The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
The Cystic Fibrosis Foundation announced a new set of research agreements to drive progress on its Path to a Cure. The nine awards will advance a variety of tools and strategies to accelerate treatments for the underlying cause of cystic fibrosis for all people with CF, regardless of their mutations.
Today, the Cystic Fibrosis Foundation announced it has awarded up to $3.3 million to Polyphor AG to develop an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis. About 17% of individuals with CF who had Pseudomonas infections last year had multi-drug resistant strains.
Potential treatment from Calithera Biosciences minimizes growth of germs in the lungs
Today, the Cystic Fibrosis Foundation announced that it awarded funding to enGene Inc. to develop a customized vehicle to deliver genetic-based therapies, such as gene therapy and gene editing, into the lung cells of people with cystic fibrosis (CF). Delivering genetic-based therapies to the lungs is a key hurdle to developing effective treatments for all people with CF, including individuals with two nonsense and rare mutations.