ultevursen

Ultevursen is a first-in-class, single-stranded antisense oligonucleotide designed to treat vision loss associated with mutations in exon 13 of the USH2A gene, which encodes the usherin protein. It works by an exon-skipping mechanism that allows retinal cells to produce functional usherin protein despite the presence of disease-causing mutations. Ultevursen is administered via intravitreal injection and is being developed primarily for retinitis pigmentosa and Usher syndrome type 2 caused by USH2A exon 13 mutations. The drug was originally developed by ProQR Therapeutics and later acquired for further development by Laboratoires Théa (and its subsidiary Sepul Bio). Ultevursen has received orphan drug designation in both the US and EU[1][2][3][5][6].

Brand names

ultevursen

Other names

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