November 02, 2018
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ProQR granted license to Ionis’ retinitis pigmentosa candidate

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ProQR Therapeutics has acquired worldwide licensing rights to Ionis Pharmaceuticals’ QR-1123, an RNA medicine for autosomal dominant retinitis pigmentosa caused by the P23H mutation in the rhodopsin gene, according to a press release.

ProQR made an upfront payment in ordinary shares in the amount of $2.5 million to Ionis and will make future milestone payments. ProQR received a worldwide license to the drug and its relevant patents.

“Unlike other molecules we are developing, QR-1123 is a gapmer with a mutant allele-specific knockdown mechanism of action,” ProQR CEO Daniel A. de Boer said in the release. “If validated, it would further broaden the potential of RNA-targeted therapies in retinal diseases. We are excited to start clinical development to explore QR-1123’s potential in helping patients with P23H adRP.”

ProQR plans to begin a phase 1/2 clinical trial in 2019 pending submission and clearance of an investigational new drug application by the FDA, the release said.