Our latest must-read developments


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# July/August Update

GENETIC THERAPIES

Foundation Invests Additional Funds in Gene Editing Therapy

We invested up to an additional $24 million in Prime Medicine to develop a
gene editing therapy ([link removed])
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 >> ([link removed])




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INFECTION RESEARCH

Clarametyx Reports Positive Interim Results of Phase 2 Trial


Clarametyx Biosciences ([link removed])
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 ([link removed])
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.

Urine Test Is Being Developed to Detect Pseudomonas Infections

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 ([link removed])
to develop a breath test for Pseudomonas detection.



POLICY & ADVOCACY

Take Action to Protect Federal Research Funding

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 ([link removed]). 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 >> ([link removed])




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BACK TO SCHOOL

Prepare for a Successful School Year


Visit our School and CF page ([link removed])
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 >> ([link removed])




ADVANCED LUNG DISEASE AND TRANSPLANT

Lung Transplant Resources

* If you are waiting for -- or recovering from -- a lung transplant, use the handout "
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* The
2024 Advanced Lung Disease and Transplant Year in Review ([link removed])
provides key updates on CF Foundation-supported research, clinical care, and transplant-related initiatives -- and is now available in Spanish. (
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NEW CLINICAL TRIALS NOW ENROLLING

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 ([link removed]):

*
PRIDE-CF ([link removed]): Exploring health outcomes among people living with CF who identify as LGBTQIA+.
*
STOP PEDS ([link removed]): Evaluating the safety and effectiveness of two antibiotic treatment approaches for mild pulmonary exacerbations in children with CF.
*
TIDES 2.0 ([link removed]): Assessing anxiety, depression, and behavioral challenges in children with CF under age 12.



Have questions about clinical trials and how to find ones relevant to you or your loved one? Contact
Katie Howe ([link removed]), the Foundation's clinical trial navigator.



GET INVOLVED

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



Stay connected with the CF Foundation via our social media channels or visit
cff.org/news ([link removed])
for the latest updates.



Have a question or topic you'd like us to explore in a future issue? Email us at [email protected] -- we'd love to hear from you.


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