Krystal Biotech Stock Price, News & Analysis
Company Description
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines for diseases with high unmet medical needs. According to the company’s public disclosures, Krystal Biotech is headquartered in Pittsburgh, Pennsylvania and operates within the biological product manufacturing and broader healthcare sectors.
The company’s first commercial product, VYJUVEK (beremagene geperpavec-svdt), is described as the first-ever redosable gene therapy and the first genetic medicine approved in the United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa (DEB). VYJUVEK is a non-invasive, topical genetic medicine designed to deliver two copies of the COL7A1 gene directly to DEB wounds. It is intended to treat DEB at the molecular level by providing skin cells with a template to make normal COL7 protein, addressing the underlying disease mechanism.
Krystal Biotech reports that VYJUVEK is approved as a herpes simplex virus type 1 (HSV‑1) vector-based gene therapy for the treatment of wounds in adult and pediatric patients with DEB who have mutations in the COL7A1 gene. A label update from the U.S. Food and Drug Administration (FDA) expanded the eligible patient population to include DEB patients from birth and allows patients and caregivers to apply VYJUVEK themselves, with greater flexibility in managing wound dressings. These changes are based on real-world data and open-label extension study results that support long-term safety and efficacy across age groups, including patient or caregiver application.
Business model and rare disease focus
Public information indicates that Krystal Biotech is a commercial-stage company with one operating segment focused on developing and commercializing pharmaceutical products. The company emphasizes rare diseases, particularly those where genetic medicines and redosable gene therapies may address significant unmet needs. Its rare disease strategy includes DEB and other indications where few or no disease-modifying therapies are available.
Krystal Biotech has articulated a rare disease strategic vision to build a global rare disease business based on its gene therapy platform. The company highlights that hundreds of millions of people worldwide are living with rare diseases, many lacking adequate therapies, and positions its genetic medicine programs as potential options for these patient populations.
Genetic medicine platform and HSV‑1 vector technology
Krystal Biotech’s programs are built on a genetically modified, non-replicating HSV‑1 viral vector platform. The company states that this platform is designed for redosable gene delivery and has been used in both topical and inhaled formulations. The HSV‑1 vector used in certain programs, including KB801, has received FDA platform technology designation, which the FDA’s program describes as intended to provide potential efficiencies in drug development, manufacturing, and regulatory review for products that use designated platform technologies.
The company notes that its HSV‑1 platform has been granted platform technology designation based on factors such as being well-understood and reproducible, being used in an FDA-approved product (VYJUVEK), and having the potential to support multiple drugs or biologic products without compromising quality, manufacturing, or safety. Krystal Biotech also reports that the platform is well suited for gene delivery to high turnover tissues such as the skin, lung, and eye.
Commercialization of VYJUVEK
Krystal Biotech’s disclosures describe VYJUVEK as its first commercial product and the foundation of its rare disease business. VYJUVEK is approved in the United States, Europe, and Japan for DEB. The company has launched VYJUVEK in multiple markets, including the United States, Germany, France, and Japan, and is pursuing additional regulatory filings and pricing discussions in other European countries and regions.
In the United States, the updated label allows treatment of DEB patients from birth and permits application by healthcare providers, caregivers, or patients. The label also allows wound dressings to be removed at the next dressing change rather than requiring a fixed waiting period, which the company describes as integrating VYJUVEK into existing wound care routines.
Pipeline: respiratory, ophthalmology, dermatology, oncology, and aesthetics
Beyond VYJUVEK, Krystal Biotech reports a broad preclinical and clinical pipeline of investigational genetic medicines across several therapeutic areas:
- Respiratory: KB407 for cystic fibrosis (CF) and KB408 for alpha‑1 antitrypsin deficiency (AATD) lung disease.
- Ophthalmology: KB803 for ocular complications of DEB and KB801 for neurotrophic keratitis (NK).
- Dermatology: KB111 for Hailey‑Hailey disease (HHD).
- Oncology: KB707 in inhaled and intratumoral formulations for solid tumors, including non‑small cell lung cancer (NSCLC).
- Aesthetics: KB304 for wrinkles of the décolleté, developed by Jeune Aesthetics, Inc., a wholly owned subsidiary.
