Here are 1-3 brief analogies for Krystal Biotech (KRYS):

• A bluebird bio for rare genetic skin diseases.

• Spark Therapeutics for rare dermatological conditions.

• A BioMarin focused on gene therapies for severe rare diseases.

• VYJUVEK (beremagene geperpavec): An FDA-approved topical gene therapy used for the treatment of dystrophic epidermolysis bullosa (DEB), a rare genetic skin blistering disorder.
• KB407: An investigational inhaled gene therapy currently in clinical trials for the treatment of cystic fibrosis (CF), a genetic disorder affecting the lungs and other organs.
• KB301: An investigational gene therapy in preclinical development targeting alpha-1 antitrypsin deficiency (AATD), a genetic condition that can lead to lung and liver disease.

Krish S. Krishnan, Chairman & Chief Executive Officer

Krish S. Krishnan is an accomplished biotech executive who co-founded Krystal Biotech in 2016 and initially self-funded the company with his wife, Suma Krishnan. He served as Chief Operating Officer and Chief Financial Officer of New River Pharmaceuticals, Inc., which was sold to Shire Pharmaceuticals, plc for $2.6 billion. He was also the Chief Operating Officer of Intrexon Corporation, Inc., playing a key role in taking it public. Krishnan previously held the role of Chief Executive Officer at Pinnacle Pharmaceuticals, Inc. His career has been closely linked to billionaire investor Randal J. Kirk, and he served as Senior Managing Director of Third Security, LLC, an investment firm. He has been involved in three IPOs, including Krystal Biotech's.

Nishant Saxena, Chief Financial Officer

Nishant Saxena was appointed Chief Financial Officer of Jeune Aesthetics, a wholly-owned subsidiary of Krystal Biotech, effective January 15, 2025. He brings over 20 years of experience in finance, strategy, capital markets, mergers and acquisitions, and corporate development. Prior to joining Jeune, Saxena was a Managing Director in Evercore's healthcare group for over 15 years, where he advised on transactions totaling more than $500 billion. Earlier in his career, he held positions of increasing responsibility in private equity, venture capital, and investment advisory firms.

Suma M. Krishnan, President, Research & Development

Suma M. Krishnan co-founded Krystal Biotech with her husband, Krish S. Krishnan, in 2016, and initially self-funded the company. A seasoned drug developer with over two decades of experience, she holds more than 70 patents for various drugs. Krishnan previously served as Senior Vice President at New River Pharmaceuticals Inc., where she led the discovery, development, and approval of the blockbuster drug Vyvanse. She also held senior leadership roles at Intrexon Corporation, Inc., including Senior Vice President of Human Health and Therapeutic Division, and Senior Vice President of Regulatory Affairs. Her career also includes senior positions at Shire, Pfizer, and Janssen Pharmaceuticals.

Kathryn Romano, Executive Vice President & Chief Accounting Officer

Kathryn Romano serves as the Executive Vice President and Chief Accounting Officer at Krystal Biotech, a position she has held since 2020. Prior to joining Krystal Biotech, she was the Corporate Controller at CNX Resources Corporation. She also served as Corporate Controller for Rice Energy from January 2013 until its acquisition in November 2017. Her earlier career involved various accounting and auditing roles at Black Box Corporation and Deloitte.

Laurent Goux, Senior Vice President & General Manager Europe

Laurent Goux has been the Senior Vice President & General Manager Europe at Krystal Biotech since September 2021. Before joining Krystal, he held multiple roles at Galderma from 2002 to 2013 and again from 2016 to 2021, most recently as Head of Global Strategic Marketing and Market Access.

1. Dependence on VYJUVEK Revenue: Krystal Biotech's revenue is substantially dependent on the commercial success of VYJUVEK, its FDA-approved gene therapy for dystrophic epidermolysis bullosa. This singular product reliance exposes the company to significant risks if unforeseen challenges arise in its production, regulation, market competition, or unpredictable patient treatment patterns. Any issues with VYJUVEK's performance could directly and severely impact the company's financial stability and growth prospects.
2. Underinvestment in Research and Development (R&D): There are concerns that Krystal Biotech's current profitability and cash flow may be inflated due to a comparatively low investment in research and development. This "R&D austerity" could lead to a neglected pipeline, making it challenging for the company to diversify its product portfolio and sustain long-term growth once VYJUVEK's revenue growth plateaus. Developing new gene therapies for larger indications, such as alpha-1 antitrypsin deficiency, cystic fibrosis, or solid tumors, would require significantly higher R&D investments than the ultra-rare disease VYJUVEK targets.
3. Regulatory and Market Competition Risks: As a biotechnology company, Krystal Biotech is subject to stringent regulatory requirements. Changes in policy or delays in approvals for current or future products could adversely affect its operations. Furthermore, the competitive landscape in the biotech industry is constantly evolving, with new entrants and technologies posing threats to the company's market position. These general industry risks are magnified by the company's dependence on VYJUVEK and its relatively limited pipeline, as competition or regulatory setbacks for its primary product or development candidates could have a disproportionate impact.

