RNA therapies developer ProQR Therapeutics is eying a future without ophthalmology. After receiving European Medicines Agency feedback for its lead program for a rare eye disorder, the biotech has decided to find a strategic partner for all of its eye drug assets and apply its RNA technology to other therapeutic areas.
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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ProQR Therapeutics has encouraging data from an early-stage study evaluating its treatment for vision loss in the rare, inherited disorder Usher syndrome. The biotech now plans to advance its RNA therapy to two pivotal studies later this year.