Program Development

Achievement of Proofs of Concept

AptamiR’s distinctive model of virtual, outsourced, and cost-effective drug research and development has resulted in the achievement of several proof of concept milestones within a few years in operations:

  • In silico screening and selection of microRNAs regulating lipid metabolism, mitochondrial activity and thermogenesis
  • In vitro validation of microRNA candidates in primary cultures of human adipocytes (clinical trial in a dish)
  • In vivo reduction of weight gain and fat mass by treatment with microRNA modulators in a mouse model of diet-induced obesity with improvement of metabolic parameters.
  • Filing of several provisional patent applications covering the use of microRNA modulators to treat obesity and related conditions
  • Regulatory first pre-IND application positively reviewed by the FDA, setting the stage for the second generation antagomir full IND application to be submitted for early clinical program