August Update

IN THIS EDITION:

Congrats to this year’s Golden Ticket competition winner, Positivo Biotechnologies. The CF Foundation selected the company to receive a year of lab space at Bakar Labs at the University of California Berkeley to help advance their genetic delivery technology and apply it to cystic fibrosis. Positivo Biotechnology is developing nonviral delivery technologies, which aim to address challenges in CF genetic therapies, including unwanted immune response and toxicity.

The CF Foundation, along with Deep Science Ventures, recently announced the creation and funding of a new company, Panthura, which is solely dedicated to developing a genetic therapy delivery technology for cystic fibrosis. Panthura is beginning to investigate nonviral methods to accurately deliver genetic therapies to cells in the lungs intravenously — a major challenge in genetic therapies. Panthura’s establishment is the outcome of an agreement with Deep Science Ventures with whom we have collaborated over the past three years. 

In June, 4D Molecular Therapeutics (4DMT) reported interim data from an ongoing Phase 1/2 trial of its genetic therapy for CF. The company is using a custom adeno-associated virus to deliver copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene into the lung cells of people with CF. 4DMT identified safe doses and evidence of delivery and expression of the CFTR gene in lung tissue. Enrollment in a Phase 2 trial to test a particular dosage is expected to begin later this year.

The U.S. Food and Drug Administration (FDA) will decide by January 2, 2025, whether to approve the new once-a-day modulator therapy developed by Vertex Pharmaceuticals. Known as the vanzacaftor (vanza) triple, the modulator would be approved for people with CF ages 6 and older who have at least one F508del mutation or one other mutation that responds to the vanza triple (which include Trikafta®-responsive mutations). Clinical trial results of the drug showed improvements in lung function similar to those seen with Trikafta and a reduction in sweat chloride levels that exceeded treatment with Trikafta.

Also, Vertex reported that it submitted an application to the FDA to approve Trikafta for additional rare mutations that have been shown to be responsive in lab tests.

Sionna Therapeutics has acquired the rights to develop three potential modulator therapies from AbbVie — two of which have been shown to be safe and well tolerated in Phase 2 studies. The company plans to combine one of these potential modulator therapies with one of several of its own compounds to target different places in the CFTR protein than existing modulators. The objective is to give people with CF additional modulator treatment options.

The CF Foundation is working to advance research and care for people with CF who have had a lung transplant or may need one. The CF Lung Transplant Consortium — a group of 15 lung transplant centers funded by the Foundation to focus on research, clinical guidance, and education — is conducting a study to collect blood, fluid samples from the lungs, and data to research a type of lung transplant complication called chronic lung allograft dysfunction. So far, the study has enrolled more than 1,000 patients in the U.S. and Canada. Learn more about the consortium and their research.

If you are considering a lung transplant, visit our updated Find a Lung Transplant Center page to find important information about lung transplant centers.

If you or a loved one are transitioning from pediatric to adult CF care, you may be interested in CF Responsibility. Independence. Self-Care. Education. (CF R.I.S.E.), a program that provides resources to help people between the ages of 10–25 develop the skills to manage their CF care independently. Ask your care team for the clinical code to enroll in the CF R.I.S.E. program.

The 2023 CF Foundation Patient Registry Highlights Handout is now available. It includes important data on people with CF who participate in the Registry, such as lung function, types of bacterial infections, and milestones such as the percentage of adults with CF with jobs.

We are excited to introduce CF Circles — topic-driven, hour-long, small-group discussions where primarily adults with CF connect virtually with others who understand the unique challenges and triumphs of living with CF. Peer-led discussions provide an opportunity to join a supportive community where you’ll find a sense of belonging and valuable guidance. Upcoming discussions include Transitioning to Financial Independence, Black People With CF, Life Without CFTR Modulators, Delayed CF Diagnosis, CF-Related Diabetes, and more. Find additional program information and register for upcoming CF Circles.

We’re adding more content to our Spanish website. Most recently, we have added general information about clinical trials, including a Spanish-language form to contact our Clinical Trial Navigator, who can help answer questions about clinical trials and help people with CF find clinical trials to participate in. Over time, we’ll continue to add information about our research, why it’s important, and how it may help people with CF and their families.