The Cystic Fibrosis Foundation is providing additional funding to ReCode Therapeutics to further develop the company’s delivery method to carry a potential gene editing therapy to the lungs of people with CF.
For gene editing and other genetic therapies to work in CF, the treatment needs to reach the right cells in the lungs. This is one of the biggest challenges in developing a treatment that could benefit all people with CF — including those who cannot benefit from or tolerate modulators — because the body’s natural defenses are designed to block germs and other foreign invaders from entering cells.
ReCode is working to adapt its lipid nanoparticles so they can better reach certain lung cells, including lung stem cells that would be an ideal target for a permanent gene editing therapy. Like a pill capsule that protects medicines, lipid nanoparticles shield and direct genetic material to cells, preventing the material from potential damage along the way. Lung stem cells are important because they create new lung cells to replace ones that are damaged or dying.
ReCode plans to combine its delivery system with a gene editing therapy designed to correct mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is caused by CFTR mutations that result in dysfunctional or missing CFTR protein. If successful, gene editing could be used to correct the mutations permanently, so that functional CFTR protein can be made.
This investment builds on the Foundation’s existing relationship with ReCode. The Foundation previously provided funding for ReCode’s development of a messenger RNA genetic therapy that is being tested in a Phase 2 trial in people with CF. ReCode’s lipid nanoparticles are being used to deliver a normal template of instructions — mRNA — to lung cells to create healthy CFTR protein. The investments in both programs demonstrate the Foundation’s commitment to strategically advance the most promising science to deliver transformative therapies for all people with CF, especially for those who cannot benefit or tolerate modulators.