RXi Pharmaceuticals, a clinical-stage biotechnology company focused on RNAi therapies, has exclusively licensed its sd-rxRNA technology to Thera Neuropharma to develop novel therapies for neurodegenerative diseases. The sd-rxRNA, or ‘self-delivering’ RNAi compounds, are designed with properties of both single-stranded antisense molecules and double-stranded RNAi, to generate highly potent compounds that are spontaneously taken up by cells. Building on work conducted at RXi in collaboration with Robert Brown of University of Massachusetts Medical School, Thera will focus initially on developing compounds that target superoxide dismutase 1 (SOD1) as drug candidates for SOD1 ALS. In addition, Thera will explore combination therapies of the SOD1 sd-rxRNAs compounds with its own broad-spectrum neuroprotective compounds.
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