FDA Designation: Usher Syndrome

ProQR have received Rare Paediatric Disease designation from the FDA for QR-421a, an investigational gene therapy which targets the USH2A gene responsible for causing Usher Syndrome Type 2 and non-syndromic Retinitis Pigmentosa.

QR-421a works to restore the functionality of the Usherin protein produced by the USH2A gene and is intended for administration through intravitreal injections into the eye. This approval means that QR-421a offers a treatment option to people living with Usher Syndrome type 2 and non-syndromic Retinitis Pigmentosa to potentially stop or reverse their vision loss.

QR-421a marks the second designation received by ProQR for rare paediatric diseases, following the approval of Sepofarsen for treating Leber Congenital Amaurosis 10, which you can read about on the ProQR website here.


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