Here are the latest news and developments in cystic fibrosis research and care from the Cystic Fibrosis Foundation:
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Investing in Potential Splicing Mutation Treatment |
We are investing up to $8.5 million in additional funds in SpliSense to support a planned Phase 2 clinical trial of its inhaled antisense oligonucleotide (ASO) drug for people with CF who have the splicing mutation 3849+10Kb C-to-T. The objective of the clinical trial is to generate a proof-of-concept for using this type of technology to treat people with CF with certain rare mutations.
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ReCode Therapeutics mRNA Therapy |
In February, ReCode Therapeutics dosed the first participants of a Phase 1 trial of an inhaled messenger RNA therapy (mRNA), which could provide a treatment option for all people with CF, regardless of their mutations. ReCode’s mRNA therapy is designed to provide a correct copy of the mRNA to lung cells to make a functional CFTR protein.
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Genetic Therapies Webinar Recording Now Available |
In case you missed it, the recording from our genetic therapies research webinar is available in English and Spanish. Starting from the basics, learn about genetic therapies in CF, including how they differ from existing therapies and the benefits they can potentially provide to any person with CF, including people with nonsense and rare mutations. Katie Howe, one of the featured speakers, is the Foundation’s new clinical trial navigator — a designated individual who provides personalized, one-on-one assistance to people with CF and their families who have questions about clinical trials. Contact Katie at [email protected] or by filling out an inquiry form.
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We invested more than $6.6 million in additional funding in Sionna Therapeutics to continue research into the development of potential new modulator therapies that could provide alternatives for people with CF who can’t tolerate current modulators.
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We provided an additional $5 million in BiomX Inc. to support a Phase 2b clinical trial to test the efficacy of bacteriophage (phage) therapy in treating chronic Pseudomonas aeruginosa infections — the second most common type of lung infection in people living with CF. Phage therapy uses phages (bacteria-specific viruses) to kill specific bacterial strains that are causing an infection.
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The Foundation has published new guidelines for CFTR-related metabolic syndrome (CRMS), a classification that describes someone whose newborn screen indicated they might have CF, but their sweat chloride test results fell into an uncertain or borderline range. These new guidelines reflect increased knowledge of CRMS since the publication of the original guidelines in 2009 and contain recommendations for CF specialists and primary care providers about the care of people with CRMS, specifically genetic testing and counseling; monitoring, testing, and treatment; and communication with people with CRMS and families.
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Share Your Thoughts About the Cost of CF
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How has the cost of living with cystic fibrosis affected your daily life and access to healthcare services? Adults with CF and caregivers of people with CF who are U.S. residents are invited to participate in our research survey and help the Foundation understand how to inform and support the CF community and care teams in addressing financial issues associated with CF.
Take the survey today >>
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Join us at ResearchCon April 30 – May 1 to hear what’s new in CF science, research, and care. Engage with those living with CF and researchers who are studying the disease while hearing the latest updates on the drug development pipeline, evolving infection research, the CF Foundation Therapeutics Lab, and more.
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Educational Video on Health Equity in CF
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Every person with CF deserves access to a timely diagnosis, quality care, and community support. The CF Foundation created an educational video featuring Jennifer Taylor-Cousar, MD, and Tanisha Cunningham, an adult with CF and parent of a child with CF, to illustrate the importance of health equity to the CF community. Together, we can make a difference in the lives of people with CF — improving care, research, and community support so that everyone benefits.
Watch the video >>
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Stay connected with the CF Foundation via our social media channels or visit cff.org/news for the latest updates.
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