In latest antisense failure, ProQR's rare eye disease treatment falls flat
It’s been a bad 12 months for antisense technology.
ProQR, an RNA-focused startup, announced Friday that its lead candidate for one form of a rare genetic eye disease called Leber congenital amaurosis 10 failed in a pivotal trial, showing no difference between placebo on any efficacy measures.
ProQR’s stock $PRQR was down 67% on the news, from $5.64 to $1.88.
The setback adds to a growing list of disappointments over the past year for antisense technology, a form of gene therapy that, after decades of development, had begun to show promise for treating a host of rare and not-so-rare diseases.
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