Selected clinical-stage programs
KB407 for cystic fibrosis: KB407 is described as a redosable gene therapy designed to deliver two copies of the full-length CFTR transgene to the lung via inhalation for the treatment of CF. The company reports that KB407 aims to enable expression of full-length, wild-type CFTR protein in the lung to restore CFTR-mediated ion transport, mucus clearance, and lung function regardless of underlying genetic mutation. Interim data from the CORAL‑1 Phase 1 study showed molecular confirmation of wild-type CFTR delivery and expression in conducting airway cells of patients with CF, including patients ineligible for modulator therapy.
KB408 for alpha‑1 antitrypsin deficiency lung disease: KB408 is being evaluated in the SERPENTINE‑1 open-label dose escalation study in adult patients with AATD with specific genotypes. The company has reported that prior data showed successful delivery of SERPINA1 to the lungs of AATD patients after a single dose, and ongoing cohorts are assessing repeat dosing.
KB803 for ocular complications of DEB: KB803 is under evaluation in IOLITE, an intra-patient, double-blind, multicenter, placebo-controlled Phase 3 study with a crossover design. The primary endpoint is the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo in DEB patients.
KB801 for neurotrophic keratitis: KB801 is a redosable eye drop gene therapy designed to enable sustained, localized expression and secretion of nerve growth factor (NGF) by epithelial cells in the front of the eye for NK. The EMERALD‑1 study is a 2:1 randomized, double-masked, multicenter, placebo-controlled Phase 1/2 trial evaluating safety, tolerability, and efficacy, including the proportion of patients with complete durable healing of the corneal epithelium.
KB111 for Hailey‑Hailey disease: KB111 uses a replication-defective, non-integrating HSV‑1-based vector to deliver two copies of the full-length wild-type ATP2C1 gene following topical application. It is designed to increase functional ATP2C1 levels in the skin to accelerate lesion healing and reduce disease burden in HHD, a rare genetic skin disease characterized by painful rash and blistering in skin folds and significant quality-of-life impact.
KB707 in oncology: Krystal Biotech is evaluating inhaled KB707 for solid tumors of the lung, including NSCLC, in the KYANITE‑1 Phase 1/2 study. Based on interactions with the FDA, the company reports that a single Phase 3 registrational study of inhaled KB707 in combination with chemotherapy versus chemotherapy alone in advanced NSCLC could potentially support registration. An intratumoral formulation of KB707 for injectable solid tumors is also being studied in the OPAL‑1 Phase 1/2 trial, although enrollment in OPAL‑1 has been paused while patients continue to be followed.
KB304 in aesthetics: Jeune Aesthetics, Inc., a wholly owned subsidiary, is advancing KB304 for the treatment of wrinkles of the décolleté. Positive safety and efficacy results from a Phase 1 study have been reported, and Jeune is preparing a Phase 2 study based on feedback from the FDA.
Rare disease strategy and global infrastructure
Krystal Biotech has outlined a strategy to build a global rare disease infrastructure for drug development and launch. The company reports that it has launched VYJUVEK in the United States, major European markets such as Germany and France, and Japan, and is building a specialty distributor network covering markets in regions including the Middle East, Turkey, and Central and Eastern Europe. The company has also stated corporate objectives related to expanding launches into additional European markets and growing its specialty distributor network.
According to recent business updates, Krystal Biotech’s strategic vision includes having multiple marketed rare disease medicines, inclusive of VYJUVEK, and treating thousands of patients worldwide. The company also indicates that it intends to invest across its preclinical and clinical pipeline, including programs targeting larger indications such as alpha‑1 antitrypsin deficiency and non‑small cell lung cancer.
Regulatory interactions and designations
In addition to the FDA’s platform technology designation for its HSV‑1 vector used in KB801, Krystal Biotech reports multiple regulatory milestones for VYJUVEK, including approval in the United States, Europe, and Japan, and a U.S. label update expanding eligibility and application flexibility. In France, VYJUVEK has received early reimbursed access under the Accès Précoce AP2 program and an Amélioration du Service Médical Rendu (ASMR) III appraisal, which recognizes added clinical benefit and is cited by the company as an important step in access discussions.
Corporate structure and listings
Krystal Biotech, Inc. is incorporated in Delaware and lists its common stock on the NASDAQ under the ticker symbol KRYS. The company’s SEC filings, including Form 8‑K reports, document material events such as financial results, regulatory approvals, clinical updates, and commercialization milestones. The company also reports activities of its wholly owned subsidiaries, including Krystal Biotech Japan KK and Jeune Aesthetics, Inc.
