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Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Published Online: 3 April 2024
Abstract
Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of nucleic acid-based therapies has been strengthened by recent regulatory approvals and tremendous clinical success. In this review, we look at the major obstacles that have hindered the field, the historical milestones that have been achieved, and what is yet to be resolved and anticipated soon. This review provides a view of the key innovations that are expanding nucleic acid capabilities, setting the stage for the future of nucleic acid therapeutics.
Introduction
It has been almost 25 years since the first nucleic acid-based therapeutic—fomivirsen—was approved in 1998 [1]. Nucleic acid therapeutics (NATs) are drugs with nucleic acids as their therapeutically active component, such as DNA or RNA of all forms [2]. Since 1998, this new ground-breaking class of drugs has expanded to more than 20 approved products, with hundreds of ongoing clinical trials worldwide [3–6]. The NAT modalities approved and under investigation harness different mechanisms of action with an ever-expanding chemistry, conjugation, and delivery platforms. In this perspective, we provide a short overview of (a) the challenges NATs must overcome, (b) the progress of NATs to date, and (c) highlight new therapeutics that may enter the clinic, hit clinical milestones, or achieve regulatory approval in 2024 and beyond. Thanks to the significant innovation opening new organs and diseases for NAT applications, we discuss the potential of NATs as a new drug class, offering a future for patients with uncurable diseases.
