AptamiR Therapeutics is focused on developing innovative oligonucleotide therapeutics (ONTs) that target microRNAs to address complex diseases such as obesity, diabetes, and epithelial ovarian cancer. Their therapeutic strategy leverages the pleiotropic concept of "One Drug-Multiple Targets," allowing their drug candidates to modulate several target genes simultaneously. This approach aims to treat conditions like cardiometabolic disorders and cancer without adversely affecting brain functions, thereby enhancing patients' quality of life. The company has achieved proof of concept for its first-generation microRNA-based ONTs in both in vitro and in vivo studies, demonstrating efficacy in reducing fat mass and improving metabolic parameters.

The company is currently advancing its second generation of microRNA antagomirs, specifically targeting miR-22-3p, which are designed for active targeting to adipocytes through conjugation with fatty acids or peptides. This targeted delivery mechanism is expected to enhance the safety and pharmacokinetic profiles of the drug candidates, allowing for lower effective doses. AptamiR's ongoing research includes preclinical studies aimed at completing the pre-Investigational New Drug (IND) phases for their lead candidates, with the ultimate goal of initiating clinical studies within the next two years. Their innovative approach positions them to potentially transform the treatment landscape for metabolic diseases and ovarian cancer, ultimately aiming to improve patient outcomes and reduce healthcare costs.

United States

AptamiR Therapeutics has created an innovative model of virtual and outsourced Drug Research & Development to cure human obesity by developing microRNA-based treatments that target peripheral adipose tissues (fat cells).

AptamiR Therapeutics

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