Nonprofit issues challenge to accelerate treatments for every person with CF
Revised Law Includes Designated Funding for Rare Diseases
Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies
Collaborative effort identifies 40 cases of COVID-19 across eight countries; outcomes for this high-risk population were reported in the Journal of Cystic Fibrosis
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
Today, the Cystic Fibrosis Foundation announced that it awarded funding to enGene Inc. to develop a customized vehicle to deliver genetic-based therapies, such as gene therapy and gene editing, into the lung cells of people with cystic fibrosis (CF). Delivering genetic-based therapies to the lungs is a key hurdle to developing effective treatments for all people with CF, including individuals with two nonsense and rare mutations.