Ionis Pharmaceuticals, Inc. is engaged in the discovery and development of RNA-targeted therapeutics. The company’s Phase 3 product candidates include:
Nusinersen: Treatment for spinal muscular atrophy (SMA)
IONIS-TTR Rx: Treatment for transthyretin amyloidosis
Volanesorsen: Treatment for conditions that are associated with elevated triglycerides including familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL)