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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Restructuring Updates: Homology implemented modest job cuts as it ended a gene therapy trial, while ProQR revealed its second round of layoffs this year and Synthetic Biologics shifted focus to oncolytic virus pipeline. Also, Bolt, MacroGenics and others deprioritized programs in their Q2 updates.
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.