When the Cystic Fibrosis Foundation’s lab opened 10 years ago, its primary focus was on developing more modulators that could treat a larger number of people with CF. Kalydeco® had just been approved, but for only 4% of the CF population.
Now with many people with CF benefiting from modulator therapies, the Cystic Fibrosis Foundation Therapeutics Lab has evolved and turned its attention to facilitating the development of genetic-based therapies that could — one day — lead to a cure for all people with cystic fibrosis.
We took another step closer to this mission in August when the lab, based in Lexington, Mass., completed work on its most recent physical expansion, growing by a third to nearly 27,000 square feet.
This expansion includes room for a business incubator — dedicated space that will allow lab scientists to mentor startup companies working on genetic therapies to treat CF — as well as space to host scientists from more established companies who can work side-by-side with lab scientists and benefit from our expertise in testing techniques and CF biology. This will help speed up the development of new treatments.
“This incubator space fits well with our strategy to lower the barrier for new companies to apply their technologies to CF,” said Martin Mense, PhD, senior vice president of drug discovery at the CF Foundation and director of the CF Foundation Therapeutics Lab. “It will help us to de-risk early-stage research and attract the most talented minds to finding a cure for CF.”
Carbon Biosciences, the first company to incubate with the lab, began working in the original lab space in May and has since moved into the new addition.
The recent expansion of the CF Foundation Therapeutics Lab also allows the flexibility to add staff members and new projects critical in the effort to learn more about CF. It also includes twice as much room for stem cell1 research, which occupied a small space in the original lab. An example of one of these new projects that Foundation researchers are undertaking at the lab is examining ways to create healthy lung or lung progenitor cells in the lab using genetic therapies. The goal is to ultimately engraft such cells into the lungs of people with CF, where healthy cells can expand into more healthy cells. The lab also will focus on developing a screening test to see what kind of additional treatment would help get gene delivery technology past the natural barriers of the lung to the native lung stem cells.
While the lab pursues work on genetic therapies, it will continue to concentrate on other potential treatments for people with nonsense and rare mutations who are not eligible to take modulators. For example, lab researchers are exploring ways to promote the development of small molecules to help the protein-making machinery of the cell override premature stop signals caused by nonsense mutations so that a full-length cystic fibrosis transmembrane conductance regulator (CFTR) protein can be made. A defective CFTR protein is the underlying cause of cystic fibrosis.
The lab’s work is part of the Foundation’s overall mission of finding a treatment for the underlying cause of cystic fibrosis for everyone with the disease. The Foundation is investing more aggressively than ever before to help people with CF live their longest, healthiest lives possible.
- 1The lab uses induced pluripotent stem cells, which are derived from adult human cells.