RNA therapy shows positive trial results for rare blindness disorders

Dutch rare diseases biotech firm ProQR Therapeutics (Nasdaq: PRQR) saw its share rise as much as 11% in early trading today, after it announced encouraging results from a planned analysis of its Phase I/II Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.

In the trial, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. QR-421a was observed to be well tolerated with no serious adverse events reported. Based on these findings, the company plans to advance QR-421a to two parallel pivotal Phase II/III trials by year end 2021 – one in early-moderate patients, one in advanced patients.

Early last year, the US Food and Drug Administration awarded a Rare Pediatric disease designation for QR-421a, designed to address the underlying cause of vision loss related to this type of genetic abnormality.