Differentiating R&D approach
InteRNA pursued a unique and new approach to identify the best miRNA drug candidate for each disease type considered. The developed miRNA discovery and functional validation technology platform enabled an unbiased genomics screening approach focusing on the elucidation of the functional roles of individual miRNAs in cancer cell biology. This approach gave a direct insight in potential functions and allowed for combinatorial screens with e.g., chemotherapy, ionizing radiation, targeted kinase inhibitors, and immunotherapy to address resistance/re-sensitization of cancer cells.
The unique features of InteRNA’s differentiating R&D approach:
- miRNA Library: Use of next generation sequencing and bioinformatics to create a broader microRNA library
- Screening: Non-biased (agnostic) phenotypic functional screening in cell-based assays (instead of descriptive expression profiling)
- Chemical Modifications: Application of chemical modification to enrich uptake of active strand in the RNA-Induced Silencing Complex (RISC)
- Delivery: Use of next-generation lipid nanoparticle (LNP) formulation selected on localized cellular delivery/delivery beyond the liver, endosomal escape, and pharmacodynamic effects
- MoA: Use of RNAseq, Ingenuity Pathway Analysis software, and bioinformatics to decipher molecular/biological Mode of Action (MoA)
- IP: Unique IP portfolio around drug candidates and formulation
Beyond oncology, we have shown this R&D approach qualifies also for the identification of miRNA drug candidates for treatment of Central Nervous System (CNS) indications, e.g., mesial Temporal Lobe Epilepsy (mTLE) and neurodegenerative disease (see Scientific Publications).