Precision medicine with neuron specific targeting
using non-invasive brain delivery technology

The challenge of delivering oligonucleotides
to the central nervous system (CNS)

  • Reaching the CSF is just the first challenge. To be effective, an oligonucleotide needs to reach specific regions and cells in the brain.
  • Of the 1.7 billion brain cells, 50% are neurons
  • 350,000 serotonergic neurons account for only 0.02% of the cells in the brain – a very small target

Our technology enables the target delivery of oligonucleotides to specific brain cell populations


  • The conjugation of small molecules to oligonucleotides enables targeted delivery to specific cells in the brain

  • The small molecule component binds to a specific cell surface neurotransmitter transporter or GPCR receptor on the target cell enabling the delivery of the therapeutic oligonucleotide
  • TPH2+ cells

  • A488-MIC-083

  • DAPI nucleous

Selective accumulation of MIC-083 in tryptophan hydroxylase2-positive (TPH2-positive) 5-hydroxytryptamine (5-HT) neurons after intranasal administration.