The clear emerging threat for Krystal Biotech is the potential market entry of a competing gene-corrected cell therapy for dystrophic epidermolysis bullosa (DEB).

• Abeona Therapeutics has submitted a Biologics License Application (BLA) to the FDA for its investigational autologous gene-corrected cell therapy, pz-cel (formerly EB-101), for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe form of DEB, which is the primary indication for Krystal Biotech's approved product, Vyjuvek.
• While Vyjuvek is an in vivo topical gene therapy, pz-cel represents an alternative gene-based treatment modality for the underlying genetic defect in RDEB. Abeona's BLA submission, occurring in November 2023, signifies that pz-cel is in advanced regulatory review and could receive approval and launch within 2024, directly competing with Vyjuvek for a significant portion of the DEB patient population.

Krystal Biotech (KRYS) is expected to drive future revenue growth over the next 2-3 years through several key initiatives:

1. Continued Growth and Market Penetration of VYJUVEK in the U.S.: VYJUVEK, Krystal Biotech's FDA-approved gene therapy for Dystrophic Epidermolysis Bullosa (DEB), is a primary revenue generator. The company anticipates sustained growth in U.S. sales as it continues to secure reimbursement approvals and expand patient access. Projections for full-year 2024 anticipate U.S. net product revenues for VYJUVEK to be between $205 million and $220 million, with management reiterating a target of over $1 billion in peak annual sales.

2. International Expansion of VYJUVEK: Krystal Biotech is actively pursuing the international commercialization of VYJUVEK, particularly in Europe and Japan, following successful regulatory approvals. VYJUVEK has already launched in Germany, France, and Japan, with early indications of demand and strong pricing. This geographical expansion is a crucial part of the company's growth strategy to broaden its market reach and capitalize on unmet medical needs in rare diseases globally.

3. Advancement of the Oncology Pipeline, Specifically Inhaled KB707: Krystal Biotech has expanded its R&D pipeline into oncology, with its lead candidate KB707 targeting solid tumor malignancies. The company has prioritized the inhaled formulation of KB707 for non-small cell lung cancer (NSCLC) and expects an End of Phase 2 meeting with the FDA in October 2025 to discuss a potential Phase 3 registration study. This program, which has received Fast Track Designation, leverages the company's HSV-1 platform to deliver genes encoding IL-12 and IL-2 to the tumor microenvironment.

4. Progress in Other Clinical Pipeline Programs: Krystal Biotech is advancing a robust pipeline of investigational genetic medicines in various therapeutic areas. Key programs expected to contribute to future growth include:

   • KB407 for Cystic Fibrosis (CF): Interim data from clinical trials for this inhaled gene therapy are anticipated in Q4 2025.
   • Ophthalmic B-VEC for Ocular Complications of DEB: A Phase 3 study (IOLITE) is underway, with top-line data expected before the end of 2025 and an anticipated launch.
   • KB408 for Alpha-1 Antitrypsin Deficiency (AATD): Clinical trial data for this inhaled gene therapy are also expected in the first half of 2026.
   • Jeune Aesthetics Products: The company's wholly-owned subsidiary, Jeune Aesthetics, is developing products using the same platform for aesthetic skin conditions, showing promise with positive Phase 1 data.

5. Leveraging its Proprietary HSV-1 Gene Delivery Platform: The FDA's platform technology designation for Krystal's HSV-1 viral vector validates its broader utility and potential. This proprietary redosable gene therapy platform allows Krystal Biotech to discover, develop, and commercialize genetic medicines across multiple therapeutic areas, offering a differentiated approach for various rare genetic disorders and potentially unlocking significant new market opportunities beyond its current indications.

Share Issuance

• Krystal Biotech's additional paid-in capital consistently increased, from $0.804 billion in 2021 to $1.127 billion in 2024, and further to $1.146 billion by June 30, 2025.
• The number of common shares outstanding grew from approximately 22.2 million as of July 30, 2021, to 28.29 million as of February 19, 2024, and reached 28.98 million as of September 30, 2025.

Inbound Investments

• In 2023, Krystal Biotech recorded a gain of $100.0 million from the sale of a rare pediatric disease Priority Review Voucher, which was received following the FDA's approval of VYJUVEK.

Capital Expenditures

• Krystal Biotech incurred significant capital expenditures for the construction of its second CGMP facility, ASTRA, acquired in March 2021. The facility began research and development operations in 2023, and the company anticipates continued capital expenditures for ASTRA throughout its operational life.
• Annual capital expenditures were reported as $68.3 million in 2021, $53 million in 2022, $11.8 million in 2023, and $4.24 million in 2024.