Jerusalem-based SpliSense, which focuses on cystic fibrosis and other genetic pulmonary diseases, closed on a $28.5 million Series B financing round. Participating in the round were OrbiMed, Israel Biotech Fund, Integra Holdings and the Cystic Fibrosis Foundation.
Roche acquired a portfolio of experimental cystic fibrosis therapies, including a candidate that has the potential to treat all patients with the disease.
Moderna Inc. and Vertex Pharmaceuticals Incorporated announced a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF).
The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.