Research & Development

Nucleic acid delivery platform

nLife Therapeutics has developed different nucleic acid chemical modifications with the aim to optimize nucleic acid cell specific delivery.

We have combined siRNAs and antisense oligonucleotides (ASOs) with some specific and potent small molecule ligands to cell membrane receptors or transporters.

These combinations proofed to deliver the nucleic acid to the selected cell in an effective way by means of an active receptor or transporter mediated endocytosis process.

The conjugated oligonucleotide is accumulated in specific cell endosomes and later released into the cytosol to perform the gene silencing.

Extension of current nOligos platform with new cargo

mRNA and Ribonucleic Protein particles (RNPs) is under way.

Route of administration

Conjugated oligonucleotides can be formulated for intrathecal or intracerebroventricular (ICV) bolus administration to access the brain area. Also, preliminary studies in mice demonstrated that intranasal delivery of the conjugate works as well as the icv direct brain administration²

The USPTO has granted the first patent family

Neurotransmitter transporters as entry neuronal entry points and PCT patent application is also under evaluation. Recently, a second patent application has been filed claiming the targeting to different brain regions to secure the development for other disease areas.

² Bortolozzi, A., Castañé, A., Semakova, J., Santana, N., Alvarado, G., Cortés, R., et al. (2012). Selective siRNA-mediated suppression of 5-HT1A autoreceptors evokes strong anti-depressant-like effects. Molecular Psychiatry, 17(6), 612–623. doi:10.1038/mp.2011.92