase to $389.1 million in 2025, and Q1 2026 revenue of $116.4 million representing 32% year-over-year growth. The company operates on a traditional pharmaceutical commercial model with direct sales force and reimbursement approvals.

Pricing tiers

Go-to-market motion1 record

Distribution channels3 records

Marketing channels6 records

Core offering

Krystal Biotech develops, manufactures, and commercializes redosable genetic medicines using its proprietary engineered replication-deficient HSV-1 vector platform. The lead commercial product is VYJUVEK (beremagene geperpavec-svdt), the first and only FDA-approved redosable gene therapy for dystrophic epidermolysis bullosa (DEB), applied topically to deliver the COL7A1 gene to skin cells. The company also maintains a pipeline of gene therapy candidates for cystic fibrosis, Alpha-1 Antitrypsin Deficiency, lung cancer, neurotrophic keratitis, and other rare diseases, plus an aesthetics subsidiary (Jeune Aesthetics).

Product overview

Krystal Biotech is a fully integrated, commercial-stage biotechnology company developing redosable genetic medicines using its proprietary HSV-1 gene delivery platform. The company's lead product is VYJUVEK (beremagene geperpavec-svdt), the first and only FDA-approved redosable gene therapy for dystrophic epidermolysis bullosa (DEB). VYJUVEK is approved in the US, Europe, and Japan, with additional approvals in the UK. The platform supports a broad pipeline of candidates across dermatology, respiratory (cystic fibrosis, Alpha-1 Antitrypsin Deficiency), oncology (KB707 for NSCLC and solid tumors), ophthalmology (KB801 for neurotrophic keratitis, KB803 for corneal abrasions), and aesthetics (Jeune Aesthetics subsidiary). The technology enables focal gene delivery with repeat dosing capabilities through engineered HSV-1 vectors that evade host immunity.

Differentiator

Problem solved

Functional benefit

Brands

• VYJUVEK: First and only redosable gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB). Contains beremagene geperpavec-svdt for topical application.
• Jeune Aesthetics

Products and services

• VYJUVEK (beremagene geperpavec-svdt) The first and only FDA-approved redosable gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder caused by mutations in the COL7A1 gene. Contains over 19 kb of genetic cargo and is applied topically to wounds to deliver functional copies of the COL7A1 gene to skin cells.

Quantifiable outcome

• 34% year-over-year revenue growth in 2025 to $389.1 million
• +4 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Scale indicators9 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• uniQure: Gene therapy company developing AAV-based treatments for rare diseases including Huntington's disease and hemophilia. Comparable platform-based rare disease gene therapy model.
• Abeona Therapeutics: Clinical-stage gene therapy company developing treatments for dystrophic and recessive epidermolysis bullosa, directly overlapping with VYJUVEK's DEB indication. Competes in the same rare dermatology gene therapy space.
• Rocket Pharmaceuticals: Late-stage gene therapy company focused on rare monogenic diseases using lentiviral and AAV platforms. Highly comparable business model of platform-based rare disease gene therapy commercialization.
• Sarepta Therapeutics: Commercial-stage gene therapy company with multiple approved rare disease genetic medicines (DMD). Comparable in commercial-stage rare disease gene therapy execution, though focused on AAV-based delivery.
• CRISPR Therapeutics: Pioneer in CRISPR/Cas9 gene-editing therapies with approved products for sickle cell disease and beta thalassemia. Comparable as a genetic medicine platform company targeting rare monogenic diseases, though using a different modality.
• bluebird bio: Gene therapy company developing lentiviral-based treatments for rare genetic diseases. Comparable as a platform-based rare disease gene therapy developer with approved products.

Broad incumbents

• Vertex Pharmaceuticals: Major rare disease pharmaceutical company with dominant position in cystic fibrosis (CFTR modulators). Directly overlaps with Krystal's KB407 cystic fibrosis gene therapy program and competes for the same patient population.
• Regeneron Pharmaceuticals: Large biotechnology company with established gene therapy capabilities and rare disease programs through Regeneron Genetic Medicines. Comparable as a broader rare disease player with gene therapy investment, though operating at much larger scale.
• BioMarin Pharmaceutical: Established rare disease biotechnology company with multiple approved genetic medicines. Comparable as a commercial-stage rare disease therapeutics company, though with a broader portfolio including enzyme replacement therapies.

Emerging players

• Ultragenyx Pharmaceutical: Commercial-stage rare disease company developing genetic medicines including gene therapies. Overlaps in rare disease focus and gene therapy pipeline development.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/KrystalBiotech
• LinkedInlinkedin.com/company/krystal-biotech-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Subsidiaries1 record

Funding overview

Funding rounds6 records

Investors6 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Krystal Biotech