ProQR readies second antisense program for pivotal testing

ProQR is readying to advance a second RNA-based antisense oligonucleotide therapy for inherited retinal diseases into pivotal testing on the back of positive early data for QR-421a late last month and a $90 million follow-on.  

ProQR Therapeutics N.V. (NASDAQ:PRQR) reported a single dose of QR-421a improved visual acuity, visual fields and retinal sensitivity in the Phase I/II Stellar trial in adults with Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations. ...