ProQR Remains Confident In Pipeline After Sepofarsen Flop, Analysts Not So Much
The company is pinning hopes on its Usher syndrome and retinitis pigmentosa programs, but the disconnect between Phase I/II success and Phase II/III failure dented analysts’ enthusiasm.
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Sio Gene, Genocea, Solid Biosciences, Saniona and Black Diamond join First Wave, Zosano, Vallon, Finch, Magenta, ProQR and Imara on the growing list of biopharma firms reducing headcount to make their cash last until valuations return to a level that may support new fundraising.
ProQR’s Axiomer platform can enable single nucleotide RNA editing, an approach that could address thousands of disease-causing genetic mutations, the company says.
The Dutch biotech has firmed up its finances as its promising pipeline of RNA therapies for rare genetic eye disease starts to move through the clinic.