BioCentury
ARTICLE | Product Development

A big year for Ionis

The RNA therapeutics company is moving from partnering to commercializing products, and from rare to common diseases

May 15, 2026 8:47 PM UTC

Ionis spent decades persuading investors, regulators and physicians that antisense oligonucleotides could become viable medicines as rivals abandoned the field or collapsed. After some false starts, approval of Spinraza for spinal muscular atrophy validated the approach, opening the door for the Carlsbad, Calif.-based biotech to assemble a portfolio of approved drugs — and for competitors to race through that door and develop their own ASOs.

Having proved the concept, Ionis Pharmaceuticals Inc. (NASDAQ:IONS) is shifting from primarily licensing its technology to larger pharmaceutical companies toward commercializing therapies itself.  ...