Hal Barron’s swan song as GSK R&D chief ends with a promise: The best is yet to come – Endpoints News

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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