The Cystic Fibrosis Foundation will provide up to $7.3 million to Ride Therapeutics to identify potential delivery vehicles to carry genetic therapies into the lungs of people with CF. Genetic therapies could one day offer a transformative treatment for everyone with CF, regardless of their mutations, but delivering those treatments into the correct lung cells is a significant challenge.

Delivery is difficult because the body’s natural defenses are designed to block germs and other foreign invaders from entering lung cells. Ride Therapeutics plans to build a library of millions of tiny particles (nanoparticles) from a variety of materials designed to work safely in the body. Similar to a pill capsule that protects medicines, the nanoparticles are designed to shield and direct genetic materials to cells without damaging the material along the way. 

Ride plans to test each of the nanoparticles, and the data from these millions of tests will be used to help train an AI system to design the best nanoparticles capable of delivering genetic therapies into the lung cells of people with CF. 

"The Foundation is committed to supporting multiple approaches to deliver transformative genetic therapies to all people with CF," said Steven M. Rowe, MD, MSPH, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. "This investment is part of our overall strategy to identify the best delivery method to reach target lung cells."

In addition to Ride’s work with nanoparticles, researchers are investigating other ways to overcome the delivery challenge by exploring options such as harmless viruses and tiny fat particles (lipid nanoparticles).

CF scientists are currently focusing on the challenge of delivering genetic therapies to the cells of the lung, because that is one of the organs most severely affected by the disease. Different delivery vehicles would be needed to deliver genetic therapies to other CF-affected organs, such as the intestine or pancreas.