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Revised Law Includes Designated Funding for Rare Diseases

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

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.

Today, the Cystic Fibrosis Foundation announced a new award of up to $2 million to Eloxx Pharmaceuticals Inc. to support the global Phase 2 clinical program of ELX-02, a potential therapy to treat people with cystic fibrosis who have nonsense mutations.

The Cystic Fibrosis Foundation announced a new set of research agreements to drive progress on its Path to a Cure. The nine awards will advance a variety of tools and strategies to accelerate treatments for the underlying cause of cystic fibrosis for all people with CF, regardless of their mutations.

Today, the Cystic Fibrosis Foundation announced that it has awarded up to $400,000 to Life Edit Therapeutics Inc. to explore the application of their unique gene editing technology in CF.

Funding will support improved detection, diagnosis, prevention, and treatment of infections related to cystic fibrosis.

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