Antisense oligonucleotide platform landscape (2026 reference)
Reference snapshot of approved ASO therapies, late-stage programs, and the delivery and chemistry platforms behind them.
Approved antisense oligonucleotide therapies in 2026 span multiple indications. Nusinersen (Spinraza) in spinal muscular atrophy, eteplirsen and follow-ons in Duchenne muscular dystrophy, inotersen and tofersen in transthyretin amyloidosis and ALS respectively, and several others.
Delivery platforms split into systemic subcutaneous administration (most ASOs), intrathecal injection (where CNS targeting is required, including SMN1 and SOD1 targeting), and emerging conjugate platforms (GalNAc for hepatic targeting, others for tissue-specific delivery). The chemistry platforms (2'-MOE, LNA, others) define delivery options, half-life, and toxicity profiles.
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