Ionis Pharmaceuticals (NASDAQ:IONS), founded in 1989 and headquartered in Carlsbad, California, is a pioneer in RNA-targeted drug discovery and development. The company’s proprietary antisense technology enables the design of therapeutic agents that selectively bind to RNA to inhibit, enhance, or modulate the production of disease‐causing proteins. Over three decades of innovation have positioned Ionis as a leader in advancing novel treatments for a wide range of serious and rare diseases.
Ionis’s product portfolio includes several approved therapies developed in collaboration with global partners. Key launches include SPINRAZA (nusinersen) for spinal muscular atrophy, co-developed with Biogen, as well as Tegsedi (inotersen) and Waylivra (volanesorsen) for rare forms of transthyretin amyloidosis and familial chylomicronemia syndrome. Beyond these marketed products, Ionis maintains a robust pipeline targeting cardiovascular, metabolic, neurological and oncology indications, with numerous candidates in late-stage clinical trials.
The company operates research and development facilities across North America, Europe and Asia, and collaborates with leading pharmaceutical organizations such as Roche, AstraZeneca and Biogen. These strategic alliances enable Ionis to leverage external expertise and resources, accelerating the transition of its RNA-targeted therapies from discovery through regulatory review. Ionis’s collaborative model ensures efficient advancement of programs while retaining control over its core antisense platform.
Under the leadership of CEO Brett P. Monia, Ph.D., Ionis continues to expand its scientific capabilities and global footprint. Supported by a board of directors with extensive industry experience, the management team remains focused on translating RNA science into therapeutic solutions. Ionis is dedicated to addressing unmet medical needs and improving patient outcomes by advancing innovative treatments based on its pioneering antisense technology.
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