Ann: Successful Major Milestone - Human Retina in a Dish Models, page-5

While it’s unclear at this stage precisely which retinal disease indication PYC will target in its clinical program, it’s interesting to compare today’s results in a retinal organoid with those of ProQR’s QR-421a for the treatment of retinitis pigmentosa due to USH2A exon 13 mutations.

Development of QR-421a is currently at Phase 1/2 stage with results expected next year. In Pro QR’s published pre-clinical study results, a single intravitreal injection in a retinal organoid or optic cup model at a dose of 5µm achieved somewhat less than 50% exon-skipping.

PYC’s result of 91% exon-skipping after a single 5µm  dose looks very promising.

https://www.proqr.com/wp-content/up...ment-of-RP-due-to-USH2A-exon-13-mutations.pdf

https://www.proqr.com/wp-content/uploads/2019/05/ProQR_QR-421a_ARVO-2019_Presentation.pdf