Profile

Ionis Pharmaceuticals is a biotechnology company based in Carlsbad, California that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza , Tegsedi , and WAYLIVRA and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis. The company was named Isis Pharmaceuticals until December 2015.