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Lookup NU author(s): Dr Othman AlmusaimiORCiD, Dr Danah Alshaer
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
In 2024, the FDA approved fifty novel drugs, including four peptides and oligonucleotides (TIDEs) (two pepTIDEs and two oligonucleoTIDEs), highlighting their increasing importance as effective alternatives to traditional drug classes. TIDEs provide essential therapies for complex diseases, such as genetic disorders, rather than merely addressing symptoms. In addition to oligonucleotide therapeutics for various genetic conditions, peptides became the first approved treatment for Rett Syndrome in 2023 and were also used to treat Niemann–Pick disease type C (NPC) in 2024. Interestingly, among the strategies employed in recent approvals to enhance stability and/or delivery, the prodrug approach, exemplified by palopegteriparatide and pegulicianine, is emerging as a more targeted and precise therapeutic strategy. Additionally, the Enhanced Stabilization Chemistry (ESC)-GalNAc platform has been expanded for hepatic delivery of a new oligonucleotide drug, olezarsen. Furthermore, novel modifications to the ribose moiety in oligonucleotides, such as the 3′-amino substitution in imetelstat, enhance their stability. This review examines the TIDES approved in 2024 based on their chemical structure, medical targets, modes of action, administration routes, and common adverse effects. In addition, it highlights how the prodrug strategy has improved targeting efficiency and extended the half-lives of the active drugs.
Author(s): Al Musaimi O, Alshaer D, de la Torre BG, Albericio F
Publication type: Article
Publication status: Published
Journal: Pharmaceuticals
Year: 2025
Volume: 18
Issue: 3
Online publication date: 20/02/2025
Acceptance date: 18/02/2025
Date deposited: 10/03/2025
ISSN (electronic): 1424-8247
Publisher: MDPI AG
URL: https://doi.org/10.3390/ph18030291
DOI: 10.3390/ph18030291
Data Access Statement: Data sharing is not applicable.
Notes: This article belongs to the Section Biopharmaceuticals.
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