RNA therapies
Two key drug approvals have re-energised the field after a period of uncertainty.
This Nature Outlook is editorially independent, produced with financial support from a third party. About this content.
Nature is pleased to acknowledge the financial support of Ionis Pharmaceuticals in producing this Outlook. The sponsor retains sole responsibility for the following message.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.
