UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...
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) ...
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 ...
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, ...
Leerink Partners Global Healthcare Conference 2026 on Tuesday, March 10 th, 2026, at 1:40 p.m. ET Live webcasts of the presentations can be accessed in the ‘Investors’ section of Sionna’s website at ...
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and ...
- 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 ...
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 ...
Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies ...
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The FDA approved a once-daily oral combination of vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis (CF) in adults and children 6 years and up ...
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 ...
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