Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...

- 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 ...

Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutationsKB407 transduction confirmed in all ...

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 ...

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 ...

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) ...

Cystic fibrosis represents one of the most common inherited genetic disorders affecting people of European descent, touching the lives of approximately 30,000 individuals in the United States alone.

MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, ...

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...

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 ...