Krystal Biotech (KRYS) — Stock, Pipeline & Market Cap

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KRYS · Stock Price

USD 296.70+164.52 (+124.47%)

Market Cap: $8.8B

Historical price data

Market Cap: $8.8BPipeline: 14 drugs (3 Phase 3)Patents: 16Founded: 2016HQ: Pittsburgh, United States

Sector Ranking#14 in Cell & Gene Therapy #20 in Rare & Orphan Diseases

Overview

Krystal Biotech's mission is to develop and deliver redosable gene therapies for severe, life-threatening rare diseases. Its landmark achievement is the 2023 FDA approval of VYJUVEK®, the first and only redosable gene therapy, which validates its proprietary HSV-1 vector platform. The company's strategy is to commercialize VYJUVEK globally while rapidly advancing a deep internal pipeline across multiple therapeutic areas, supported by in-house GMP manufacturing capabilities. This integrated approach positions Krystal as a leader in next-generation, repeat-administration genetic medicines.

DermatologyPulmonologyRare Genetic Diseases

Technology Platform

Proprietary STAR-D platform using an engineered, replication-defective, non-integrating herpes simplex virus type 1 (HSV-1) vector, designed to enable safe, repeat administration of therapeutic genes.

Pipeline

14

14 drugs in pipeline3 in Phase 3

Drug	Indication	Stage
KB803 + Placebo	Dystrophic Epidermolysis Bullosa	Phase 3
Topical Beremagene Geperpavec	Dystrophic Epidermolysis Bullosa	Phase 3
Open Label Topical Beremagene Geperpavec (B-VEC)	Dystrophic Epidermolysis Bullosa	Phase 3
KB105	Autosomal Recessive Ichthyosis	Phase 2
Topical beremagene geperpavec + Placebo gel	Dystrophic Epidermolysis Bullosa	Phase 1/2

Funding History

4

Total raised:$297M

PIPE$200MUndisclosedJun 15, 2021

IPO$75MUndisclosedJul 25, 2019

Series B$17MRedmile GroupJun 15, 2017

Series A$5MJAFCOJun 15, 2016

Opportunities

The primary opportunity is the global commercialization of VYJUVEK® in DEB, a first-in-class therapy with no competition, followed by expansion into larger markets like cystic fibrosis and aesthetic medicine.

The redosable HSV-1 platform itself represents a significant opportunity to develop multiple high-value genetic medicines for localized disorders.

Risk Factors

Key risks include commercial execution in a rare disease market, clinical failure of pipeline candidates (notably in cystic fibrosis), potential long-term safety findings with the HSV-1 vector, and future competition from next-generation gene delivery technologies.

Competitive Landscape

Krystal's main competition in DEB is palliative care, with no approved gene therapy alternatives. In the broader gene therapy platform space, it competes with dominant AAV vectors, differentiating on redosability and payload size. In cystic fibrosis, it will compete with Vertex's modulators and other gene therapy approaches for the residual patient population.