• ProQR Therapeutics

    An RNA-based gene therapy made by Dutch biotech company ProQR Therapeutics has received Orphan Drug Designation from the FDA and European Medicines Agency for treatment of Usher syndrome type II, a form of retinitis pigmentosa.

    The investigational product, called QRX-411, is an RNA oligonucleotide designed to restore a wild-type version of the USH2A messenger RNA, leading to production of functional copies of the USH2A gene. Some individuals with Usher II possess a known substitution mutation in this gene, which is involved in both photoreceptor cell homeostasis and inner ear cell differentiation. This form of the disease causes progressive central vision loss and impaired hearing.

    QRX-411 demonstrated promise in preclinical studies using patient fibroblasts and an induced pluripotent stem cell-derived optic cup model, according to data presented at ARVO 2017.

    Currently, there are no other therapies approved or in development to slow or reverse Usher II.

    “Securing orphan drug designations from the FDA and EMA for QRX-411 is a milestone for the program and highlights the importance of addressing the unmet need of this debilitating disease,” said Daniel A. de Boer, CEO of ProQR. “We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.”

    The company has stated the product is ready for Investigational New Drug (IND) application-enabling studies.