The Massachusetts-based company will use the funds to develop a customized vehicle for the delivery of gene-based therapies into the lungs of individuals with cystic fibrosis. According to the foundation, traditional gene therapy approaches use harmless viruses, called vectors, to deliver healthy genes into cells. But viral vectors are particularly susceptible to mechanisms in the lungs that protect against foreign intruders, severely impairing their efficacy. The technology developed by enGene is non-viral and includes small, dense structures of DNA that are coated with molecules to make them more effective at penetrating mucus.
"Today's announcement reflects yet another milestone on our path to delivering transformative therapies for people with CF," said CFF executive vice president and chief scientific officer William Skach. "This is a very early but important step in advancing a viable way to produce healthy CFTR genes in the lungs."