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ProQR Experimental RNA Therapeutic for Rare Eye Disease Misses Endpoints in Late-Stage Trial

ProQR Experimental RNA Therapeutic for Rare Eye Disease Misses Endpoints in Late-Stage Trial
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Outlook dims for ProQR s RNA therapy for vision loss following failure in key test

The lead RNA therapy from ProQR Therapeutics has failed the main and secondary goals of a pivotal study testing it as a treatment for a rare, inherited vision disorder. Despite the disappointing preliminary data, the biotech said it remains confident in the technology that produced the experimental therapy, sepofarsen, and the Phase 2/3 study will continue.

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