QR-1123, a first-in-class investigational antisense oligonucleotide for the therapy of vision loss associated with autosomal leading retinitis pigmentosa, obtained an orphan medicine classification from the FDA, ProQR announced in a press release.
” We are pleased to have orphan drug classification for our QR-1123 program targeting autosomal leading to retinitis pigmentosa, or adRP,” said Daniel de Boer, Chief Executive Officer of ProQR. “It highlights the unmet need for individuals with this progressive disease, causing blindness. Our objective is to develop and actively break through a pipe of programs that can treat inherited retinal diseases like adRP in a targeted way.”
QR-1123, which is made to be administered via intravitreal injections, intends to prevent the harmful variation of the rhodopsin protein by binding the P23H mutation in the RHO genetics.
Discovered and also developed by Ionis Pharmaceuticals, it was licensed by ProQR in 2018 as well and has since received IND clearance and a fast lane designation from the FDA.