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news.ProQR Therapeutics has started enrollment in its Phase Ib clinical trial (PQ-010-001) of QR-010, a new investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of cystic fibrosis (CF) patients due to the DF508 mutation.
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QR-010, a RNA-based oligonucleotide, is designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of these patients.
The 28-day randomized, double-blind, placebo-controlled trial will be conducted in 20 centers across the world and will evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 in 64 CF patients carrying two copies (homozygotes) of the DF508 mutation.
The trial will also assess sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function.
ProQR chief development officer Noreen Henig said: "The Phase Ib study and the NPD proof-of-concept study will provide a strong, early signal as to the therapeutic potential of QR-010."
During the trial, QR-010 will be administered through inhalation for up to three times a week for a maximum period of four weeks.
This Phase Ib trial will be carried out in parallel with a proof-of-concept Nasal Potential Difference (NPD) study, which will begin enrollment of 16 CF patients that are either homo- or heterozygous for the DF508 mutation in third quarter of 2015.