The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals Inc.’s Trikafta to treat cystic fibrosis in children aged 6 to 11 years old, making the cocktail therapy the first approved treatment for CF in that age group.
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.
At the 2021 virtual JP Morgan Healthcare Conference, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”
Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing.
The U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for use in people with cystic fibrosis with certain rare mutations.
Roche acquired a portfolio of experimental cystic fibrosis therapies, including a candidate that has the potential to treat all patients with the disease.
Topline results from a 28-week Phase IIb trial show that lenabasum, a novel oral small molecule developed by Massachusetts-based Corbus Pharmaceuticals, did not reduce the rate of new pulmonary exacerbations compared with placebo in patients with cystic fibrosis (CF).
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).
Teens and tweens with cystic fibrosis are trying to teach other teens a thing or two about social distancing as part of The Social Distance Squad, a program developed by AREA 23, an FCB Health Network company, in partnership with five leading CF nonprofits.
Chicago-based AbbVie announced plans for a strategic collaboration to develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Cystic Fibrosis Foundation.