ProQR Therapeutics (NASDAQ:PRQR) announces that it received Orphan Drug designation from the FDA for QR-1123.
The company says QR-1123 is a first-in-class investigational antisense oligonucleotide designed to address the underlying cause of vision loss associated with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene.
ProQR’s stated goal is to develop and actively advance a pipeline of programs that can treat inherited retinal diseases like adRP in a targeted manner.
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