Krystal Biotech

KRYSPhase 3

Pittsburgh, United StatesFounded 2016krystalbio.com

Krystal Biotech is a NASDAQ-listed gene therapy company that has successfully transitioned from development to commercial stage with VYJUVEK, their FDA-approved treatment for dystrophic epidermolysis bullosa. Built on a clinically-proven HSV-1 vector platform that is replication-defective and non-integrating, the company focuses on redosable gene therapies for rare genetic diseases. With two GMP manufacturing facilities and global operations spanning the US, Europe, and Japan, Krystal is advancing an extensive pipeline targeting genetic conditions with high unmet medical needs.

Market Cap

$7.4B

Founded

2016

Focus

RNA & Gene Therapy

KRYS · Stock Price

USD 251.68+187.33 (+291.11%)

Historical price data

AI Company Overview

Technology Platform

Proprietary HSV-1 based gene delivery platform that is replication-defective, non-integrating, and redosable, enabling targeted focal gene delivery for treating genetic diseases.

Pipeline Snapshot

14 drugs in pipeline, 3 in Phase 3

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

Funding History

Total raised: $297M

PIPE$200MUndisclosedJun 15, 2021

IPO$75MUndisclosedJul 25, 2019

Series B$17MRedmile GroupJun 15, 2017

Series A$5MJAFCOJun 15, 2016

Opportunities

Significant expansion potential through pipeline development using validated HSV-1 platform, international market expansion for VYJUVEK, and potential partnerships in rare disease therapeutics.

The company's proven commercial execution and manufacturing capabilities position it well for scaling operations.

Risk Factors

Primary risks include dependence on single commercial product VYJUVEK, complex gene therapy manufacturing requirements, evolving regulatory landscape, and increasing competition from large pharmaceutical companies entering the gene therapy space.

Competitive Landscape

Krystal competes in the gene therapy space with a differentiated HSV-1 platform against companies using AAV or lentiviral approaches. The company's first-in-class redosable gene therapy VYJUVEK and proven commercial execution provide competitive advantages in the rare disease therapeutics market.

Publications

Patents

Pipeline