Therapeutic oligonucleotides as genetic medicines: The current state and future perspective
- 1Board of Directors, Oligonucleotide Therapeutics Society, San Diego, CA, USA
- 2Massachusetts General Hospital, Boston, MA, USA
- 3Leiden University Medical Center, Leiden, ZH, the Netherlands
Abstract
Antisense therapeutic oligonucleotides are entering a transformative era in clinical development, with a total of 21 oligonucleotides approved and a robust pipeline poised for the coming years. These drugs have emerged as a cornerstone of genetic medicine, providing a programmable platform to modulate gene expression and treat otherwise untreatable disorders. This review covers the different mechanisms of action of these drugs and highlights the key advances in oligonucleotide chemistry and delivery that have supported the clinical successes witnessed in the last decade. It also provides a historical account and overview of the growing number of approvals and key phase 3 studies. Finally, it offers a perspective on the future of the field, initially focused only on rare diseases and now expanding to common disorders.
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BibTeX
@article{godinho2026therapeutic,
title = {{Therapeutic oligonucleotides as genetic medicines: The current state and future perspective}},
author = {Bruno Miguel Da Cruz Godinho and Chantal M. Ferguson and Annemieke Aartsma-Rus},
journal = {Cell Reports Medicine},
year = {2026},
month = sep,
doi = {10.1016/j.xcrm.2026.103058},
url = {https://doi.org/10.1016/j.xcrm.2026.103058},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Therapeutic oligonucleotides as genetic medicines: The current state and future perspective
AU - Bruno Miguel Da Cruz Godinho
AU - Chantal M. Ferguson
AU - Annemieke Aartsma-Rus
PY - 2026
DA - 2026/09/22
JO - Cell Reports Medicine
DO - 10.1016/j.xcrm.2026.103058
UR - https://doi.org/10.1016/j.xcrm.2026.103058
AB - Antisense therapeutic oligonucleotides are entering a transformative era in clinical development, with a total of 21 oligonucleotides approved and a robust pipeline poised for the coming years. These drugs have emerged as a cornerstone of genetic medicine, providing a programmable platform to modulate gene expression and treat otherwise untreatable disorders. This review covers the different mechanisms of action of these drugs and highlights the key advances in oligonucleotide chemistry and delivery that have supported the clinical successes witnessed in the last decade. It also provides a historical account and overview of the growing number of approvals and key phase 3 studies. Finally, it offers a perspective on the future of the field, initially focused only on rare diseases and now expanding to common disorders.
SN - 2666-3791
ER -