ProQR granted license to Ionis’ retinitis pigmentosa candidate

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.

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.