WALTHAM, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will participate in fireside chats at the following upcoming investor events: TD Cowen 46th Annual Health Care Conference on M ...

Core Viewpoint - The healthcare sector is expected to rebound and deliver above-average returns, with Intuitive Surgical and Vertex Pharmaceuticals identified as attractive investment opportunities for the long term [1]. Group 1: Intuitive Surgical - Intuitive Surgical has faced challenges such as high tariffs and increased competition in the robotic-assisted surgery (RAS) market, but its long-term outlook remains strong due to its leadership in the underpenetrated RAS market [3]. - The company develops leading robotic surgery devices, including the da Vinci system, which supports minimally invasive procedures [3]. - Intuitive Surgical has a market cap of $173 billion, with a current price of $488.46 and a gross margin of 65.99% [4][5]. - The installed base of Intuitive Surgical's devices increased by 12% year-over-year, reaching 11,106 units, which is expected to drive procedure volume and growth [5]. - Despite a 13% decline over the past year, Intuitive Surgical's stock is considered a top pick for buy-and-hold investors due to its long-term prospects [6]. Group 2: Vertex Pharmaceuticals - Vertex Pharmaceuticals is expanding its portfolio beyond its successful cystic fibrosis treatments, with plans to submit regulatory applications for zimislecel, a therapy for type 1 diabetes [7]. - The company has several upcoming catalysts, including late-stage data readouts for inaxaplin, targeting APOL-1 mediated kidney disease, and top-line data for povetacicept, aimed at IgA nephropathy [9]. - Vertex's market cap is $121 billion, with a current price of $477.91 and a gross margin of 86.29% [8]. - The anticipated success of new therapies, alongside the continued growth of its core cystic fibrosis franchise, positions Vertex's shares as attractive investments [10].

Company Overview - Sionna Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel medicines to treat cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [3] - The company aims to deliver differentiated medicines that restore CFTR function to near-normal levels by stabilizing CFTR's nucleotide binding domain 1 (NBD1), which is believed to be crucial for improving clinical outcomes and quality of life for CF patients [3] - Sionna is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation in NBD1 and is also developing complementary CFTR modulators to enhance CFTR function synergistically [3] Upcoming Events - Management will present at the Guggenheim Securities Emerging Outlook: Biotech Summit 2026 on February 11, 2026, at 2:00 p.m. ET [1] - A live webcast of the presentation will be available on Sionna's Investor Relations website, with a replay accessible after the event [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.

Core Insights - Hardman Johnston Global Advisors reported underperformance in its portfolio for Q3 2025, with a return of 5.02% net of fees, compared to 7.62% for the MSCI AC World Net Index [1] Company Performance - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is highlighted as a key stock, with a one-month return of 8.76% and a 52-week gain of 15.91% [2] - As of December 26, 2025, Vertex's stock closed at $462.90 per share, with a market capitalization of $118.683 billion [2] Challenges and Opportunities - Vertex Pharmaceuticals faced challenges in Q3 2025, with underperformance attributed to macro uncertainties from a Section 232 tariff investigation into medical devices and setbacks in chronic pain treatment developments [3] - The company ended its pursuit of a broad chronic neuropathic pain label for Journavx, which limited its total addressable market (TAM) in chronic pain, despite the drug being approved for acute pain with positive initial indications [3] - Vertex's next-generation pain asset, VX-993, failed in phase II development, impacting stock performance [3] - Positive updates were noted in other areas, including progress in treatments for kidney disease, type 1 diabetes, and gene-editing therapy, indicating a strong pipeline [3] - The launch of Alyftrek, a next-generation cystic fibrosis treatment, is underway, with patients transitioning from Trikafta, which is expected to improve margins due to a lower royalty burden [3] - The long-term outlook for Vertex remains positive, supported by its high-margin cystic fibrosis franchise and a robust balance sheet [3]

Core Viewpoint - The company is discussing interim Phase I data from the AEROW trial focused on cystic fibrosis, indicating progress in their research and development efforts [2]. Group 1: Company Overview - The call features key personnel including the Co-Founder and CEO, the Lead Principal Investigator from National Jewish Health, and a Co-Head from the Cystic Fibrosis Center at SickKids Research Institute [4]. - The company encourages stakeholders to visit their Investor Relations website for further details, including a press release and access to the webcast recording [2]. Group 2: Research and Development - The AEROW trial is a significant focus for the company, highlighting their commitment to advancing treatment options for cystic fibrosis [2]. - The involvement of leading experts in the field, such as Dr. Jennifer Taylor-Cousar and Dr. Felix Ratjen, underscores the trial's credibility and potential impact [4].

