This program is a first-of-its-kind collaboration to spur the development of genetic-based therapies for cystic fibrosis. Through this agreement, Pioneering Medicines will combine technologies from several companies to develop potential treatments for CF.

At the onset, the program will focus on developing technology to determine whether it can create a functional CFTR protein in lung cells, and a gene writing approach that may enable the simultaneous correction of numerous types of mutations in the CFTR gene. These two strategies will be combined with a delivery approach focused on targeting the proper cells in the lung and potentially other tissues.

Status

Laboratory studies to develop and test these technologies are underway.

Sponsor

This program is sponsored by Pioneering Medicines, an initiative of Flagship Pioneering, and partially funded by the Cystic Fibrosis Foundation.