Ionis Pharmaceuticals

Ionis Pharmaceuticals is a biotechnology company focused on developing RNA-targeted drugs. Its technology platform in the antisense space allows for the control of protein production and reduction at a genetic level. Ionis´ antisense drugs are designed to interact precisely with RNA, allowing them to create treatments that disrupt the disease process and change its course. The company – on its own and in collaboration with its partners – has brought five drugs to the market already and currently has more than ten drug candidates in mid- to late-stage clinical development. The five approved drugs are Spinraza (partnered with Biogen) for spinal muscular atrophy, Tegsedi for hereditary transthyretin amyloidosis polyneuropathy, Waylivra for familial chylomicronemia syndrome, Qalsody (partnered with Biogen) for ALS and Wainua (partnered with AstraZeneca) for polyneuropathy.