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Revised Law Includes Designated Funding for Rare Diseases

Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies

Nonprofit issues challenge to accelerate treatments for every person with CF

The Cystic Fibrosis Foundation has awarded up to $3 million to Kinnear Pharmaceuticals to conduct preclinical testing of a broad-spectrum anti-infective that has the potential to treat multi-drug resistant Pseudomonas and other infections in people with cystic fibrosis.

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.