DRUG DEVELOPMENT PIPELINE

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QR-010

Phase One

Phase One

Therapeutic Approach

Restore CFTR Function

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.

Status

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. 

Sponsor

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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