Ionis Pharmaceuticals (IONS)

Ionis has played a central role in bringing multiple RNA-targeted medicines to patients. Its collaboration with Biogen produced Spinraza, a treatment for spinal muscular atrophy that became an important proof point for antisense technology after its 2016 launch. The Biogen partnership also led to Qalsody, an ALS therapy launched in 2023. Through Akcea, its cardiovascular-focused subsidiary, Ionis commercialized Tegsedi for hereditary transthyretin amyloidosis and Waylivra for certain lipid disorders in Europe. The company also works with AstraZeneca, including on Wainua, a treatment for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

A key feature of Ionis is its hybrid model: it develops medicines both independently and with larger pharmaceutical partners. This allows it to share development risk while retaining opportunities to build its own commercial infrastructure. Its first independent launches, Tryngolza for familial chylomicronemia syndrome and Dawnzera for hereditary angioedema, mark a shift toward becoming a more fully integrated biotech company. For investors, Ionis represents exposure to the expanding field of RNA-based therapeutics, with value tied to clinical execution, regulatory outcomes, partner economics, and the adoption of its marketed rare-disease medicines.