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.
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