Summary of 4D Molecular Therapeutics Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (NasdaqGS:FDMT) - **Focus**: Development of gene therapies for cystic fibrosis (CF) using AAV vector technology Key Industry Insights - **Cystic Fibrosis**: A life-shortening genetic disease affecting over 100,000 people globally, with 40,000 in the U.S. The disease leads to chronic lung infections and progressive respiratory failure. - **Current Treatment Landscape**: While CFTR modulators have improved outcomes for some patients, many still face high unmet needs and burdensome daily treatments. Core Findings from the ARROW Trial 1. **Safety and Tolerability**: - 4D-710 was well tolerated with adverse events typical of nebulized therapies, resolving quickly [4][13][14]. - No serious adverse events related to 4D-710 beyond day 14, with one mild liver enzyme elevation noted [14]. 2. **Clinical Efficacy**: - Significant improvements in lung function and quality of life were observed in participants receiving the 2.5 x 10^14 vector genomes dose compared to the 5 x 10^14 vector genomes dose [34][38]. - The trial demonstrated durable CFTR expression in lung tissue, persisting for at least one year [41]. 3. **Study Design**: - The phase I trial focused on dose finding, safety, and CFTR expression levels through lung biopsies and bronchoscopy [10][11]. - The trial included multiple endpoints, such as FEV1, lung clearance index (LCI), and CFQ-R quality of life measures [24][39]. 4. **Durability of Treatment**: - Paired biopsy data indicated that CFTR expression remained within therapeutic range for one to three years post-treatment, validating the AAV platform's effectiveness [41]. 5. **Future Directions**: - Phase II enrollment is expected to complete in the first half of 2026, with ongoing collection of paired biopsy data to inform redosing strategies [43]. - The company anticipates that repeat dosing will be feasible due to the natural turnover of lung epithelial cells [42]. Regulatory and Market Considerations - **Regulatory Engagement**: Productive discussions with the FDA regarding the use of LCI as a primary efficacy endpoint, especially in pediatric studies [50][54]. - **Market Need**: There is a significant demand for effective therapies among patients who do not currently have access to modulators, highlighting the urgency for 4D-710 [63]. Financial Aspects - The program is fully funded by the Cystic Fibrosis Foundation through the second half of 2026, minimizing financial strain on the company [60]. Additional Insights - **Endpoint Sensitivity**: LCI is recognized as a more sensitive measure for detecting early disease changes compared to FEV1, which is effort-dependent and less sensitive to small airway disease [28][29]. - **Patient Variability**: The trial acknowledges variability in patient responses, emphasizing the need for a diverse participant pool in future studies [47][51]. Conclusion 4D Molecular Therapeutics is advancing its gene therapy program for cystic fibrosis with promising early data on safety, efficacy, and durability. The company is positioned to address significant unmet needs in the CF patient population while navigating regulatory pathways for potential accelerated approval. Further data collection and analysis will guide future development and redosing strategies.

Core Insights - Sionna Therapeutics is focused on developing innovative treatments for cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [2][4] - The company is advancing a pipeline of small molecules aimed at correcting defects caused by the F508del genetic mutation, which is significant for improving clinical outcomes for CF patients [2] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing CF treatment through novel medicines that stabilize CFTR's nucleotide binding domain 1 (NBD1) [2] - The company aims to deliver differentiated medicines that restore CFTR function to near-normal levels, potentially enhancing the quality of life for individuals with CF [2] - Sionna is also developing a portfolio of complementary CFTR modulators designed to work synergistically with its NBD1 stabilizers [2] Investor Engagement - Sionna will participate in upcoming investor events, including the Annual Evercore Healthcare Conference on December 3, 2025, and the Citi 2025 Global Healthcare Conference on December 4, 2025 [4] - Live webcasts of these presentations will be available on Sionna's Investor Relations website, with replays accessible after the events [1][3]

Core Insights - Sionna Therapeutics is focused on developing novel medicines to improve the treatment of cystic fibrosis by normalizing the function of the CFTR protein [2][4] - The company is advancing a pipeline of small molecules aimed at correcting defects caused by the F508del genetic mutation, which is significant for cystic fibrosis patients [2] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing cystic fibrosis treatment [2] - The company's approach involves stabilizing the nucleotide binding domain 1 (NBD1) of the CFTR protein, which is believed to be crucial for enhancing clinical outcomes and quality of life for cystic fibrosis patients [2] - Sionna is also developing complementary CFTR modulators to work synergistically with its NBD1 stabilizers [2] Investor Engagement - Sionna will participate in upcoming investor events, including the Guggenheim 2nd Annual Healthcare Innovation Conference and the Stifel 2025 Healthcare Conference [4] - Live webcasts of these presentations will be available on Sionna's Investor Relations website, with replays accessible after the events [1][4]

Core Viewpoint - Sionna Therapeutics is focused on transforming the treatment of cystic fibrosis (CF) by developing innovative medicines that normalize the function of the CFTR protein [2]. Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing CF treatment through novel medicines aimed at restoring CFTR function [2]. - The company aims to deliver differentiated therapies that stabilize the nucleotide binding domain 1 (NBD1) of the CFTR protein, which is believed to be crucial for improving clinical outcomes and quality of life for CF patients [2]. - Sionna is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation in NBD1 and is also developing complementary CFTR modulators to enhance CFTR function [2]. Investor Engagement - Sionna will participate in investor events, including the Cantor Global Healthcare Conference on September 3, 2025, and the Morgan Stanley 23rd Annual Global Healthcare Conference on September 8, 2025 [4]. - Live webcasts of these presentations will be available on the company's Investor Relations website, with replays accessible after the events [1][3].