QR-1123, a first-in-class investigational antisense oligonucleotide for the treatment of vision loss associated with autosomal dominant retinitis pigmentosa, received an orphan drug designation from the FDA, ProQR announced in a press release.
“We are pleased to have orphan drug designation for our QR-1123 program targeting autosomal dominant retinitis pigmentosa, or adRP,” said Daniel de Boer, CEO of ProQR. “It highlights the unmet need for patients with this progressive disease-causing blindness. Our goal is to develop and actively advance a pipeline of programs that can
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