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
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