Shots:

• The US FDA has granted RPD to the QR-421a for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene
• The RPD designation provides PR voucher by the FDA to the sponsor- that voucher can be redeemed to receive FDA’s PR designation for any subsequent MAA for a different product
• QR-421a (IVT) is a first-in-class investigational RNA-based oligonucleotide addressing the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic RP due to mutations in exon 13 of the USH2A gene and has received FDA’s ODD and EU’s FT designation

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