Shares of ProQR Therapeutics (PRQR) have lost half of their value since my initial article was published in October of last year, which detailed impressive interim data for lead candidate QR-110 in patients with LCA10 and expressed my optimism over potential for their RNA treatments to address many other opportunities in the inherited retinal disease space. The stock was a loser for us in the ROTY model account the first time around as well, as I underestimated the negative impact of certain safety/tolerability concerns for sepofarsen.
As pointed out before, QR-421a for Usher