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Criteria for developing active cellular targeting miRNA oligonucleotide therapeutics with a peptide nucleic acid backbone: Combating cardiometabolic pandemics

Marc Thibonnier1*
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1 Aptamir Therapeutics, Inc., Naples, Florida, United States of America
INNOSC Theranostics and Pharmacological Sciences 2024, 7(3), 3025 https://doi.org/10.36922/itps.3025
Submitted: 26 February 2024 | Accepted: 25 April 2024 | Published: 16 July 2024
© 2024 by the Author(s). This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution 4.0 International License ( https://creativecommons.org/licenses/by/4.0/ )
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Abstract

Oligonucleotide therapeutics (ONTs) represent a growing new class of therapeutic agents aimed at addressing chronic diseases that remain untreatable by small molecules and antibodies. Our goal was to establish a selection of several criteria to design and develop miRNA-based ONTs, focusing on improved chemistry, pharmacokinetics/pharmacodynamics (PK/PD) profiles, and safety characteristics to combat cardiometabolic pandemics. By leveraging our own experimental data obtained from experiments involving miR-22-3p antagomirs and a careful review of the literature, we established a set of seven criteria to optimize the design of miRNA ONTs. These criteria prioritize simplified drug synthesis, optimized PK/PD properties, and reduced potential toxicities. This proposed set of seven criteria represents a novel strategy for developing active cellular targeting miRNA ONTs for various therapeutic indications.

Keywords
MiRNAs
Antagomirs
Oligonucleotide therapeutics
Active cellular targeted delivery
Cardio-metabolic pandemics
Funding
This research received no external funding. Funding for this R and D work was provided by AptamiR Therapeutics, Inc.
Conflict of interest
Marc Thibonnier is the founder and a shareholder of AptamiR Therapeutics, Inc.
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INNOSC Theranostics and Pharmacological Sciences, Electronic ISSN: 2705-0823 Print ISSN: 2705-0734, Published by AccScience Publishing
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