Narrow-Moat Ionis' Late-Stage Candidates Continue to Make Progress; Positive Long-Term Outlook

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.