Read our 2024 annual report to see how we are supporting every person with cystic fibrosis, no matter where they are on their journey.
Message From Our CEO
At the heart of our progress is purpose. Take a moment to hear from our CEO, Michael P. Boyle, MD, as he reflects on what drives our mission forward.
We’re committed to developing a transformative treatment – and ultimately, a cure – for all people with cystic fibrosis.
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Genetic therapies have potential to treat the underlying cause of CF, no matter what mutations a person has. We’re supporting half a dozen potential genetic therapies in clinical trials, using several complementary approaches, and 95 total research projects for genetic therapies and rare and nonsense mutations.
We are investing up to $15 million in Prime Medicine to research the application of two of the company’s unique prime editing technologies in cystic fibrosis.
We provided additional funding for ReCode Therapeutics to develop a delivery system to get a gene editing therapy to lung stem cells. We also continue to support ReCode’s mRNA therapy for CF, which is currently in Phase 2 clinical trials.
Genetic Therapy Clinical Trials Progress
Potential mRNA therapies from ReCode Therapeutics, Arcturus Therapeutics, and Vertex Pharmaceuticals entered Phase 2 trials.
4D Molecular Therapeutics is in early stage clinical trials.
We are investing up to an additional $8.5 million in SpliSense for its Phase 2 trial of a type of genetic therapy for certain CF splicing mutations.
Clinical trials require many volunteers, which is challenging when recruiting from the small pool of people with CF who aren’t taking modulators. That’s why we are funding the REACH study, which is enrolling people with CF not taking modulators. Study participants will provide health information and specimens (like blood samples) for researchers to compare to new treatments in development, including genetic therapies.
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We are pioneering new research to develop genetic therapies, with a focus on their future potential to cure CF. To do that, we’re:
Conducting new research at the CF Foundation Therapeutics Lab.
Prioritizing our investments in therapeutic development companies using strategic selection criteria and supporting them with robust resources and direct input from the CF community.
Advocating for policies and funding that support the development of genetic therapies.
Funding and supporting trials conducted in the Cystic Fibrosis Therapeutics Development Network, the largest CF clinical trials network in the world.
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Drug Development Pipeline
To advance drug development and a search for a cure, the Cystic Fibrosis Foundation (CFF) has contracts with several companies to help fund the development of potential treatments and/or cures for cystic fibrosis. Pursuant to these contracts, CFF may receive milestone-based payments, equity interests, royalties on the net sales of therapies, and/or other forms of consideration. Resulting revenue received by CFF is used in support of our mission. See “How Drugs Get on the Pipeline” for more.
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CFTR modulators have dramatically improved life for many with CF. We’re supporting new alternatives and expanded access to provide additional treatment options.
The U.S. Food and Drug Administration (FDA) approved Alyftrek, a once-a-day CFTR modulator for people ages 6 and older who are already eligible for Trikafta or who have one of 31 rare mutations not treated by Trikafta.
The FDA expanded access to Trikafta for people ages 2 and older with at least one of 94 mutations. This approval enables approximately 300 people with CF in the U.S. to access a treatment for the underlying cause of their disease for the first time.
We invested up to $6.6 million of additional funding in Sionna Therapeutics toward developing a new modulator as an alternative that could be more effective or cause fewer side effects for some with CF.
Specialized care is the hallmark of the CF care model. Today, we’re adapting to tailor care to a widening range of health needs so that we can best support individual priorities and well-being.
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We believe in identifying and promoting best practices that support the foundational relationships between people with CF, their support system, and the CF care team.
Following a nationwide survey to learn about the needs of people with CF and how they receive care, we published journal articles with interim clinical guidance informed by the survey findings. This guidance provides considerations and best practices for CF care and the care team structure. Additional research is ongoing as we aim to support individualized care.
We adapted how we report lung function in the CF Foundation Patient Registry and Foundation-supported trials to no longer use race as a factor in comparison calculations. Based on scientific evidence and recommendations from the American Thoracic Society, the race-neutral lung function calculations avoid underestimating the extent of lung disease in certain groups.
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After 22 years leading clinical affairs at the CF Foundation, Bruce Marshall, MD retired in July. Dr. Marshall’s legacy as our chief medical officer is felt across CF care. By spearheading the development of quality improvement initiatives and clinical practice guidelines, and supporting clinical research through the CF Patient Registry, Dr. Marshall made a lasting difference for people with CF.
Albert Faro, MD, chief medical officer, and A. Whitney Brown, MD, vice president of clinical affairs, are now leading the Foundation’s Clinical Affairs team, supporting care center networks through a changing time in CF care.
We continue to learn about the CF population through our Patient Registry — representing about 80% of people with CF in the U.S. — including health status and their lives beyond CF. Here are some highlights from the latest report:
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We are driving progress on a wide range of complications that often impact people with CF.
