Explore our progress to find a cure for CF


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

www.cff.org ([link removed])

It's been a little over two years since we launched our $500 million Path to a Cure initiative, an ambitious research agenda to deliver treatments for the underlying cause of cystic fibrosis and, one day, a cure for every person with CF. We've made tremendous progress toward our goal this year, executing 16 new Path to a Cure industry contracts thus far.

We remain committed to exploring therapies focused on nonsense and rare mutations with investments in several different strategies:

Up to $8.4 million to SpliSense ([link removed]) to develop a therapy for people with CF who have splicing mutations and potentially other rare mutations

Up to $2.6 million to Eloxx Pharmaceuticals Inc. ([link removed]) to identify potential therapies for CF nonsense mutations and up to $3.6 million to support the global Phase 2 clinical program of a more advanced compound, ELX-02 ([link removed]), for treatment of people with CF who have at least one G542X mutation

Up to $400,000 to Life Edit Therapeutics Inc ([link removed]). ([link removed]) to explore gene editing tools that may correct the six most common nonsense mutations in people with CF

We continued to invest in essential research that will lay the groundwork for genetic therapies, such as RNA therapy, gene delivery and gene editing in CF. This included more than $1.8 million to three companies ([link removed]) for early-stage research into potential genetic therapies.

Additionally, we're thinking innovatively about how we can attract new companies into the CF space. We partnered with Pioneering Medicines ([link removed]), an initiative of Flagship Pioneering, to bring together three different companies in a first-of-its-kind collaboration where each company will provide a piece of the puzzle for at least two new CF therapies. We have agreed to invest up to $110 million in the collaboration with an initial commitment of $20 million. We also announced an exciting collaboration with Deep Science Ventures ([link removed]) to uncover and design new technologies with the potential to overcome challenges to developing genetic therapies for CF.

In parallel with these industry partnerships, we're supporting researchers across the country studying cutting-edge science in the lab. This year, we awarded the first Pioneer Award supported through Path to a Cure to academic researchers who are studying a novel technology for programmable gene insertion. The award will provide $1.3 million in support over three years to advance this approach, which would insert a healthy CFTR gene and replace the mutated version.

We've made great strides along the Path to a Cure – but we're not done yet. We intend to allocate a half billion dollars to the effort through 2025, and we will not rest until CF stands for Cure Found.

To learn more about genetic therapies and upcoming clinical trials, watch the recording ([link removed]) from our most recent webinar, "Genetic Therapies 101."





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

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