UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...
Researchers engineered lipid nanoparticles to deliver a full CFTR gene into human airway cells, restoring near-normal ...
News Medical on MSN
New gene editing approach offers hope for cystic fibrosis patients
UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...
- 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 ...
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 ...
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 ...
WALTHAM, Mass., June 06, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for ...
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, ...
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 ...
- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
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