News

NACFC 2023: Sionna’s Series 2 molecules can restore CFTR protein

Sionna Therapeutics’ second-generation molecules, combined with standard CFTR modulators, fully restored the production and function of the CFTR protein with the most common mutation that causes cystic fibrosis (CF), according to preclinical data. The Series 2 molecules, SION-719 and SION-451, are undergoing investigational new drug (IND) enabling…

First CF patient dosed with molecular prosthetic CM001

The first person with cystic fibrosis (CF) has been dosed with CM001, an experimental inhaled therapy being developed by Cystetic Medicines to treat all people with CF, including those who aren’t eligible for modulators. “Dosing the first person with CF represents a critical milestone in our mission to…

Trikafta leads to clinical gains for 2 adults with rare M1101K mutation

Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly improved lung function and nutritional status in two adults with cystic fibrosis (CF) caused by the rare mutation M1101K, according to two cases described in Canada. “Our cases not only demonstrate the therapeutic benefit of [Trikafta] in individuals with non-F508del…

Complex genetic diversity found among P. aeruginosa strains

A high degree of genetic diversity was found among strains of Pseudomonas aeruginosa bacteria initially infecting the lungs of people with cystic fibrosis (CF), a study reports. Over a seven-year follow-up, the majority of CF patients experience a recurrence of the bacteria, with one-third of those being infected…