Therapeutic Strategy

Delivery Platform

In order to optimize the benefit-risk ratio and the PK/PD profile of our drug candidates, AptamiR is pursuing multiple approaches to achieve targeted delivery of microRNA modulators to adipose tissues. Hence, the trademarked name of our Company, AptamiR™, that combines the word apta (from the Latin verb aptare “fit together”) with the microRNA abbreviation miR. We are currently focusing on two approaches:

Lipid Conjugates: The first method shown to improve the in vivo delivery of miRNA analogs was 3’ conjugation with cholesterol. We are taking advantage of the fact that the human adipocyte membrane is rich in receptors and transporters for fatty acids to conjugate our miRNA candidates with distinct fatty acids that are actively transported across the adipocyte membrane.

Peptide Conjugates: Short peptides have been shown to facilitate the delivery of oligonucleotide therapeutic agents inside cells.  We are taking advantage of the fact that the adipocyte membrane is rich in specific receptors that transport peptides to conjugate our miRNA candidates with distinct peptides that are actively transported across the adipocyte membrane.

The Adipocyte-Targeting Delivery platform developed by AptamiR could be used not only for oligonucleotides but also for small molecules, peptides, peptidomimetics, nutraceuticals and gene editing systems.