Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many people living with cystic fibrosis and their families face complicated issues related to getting the care they need. Cystic Fibrosis Foundation Compass makes sure that no one has to do it alone.
CF Foundation Compass is a service that helps people with CF and their families with navigating insurance options, connecting to legal information and experts, finding available financial resources, and tackling other life issues.
CF care team members are paramount in providing highly specialized care to people living with CF. CF Foundation Compass can help by serving as a strategic ally for care teams, so team members can focus on their patients’ care.
CF Foundation Compass can help you navigate insurance, financial, legal, and other issues you are facing. Use this online form to start your conversation with a Compass case manager today.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
Published on May 13, 2021
BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide (ASO) therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations. The investment is part of the Foundation's Path to a Cure, a research agenda to accelerate treatments for the underlying cause of cystic fibrosis for every person with CF.
The CF Foundation's funding will support SpliSense's splicing mutation correction program, including preclinical development and a Phase 1/2a clinical trial to test the safety and tolerability of the ASO as a potential treatment for people with the splicing mutation 3849+10Kb C-to-T. The objective is to generate a proof-of-concept for the ASO technology in cystic fibrosis. If the trial is successful, it could potentially validate other similar therapeutic approaches in SpliSense's pipeline that address rare mutations in cystic fibrosis which do not respond to current therapies that target the underlying cause of CF.
“This agreement will enable the exploration of synthetic antisense oligonucelotides as a potential treatment for CF in the clinic, opening exciting new opportunities for therapeutic development and further diversifying the approaches we are pursuing as part of our Path to a Cure,” said William Skach, MD, executive vice president and chief scientific officer for the CF Foundation. “As such, these studies will provide important information for the future development of these potential therapies for additional people with rare mutations.”
Splicing mutations disrupt the production of functional CFTR messenger RNA (mRNA), which is needed to make CFTR proteins. As a result, splicing mutations block the synthesis of normal CFTR proteins. If successful, this potential therapy would enable the creation of a full-length normal CFTR protein, helping restore the proper balance of salt and fluids in the lungs to improve lung function.
A splicing mutation occurs when there is an alteration in the DNA sequence that changes the instructions needed to properly generate mRNA. Oligonucleotides work by binding to the RNA molecule and correcting these instructions. Oligonucleotides, including ASOs, provide a potential therapeutic approach for several types of CFTR mutations and are already used in the treatment of spinal muscular atrophy, certain forms of muscular dystrophy, and other genetic diseases.
SpliSense's technology is based on the research of Professor Batsheva Kerem from the Hebrew University of Jerusalem. Professor Kerem was part of the team that identified and cloned the CFTR gene which, when mutated, is responsible for causing cystic fibrosis.
The Foundation awarded $400,000 to SpliSense in 2017 to develop this ASO therapy.
About the Path to a Cure
The CF Foundation launched its $500 million Path to a Cure initiative in October 2019. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF. Each approach requires a different set of scientific tools and knowledge, leading the Foundation to bring together researchers and industry leaders from a range of disciplines to advance multiple areas of research in parallel. Learn more about the Path to a Cure and related funding opportunities on cff.org.
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.
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