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ProQR’s retinitis pigmentosa candidate gets investigational NDA clearance

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The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.

The company plans to begin a first-in-human phase 1/2 trial of QR-1123 by the end of this year, with a single-dose escalation open-label arm and a multiple-dose double-masked arm, the release said. Safety, tolerability, pharmacokinetics and efficacy will be evaluated.

“We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,” ProQR CEO Daniel A. de Boer said in the release. “This represents our fifth IND in less than 5 years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with [autosomal dominant retinitis pigmentosa].”

QR-1123 aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by binding the mutated RHO mRNA, casing allele specific knockdown of the mutant mRNA. It is intended to be administered through intravitreal injection.