About cystic fibrosis—
& the final 10%
Cystic fibrosis, affecting about 80,000 people worldwide, is a progressive disease primarily affecting the lungs. It is caused by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene, a key protein that helps move bicarbonate move from the inside of cells to the outside.
The treatment outlook
90% of people with cystic fibrosis have mutations that cause the CFTR protein to malfunction, and can benefit from recently introduced CFTR modulators. But in about 10% of cases, people with cystic fibrosis produce little-to-no CFTR. In these cases, modulators can’t help.
For people with CF not on modulators, treatments that address the missing CFTR protein remain unavailable.
At cystetic Medicines, we believe that together we can change that.
You simply can’t affect the behavior of a protein if there is no protein to affect – so we had to find a different solution.
A community that has held its
breath for far too long
The cystic fibrosis final 10% community is one of hope and inspiration. A group that has watched others with their disease get access to lifesaving care, members of the final 10% have applauded those breakthroughs, while waiting for their own.
But they do more than wait. The final 10% is made of change makers. They are standing on the precipice of hope, counting the time to treatment not in decades, but in months and short years. And they are committed to driving change and making comprehensive CF care a reality.
Meet the community
Emily’s Entourage is the life work of Emily Kramer-Golinkoff, who was diagnosed with cystic fibrosis at six weeks old and is a member of the final 10% community that does not benefit from currently available CFTR modulators. Emily founded Emily’s Entourage with her family and friends in 2011 as an engine to drive research and drug development for those in the final 10% of the CF community. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development. Emily’s Entourage was a critical early funder of the molecular prosthetics approach that underlies cystetic Medicines and CM001.
cystetic Medicines clinical trials
cystetic Medicines is launching phase 1 clinical trials with their first clinical candidate, CM001, in the spring of 2023. A phase 2 clinical trial will follow as soon as possible thereafter. The phase 2 clinical trial will be open to people with cystic fibrosis who aren’t using CFTR modulators.