Our laboratory

allows the development of nuclear microRNA drugs for RNA activation of therapeutic genes from discovery to pre-clinical proof-of-concept.

In vitro validation

The best RNA drug candidates are synthesized and taken into in vitro testing in our own BSL2-level cell culture and molecular biology laboratories. At this point, we test the functionality of the lead microRNAs in relevant cell culture models, followed by modern molecular biology methods, including:

MicroRNA drug leads are tested in relevant cell culture models by knock-in/out analyses
Target protein expression is verified
Next-generation sequencing analysis is done to verify the specificity of RNA activation
Confirmed leads are taken forward to formulation selection

EV production

RNatives has long experience in working with extracellular vesicles (EVs), such as exosomes. These vesicles are natural carriers of molecular messages between the tissues in the body, and as such are a natural choice for introducing candidate drugs microRNAs into cells. RNatives developed a process for R&D level production and downstream purification of EVs.
The key steps are:

The stable cell line is produced to overexpress microRNA for loading to EVs
Upstream production of EVs, small or large scale
Downstream purification and concentration of EVs
Analysis and quality control of EV/miRNA product
Functional testing of EV/miRNA drug in target cells (release and potency)

IN VIVO PROOF-OF-CONCEPT

Combining in silico and in vitro work results in a confined number of leads, that are scientifically relevant to the desired indication and have shown functional effects in relevant cell lines. RNatives work together with trusted, validated, and high-quality contract research organizations (CROs) to shift the initial molecular findings into physiological, meaningful results in a disease model. These proofs-of-concept (POC) experiments drive the selection of RNatives drugs towards IND-enabling studies.