From Partnered ASO Therapies To A Wholly-Owned Pipeline With Ionis's Brett Monia, Ph.D.

Ionis Pharmaceuticals, a pioneer in RNA therapeutics, has successfully transitioned from a partnership-dependent research organization to a fully integrated biotech company with its own commercialization capabilities. Founding scientist and CEO Brett Monia details the evolution of antisense oligonucleotide (ASO) technology, which targets RNA to prevent the production of disease-causing proteins. The company overcame significant scientific and financial hurdles over three decades to prove the efficacy of this modality. By shifting focus toward a wholly owned pipeline in cardiometabolic and neurological diseases, Ionis has moved from relying on large pharmaceutical partners to delivering its own breakthrough medicines directly to patients. With multiple FDA approvals expected this year, including treatments for severe hypertriglyceridemia and Alexander disease, the company is scaling its manufacturing and commercial infrastructure to support a broader patient population while maintaining its core commitment to scientific innovation.