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Research and Care August Update

# August Update

IN THIS EDITION:

* Golden Ticket winner announced
* FDA reviewing new triple-combination modulator
* Patient Registry Highlights Report available
* New content on our Spanish website



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Genetic Therapies

Congrats to this year's Golden Ticket competition winner,
Positivo Biotechnologies ([link removed]). 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 ([link removed]), 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 ([link removed])
(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.



CFTR Modulator News

The U.S. Food and Drug Administration (FDA) will decide by January 2, 2025, whether to approve the new once-a-day
modulator therapy ([link removed])
developed by Vertex Pharmaceuticals. Known as the
vanzacaftor (vanza) triple ([link removed]), 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 ([link removed])
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 ([link removed])
than existing modulators. The objective is to give people with CF additional modulator treatment options.



Improving Lung Transplant Outcomes

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 ([link removed]).

If you are considering a lung transplant, visit our updated
Find a Lung Transplant Center page ([link removed])
to find important information about lung transplant centers.



Get Help With the Transition From Pediatric to Adult Care

If you or a loved one are transitioning from pediatric to adult CF care, you may be interested in
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(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.



Patient Registry Highlights Report Available

The
2023 CF Foundation Patient Registry Highlights Handout ([link removed])
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.



CF Circles

Join Small-Group Discussions

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 ([link removed])
for upcoming CF Circles.



Expanding our Spanish-Language Content

We're adding more content to our
Spanish website ([link removed]). Most recently, we have added
general information about clinical trials ([link removed]), including a
Spanish-language form ([link removed])
to contact our
Clinical Trial Navigator ([link removed]), 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.



Other News

*
Anagram Therapeutics ([link removed])
reported that its potential enzyme therapy was well tolerated in people with CF who participated in its Phase 1 study. Data from the study will be presented at the
North American Cystic Fibrosis Conference ([link removed])
in September.
* The first results from the 2022 Wellness in the Modulator Era survey have been published in the Journal of Cystic Fibrosis.
The article ([link removed])
reports findings on the impact of modulator therapy on the well-being of people with cystic fibrosis. Future articles will cover other study results including mental and financial well-being.



DRUG DEVELOPMENT PIPELINE ([link removed])


CLINICAL TRIAL FINDER ([link removed])


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


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