ProQR receives exclusive worldwide license for IONIS-RHO-2.5Rx, now QR-1123, for autosomal dominant retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy
The trial, called WINGS, will evaluate the safety and efficacy of QR-313 in subjects with recessive dystrophic epidermolysis bullosa due to mutations in exon 73 of the COL7A1 gene.
… for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. The company will also … RNA medicines for the treatment of severe genetic rare diseases, today announced that eight out of twelve …
… RNA medicines for the treatment of severe genetic rare diseases, today announced results from a planned interim … utility of RNA-based therapeutics in a human photoreceptor disease, particularly one with a severe unmet medical need. …
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra