technology
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
