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Cystic Fibrosis Foundation
4550 Montgomery Ave, Suite 1100N
Bethesda, Maryland 20814

www.cff.org ([link removed])

Latest Progress in the Path to a Cure

Learn more about recent news to develop therapies for all people with CF.

The Cystic Fibrosis Foundation continues to make progress in its $500 million Path to a Cure ([link removed]) initiative to accelerate treatments for the underlying cause of cystic fibrosis and develop a cure. So far this year, the Foundation has made three significant investments to further research into genetic therapies and potential treatments for nonsense mutations.

Those investments include:

A $6 million investment in June in Carbon Biosciences ([link removed]) to support the company's preclinical research into an innovative gene therapy approach that could temporarily “fix” CF in the lungs for all people with CF regardless of their mutations.

Funding for SalioGen Therapeutics ([link removed]) to support the company’s preclinical research into a novel genetic therapy that could help all people with CF regardless of their mutations.

Up to $15.9 million in additional funding to Eloxx Pharmaceuticals Inc ([link removed]). to expand clinical studies of ELX-02 ([link removed]), a potential therapy for people with CF who have nonsense mutations.

In other news:

We recently announced the winners of our inaugural Golden Ticket Competition ([link removed]). The winners will receive one year of free laboratory space and support to help advance early-stage research into genetic therapies for CF.

Several clinical trials for CF genetic therapies either are already underway or are scheduled to begin in the next 18 months. One gene therapy trial that has already started is sponsored by 4D Molecular Therapeutics ([link removed]). The early-stage clinical trial ([link removed]), which the Foundation helped fund, is for 4D’s potential gene therapy for people with CF who are ineligible for or unable to tolerate modulators.

To learn more about genetic therapies and upcoming clinical trials, watch these videos:

The closing keynote from ResearchCon 2022, “Something for Everyone: A look at possibilities and challenges of genetic therapies on the horizon. ([link removed])”

A webinar in Spanish ([link removed]) explaining the basics of genetic therapies.

CLINICAL TRIAL FINDER ([link removed])

RESEARCH INTO GENETIC THERAPIES ([link removed])





4550 Montgomery Avenue, Suite 1100N | Bethesda, MD 20814 US

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