CF Foundation Awards $1 Million to Felix Biotechnology to Develop Novel Phage Therapy

The Cystic Fibrosis Foundation awarded $1 million to Felix Biotechnology to develop a novel bacteriophage therapy approach to treat chronic Pseudomonas aeruginosa infections in people with cystic fibrosis.

Photo: JP Clancy, vice president of clinical research for the Cystic Fibrosis Foundation

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by mutations in the CFTR gene that lead to a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein. Children must inherit two defective CFTR genes—one from each parent—to have CF. There are approximately 2,000 known mutations in the CFTR gene. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

Pseudomonas infections are the third most common type of lung infection in people with cystic fibrosis and are very difficult to eliminate once established in the airways and a main contributor to morbidity and mortality in these patients. Approximately 32 percent of the nearly 31,000 people with CF in the Cystic Fibrosis Foundation Patient Registry had Pseudomonas infections in 2020, and more than 13 percent of those infected had multi-drug resistant Pseudomonas infections.

The CF Foundation award will support preclinical studies of a potential bacteriophage (phage) therapy to treat resistant Pseudomonas infections. Phages are specialized viruses that kill very specific bacterial strains.

Felix Biotechnology is developing an inhalable treatment licensed from the lab of Yale University Professor Paul Turner that is designed to address one of the key shortcomings of traditional phage therapy: bacterial resistance in chronic infections.

“Developing new treatments for Pseudomonas infections is a priority because they affect a large number of people with CF and can be difficult to treat,” said JP Clancy, vice president of clinical research for the Cystic Fibrosis Foundation. “Phage therapy has shown some promising results in several individual cases, but more research is needed to determine whether this potential therapy is safe and effective.”

The Foundation has supported several phage studies to explore the feasibility of phage therapy as a treatment for drug-resistant infections in CF, most recently investing up to $5 million in an equity investment in BiomX to support the clinical development of its experimental therapy targeting chronic Pseudomonas aeruginosa infection in CF patients.

Currently, people with cystic fibrosis can only access phage treatment in the United States by participating in a clinical trial or using the Food and Drug Administration emergency Investigational New Drug process, which only allows the use of experimental therapies for life-threatening conditions.

Author: Rare Daily Staff