Risk considerations
As a biotechnology company focused on genetic medicines, Krystal Biotech’s business is subject to typical sector risks, including clinical development outcomes, regulatory decisions, manufacturing considerations, commercialization performance, and reimbursement dynamics. The company’s public statements and SEC filings provide detailed risk factor discussions and forward-looking statements that investors may review for a fuller understanding of these considerations.
FAQs about Krystal Biotech, Inc. (KRYS)
- What does Krystal Biotech, Inc. do?
Krystal Biotech, Inc. is a commercial-stage biotechnology company that focuses on discovering, developing, and commercializing genetic medicines for diseases with high unmet medical needs. Its first approved product, VYJUVEK, is a redosable gene therapy for dystrophic epidermolysis bullosa, and the company is advancing a pipeline of investigational programs in respiratory, ophthalmology, dermatology, oncology, and aesthetics. - What is VYJUVEK and who is it for?
VYJUVEK (beremagene geperpavec-svdt) is a non-invasive, topical, redosable genetic medicine designed to deliver two copies of the COL7A1 gene directly to DEB wounds. It is approved in the United States, Europe, and Japan for the treatment of wounds in adult and pediatric patients with dystrophic epidermolysis bullosa who have mutations in the COL7A1 gene, and the U.S. label allows treatment from birth. - How is VYJUVEK administered?
According to the company’s description and U.S. label update, VYJUVEK gel may be applied by a healthcare provider, a caregiver, or the patient. The label allows flexibility in wound dressing management, including removal of dressings at the next dressing change, and supports integration into existing wound care routines. - What is Krystal Biotech’s gene therapy platform?
Krystal Biotech uses a genetically modified, non-replicating HSV‑1 viral vector platform designed for redosable gene delivery. This platform underlies VYJUVEK and multiple pipeline candidates. The HSV‑1 vector used in KB801 has received FDA platform technology designation, which is intended to provide potential efficiencies in drug development and regulatory review for products that use the designated technology. - Which therapeutic areas does Krystal Biotech’s pipeline cover?
The company’s pipeline spans several areas: respiratory (KB407 for cystic fibrosis and KB408 for alpha‑1 antitrypsin deficiency lung disease), ophthalmology (KB803 for ocular complications of DEB and KB801 for neurotrophic keratitis), dermatology (KB111 for Hailey‑Hailey disease), oncology (KB707 for solid tumors, including non‑small cell lung cancer), and aesthetics (KB304 for wrinkles of the décolleté through Jeune Aesthetics, Inc.). - What is KB407 for cystic fibrosis?
KB407 is a redosable gene therapy designed to deliver two copies of the full-length CFTR transgene to the lung via inhalation for the treatment of cystic fibrosis. Interim data from the CORAL‑1 Phase 1 study have shown molecular confirmation of wild-type CFTR delivery and expression in conducting airway cells of patients with CF, including those ineligible for modulator therapy. - What is KB801 for neurotrophic keratitis?
KB801 is a redosable eye drop gene therapy intended to enable sustained, localized expression and secretion of nerve growth factor by epithelial cells in the front of the eye for the treatment of neurotrophic keratitis. It is being evaluated in the EMERALD‑1 randomized, double-masked, placebo-controlled Phase 1/2 study, which assesses safety, tolerability, and efficacy based on corneal epithelial healing. - How is Krystal Biotech expanding internationally?
Krystal Biotech has launched VYJUVEK in the United States, Germany, France, and Japan and is pursuing additional regulatory filings and pricing negotiations in other European markets. The company is also building a specialty distributor network that covers markets in regions such as the Middle East, Turkey, and Central and Eastern Europe to support VYJUVEK commercialization. - What is Jeune Aesthetics, Inc.?
Jeune Aesthetics, Inc. is a wholly owned subsidiary of Krystal Biotech focused on aesthetics. Jeune is developing KB304 for the treatment of wrinkles of the décolleté and has reported positive safety and efficacy results from a Phase 1 study, with plans for further clinical development. - On which exchange does Krystal Biotech trade and what is its ticker symbol?
Krystal Biotech, Inc. trades on the NASDAQ under the ticker symbol KRYS. The company files reports with the U.S. Securities and Exchange Commission, including Form 8‑K filings that disclose material events such as financial results, regulatory approvals, and clinical updates.