New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
Researchers' catalog of airway cell types could reveal targets for future genetic therapies
Today, the Cystic Fibrosis Foundation announced that it has awarded up to $2.17 million to Beyond Air® to support the development of a portable inhaled nitric oxide treatment for nontuberculous mycobacteria, difficult-to-treat bacteria that infect the lungs of people with cystic fibrosis.
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
The Cystic Fibrosis Foundation has awarded up to $3 million to Kinnear Pharmaceuticals to conduct preclinical testing of a broad-spectrum anti-infective that has the potential to treat multi-drug resistant Pseudomonas and other infections in people with cystic fibrosis.
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