Welcome to our bimonthly updates


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MEDICAL AND RESEARCH

February Update

Welcome to our first bimonthly medical and research update email, which combines the latest news and developments in cystic fibrosis research and care from the CF Foundation.

Genetic Therapies


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# Research Webinar Tomorrow


Join us from 7-8 p.m. ET Wednesday, February 21 ([link removed]), to learn about
genetic therapies ([link removed])
in CF, hear the latest on current genetic therapy clinical trials, and participate in the audience Q&A. Can't attend live?
Register ([link removed])
to receive a link to the webinar recording after the event.

Register now >> ([link removed])


# Investing in Genetic Therapies

We will continue to provide funding for the development of genetic therapies that promise to - one day - offer treatment for the underlying cause of cystic fibrosis in all people with the disease, including people with rare and nonsense mutations.

We are providing up to $15 million to
Prime Medicine ([link removed])
for early-stage research into
gene editing for cystic fibrosis. ([link removed])
Prime Medicine is using a gene editing technology called prime editing -- a technology that enables insertions or deletions of small segments of DNA at precise sites. This technology has the potential to enable many types of CF mutations to be corrected with a single type of genetic therapy.

CFTR Modulator News

Earlier this month, Vertex Pharmaceuticals announced positive results of its late-stage clinical trials for its once-daily triple-combination CFTR modulator made up of vanzacaftor, tezacaftor, and deutivacaftor (the "vanzacaftor triple"). The results showed improvements in lung function that were comparable to those seen with Trikafta® and a reduction in sweat chloride levels that exceeded treatment with Trikafta.

The trials evaluated the potential treatment in people with CF who have at least one copy of the F508del mutation or another mutation responsive to CFTR modulators. Vertex plans to apply for U.S. and European approval of the modulator for people with CF ages 6 years and older by mid-2024.



In January,
Sionna Therapeutics ([link removed])
dosed its first patient in an early-stage clinical trial of its modulator, SION-109. This modulator differs from those currently on the market in that it targets regions of the cystic fibrosis transmembrane conductance regulator (CFTR) protein affected by the F508del mutation that haven't been treated before. If successful, this modulator could be combined with another one from Sionna that also is in early-stage clinical trials to form a combination therapy.

CF Liver Disease

The Foundation has published new
guidelines for CF liver disease, ([link removed])
updating previous guidelines published in 1999. The new guidelines aim to help promote more effective screening, monitoring, and treatment of liver disease to improve outcomes.

Find Important Information About Lung Transplant Centers


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People who are considering a lung transplant or learning how to maintain their health post-transplant can find important information about transplant centers, including how many transplants each center has performed in the past four years -- overall and for people with cystic fibrosis.

Find a lung transplant center >> ([link removed])


Colorectal Health

March is Colorectal Cancer Awareness Month. Adults with cystic fibrosis face a colorectal cancer risk that is 5-10 times greater than the general population -- and even higher for people with CF who receive a lung or other organ transplant. Colonoscopies are the most effective screening method and need to be started at age 40 for people with CF and often at age 30 for those who have had a transplant (depending on when the transplant occurs).

The
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% link:[link removed] name="NICE-CF study link" content="NICE-CF study" %], which is enrolling, will assess whether stool-based testing, a non-invasive option, is as effective as a colonoscopy in detecting colorectal cancer.
Learn more about colorectal cancer and how to reduce your risk ([link removed]).

Infection Research

In January, we announced our investment of up to $5 million in
Clarametyx Biosciences ([link removed])
to develop a drug that could help address difficult-to-treat bacteria, such as chronic Pseudomonas aeruginosa -- a major cause of lung infections in people with cystic fibrosis.



Sound Pharmaceuticals reported positive interim results from its Phase 2b trial of its drug
ebselen ([link removed])
which aims to prevent and treat hearing loss and other inner-ear side effects in people with CF who are taking aminoglycosides, a type of antibiotic. The study showed that the 400 mg and 600 mg doses had ototoxicity (damage to the inner ear) rates of 44% and 43% respectively at four weeks after aminoglycoside treatment. These rates are significantly lower than the 93% aminoglycoside-associated ototoxicity rate shown in a Phase 1b observational study without ebselen treatment.

Health Equity

The Foundation established the
Health Equity Team Science Awards ([link removed])
in 2023 to provide support to research projects that aim to advance our understanding of barriers to care for individuals with CF from underrepresented communities.



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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