We invested up to $5 million in Clarametyx Biosciences for a new approach to help address chronic CF infections. The potential therapy entered a Phase 2 trial in 2024.
We invested an additional $5 million in BiomX Inc. to support their Phase 2b trial to test the efficacy of a phage therapy — a specialized virus that attacks specific bacteria — in treating Pseudomonas aeruginosa infections.
Our lung transplant biorepository and registry, which collects patient samples to research factors that affect lung transplant outcomes, now has more than 1,000 participants.
We convened a committee of clinicians and people with CF to develop clinical care guidelines for pain and CF, which includes pain from disease complications, as well as certain medical procedures, tests, and treatments.
The CF community thrives on connection and support. Whether you’re walking at Great Strides, joining a support program, or using your voice to advocate with elected officials, your participation matters and we are deeply grateful for the support you give us.
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Following the passing of Joe O’Donnell, a lion of CF fundraising and chair of the Milestones III: Driven by a Dream campaign, Joe’s daughter and CF Foundation Board of Trustees member Kate O’Donnell, and National Leadership Council member Amy Barry were named the campaign’s new co-chairs. The campaign, which started in 2020, has raised more than $192 million toward its $200 million goal and is poised to surpass it in 2025.
From walks and hikes to galas and golf outings, our volunteers and donors came out in full force to raise nearly $97 million in 2024.
Five years after three adults with CF created ROSE UP, this special campaign continues to inspire the CF community to find creative ways to support a cure. This year, people ROSE UP by hosting virtual chess matches, hitting 65 golf balls, painting 50 disco balls, and more.
We celebrated more than 400 volunteers at the Volunteer Leadership Conference, titled Hope in Action. Participants connected with volunteers from across the country, shared their experiences and support stories, and got energized for their volunteer work ahead.
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At March on the Hill and Teen Advocacy Day, a combined more than 400 teens and adults, some who have cystic fibrosis, gathered on Capitol Hill or participated in virtual meetings with their representatives and legislative staff. They advocated for passage of the PASTEUR Act, proposed legislation that would encourage development of new antibiotics that people with CF — and the world at large — need.
We advocated for the HELP Copays Act, bipartisan legislation that would help lower out-of-pocket costs for people with CF by restricting co-pay accumulator programs and ensure any item or service covered by insurance is considered part of their essential health benefits package. This legislation, as well as efforts to ban co-pay accumulator practices in the states, would potentially reduce the financial burden that results from complex and hard-to-navigate health insurance plans.
2024 State-Level Advocacy Wins
Alongside the CF community, we advocated for policies that would benefit people with CF in 30 states, with 480 advocates sending nearly 900 messages to state elected officials. Below are just some of the wins our community made possible in 2024.
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CF Foundation Compass helps people with CF and their families with day-to-day challenges. In 2024, Compass:
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To more fully support the CF community, Compass added additional Spanish-speaking case managers. In 2024, Compass managed 68% more cases from Spanish-speakers compared with last year.
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To offer opportunities for people with CF to connect to their peers, we established CF Circles, which are topic-based, small-group discussions that give primarily adults with CF a virtual space to discuss their unique experiences including delayed diagnoses, living as a Black person with CF, living with advancing lung disease, and CF-related diabetes.
In 2024, Community Voice members participated in projects focused on health and clinical research equity, financial wellness, sexual and reproductive health, and clinical care guidelines, among other topics.
Thank you to our corporate champions who provide support of $100,000 or more.
Platinum
Gold
Silver
Bronze
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| KC Bryan White Chair |
Teresa L. Elder Vice Chair |
Eric R. Olson, PhD Vice Chair |
| Michael L. Beatty, Esq. nonvoting, Leadership Council Chair |
Carole B. Griego, MD | Eric C. Schneider, MD |
| Jessica H. Boyd, MD, MPH | Catherine C. McLoud nonvoting, Chair Emeritus |
Steven Shak, MD |
| Michael P. Boyle, MD | Peter J. Mogayzel Jr., MD, PhD nonvoting, Pediatric CF Care Center Representative |
Dodzie Sogah, PhD |
| James (Jamie) R. Butler, II nonvoting, Advisor |
Paul A. Motenko | Eric J. Sorscher, MD nonvoting, Medical Advisory Council Chair |
| Dominic J. Caruso | David A. Mount Treasurer |
Jennifer Taylor-Cousar, MD nonvoting, CF Adult Care Center Representative |
| Louis A. DeFalco Vice Chair |
Robert H. Niehaus Vice Chair |
Doris F. Tulcin nonvoting, Chair Emeritus |
| Elise Eberwein nonvoting, Advisor |
Kate O'Donnell | John S. Weinberg Executive Vice Chair |
Corporate Officers
| President and CEO
Michael P. Boyle, MD |
Executive Vice President and Chief Operating and Financial Officer
Irena Barisic |
To come in May.