Foundation to fund up to $110 million for collaboration to accelerate the development of genetic therapies for CF
The Cystic Fibrosis Lung Transplant Consortium Biorepository and Patient Registry, in collaboration with Cleveland Clinic, will provide critical clinical data and samples to support future research investigating complications of lung transplant.
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, 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.