Latest news with #CFTR
Yahoo
03-06-2025
- Business
- Yahoo
Sionna Therapeutics to Present at the Goldman Sachs 46th Annual Global Healthcare Conference
WALTHAM, Mass., June 03, 2025 (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 a fireside chat at the Goldman Sachs 46th Annual Healthcare Conference in Miami, Florida on Wednesday, June 11th 2025 at 8:00 a.m. ET. A live webcast of the presentation will be available the day of the event on the 'Events' page within the Investors section of Sionna's website at A replay will also be available following the event. About Sionna TherapeuticsSionna Therapeutics is a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for CF by developing novel medicines that normalize the function of the CFTR protein. Sionna's goal is to deliver differentiated medicines for people living with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1), which Sionna believes is central to potentially unlocking dramatic improvements in clinical outcomes and quality of life for people with CF. Leveraging more than a decade of the co-founders' research on NBD1, Sionna is advancing a pipeline of small molecules engineered to correct the defects caused by the F508del genetic mutation, which resides in NBD1. Sionna is also developing a portfolio of complementary CFTR modulators that are designed to work synergistically with its NBD1 stabilizers to improve CFTR function. For more information about Sionna, visit Sionna intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company's Investor Relations website, in addition to following the Company's press releases, SEC filings, public conference calls, presentations, and webcasts. Media ContactAdam DaleyCG Life212.253.8881adaley@ Investor ContactJuliet Labadorfir@
Associated Press
03-06-2025
- Business
- Associated Press
Sionna Therapeutics to Present at the Goldman Sachs 46th Annual Global Healthcare Conference
WALTHAM, Mass., June 03, 2025 (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 a fireside chat at the Goldman Sachs 46th Annual Healthcare Conference in Miami, Florida on Wednesday, June 11th 2025 at 8:00 a.m. ET. A live webcast of the presentation will be available the day of the event on the 'Events' page within the Investors section of Sionna's website at A replay will also be available following the event. About Sionna Therapeutics Sionna Therapeutics is a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for CF by developing novel medicines that normalize the function of the CFTR protein. Sionna's goal is to deliver differentiated medicines for people living with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1), which Sionna believes is central to potentially unlocking dramatic improvements in clinical outcomes and quality of life for people with CF. Leveraging more than a decade of the co-founders' research on NBD1, Sionna is advancing a pipeline of small molecules engineered to correct the defects caused by the F508del genetic mutation, which resides in NBD1. Sionna is also developing a portfolio of complementary CFTR modulators that are designed to work synergistically with its NBD1 stabilizers to improve CFTR function. For more information about Sionna, visit Sionna intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company's Investor Relations website, in addition to following the Company's press releases, SEC filings, public conference calls, presentations, and webcasts. Media Contact Adam Daley CG Life 212.253.8881 [email protected] Investor Contact Juliet Labadorf [email protected]
Yahoo
22-05-2025
- Business
- Yahoo
Sionna Therapeutics to Present Preclinical Data During Oral Session at the 48th European Cystic Fibrosis Conference
WALTHAM, Mass., May 22, 2025 (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 preclinical data assessing combinations of Sionna's nucleotide-binding domain 1 (NBD1) stabilizers, SION-451 and SION-719, with complementary Sionna CFTR modulators, galicaftor (SION-2222) and SION-109, will be featured in an oral presentation at the European Cystic Fibrosis Society's (ECFS) 48th European Cystic Fibrosis Conference, being held June 4-7, 2025 in Milan, Italy. Details of the oral presentation are as follows: Abstract Title: Stabilizers of CFTR NBD1 synergize with galicaftor (SION-2222) or SION-109 to enable full correction of ΔF508-CFTRAbstract Number: WS19.01Presenting Author: Gregory Hurlburt, Ph.D., Co-Founder and Senior Vice President, Discovery Research, SionnaSession Title: WS19 - Upstream/downstream: new therapies for people with cystic fibrosisDate and Time: Friday, June 6, 2025, 5:00-5:15 p.m. CET/11:00 -11:15 a.m. ET The presentation will be made available the day of the event under the 'Scientific Presentations' section within the Science page of Sionna's website at About Sionna TherapeuticsSionna Therapeutics is a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for CF by developing novel medicines that normalize the function of the CFTR protein. Sionna's goal is to deliver differentiated medicines for people living with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1), which Sionna believes is central to potentially unlocking dramatic improvements in clinical outcomes and quality of life for people with CF. Leveraging more than a decade of the co-founders' research on NBD1, Sionna is advancing a pipeline of small molecules engineered to correct the defects caused by the F508del genetic mutation, which resides in NBD1. Sionna is also developing a portfolio of complementary CFTR modulators that are designed to work synergistically with its NBD1 stabilizers to improve CFTR function. For more information about Sionna, visit Sionna intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company's Investor Relations website, in addition to following the Company's press releases, SEC filings, public conference calls, presentations, and webcasts. Media ContactAdam DaleyCG Life212.253.8881adaley@ Investor ContactJuliet Labadorfir@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
