July/August Update

We invested up to an additional $24 million in Prime Medicine to develop a gene editing therapy for people with cystic fibrosis. This new investment focuses on targeting the nonsense mutation G542X — one of the most prevalent CF-causing nonsense mutations, and one for which there are no available therapies. Insights from this work could speed up the development of prime editing therapies for other mutations in the future.The funding builds on our initial support, which focused on the potential of prime editing to correct multiple CF mutations in the lab.

Learn more about our investment for Prime Medicine >>

Clarametyx Biosciences announced positive interim results of a Phase 2a study evaluating a drug that aims to disrupt bacterial biofilms, one of the primary causes of antibiotic resistance. Biofilms are protective structures that shield bacteria from both the immune system and antibiotics. The average amount of Pseudomonas in sputum was lower in the first 21 participants after receiving this biofilm-disrupting compound, which was well tolerated. The next part of the Phase 2 trial will test two different doses of the drug in approximately 20 additional participants.

We recently provided $500,000 to Pearl Diagnostics to develop a urine test to detect Pseudomonas infections in people with CF. This investment reflects our focus on developing diagnostic tests for infections that do not rely on sputum — which is often difficult to collect from children and from adults taking modulator therapies that reduce mucus. Sputum cultures also can miss early signs of infection. Earlier this year, we invested up to $2.3 million in Owlstone Medical to develop a breath test for Pseudomonas detection.

With a critical funding deadline approaching, Congress is making decisions that could directly impact cystic fibrosis research at the National Institutes of Health and the U.S. Food and Drug Administration — both key drivers of progress toward a cure. 

Join our Policy & Advocacy team and Senior Director of Research, Katherine Tuggle, for a live webinar from 3-4 p.m. ET Sept. 10. We’ll discuss what’s at stake, what it means for the CF community, and how you can take action to protect essential federal funding for research and drug development in fiscal year 2026.  

Register now >>

Visit our School and CF page for guidance on 504 plans and Individualized Education Programs (IEPs) that outline educational needs and accommodations, such as how the school will manage your child’s absences or medical treatments. The section also contains tips for building healthy routines, helping children take more responsibility for managing their care, and obtaining accommodations in college.  

Learn more about managing school and CF >>

Do you want to help advance CF research? Below are some of the clinical trials enrolling participants. You can explore other trials on our Clinical Trial Finder: 

Have questions about clinical trials and how to find ones relevant to you or your loved one? Contact Katie Howe, the Foundation’s clinical trial navigator.

CF Circles: Adults with CF can register now for our next virtual CF Circles session Sept. 30, which will focus on pain management for people with CF.