Inhalable modified tRNA restores lung protein function in cystic fibrosis mice, shows promise for genetic diseases

University of Toronto scientists have shown that an inhalable form of modified transfer RNA can safely reach the lungs of mice carrying a nonsense mutation that causes cystic fibrosis. The treatment remained in the lung tissue for up to 40 days and restored the function of the defective protein.

In organoid experiments using cells from cystic fibrosis patients, the researchers compared the new tRNA therapy with Vertex Pharmaceuticals' FDA‑approved drug Trikafta. Trikafta alone did not fully restore protein activity, but when combined with the modified tRNA, the cells regained normal function.

The team plans to advance the inhalable tRNA into clinical trials within the next two to three years. They are also evaluating the approach in other genetic conditions such as Duchenne muscular dystrophy and Rett syndrome, which currently lack effective treatments.