This program is developing a potential inhaled therapy to deliver normal CFTR messenger RNA (mRNA) to the lungs. Lung cells would then use the instructions in the mRNA to create functional CFTR protein. This type of therapy could work for any person with CF, regardless of their CFTR mutations.

Status

A Phase 1 study to test the safety and tolerability of this potential therapy is currently underway in New Zealand. The study initially enrolled healthy volunteers and is planned to expand to adults with CF later this year.

Sponsor

This program is sponsored by Arcturus Therapeutics and is partially funded by the Cystic Fibrosis Foundation.