Proof of concept

Netia MedTech is developing a single-stranded RNAi (ss-RNAi) platform that generates drugs capable of penetrating nearly all cell types in vivo and degrading the mRNA that encodes currently ‘undruggable’ protein targets.

PTP1B is a classic example of an ‘undruggable’ target. It is considered an important therapeutic target for numerous medical disorders, based primarily on gene knockout studies.

Netia MedTech initially established Proof of Concept for its ss-RNAi (guide strand-only) drug development platform by generating ss-RNAi drugs targeting the mRNA that encodes the PTP1B protein.

Therapeutic levels of target engagement by ss-RNAi drugs in high oligonucleotide uptake tissues, such as kidney and liver, are achieved via a single i.p. bolus dose of under 30mg/kg. At higher doses, additional drug does not enter cells and clears rapidly from the body.

Netia MedTech’s ss-RNAi compound development to date has focused on those causing degradation of the mRNA target for the protein selected. Up to 95% target degradation in the kidney, for example, is achieved after a single ss-RNAi administration to mice.

The drugs we develop are oligonucleotides and are, therefore, readily and rationally designed on the basis of the nucleotide sequence of their RNA target. Manufacturing is identical to ASO and siRNA drugs.