Krystal Biotech, Inc. (NASDAQ:KRYS) is one of the 10 best NASDAQ growth stocks to buy for the next 10 years. Andrea Tan, an analyst at Goldman Sachs, raised the firm’s price target on Krystal Biotech, Inc. (NASDAQ:KRYS) from $206 to $327 on January 30 while keeping a Buy rating. The firm’s revised price target implies an additional 16.7% upside from current levels. The company is positioning itself for short-term growth through three registrational programs. These programs include KB801, KB803, and KB407. ...

Market Overview - The AI trade has become overcrowded, with only Google and Nvidia outperforming the S&P 500 index in 2025, indicating a challenging environment for investors seeking alpha stocks in 2026 [1] - Goldman Sachs suggests that diversification may yield better risk-adjusted returns in 2026, recommending a focus on emerging markets and a mix of growth and value stocks across sectors [2] Investment Trends - Large companies, particularly AI "hyperscalers" like Meta, Amazon, Alphabet, Microsoft, and Oracle, invested over $400 billion in capital expenditures in 2025, significantly exceeding previous forecasts by nearly $150 billion [3] - These companies are expected to continue facing challenges due to the weight of their investments, which may create opportunities in other sectors [4] Stock Recommendations - SiTime Corporation (NASDAQ:SITM) has a potential upside of 5.96% with 35 hedge fund holders, and recent price target increases from UBS and Barclays suggest strong growth prospects driven by AI ventures [7][8] - Krystal Biotech, Inc. (NASDAQ:KRYS) shows a potential upside of 10.5% with 26 hedge fund holders, and multiple analysts have raised their price targets, indicating confidence in the company's growth through its registrational programs and existing product pipeline [11][13][15]

Core Insights - Krystal Biotech, Inc. (KRYS) anticipates preliminary unaudited net product revenues of $106-$107 million from its lead drug Vyjuvek in Q4 2025, raising the full-year 2025 revenue forecast to $388-$389 million, driven by strong uptake in the U.S. and successful launches in Europe and Japan [1][11] Financial Performance - At the end of 2025, Krystal reported approximately $955 million in cash, cash equivalents, and investments, providing substantial financial flexibility for clinical programs [2] - For 2026, the company expects combined non-GAAP R&D and SG&A expenses to be between $175-$195 million [2] Pipeline Development - Krystal is advancing several rare disease programs utilizing its redosable HSV-1 gene therapy platform, targeting high-turnover tissues such as skin, lung, and eye [3] - Key pipeline candidates include KB803 for ocular complications of DEB, KB801 for neurotrophic keratitis, KB407 for cystic fibrosis, and KB111 for Hailey-Hailey disease [3] Clinical Trials and Updates - Enrollment in the registrational KB801 study has increased to 60 patients, indicating potential for expedited development, with a detailed update expected in February 2026 [4] - Top-line data from the KB801 and KB803 registrational studies are anticipated in 2026, serving as important near-term catalysts [6] - Other targets for 2026 include initiating and completing enrollment in a registrational repeat dose study for KB407 and starting a double-blind, placebo-controlled study for KB111 [7] Vyjuvek Developments - Vyjuvek received FDA approval in May 2023 as the first revocable gene therapy for treating patients aged six months or older with dystrophic epidermolysis bullosa (DEB) [8] - In September 2025, a label update expanded the treatment-eligible population to include DEB patients from birth and allowed for greater dosing flexibility [9][12] Long-term Strategy - The company aims to have at least four marketed rare disease therapies by 2030, targeting over 10,000 patients globally while maintaining profitability [5] - Krystal continues to invest in earlier-stage programs for larger indications, including KB408 for alpha-1 antitrypsin deficiency and KB707 for non-small cell lung cancer, which could provide long-term growth potential [5] Stock Performance - Shares of KRYS increased by 5% on January 9, with a notable 73.9% rise over the past year compared to the industry growth of 19.6% [16]

This presentation and our discussion contain forward-looking statements that involve substantial risks and uncertainties. Any statements about future expectations, plans, and prospects for Krystal Biotech, Inc. (together with its subsidiaries, the "Company"), including but not limited to, statements about our global VYJUVEK launch; selected preliminary 2025 unaudited results; our 2030 vision and the market potential of the product candidates that could be launched over the next four years; the development a ...

