Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
EurekAlert!

A fundamentally new therapeutic approach to cystic fibrosis: Nanobody repairs cellular defect

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
Nature

Discovering CFTR modulators

Dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) — an anion channel found on epithelial cells of various tissues — causes cystic fibrosis and plays an important role in ...
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 ...
Nanowerk

Nanoparticle-based gene editing could expand treatment options for cystic fibrosis

Researchers have demonstrated that lipid nanoparticles can package and deliver an entire therapeutic gene along with gene-editing machinery - a combination of large, complex components that non-viral ...
Seeking Alpha

Arcturus: H1 2024 Rare Disease Drug Data On Deck

Arcturus Therapeutics Holdings Inc. (ARCT) is gearing up to report results from its phase 1b study using its inhaled messenger RNA [mRNA] treatment known as ARCT-032 for the treatment of patients with ...