AptamiR Therapeutics, Inc.

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.

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.