VALIDATION OF THE ANTISENSE DRUG.
Schmidt believes, “Ionis Pharmaceuticals has validated its antisense platform by successfully shepherding systemic antisense drugs to commercial feasibility. We believe antisense candidates have the best chance of success when directed at treating orphan diseases that lack treatment alternatives. Prime examples within Ionis’s pipeline include candidates for spinal muscular atrophy (SMA), TTR amyloidosis, and familial chylomicronemia syndrome (FCS).”
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