Proqr Therapeutics B.V. (PRQR)

[JohnLocke101]

“QR-421a was observed to be well tolerated at all doses in participants with exon 13 mutations in USH2A. QR-421a demonstrated encouraging evidence of disease stabilization in visual acuity and clinically significant improvements in retinal sensitivity, supported by objective retinal structural imaging data, after a single dose compared with UE and SE. An open-label extension trial is planned to follow up the Stellar trial participants, and 2 Phase 2/3 trials are planned."