QR-010 is an oligonucleotide designed to repair CFTR-encoded mRNA, which could result in a normal CFTR protein in people who have one or two copies of the F508del mutation. It is delivered via inhalation.
A Phase 1b trial to assess the safety and tolerability of QR-010 is underway. Another Phase 1 trial that is underway is a proof of concept study to evaluate the effect of QR-010 on nasal CFTR function.
This program is sponsored by ProQR Therapeutics and partially funded by Cystic Fibrosis Foundation Therapeutics (CFFT). It is being conducted within CFFT's Therapeutics Development Network.
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