News

CF gene therapy 4D-710 improves lung function, data show

A single dose of 4D Molecular Therapeutics’ inhaled gene therapy candidate 4D-710 improved or stabilized lung function in adults with cystic fibrosis (CF), according to interim data from a Phase 1/2 clinical trial. Additional data from the AEROW (NCT05248230) study, which is evaluating the therapy in CF…

Long-term Kaftrio increases gut microbiota diversity in CF

Extended treatment with Kaftrio progressively increases the diversity of the microorganisms in the gut in people with cystic fibrosis (CF), a recent study suggests. However, gut microbiota diversity remained significantly different from healthy people after about 1.5 years on the CFTR modulator, probably due to the use…

SPL84 therapy on FDA fast track for CF due to splicing mutation

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SPL84, an experimental inhalation therapy for people with cystic fibrosis (CF) caused by the 3849+10 kb C-to-T mutation in the CFTR gene. The designation is given to accelerate the development and review of therapies meant to address unmet…

Nanite, Saliogen teaming up to develop genetic medicine for CF

Two Massachusetts-based companies, Nanite and Saliogen Therapeutics, are teaming up on a new research project that aims to create a genetic medicine with the potential to treat cystic fibrosis (CF). “Nanite’s relationship with SalioGen is consequential in discovering safe polymeric nanoparticles for a transformative treatment for CF,”…