Strategy for Pre-Clinical Development of Active Targeting MicroRNA Oligonucleotide Therapeutics for Unmet Medical Needs

Marc Thibonnier; Sujoy Ghosh

doi:10.3390/ijms24087126

Strategy for Pre-Clinical Development of Active Targeting MicroRNA Oligonucleotide Therapeutics for Unmet Medical Needs

Int J Mol Sci. 2023 Apr 12;24(8):7126. doi: 10.3390/ijms24087126.

Authors

Marc Thibonnier¹, Sujoy Ghosh²

Affiliations

¹ AptamiR Therapeutics, Inc., Austin, TX 78759, USA.
² Duke-NUS Medical School, Singapore and Pennington Biomedical Research Center, Baton Rouge, LA 70808, USA.

Abstract

We present here an innovative modular and outsourced model of drug research and development for microRNA oligonucleotide therapeutics (miRNA ONTs). This model is being implemented by a biotechnology company, namely AptamiR Therapeutics, in collaboration with Centers of Excellence in Academic Institutions. Our aim is to develop safe, effective and convenient active targeting miRNA ONT agents for the metabolic pandemic of obesity and metabolic-associated fatty liver disease (MAFLD), as well as deadly ovarian cancer.

Keywords: MAFLD; active targeted delivery; diabetes; microRNAs; obesity; oligonucleotide therapeutics; ovarian cancer.

MeSH terms

Humans
MicroRNAs* / genetics
MicroRNAs* / metabolism
Non-alcoholic Fatty Liver Disease*
Oligonucleotides / therapeutic use

Substances

MicroRNAs
Oligonucleotides

Grants and funding

This research received no external funding. Funding for this R&D work came from AptamiR Therapeutics, Inc. Sujoy Ghosh was partially supported by Louisiana Clinical and Translational Science Center grant (NIGMS 2U54GM104940) and by the National Medical Research Council, Singapore.