IONS
Ionis Pharmaceuticals is a biotech company specializing in RNA-targeted therapies, using antisense technology to treat genetic diseases. Based in Carlsbad, California, Ionis developed key drugs like Spinraza (for SMA), Wainua (for amyloidosis), and Tryngolza (for FCS). It has a broad pipeline in neurology, cardiology, and rare diseases, and is shifting toward independent drug commercialization.