Genetic therapies, NTM spotlight, modulator news, and more


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CF RESEARCH UPDATE

Summer 2023

A dark-skinned man sits inside wearing a therapy vest and doing nebulizer treatment.

The Cystic Fibrosis Foundation is excited to share the latest news and developments in CF research.

# Potential Treatments for Those Who Can't Take Modulators

Genetic therapies, such as gene therapies and messenger RNA therapies, offer the potential to treat all people with CF, including people who are not eligible for or cannot tolerate modulators. A key challenge is delivering genetic therapies into the appropriate lung cells without triggering an immune response that would potentially prohibit redosing.

To help overcome this obstacle, we recently invested up to $2 million in
Nosis Bio ([link removed])
to explore the use of specialized molecules to improve the delivery of gene therapies to specific cells in the lung. In CF gene therapy, a healthy cystic fibrosis transmembrane conductance regulator (CFTR) gene would be delivered to lung cells so they can produce functional CFTR protein.

In other news,
4D Molecular Therapeutics ([link removed])
announced positive interim data from the Phase 1 portion of a Phase 1/2 trial of its CF gene therapy. The trial participants are people with CF who are not eligible for or cannot tolerate modulator treatment. In this phase of the trial, researchers looked for evidence of the gene therapy in the lung. Healthy copies of CFTR DNA, RNA, and protein resulting from the gene therapy were detected in lung cells from all three trial participants.
A second group ([link removed])
of people with CF is being administered a higher dose of the gene therapy. If the gene therapy continues to be safe, a larger Phase 2 trial is expected to begin later this year.



# To learn more about the different types of genetic therapies and how they could work:

* Take a look at our
genetic therapies section ([link removed])
on our website.
* Download our
Understanding Genetic Therapies ([link removed])
infographic.
* Find genetic therapy clinical trials that are currently enrolling patients on the
Clinical Trial Finder ([link removed]).
* Browse the
Drug Development Pipeline ([link removed])
to see more of the genetic therapy research we're funding.





# We are committed to helping all people with CF

If you are an adult with cystic fibrosis who is not eligible for or cannot tolerate a CFTR modulator, you are invited to:

*
Join a supportive small group discussion ([link removed])
on Life Without Modulators, from 7-8:15 p.m. ET Thursday, August 24. During this discussion, adults with CF in the same situation will share experiences, discuss challenges and successes, and build connections. Email Gillian Mocek link in text (mailto:[email protected]) if you have questions.

* Help inform future research in genetic therapies by
joining Community Voice ([link removed]).






# NTM Spotlight


We are providing up to $3.9 million to
TB Alliance ([link removed])
to further develop a potential drug that could eventually be used to treat infections caused by
nontuberculous mycobacteria ([link removed])
(NTM) in people with cystic fibrosis. NTM infections are difficult to treat, requiring antibiotics for a year or longer with no guarantee of success and with a risk of serious side effects.

In addition, finding a standardized way to diagnose and treat NTM is a critical unmet need for people with CF. There are two studies that aim to address these issues.
The PREDICT study ([link removed]), which is enrolling people with CF who have positive NTM cultures, is evaluating the current standard of diagnosing NTM. Participants who are referred to treatment are enrolled in the PATIENCE study, which will evaluate current treatments.

An early-stage clinical trial is testing a non-traditional treatment for people with CF who have persistent NTM infections. Participants in
the ABATE study ([link removed])
will receive two separate infusions of a drug called gallium nitrate, which is structurally similar to iron. Because iron is essential for bacterial growth, researchers believe that substituting iron with gallium may help fight NTM infections.



# Other Infection News


Armata Pharmaceuticals ([link removed])
announced
positive topline results ([link removed])
from its clinical trial of AP-PA02, an experimental bacteriophage (phage) therapy that targets Pseudomonas aeruginosa infections. Phages are specialized viruses that kill very specific bacterial strains. Phage therapy could provide an alternative to antibiotics and help fight difficult-to-treat infections in CF.



# The Importance of Early Diagnosis

A recent study funded by the Foundation underscored the importance of evaluating infants for cystic fibrosis soon after a positive
newborn screening ([link removed])
test. Researchers found that a delay in initial care at a CF center from just 10 days of life to 47 days of life was associated with reduced weight-for-age through the baby's first year and reduced height-for-age through their first 5 years, according to the study published in
The Journal of Pediatrics ([link removed]).

Because of the results of this study, the Ann and Robert H. Lurie Children's Hospital of Chicago has launched a new
awareness campaign ([link removed])
that aims to reduce missed or delayed diagnosis of CF after newborn screening, especially in infants of color, who are disproportionately diagnosed later than non-Hispanic white infants.



# Modulator News

The U.S. Food and Drug Administration (FDA) approved two label expansions for CFTR modulator therapies:

* The use of
Trikafta ([link removed])® (elexacaftor/tezacaftor/ivacaftor) for children with cystic fibrosis ages 2 through 5 years who have at least one copy of the F508del mutation or certain mutations that are responsive to Trikafta based on lab data.
* The use of
Kalydeco ([link removed])® (ivacaftor) for infants with CF ages 1 month to 4 months who have certain mutations.

Unfortunately, AbbVie decided to
discontinue development ([link removed])
of its triple-combination modulator after disappointing interim results in a Phase 2 trial of galicaftor/navocaftor/ABBV-576.


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