IONIS PHARMACEUTICALS INC

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a biopharmaceutical company pioneering RNA-targeted medicines to treat serious diseases. The company's website is www.ionis.com.

Founded in 1989 and incorporated in Delaware, Ionis has evolved into a fully integrated commercial-stage biotechnology company with a portfolio of seven marketed medicines and a robust pipeline of investigational therapies. The company specializes in discovering and developing life-changing medicines that target RNA, a molecule carrying genetic instructions encoded in DNA, enabling precise modification of gene function and expression to address disease root causes.

Ionis' marketed medicines address serious conditions across multiple therapeutic areas. TRYNGOLZA treats familial chylomicronemia syndrome, DAWNZERA prevents hereditary angioedema attacks, and WAINUA addresses hereditary transthyretin-mediated amyloidosis polyneuropathy. The company also commercializes SPINRAZA for spinal muscular atrophy, QALSODY for SOD1-ALS, TEGSEDI for transthyretin-mediated amyloidosis, and WAYLIVRA for familial chylomicronemia syndrome and familial partial lipodystrophy. The company generated revenues of $944 million in 2025 and maintained a cash position of $2.7 billion in cash, cash equivalents and short-term investments.

Beyond marketed medicines, Ionis operates a substantial pipeline including late-stage programs for severe hypertriglyceridemia, Alexander disease, and Angelman syndrome. The company has established strategic partnerships with leading pharmaceutical companies including AstraZeneca, Biogen, GSK, Novartis, Otsuka, and Roche, generating both commercial revenue through royalties and R&D revenue through milestone payments and licensing fees.

Ionis' competitive advantages stem from its proprietary RNA-targeting technology platform and recent advancements including mesyl phosphoramidate backbone chemistry, Bicycle technology for tissue-specific delivery, and Vect-Horus platform technology for blood-brain barrier penetration. The company has also entered collaborations for gene editing capabilities through Metagenomi. These technological innovations position Ionis to address an expanding range of genetic and rare diseases with limited treatment options, supporting its strategy of independent and partnered product launches anticipated over the coming years.