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Pioneering Medicines



Therapeutic Approach

Restore CFTR Function

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 a separate company specifically focused on potential treatments for CF. At the onset, the new company 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.


Laboratory studies to develop and test these technologies are underway.


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

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