Guided by world-class scientists and business leaders, Ionis is THE leading innovator in RNA-targeted therapeutics. The company was designed to create a better, more efficient drug discovery platform; to maximize the value of every medicine they create; and to get these medicines to the sick people who are depending on them as quickly and as efficiently as possible.
Ionis Pharmaceuticals antisense RNA-targeted therapies target the underlying cause of many human diseases – proteins that are over or under produced, or mutated. Traditional medicines focus on these proteins after they are produced and begin to damage the body, whereas the Ionis antisense therapies identify, select and destroy mRNA specifically, so that the disease-causing protein is eliminated before being produced. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normal levels for disease caused by loss of gene function. Ionis has brought this solution to patients with approved products SPINRAZA (Spinal Muscular Atrophy), TEGSEDI (polyneuropathy caused by hereditary transthyretin-mediated amyloidosis) and WAYLIVRA (familial chylomicronemia syndrome). Ionis has a deep pipeline with four Phase 3 trial initiations this year – IONIS-HTTRx for Huntington’s disease, Tofersen for a familial form of ALS, AKCEA-APO(a)-LRx cardiovascular disease caused by elevated levels of Lp(a), and AKCEA-TTR-LRx for TTR amyloidosis and has up to 10 therapies entering into Phase 3 trials by the end of 2020.
