Progress and promise of antisense oligonucleotide therapeutics for central nervous system diseases

- Antisense oligonucleotides (ASOs) are an emerging class of therapeutics to treat diseases of the central nervous system.
- ASOs for a variety of targets and mechanisms are currently being investigated for a number of CNS diseases.
- To date, five ASO therapeutics have entered clinical development.
- These include nusinersen for Spinal Muscular Atrophy, which recently reported positive results in two Phase 3 studies.
- This review examines central ASO delivery, distribution, pharmacokinetics, pharmacodynamics and therapeutic opportunities.

Antisense oligonucleotide (ASO) drugs are an emerging class of therapeutics that have recently demonstrated progress and promise to treat diseases of the central nervous system (CNS). ASOs for a variety of targets and mechanisms are currently being investigated in clinical trials and pre-clinically for a number of CNS diseases. This review examines the available data regarding central ASO delivery, distribution, pharmacokinetics, pharmacodynamics and therapeutic opportunities.This article is part of the Special Issue entitled “Beyond small molecules for neurological disorders”.