ProQR Therapeutics N.V. (PRQR +12.9%) is up on slightly higher volume ahead of a presentation on candidate QR-421a in Usher syndrome tomorrow at the Annual Usher Connections Conference in Philadelphia.
QR-421a is an investigational RNA-based therapeutic designed to exclude exon 13 from the USH2A gene’s messenger RNA, thereby removing the mutation that causes retinitis pigmentosa associated with Usher syndrome type 2.
Usher syndrome is an inherited disorder characterized by hearing and vision loss that worsens over time.
A Phase 1/2 study is in process with a primary completion date in December.
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