Ionis

Ionis Pharmaceuticals, Inc., commonly referred to as Ionis, is an American biotechnology company that develops antisense therapies. It is headquartered in Carlsbad, California, and trades on the NASDAQ under the ticker IONS. The company originated in 1992 as Isis Pharmaceuticals and changed its name to Ionis in 2015 to reflect its RNA-targeting focus.

Ionis specializes in antisense oligonucleotides (ASOs), short strands of synthetic nucleic acids designed to bind RNA transcripts and modulate gene expression. The platform has evolved with chemical modifications to improve stability, potency, and delivery, enabling systemic and central nervous system applications. Ionis pursues a broad pipeline addressing neurological, cardiovascular, metabolic, and other diseases, often through collaborations with larger pharmaceutical partners.

A number of Ionis programs have achieved clinical and regulatory milestones through collaborations. Spinraza, nusinersen, is a leading example developed with Biogen for spinal muscular atrophy and marketed by Biogen. Akcea Therapeutics, a wholly owned Ionis subsidiary, markets Volanesorsen (Waylivra) for familial chylomicronemia syndrome in some markets, with a regulatory and safety history that includes significant scrutiny. Ionis maintains additional collaborations with Biogen and other partners to develop multiple antisense therapies, including tofersen (IONIS-SOD1Rx) for SOD1-related amyotrophic lateral sclerosis and other candidates in late-stage development.

Financially, Ionis funds its research and development through collaboration agreements, upfront payments, milestone incentives, and potential royalties from partner programs. The company emphasizes its antisense platform as a core capability, aiming to expand RNA-targeted therapies across diverse disease areas.