20-05-2025
- Health
- Yahoo
ATS 2025: ETI-linked mucus clearance tied to lung function gains in paediatric CF
On 19 May, at the 2025 American Thoracic Society (ATS) International Conference, researchers from Cincinnati Children's Hospital presented compelling evidence that functional improvement in paediatric cystic fibrosis (CF) patients receiving elexacaftor/tezacaftor/ivacaftor (ETI) therapy is driven by regional clearance of mucus plugs. Using advanced imaging techniques—ultrashort echo-time (UTE) magnetic resonance imaging (MRI) and hyperpolarised xenon-129 (¹²⁹Xe) ventilation MRI—the study offered a granular view of structural and functional lung changes, offering insight into why certain patients derive greater clinical benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. The prospective study enrolled 30 paediatric CF patients who underwent imaging and spirometry both before and following the initiation of CFTR modulator therapy. A total of 19 patients received ETI while 11 were started on lumacaftor/ivacaftor (LI). Each lung lobe was scored for structural abnormalities, including mucus plugging, bronchiectasis, wall thickening, and opacities. Functional outcomes were assessed via ventilation defect percentage (VDP) and percent predicted FEV₁ (ppFEV₁). ETI-treated patients demonstrated significant clinical and structural improvement across multiple domains. VDP improved by a mean of 6±6% (p<0.001), while ppFEV₁ increased by 9±12% (p=0.01). These changes were tightly correlated with a decrease in mucus scores as observed via UTE MRI. Specifically, ETI patients with a 0.5-point or greater reduction in mucus burden experienced the most substantial functional gains—12±14% increase in FEV₁ and 8±4% reduction in VDP (both p<0.01). Conversely, patients on LI showed no statistically significant improvement in either imaging or functional metrics. Other imaging markers, including consolidation, air trapping, and ground-glass opacities, improved significantly in the ETI group, while bronchiectasis and wall thickening—typically associated with irreversible airway damage—remained unchanged. In one illustrative case, a paediatric patient with the largest improvement in mucus score (−4.5) experienced a 34% rise in FEV₁ and a 14% drop in VDP, underscoring the mechanistic link between mucus clearance and restored pulmonary function. Clinically, these findings support a more nuanced understanding of how ETI therapy improves outcomes, highlighting mucus clearance as a key driver of therapeutic efficacy rather than merely a downstream effect. The use of UTE and ¹²⁹Xe MRI, which offer high-resolution, radiation-free imaging of airway pathology and ventilation, respectively, adds a valuable dimension to conventional spirometry and could play a growing role in stratifying treatment response in both clinical trials and real-world practice. This data reinforces ETI's dominant profile, particularly in the paediatric setting, where long-term structural preservation is critical. The ability to directly correlate mucus plug resolution with improvements in FEV₁ and VDP adds scientific credibility to ETI's clinical value and may inform payer decision-making by providing objective, imaging-based endpoints. With Vertex's ETI franchise continuing to set the standard, these findings raise the bar for emerging competitors in the CFTR modulator space. Future entrants, whether oral, inhaled, or gene-editing-based, will be expected not only to match ETI's functional benefits but to demonstrate similar or superior regional lung clearance and structural remodelling. The integration of advanced imaging into clinical development pipelines may prove critical for differentiation, particularly as value-based care models demand measurable outcomes tied to cost-effectiveness. Key opinion leaders interviewed by GlobalData have emphasised that non-invasive, radiation-free imaging tools such as UTE and Xe MRI will be instrumental in guiding therapy in the next era of CF care. Their use may enhance treatment personalisation and enable more precise forecasting of long-term benefit, critical factors in an increasingly competitive and outcomes-driven market. "ATS 2025: ETI-linked mucus clearance tied to lung function gains in paediatric CF" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
30-04-2025
- Business
- Yahoo
Sionna Therapeutics to Present at The Citizens Life Sciences Conference 2025
WALTHAM, Mass., April 30, 2025 (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 present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the presentation will be available the day of the event on the 'Events' page within the Investors section of Sionna's website at A replay will be available for 90 days following the event. About Sionna TherapeuticsSionna Therapeutics is a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for CF by developing novel medicines that normalize the function of the CFTR protein. Sionna's goal is to deliver differentiated medicines for people living with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1), which Sionna believes is central to potentially unlocking dramatic improvements in clinical outcomes and quality of life for people with CF. Leveraging more than a decade of the co-founders' research on NBD1, Sionna is advancing a pipeline of small molecules engineered to correct the defects caused by the F508del genetic mutation, which resides in NBD1. Sionna is also developing a portfolio of complementary CFTR modulators that are designed to work synergistically with its NBD1 stabilizers to improve CFTR function. For more information about Sionna, visit Sionna intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company's Investor Relations website, in addition to following the Company's press releases, SEC filings, public conference calls, presentations, and webcasts. Media ContactAdam DaleyCG Life212.253.8881adaley@ Investor ContactJuliet Labadorfir@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