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Core Insights - Krystal Biotech, Inc. reported preliminary unaudited net product revenue for VYJUVEK® of $106 million to $107 million for Q4 2025, and $388 million to $389 million for the full year 2025, indicating strong financial performance [3][4] - The company aims to establish itself as a global leader in rare diseases, leveraging its HSV-1 platform to develop differentiated genetic medicines [2][5] - A robust clinical pipeline is in place, with multiple potential blockbuster launches anticipated in the next four years, positioning the company for sustained growth [2][5] Financial Performance - Preliminary net product revenue for VYJUVEK is projected to be between $106 million and $107 million for Q4 2025, and between $388 million and $389 million for the full year 2025 [3] - As of December 31, 2025, the company had approximately $955 million in cash, cash equivalents, and investments, indicating a strong balance sheet [3] Strategic Vision - The company’s strategic vision includes having at least four marketed rare disease medicines by the end of 2030, aiming to treat over 10,000 patients globally [7] - The company plans to accelerate clinical development for its rare disease pipeline, including increasing enrollment targets for ongoing studies [6][8] Corporate Objectives for 2026 - Key objectives include launching VYJUVEK in additional major European markets, expanding the specialty distributor network to over 40 countries, and reporting top-line results from various registrational studies [14] - The company expects combined non-GAAP R&D and SG&A expenses for 2026 to be between $175 million and $195 million [9]

Core Viewpoint - Krystal Biotech announced positive interim clinical results for its cystic fibrosis program, KB407, highlighting the molecular confirmation of wild-type CFTR protein expression in patients' lungs [2]. Group 1: Clinical Update - The conference call was held to discuss the clinical update for the cystic fibrosis program, KB407 [2]. - The positive interim results were shared in a press release available on the company's website and filed as an 8-K with the SEC [2]. Group 2: Leadership and Participation - The call featured key executives including Krish Krishnan (Chairman and CEO), Suma Krishnan (President of Research and Development), Trevor Parry (Vice President of Product Development), David Sweet (Director of Clinical Development), and Dr. Jorge Lascano (Professor of Medicine and Director of the Cystic Fibrosis Therapeutics Development Center at the University of Florida) [2].

Summary of Krystal Biotech's Clinical Update Call for Cystic Fibrosis Program KB407 Company Overview - **Company**: Krystal Biotech (NasdaqGS:KRYS) - **Program**: Cystic Fibrosis Program KB407 Key Industry Insights - **Cystic Fibrosis (CF)**: A genetic disorder caused by mutations in the CFTR gene, leading to severe respiratory issues. Current treatments, particularly CFTR modulators, do not benefit all patients, leaving a significant unmet medical need. Core Points and Arguments 1. **Positive Interim Clinical Results**: Krystal Biotech announced successful delivery and expression of wild-type CFTR protein in patients' lungs, marking a significant milestone for the KB407 program [2][5][36]. 2. **Unmet Need**: An estimated tens of thousands of CF patients are ineligible for or underserved by existing modulator therapies, highlighting the urgent need for alternative treatments [6][34]. 3. **Platform Technology**: The HSV-1 platform used for KB407 has shown potential for treating various lung diseases, supported by previous successes in other programs (KB408 for Alpha-1 antitrypsin deficiency and KB707 for lung cancer) [5][15]. 4. **Clinical Study Design**: The Phase I CORAL-1 study evaluated the safety and tolerability of KB407, with a focus on molecular correction in patients with CF. The study involved nebulized administration of KB407 and subsequent bronchoscopy for biopsy analysis [18][19]. 5. **Molecular Correction Evidence**: In the interim analysis, 42.1% of conducting airway cells in one patient were positive for the KB407-encoded human CFTR, indicating successful transduction and expression [24][30]. 6. **Safety Profile**: KB407 demonstrated a favorable safety profile, with all adverse events being transient and mild to moderate in severity. No significant neutralizing antibody response was observed, suggesting potential for long-term efficacy [31][32][56]. 7. **Next Steps**: Krystal Biotech is working with the Cystic Fibrosis Foundation and the FDA to design a registrational repeat dosing study (CORAL-3) aimed at assessing functional impact on lung function (FEV1) [33][34][52]. Additional Important Insights 1. **Gene Therapy Evolution**: Advances in gene therapy, including vector engineering and understanding of pulmonary biology, have improved the potential for effective treatments for CF [12][13]. 2. **Patient Population**: The focus is on patients with rare or minimal function mutations who do not benefit from current therapies, emphasizing the need for mutation-agnostic approaches [9][10][11]. 3. **Market Potential**: Successful development of KB407 could open a market exceeding $2 billion, addressing a significant gap in CF treatment options [36]. 4. **Regulatory Strategy**: The company is exploring accelerated approval pathways with the FDA, aiming to expedite the development and potential market entry of KB407 [45][56]. Conclusion Krystal Biotech's KB407 program represents a promising advancement in the treatment of cystic fibrosis, addressing a critical unmet need for patients who do not respond to existing therapies. The positive interim results and favorable safety profile position the company for potential accelerated development and market entry.

