Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases

RNA therapeutics comprise a diverse group of oligonucleotide-based drugs such as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and short hairpin RNAs (shRNAs) that can be designed to selectively interact with drug targets currently undruggable with small molecule-based drugs or monoclonal antibodies. Furthermore, RNA-based therapeutics have the potential to modulate entire disease pathways, and thereby represent a new modality with unprecedented potential for generating disease-modifying drugs for a wide variety of human diseases, including central nervous system (CNS) disorders. Here, we describe different strategies for delivering RNA drugs to the CNS and review recent advances in clinical development of ASO drugs and siRNA-based therapeutics for the treatment of neurological diseases and neuromuscular disorders.

Automated summary

RNA therapeutics are a diverse group of oligonucleotide-based drugs that can selectively interact with undruggable drug targets. They have the potential to modulate disease pathways and represent a new modality for generating disease-modifying drugs. This article describes different strategies for delivering RNA drugs to the CNS and reviews recent advances in ASO drugs and siRNA-based therapeutics for neurological diseases and neuromuscular disorders.