QR-421a has received rare pediatric disease designation from the FDA for the treatment of retinitis pigmentosa, according to a press release from ProQR Therapeutics.
QR-421a, an RNA-based oligonucleotide, is designed to address mutations in exon 13 of the USH2A gene, which cause vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa.
“We are pleased to have received the second rare pediatric designation for our clinical-stage development programs,” ProQR CEO Daniel A. de Boer said in the release. “Both sepofarsen for LCA10, the most common cause of blindness due to genetic disease in children, and QR-421a have received rare pediatric designation, underscoring the high unmet need for patients. We aim to make a difference for these patients as we work to advance our pipeline of programs to treat inherited retinal diseases.”