Nasdaq

Sionna Therapeutics Presents Preclinical Data on Novel CFTR Modulators at the 48th European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
Business Wire

Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs

SAN DIEGO--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease ...

Sionna Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference

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 ...
Business Wire

Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
Healio

Cystic fibrosis triple therapy outcomes improve regardless of prior modulators

Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...
Healio

CFTR modulator use during pregnancy preserves maternal lung function

Please provide your email address to receive an email when new articles are posted on . “[These findings] help clinicians be more supportive of a female with CF becoming pregnant,” Raksha Jain, MD, ...
Seeking Alpha

Vertex Presents New Data Across Portfolio of Cystic Fibrosis Medicines Including ALYFTREK® at the North American Cystic Fibrosis Conference

- Data presented on outcomes following treatment with CFTR modulators add to growing body of evidence that reduced level of sweat chloride is associated with improved clinical outcomes, particularly ...
Yahoo Finance

Krystal Biotech Announces Positive Interim Clinical Update from KB407 Phase 1 CORAL-1 Study with Confirmation of Wild-Type CFTR Delivery to the Lungs of Patients with Cystic ...

PITTSBURGH, Jan. 08, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced a positive interim clinical update from the highest dose cohort of CORAL-1, the ...
EurekAlert!

Research shows positive impact of CFTR modulators on pregnancy outcomes in people with cystic fibrosis

DENVER - A recent retrospective study published in CHEST(Opens in a new window) evaluated the effects of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use on pregnancy outcomes ...
Nasdaq

Sionna Therapeutics to Present Preclinical Data on CFTR Modulators at European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...
TMCnet

Krystal Biotech Announces Positive Interim Clinical Update from KB407 Phase 1 CORAL-1 Study with Confirmation of Wild-Type CFTR Delivery to the Lungs of Patients with Cystic ...

Positive interim safety results from the first two dose escalation cohorts, referred to as Cohorts 1 and 2, were reported in 4Q 2024. Today’s update is focused on safety and molecular findings from ...