The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
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The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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.
The studies aim to improve early detection of chronic lung allograft dysfunction (CLAD), the leading cause of lung transplant failures.
New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
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.