Antisense technology at Ionis has advanced to the state in which we have 38 drugs in development and three significant programs in different patient populations completing phase 3 studies. The pipeline is comprised of three different chemical classes and exploits several different mechanisms of action and routes of administration which demonstrate the value and versatility of the technology. Unlike most drug discovery platforms, antisense technology continues to advance. We hope to have drugs with a human ID50 dose of 1–5mg/week for liver targets next year. The longer-term future is even more exciting. As we exponentially add new knowledge about the molecular pharmacology of ASOs, the future is created. The translation of these advances into drugs promises an even more substantial enhancement of therapeutic index and patient convenience. In this presentation, the exciting present and even the more compelling future of antisense technology will be discussed.

Award Address (E. B. Hershberg Award for Important Discoveries in Medicinally Active Substances sponsored by Merck Research Laboratories). Present and future of antisense technology

253rd National Meeting (2017)

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