Work

Therapeutic oligonucleotides as genetic medicines: The current state and future perspective

Year: 2026

Type: review

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.

Source: Cell Reports Medicine

DOI: 10.1016/j.xcrm.2026.103058

Authors: Bruno Miguel Da Cruz Godinho, Chantal M. Ferguson, Annemieke Aartsma-Rus

Institutions: Massachusetts General Hospital, Leiden University Medical Center

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $5,620