A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...
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) ...
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, ...
Sionna Therapeutics targets cystic fibrosis with novel CFTR/NBD1 stabilization; key 2026 readouts and cash runway to 2028.
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 ...
UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...
Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is a combination medicine approved by the FDA in 2024. Alyftrek is approved to treat people ages 6 and older who have ...
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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