CORAL-1 Clinical Data Update - Krystal Biotech's KB407 is a gene therapy for cystic fibrosis (CF) and has the potential to become a leadership opportunity in the treatment of CF[9] - The company plans to initiate the CORAL-3 study in the first half of 2026 to evaluate the safety and efficacy of repeat KB407 administration, which could support registration of KB407[4, 93] - The company estimates a $2 billion+ market opportunity in modulator-ineligible or refractory CF patients[12] KB407 Phase 1 CORAL-1 Highest Dose Cohort Results - In patient 03-01 with CFTR genotype 2184delA/W1282X, 42.1% of conducting airway cells expressed CFTR[55] - In patient 03-02 with CFTR genotype R553X/M1V, 29.4% of conducting airway cells expressed CFTR[68] - In patient 03-03 with CFTR genotype C1210-12T/1408A>G, 36.5% of conducting airway cells were viral marker positive[76] - In patient 03-04 with CFTR genotype R334W/R1162X, 33.8% of conducting airway cells were viral marker positive[79] - In patient 03-05 with CFTR genotype F508del/F508del, 36.8% of conducting airway cells were viral marker positive[81] - In patient 03-06 with CFTR genotype F508del/F508del, 31.4% of conducting airway cells were viral marker positive[83]

Core Insights - The company announced a positive interim clinical update from the highest dose cohort of the CORAL-1 study, confirming successful lung delivery and expression of wild-type CFTR protein in cystic fibrosis patients following inhaled administration of KB407 [1][2][13] - The CORAL-3 study design has been submitted to the FDA, with enrollment expected to start in the first half of 2026 [1][13] Clinical Study Details - KB407 is being evaluated in the CORAL-1 study, which is a multi-center Phase 1 study with three dose escalation cohorts [4] - As of January 6, 2026, seven patients have been dosed in the highest dose cohort, with successful bronchoscopies yielding biopsies suitable for molecular analysis [6][12] - The percentage of conducting airway cells transduced with KB407 ranged from 29.4% to 42.1% across six patients with successful bronchoscopies [7][9] Patient Demographics and Results - Among the four modulator ineligible patients, all biopsies were positive for CFTR or viral marker expression, indicating widespread dissemination of KB407 [9] - For modulator eligible patients, the percentage of conducting airway cells positive for viral markers ranged from 31.4% to 36.8% [11] Safety Profile - Inhaled KB407 was well tolerated in the highest dose cohort, with most adverse events being mild to moderate [12] - One serious adverse event of asthma exacerbation was reported but deemed procedure-related and not related to KB407 [12] Future Plans - The company is moving forward with the CORAL-3 study, designed to evaluate the safety and efficacy of repeat KB407 administration [13] - The company expects to align on the CORAL-3 study design with the FDA in the first quarter of 2026 and start enrollment in the second quarter of 2026 [13]