Antisense drugs for rare and ultra-rare genetic neurological diseases

Madelyn E McCauley; C Frank Bennett

doi:10.1016/j.neuron.2023.05.027

Antisense drugs for rare and ultra-rare genetic neurological diseases

Neuron. 2023 Aug 16;111(16):2465-2468. doi: 10.1016/j.neuron.2023.05.027. Epub 2023 Jun 23.

Authors

Madelyn E McCauley¹, C Frank Bennett²

Affiliations

¹ Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.
² Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA. Electronic address: fbennett@ionisph.com.

PMID: 37354903
DOI: 10.1016/j.neuron.2023.05.027

Abstract

The regulatory approvals of nusinersen and tofersen, plus the large body of clinical and preclinical data from other drugs, have significantly de-risked antisense technology for neurological diseases. The platform learnings over the last 2 decades can be applied to subsequent drugs to improve the efficiency of discovering effective neuro-therapeutics.

MeSH terms

Humans
Nervous System Diseases* / drug therapy
Nervous System Diseases* / genetics
Oligonucleotides, Antisense* / therapeutic use

Substances

Oligonucleotides, Antisense